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Life after cancer: Navigating survivorship and mental health

Life after cancer: Navigating survivorship and mental health

When we think of cancer, we think of a fight: a battle between a patient and a mutation to their biology, a transformation – a hijacking – on the cellular level. Indeed, cancer doesn’t just take over a person’s physical body, but their very life. 

The word “cancer” brings to mind diagnosis – and the mental impact of that on the person, who becomes “patient” thereafter, and their families – as well as the treatment possibilities, and the recovery, the regathering of strength post those therapies, and – sometimes, hopefully, when effective – pulling through and making it to “the other side”, so to speak: the cancer in remission. 

In short, when it comes to journeying through cancer care, there seems to have been prolific, yet strangely finite, consideration of that path, ceasing when it comes to post-treatment. Screening and diagnosis to treatment and remission have been covered, but what about what happens after that? What about cancer survivorship? The good news is that a shift in the state of affairs is occurring.

In the aftermath of cancer: Fear of the unknown

June was National Cancer Survivors’ month. As the National Cancer Institute (NCI) defines it, cancer survivorship as a term applies to those who have faced cancer, undergone treatment, and survived – but, importantly also, continued onwards “through the balance of [their] life”.

This notion of balance is key. To survive cancer is not necessarily to be free of the disease; life, indeed, can very much be lived with cancer. After all, the essential nature of “survival” is to continue to live or exist in spite of an accident, ordeal, or challenging circumstances. It is under such stresses, though, that we change. Therefore, it stands to reason that post-treatment care for cancer patients should evolve to parellel these changes.

After cancer treatment has “completed”, real-life adjustments can be considerable, taking a toll not only on the body, but on a patient’s mental health. A study published in Medical Clinics of North America as far back as 2017 noted the negative mental health aspects of survivorship: that there was fear and hypervigilance to physical sensations – known as “fear of recurrence” (FOR) – and that cancer survivors “have numerous sources of distress that increase their potential for adjustment reactions”. That “distress” becomes an umbrella term for myriad and divergent symptoms, ranging, according to the National Comprehensive Cancer Network (NCCN), “from common feelings of vulnerability, sadness, and fears of recurrence to disabling depression, anxiety, trauma, panic, and existential crisis.” That’s a pretty broad remit, and certainly not insignificant when it comes to consideration of cancer survivorship care.

The same study suggested screening, not only for cancer recurrence, but for mental health needs. The NCCN itself has guidelines for screening distress to assist healthcare professionals with this, a commonly recommended measure being the Distress Thermometer (DT), a simple oral or printed measure that asks the patient to rate their distress from 0 = no distress to 10 = extreme distress. If a patient scores four or higher, further screening may be needed. 

The burgeoning field of cancer survivorship research

Cancer survivorship research, such as that conducted at Imperial College London, focuses on the health and wellbeing of people who are living with, through, and beyond a cancer diagnosis, recognising at the same time the impact of cancer on family members, friends, and caregivers. 

This field of research has sprung up because – happily – cancer survival in the UK has doubled over the last 40 years. It is estimated that some four million people will be living with or after cancer by 2030. However, it is a double-edged sword: a greater number of cancer survivors means a greater number of people living with the after-effects of the disease and its treatment. It is for this reason that supporting people to live with and beyond cancer is a major focus of the NHS Long Term Plan for Cancer. Indeed, the National Cancer Research Initiative has recently published Cancer Research Priorities for Living With and Beyond Cancer.  

Fear of recurrence (FOR) is palpable, a beastly nightmare that poses a threat in waking and in sleep, seeping through the fabric of the family unit – exacerbated by the fact that late effects might not exhibit for months or years following the last treatment. These effects can include cardiovascular and pulmonary issues, bone loss, changes to sight and hearing, lymphoedema, and additional problems.

The same Medical Clinics of North America study mentioned above also noted the extensive Childhood Cancer Survivor Study (CCSS), which tracked over 20,000 survivors across the US. While most survivors did not meet the criteria for a diagnosis of post-traumatic stress disorder (PTSD), many survivors did report post-traumatic stress symptoms. Certainly, adult and young adult (AYA) survivors (normally, 15 to 39 years of age) are increasingly recognised as a group with emotional needs that differ from either childhood cancer survivors or older adults, and FOR is common in this bracket. 

What is important to note is that physical symptoms can be associated with depressive symptoms. In other words, FOR and its ilk can become a case of “directed energy”: think it, and it shall happen. One example in the Medical Clinics of North America study was oral cancer survivors, who reported dental health, problems with smelling, and issues with range of motion – all associated with both depressive and anxiety symptoms. In terms of treatment for cancer survivors, however, when it comes to mental health conditions, which the study notes "can linger for up to 10 years after treatment” – cognitive behavioural therapy (CBT) has been shown to be effective in reducing mood symptoms, while mindfulness-based approaches have demonstrated efficacy in reducing anxiety and depressive symptoms. 

Furthermore, according to a Phase 3 study led by researchers at the University of Pittsburgh School of Medicine, and supported by the National Cancer Institute, run at the UPMC Hillman Cancer Center in Pittsburgh and published in The Lancet, cancer patients who receive specialised mental health support as part of their treatment plan are more likely to see improvement in quality of life and pain reduction, as well as fatigue and depression. 

Importantly, the study showed that, even if the collaborative care intervention was offered to patients for free, hospitals could expect to save some $4 million or more for every 250 patients. Mental health treatment for cancer survivors, it suggested, results in fewer emergency room visits, fewer readmissions to hospital within 90 days, and a shortening of the length of hospital stays themselves – all benefits that reduce costs to healthcare systems.

Shaping the future of cancer survivorship care and support

The National Cancer Survivorship Resource Center (The Survivorship Center) is a collaboration between the American Cancer Society and the George Washington University Cancer Institute. Funded by a five-year cooperative agreement from the Centers for Disease Control and Prevention (CDC), its goals are to shape the future of cancer survivorship care and improve the quality of life of cancer survivors as they transition from treatment to recovery.

Merck & Co (known as MSD outside of the US and Canada) is a pharma company striving to provide similar support. The World Cancer Day motto, “Alone we are strong. Together we can be unstoppable.”, urges the necessity of collaboration, and 2024 has been an important year for the Close The Care Gap campaign, which advocates equitable access to cancer treatment. Notably, Merck has collaborated with the American Cancer Society’s Building Expertise, Advocacy, and Capacity for Oncology Navigation (BEACON) initiative for several years, also, supporting global health institutions in low- and middle-income countries (LMICs) by fostering the creation and integration of cancer patient navigation programmes to address disparities in cancer care. Additionally, by harnessing AI, ML, and other technologies, the company insists personalised cancer care can be supported in ways that were not possible before; for example, digital healthcare platforms, which can enhance patient education and help support adherence, while cultivating community support, including such CBT programmes as mentioned above.

A case study – Breast cancer and the enlightened patient experience

Let’s turn, though, to a real-life example of cancer survivorship: Bethan Brookes, a journalist who wrote a personal blog about her cancer, and who permitted reference to her journey in this feature, is now in remission. However, her life was turned upside down on the 13th January 2021, when she was diagnosed with advanced HER2-positive Stage 3 breast cancer. Three weeks later, she had had a mastectomy (“Show up. Breathe. Trust”, the name of the blog, was Brookes’ mantra on the morning of her mastectomy); all of the lymph nodes on her left-hand side were also removed (15 out of 20 had shown signs of cancer). This was followed over subsequent months by gruelling chemotherapy rounds, radiotherapy, and drug therapy. 

The blog, in fact, became Brookes’ own form of therapy, her mental health journey through cancer survivorship – and an online location to which other cancer survivors could come, read and relate, share their stories, and build a community of care.

To follow Brookes’ journey through her blog is to accompany her through “spiralling fears” following a mere “four-minute call (rather than a face-to-face appointment)” due to the COVID-19 pandemic, to understand that “nothing can prepare you for the diagnosis”, but that there is a calming effect within simple, clearly defined instructions for patients undergoing surgery and post-operative care. Of course, an operation is just “a launch pad” for the “odyssey” that cancer is. The diagnosis changes everything and “fear loomed large in the shadows”.

After an all-clear post-mastectomy, there was a separation that Brookes felt from normalcy, feeling that she had “finally stepped over the threshold into a different space. The club that no-one wants to be part of. The cancer club. And through the cancer club lens, the world looks different.” Indeed, that club contains “a seemingly endless continuum of tests and scans, nail-biting waits for results, and follow-up appointments.” What saw her through the early days, though, was the allocation of a MacMillan breast cancer nurse.

Such support is deeply required by patients who have no clear inkling of the parameters of time left them post-treatment, as much as during: Will it be five years? Five months? The psychological impact is intense. The expression “every moment counts” is a pressing one for the survivorship group, and for their nearest and dearest. In Brookes’ case, she shares that there came a point where, with hesitancy, her 16-year-old son asked whether he could see his mother’s mastectomy, asked with “courage and emotional maturity” – highlighting that, when it comes to adapting to the cancer journey, it is not just the patient who is forced into accepting transformation – a “brutal transition” – of life as they had come to know it.

While Brookes herself is stoical about the loss of one of her breasts – “They fed my children and sustained new life. They have done their job, and, for me, losing one will not significantly diminish my experience of life.” – she is also consciously aware of the stigma and, sometimes assumed, sympathetic distress of “other people” about such a loss for a woman. And that can include survivors’ own partners and children. But it is, as Brookes states, all too easy “to choose to retreat into a place of separateness”, to seek refuge in the “quiet loneliness” of the disease. The cancer survivor’s journey is frequently not a solo one; rather, it is one embarked upon hand in hand with a loved one, there to provide support in the darkest hours. 

Sadly, that is not always the case, however, and a retrospective longitudinal study conducted over 10 years and published in the Journal of the National Comprehensive Cancer Network found that loneliness and social isolation were associated with a higher risk of mortality among cancer survivors, according to the UCLA Loneliness Scale. A total of 3,371 cancer survivors with 5,711 person-years of observation were included in the study and most of them were long-term survivors, diagnosed over two years prior to the survey. The variables of male sex, non-White race/ethnicity, unmarried status, less education, more health conditions (other than cancer), and feeling depressed in the past year were linked to a higher probability of feeling or being “lonelier”.

Survivorship: No battles, but acceptance

Support and community are key words, then, in cancer survivorship. So, while naming her drugs after her children’s favourite Avengers characters and glutting on Schitt’s Creek to see her through long and gruelling chemotherapy sessions seems formidable in the concept of fighting cancer, it is notable that Brookes would beg to differ with the notion of “fighting”, per se. Instead, for her “using the language of war feels neither helpful, nor wise […] To be at war with the cancer implies a zero sum game. Cancer or no cancer. Win or lose. You beat it or it beats you.” What is to be submitted to, rather, is an acceptance, a “surrender to the process.” 

Survivorship for Brookes is a crossroads of an ending and a beginning. After “one mastectomy with full lymph clearance, 27 intravenous infusions, 21 sessions of radiotherapy, 16 doses of targeted therapy, 36 self-administered injections, [and] a bucket full of drugs” she finished her treatment, yet noting that NICE guidelines state she should not receive any monitoring scans. Rather, she – as are other such survivors – is advised to carry on with life, “hope for the best, and ‘wait for any symptoms of secondaries’ (which will mean […] Stage 4 cancer).” The onus, then, falls on the patient, a health responsibility brusquely handed back. So, while Brookes takes on the importance of stress reduction, a largely plant-based diet, supplementation, and exercise – and prioritises tasks dependent on energy available on any one day whilst in recovery, from treatment, from trauma – it’s all too clear that “modern oncology focuses on treating us once we have been diagnosed with cancer,” looking ever for a cure, rather than prevention, or healing post-fact.

Brookes’ automatic and “underlying suspicion” was that it was all about cost-saving. The reality is, however, that repeated scans are not good for the body: the risks of developing a fatal cancer from a CT scan are about one in 2,000. Yet, the risks are not the same for every cancer, nor each individual. Personalisation is, indeed, also key. There is, furthermore, the consideration that, with any secondary cancer that might be found via a scan, care then becomes palliative, focused on improving quality of life, as it is “incurable”. 

Brookes has since opted for a contralateral mastectomy, not wanting reconstruction. It’s not about risk reduction, but about “symmetry” – again, this notion of balance, of stability, of control over one’s body once more. Yet, while Brookes notes the sense of “an identity in free fall” and repeated moments of return to the trauma of her experience, survivorship – like the cancer experience itself – is unique to each individual. 

Cancer survivorship: Personal in so many ways

In a landmark 1985 report published on cancer survivorship, physician Fitzhugh Mullan described his own battle with cancer and the unique issues that lay ahead for survivors of malignancies. In the report, he observed three “seasons of survival”: acute survival (diagnosis to completion of initial treatment); extended survival (the period of anxiety over possible recurrence); and permanent survival (recognising the likelihood of having been cured). 

The outcomes for cancer survivors vary on many levels. As a 2021 Canadian study published in Current Oncology noted, of the 44 participants who took part – 11 survivors, seven family/friend caregivers, 18 healthcare providers, and eight decision-makers – 13 stakeholder-relevant outcomes were identified and categorised into the domains of psychosocial, physical, economic, informational, and patterns and quality of care. 

In 1996, the Office of Cancer Survivorship (OCS) was established to promote a better understanding of and the ability to address the unique needs of the growing population of cancer survivors. Housed within the NCI’s Division of Cancer Control and Population Sciences, the OCS works to enhance the quality and length of survival of those diagnosed with cancer and to minimise or stabilise adverse effects experienced during cancer survivorship. The office supports research that both examines and addresses the long- and short-term physical, psychological, social, and economic effects of cancer and its treatment among cancer survivors and their families.

Cancer survivorship research under the OCS includes a variety of funding mechanisms, such as Investigator Initiated Applications (R01s), Small Grant Programs (R03s), and Requests for Applications (RFAs), as well as collaborations within NCI and other organisations regarding survivors' needs for education, communication, and appropriate medical and supportive care.

Refocusing the cancer club lens

There yet exist further barriers to accessing what is clearly necessary mental health support as a cancer survivor, including stigma, lack of integration between oncology and mental healthcare systems, and financial constraints. To this end, endeavours such as the Working With Cancer Pledge, for example, ensure a supportive and positive work environment that is inclusive, respectful, and safe, providing beneficial, informational resources.

Additionally, if a cancer survivor goes on to develop another condition separate from the oncological, the impact on their care is considerable. Nonetheless, despite these challenges, there exist evidence-based interventions for mental health in survivorship, too, such as psychotherapy, pharmacological treatments, peer support, and lifestyle interventions. Therefore, there needs to be a call for greater collaboration – again, one of those key words – between oncology and mental health professionals; and a greater call for education on the availability of these interventions.

At the end of the day, it should be remembered that many cancer survivors spotlight the gratitude they feel for each new day that dawns: the value in life and living, in friends and family, and the necessity of community in fighting the fight (or the process of acceptance and submission) and carrying on – through treatment, and beyond – is invaluable.

Nicole Raleigh

About the author

Nicole Raleigh is pharmaphorum’s web editor. Transitioning to the healthcare sector in the last few years, she is an experienced media and communications professional who has worked in print and digital for over 18 years.

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At ASCO, a focus on care as well as cure

At ASCO, a focus on cancer care as well as cure

At ASCO 2024, integrated palliative care was a presidential theme, as doctors and patients ruminated on the art and science of caring for the whole patient through the unique experience of cancer treatment. 

As cancer doctors gathered in Chicago earlier this month for the ASCO annual conference, their focus was not only on the newest innovations in cancer care, but also looking back on how far the space has come. Cancers that once had bleak prognoses now have high survival rates and multiple treatment options. 

ASCO President Dr Lynn Schuchter experienced this transformation in the space firsthand as a melanoma specialist. 

“When I began my fellowship, people often asked why I chose melanoma,” she said in her opening address. “There were no treatments beyond surgery. Outcomes were bleak. In the early days, I was essentially a hospice doctor. But even then, there were clues from emerging science that suggested the potential and power of immunotherapy and targeted therapy directed at specific driver mutations. And indeed, gradually, over decades, laboratory and clinical researchers, many of you here today, translated this promising basic science into powerful therapies, turning what was once a treatment desert into a rainforest of options for our patients with melanoma.” 

Lynne Schucter at ASCO 2024

All photographs courtesy of Jonah Comstock

It’s a welcome change for doctors and patients. Yet, as more and more people survive cancer, what it means to care for a cancer patient changes. Palliative care, which was once synonymous with end-of-life care, now represents a treatment modality that can help every cancer patient live with the life-changing realities of diagnosis, treatment, and survivorship – or help them and their family face the end with comfort and dignity. 

Schuchter shared that this is the first time in 25 years an ASCO presidential theme has focused on palliative care, but she believes now is the time to emphasise this important aspect of what oncologists do.

“What I’m advocating is a reemphasis on a fundamental concept in medicine. It dates back to Hippocrates, who said, ‘to cure sometimes, to relieve often, to comfort always, is all that may be reasonably expected of medicine.’ These words, to comfort always, tell us that whether or not we cure a disease or even succeed in relieving symptoms, comfort is not optional. It’s an essential part of patient care.” 

Concrete progress at ASCO

The conference featured a number of compelling presentations on the importance of palliative care, from Schuchter’s address to keynote speaker Dr Abraham Verghese to the ASCO Voices session where patients and doctors shared deeply personal stories. 

But the theme wasn’t only a matter of conference content. In May, ASCO updated its palliative care guidelines for the first time since 2016.  

“Updated ASCO palliative care guidelines have just been published to emphasise the essential role of all oncologists in providing palliative care as an integrated, holistic part of treatment, beginning with diagnosis,” Schuchter stressed. “Let me I’ll say that again. Integrating palliative care early from the time of diagnosis is key.”

One of the plenary sessions at ASCO also focused on palliative care, specifically looking at virtual delivery of palliative care via telehealth. The study, which included a randomised sample of 1,250 patients with advanced lung cancer, showed that virtual palliative care was just as effective as in-person care, measuring quality of life, satisfaction with care, and patient mood.

Abraham Verghese at ASCO 2024

All photographs courtesy of Jonah Comstock

“The main takeaway from this national multi-site randomised study is that palliative care led to equivalent benefits for patient-reported quality of life whether delivered via video or in-person visits among adults with advanced lung cancer,” study author Joseph Greer, PhD, of Massachusetts General Hospital, said in a press briefing. “The study findings underscore the potential to increase access to evidence-based early palliative care through telehealth delivery.”

Additionally, Schuchter shared that ASCO is working to increase the number of physicians dual-certified in palliative care and oncology, a number that now stands at only 3% to 4% of oncologists. A new pilot fellowship, led by Dr Jamie Von Roenn, graduated its first class last month. 

Palliative care and living with cancer

Conversations about palliative care at the show fell into two buckets: palliative care’s traditional role as end-of-life care, and the growing understanding of palliative care as a holistic part of cancer care throughout treatment, including for survivors. 

“Research indicates that early integration significantly improves patients’ quality of life, of well-being, and some studies suggest potential for longer survival times,” Schuchter said. “This tells me that patients on the road to cure need symptom palliation just as much as those with more advanced cancer.”

During the ASCO Voices session, Phuong Gallagher, a 17-year survivor of stage 4 rectal cancer, spoke earnestly about her own experience and how it showed her the importance of this care.

“When I was first diagnosed, there was suddenly this rush of information in front of me,” she said. “All these decisions that had to be made immediately, because I had to get into treatment. My doctors were focused on giving me the best chance I had at a cure and finding the best options for me. […] The problem was that all these things addressed the immediate of what was happening. It didn’t talk about what I would be living with years down the road. And now, as we have more and more patients living longer and longer, we have the opportunity to look at the different long-term effects that we have to deal with.” 

Gallagher’s life has been impacted immensely by the long-term complications of her cancer.

Phuong Gallagher at ASCO 2024

All photographs courtesy of Jonah Comstock

“We didn’t talk about chemotherapy and how that could cause cognitive issues with chemo brain. We didn’t talk about surgery and how that would leave me with LARS syndrome, where I felt tied to the bathroom wherever I went, having to scope out immediately, ‘Where is the bathroom? How accessible is it? Can I get out of my seat fast enough to get there?’ And then the reversal of that with ostomy surgery and as a permanent ostomate, how that changed my ability to live my life and not have to be tied to the bathroom now. I could go on long trips and not worry. So that was a positive change. But we didn’t talk about that either,” she said.

Better communication earlier on might not have changed the treatment decisions she made or the outcomes of those treatments, Gallagher said. But it would have given her a chance to prepare for those realities and feel more in control of her treatment journey. 

“Now that we have people who are living longer, we can talk about the effects of it and how to address it because quality of life is just as important as quantity of life,” she said.

Palliative care at the end of life 

Several speakers at the show also talked about how important palliative care continues to be at the end of life. 

“When I was a young physician, I was caught up in what I call the conceit of cure,” physician and author Dr Abraham Verghese said in his keynote address. “The sense that we could fix just about anything, and if we couldn’t, it was because people had come in too late.” 

As an infectious disease doctor and not an oncologist, Verghese said, it was easier for him to keep up that illusion for a time – until the AIDS epidemic. But when a young patient’s mother called the office to report that he was too weak to come in, Verghese decided to pay the patient a visit at home. 

“I drove out to where he lived in the country in a mobile home, and I was doing it for my own purposes,” he said. “Some sense of closure, if you like. When I got there, it was profound how powerful my visit was to the family, helping them come to terms with this, but especially on him, making him feel that I hadn’t quite abandoned him and that I was going to be on this journey with him. And I remember leaving the house and thinking, ‘This is what the horse and buggy doctors of hundreds of years ago did so well in the absence of all the cures we have now. When they could not cure, they could still heal.” 

Richard Leiter at ASCO 2024

All photographs courtesy of Jonah Comstock

In the ASCO Voices session, Dr Richard Leiter, a palliative care physician at the Dana Farber Cancer Institute and Brigham and Women’s Hospital, told his own story about a young patient named Carlos, who died of kidney failure after a failed stem cell transplant for AML. 

“I was a newly minted palliative care fellow just over a month into my training. I was progressing quickly, but, as happens with so many of us in medicine, my confidence set a few steps ahead of my skills,” he said. 

To Leiter’s mind, Carlos’s mother was in denial of her son’s situation and he needed to get through to her. 

“I don’t understand why this is happening. He was cured of his leukaemia. I hear you telling me his liver and his kidneys aren’t working, but I have hope, hope they’ll get better. He is going to get better,” the mother told Leiter in his retelling.

“‘I hope they will,’ I told her,” Leiter said. “I should have stopped there. But in my eagerness to show my attending and myself that I could handle this conversation on my own, I kept going, mistakenly. ‘But none of us think they will.’” 

The mother reacted badly, accusing Leiter of wanting her son to die. His attending took over the case. But Leiter learned an important lesson about his role as a palliative care doctor. 

“I could have acted as her guide, rather than as her cross-examiner,” he said. “And I could have hoped to ease Carlos’ suffering and genuinely hoped that he got better, even though I knew it was next to impossible. I know now that hope is a generous collaborator, and it can coexist with rising creatinine, failing livers, and fears about intubation. As clinicians, we can always find space to hope with our patients and their families if we look for it.” 

About the author

Jonah Comstock is a veteran health tech and digital health reporter. In addition to covering the industry for nearly a decade through articles and podcasts, he is also an oft-seen face at digital health events and on digital health Twitter.

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Commercialisation in oncology: Challenges and innovative solutions for pharma manufacturers

Commercialisation in oncology: Challenges and innovative solutions for pharma manufacturers

Every day, manufacturers continue to develop new, life-saving cancer treatments, and breakthroughs in oncology promise to transform patient outcomes. Last month, the American Society of Clinical Oncology (ASCO) had its Annual Meeting in Chicago, highlighting groundbreaking advancements in cancer care and the rapid development of new drugs and therapies during the plenary session. Yet, behind these achievements, small and midsize manufacturers face a labyrinth of challenges that threaten to stall their progress. 

Limited resources, a lack of expertise, and financial hurdles often impede drug manufacturers’ ability to navigate the complexities of bringing new therapies to market. As we look ahead to the second half of 2024, what obstacles must manufacturers overcome, and how can they navigate this complex landscape?

Anne Marie Robertson, executive vice president of EVERSANA Oncology Commercialisation, and Maneesh Gupta, senior partner at EVERSANA Consulting, offer their insights on the most pressing issues in oncology commercialisation and present solutions to help brands and patients alike.

What oncology brands are up against in 2024

Accelerated approvals: A double-edged sword

Anne Marie Robertson: The unmet needs in oncology, coupled with the biotechnology industry's development pipeline, create unprecedented pressure for rapid approvals. This urgency often results in more teams participating in the accelerated approval process.

The fast pace of drug development, while promising in terms of development timelines, often leads to substantial risk in launch planning. Brands are required to meet stringent regulatory requirements and ensure that their products demonstrate substantial clinical benefits to patients. 

The need for accelerated approval often means that drugs are brought to market based on surrogate endpoints and require confirmatory trials. This approach, while beneficial for getting treatments to patients faster, creates significant challenges in recruiting the confirmatory trial and in meeting the clinical endpoints. The Food and Drug Omnibus Reform Act of 2022 requires that the confirmatory trial be underway at the time of approval and allows the Food and Drug Administration (FDA) to reject applications without confirmatory trials.

Maneesh Gupta: As of April 2023, 23 oncology drugs with accelerated approvals had withdrawn specific cancer indications, with 74% of these withdrawals occurring in the last three years. These withdrawals highlight the challenge of validating clinical benefits beyond surrogate endpoints through confirmatory trials. 

Manufacturers must assess the unmet need in the disease area to justify seeking accelerated approval. Also, manufacturers have a hard time recruiting for the trials to meet the FDA timelines. Now, manufacturers can leverage integrated datasets, including longitudinal claims, EMRs, and prescriptions, to find hospitals and clinics that have the maximum number of potential patients who meet the inclusion and exclusion criteria.

Secondly, manufacturers that receive accelerated approvals do not need to shortchange their launch preparations or rush to out-license, fearing that the drugs will not be ready in time. They can leverage the prebuilt infrastructure of commercial partners that can bring these to market in less than a year while reducing the upfront costs of commercialisation by 25% or more.

Building a commercial infrastructure

Robertson: Drug developers often have no in-house expertise or physical infrastructure to build commercial operations and facilitate access to innovative solutions for supporting product commercialisation. The need to scale rapidly becomes paramount as a compound advances toward accelerated approval. 

The transition from clinical development to commercial launch involves numerous growth challenges. Development-phase companies need to establish distribution networks, ensure compliance with regulatory requirements and build effective commercial teams. This phase demands a versatile infrastructure capable of adapting to the changing demands of the market. Development-phase companies need to create a value story for their development compounds prior to launch.

Gupta: Traditional third-party partners often lack the agility needed to navigate the complexities of the unpredictable oncology landscape. These models cannot be customised to address the unique needs of each partner, tumour type, and patient journey. Outsourcing efforts provide emerging companies access to expertise and resources they may lack internally. 

Leveraging next-generation solutions such as omnichannel customer engagement platforms, electronic benefit verification (eBV), and direct-to-patient initiatives can optimise patient engagement and access. Commercialisation partners, which go beyond traditional service providers, can provide emerging companies with the same platform as large pharmaceutical manufacturers while adding the advantages of scalability, speed, and risk sharing.

Additionally, the integration of data analytics and artificial intelligence can significantly enhance the ability to predict physician behaviours and patient needs, thereby allowing for more informed decision-making. These technologies can help identify potential issues before they become significant problems, as well as raise promotional effectiveness significantly, enabling a proactive and precise approach to commercialisation.

Navigating financial risks

Robertson: Manufacturers grapple with the uncertainty of technical and regulatory success of FDA approval. Despite significant upfront investments, there is no assurance that products will reach the market. A new risk that must be considered is the potential for product withdrawal due to the results or accrual of the confirmatory trial.  

A large biotech company recently received a complete response letter when the confirmatory trial was still in the safety phase at the time of product approval. We have also seen products withdrawn from the market and close the confirmatory trial, jeopardising years of research and corporate cash flow. 

While large pharmaceutical companies may recalibrate and relaunch therapies, emerging and small manufacturers struggle with redesigning clinical plans and completing additional trials. Smaller manufacturers may find it more challenging to absorb the costs of additional trials and regulatory setbacks. These financial constraints can hinder innovation and delay the introduction of potentially life-saving therapies.

Gupta: By leveraging innovative solutions and strategic partnerships, manufacturers can mitigate financial risks. Access to expertise, resources, and advanced technologies help to navigate regulatory challenges and ensure a more efficient and successful commercialisation process. 

Collaborating with third-party organisations that specialise in specific aspects of the commercialisation process can provide the necessary support without the need for significant capital investment. Also, these collaborations can enable manufacturers to scale up or down quickly and cost-efficiently.

The first five years of commercialisation typically cost upwards of $265 million. Manufacturers should not be forced to lose ownership, sacrifice their company value, or invest more than $200 million to commercialise on their own. Risk-sharing agreements and value-based pricing models are also emerging as viable strategies to manage financial risks. 

These approaches align the incentives of manufacturers and commercialisation partners, ensuring that financial rewards are tied to partner success, clinical outcomes, and patient benefits.

Articulating and communicating value

Robertson: For first-to-market treatments, companies must raise awareness about how the therapy works and its benefits. Clearly articulating the value proposition allows the organisation to define and segment the landscape, negotiate pricing and reimbursement, and support a go-to-market strategy. This approach helps remove market access and affordability barriers, enabling patients to access the therapy quickly.

Effective communication strategies are essential to educate healthcare providers and patients about the benefits and potential risks of new therapies. This involves developing communication campaigns that reach all stakeholders, creating educational materials for patients and providers, and ensuring the community has a clear understanding of the benefits of the therapy as well as the best way to administer it to patients.

Gupta: Technological advances and personalised therapies driven by new tumour biology discoveries necessitate marketing efforts that can adapt to a changing landscape. Mapping diagnostic and treatment journeys through the unique lens of patient and provider archetypes is crucial. Without internal resources and financing, pharma innovators struggle to carry out concurrent planning and strategic development, which hinders successful commercialisation.

Leveraging omnichannel communications, including community-based digital platforms and social media, can enhance communication efforts, allowing for real-time interaction with healthcare providers and patients. These platforms provide an avenue for sharing the latest research findings, treatment protocols, and patient testimonials, thereby building trust and credibility in the market.

What's next?

The oncology market presents both significant opportunities and formidable challenges for pharma manufacturers. Robertson and Gupta's insights underscore the need for innovative solutions, strategic partnerships, and agile approaches to navigate the complexities of commercialisation. By leveraging expertise, advanced technologies, and customised strategies, manufacturers can overcome hurdles, optimise patient and provider engagement, and ensure the successful launch of lifesaving oncology therapies.
The future of oncology is bright, with continuous advancements promising improved patient outcomes and extended survival rates, including radiopharmaceuticals, cell and gene therapies, antibody drug conjugates, and biospecifics. However, realising this potential requires a concerted effort to address the multifaceted challenges of commercialisation. With the right strategies and support systems in place, manufacturers can navigate these challenges effectively, bringing new hope to patients battling cancer.

About the authors

Anne Marie Robertson
Executive vice president, EVERSANA Oncology Commercialisation

LinkedIn

Anne Marie Robertson is helping EVERSANA deliver value to stakeholders across the life sciences industry. Through her multi-decade career at both large and small pharmaceutical companies, she has become a proven biopharma leader driving cross-functional teams. Specifically, she has a record of successfully commercialising oncology therapies, such as Erbitux, Inlyta, Onivyde, and Akynzeo, as well as developing new indications for in-market therapies. Her ability to set a launch strategy and create science-based differentiation allows her to leverage marketing strategies for clients globally while establishing and meeting launch metrics and driving overall brand value.

Robertson has an MBA from Baruch College of the City University of New York and a Bachelor of Science in Biology from Rensselaer Polytechnic Institute. Early in her career, she was a US Peace Corps volunteer in Central Africa.

Maneesh Gupta
Senior partner, EVERSANA Consulting

Linkedin

Maneesh Gupta is the practice lead for launch and complete commercialisation with the EVERSANA Consulting team. He is focused on helping clients and partners succeed at launches strategically, operationally, and financially by leveraging experience gained across more than 100 launches over the last 25 years.

Gupta comes to EVERSANA management consulting from IQVIA. He held a number of leadership roles over his 10 years at the firm, serving as practice lead, launch excellence and brand and commercial strategy. In this role, he led the practice in the United States, focusing on helping clients optimise their strategy, planning, implementation, and tracking. Among his many accomplishments, he conceptualised and developed IQVIA’s patient-centricity/patient journey offering and was recognised with an Outstanding Achievement of the Year award by IQVIA’s US president and honoured by a client with its Consulting Partner of the Year award.

Gupta holds an MBA in business administration from The University of North Carolina at Chapel Hill and an MBA in marketing and supply chain management from UNC Kenan-Flagler Business School. He earned his Bachelor of Chemical Engineering from Jadavpur University in Calcutta, India.

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Insider insights: Vittoria CEO Nicholas Siciliano on the future of CAR-T

Nicholas Siciliano

Insider insights: Vittoria CEO Nicholas Siciliano on the future of CAR-T 

Unlocking the full potential of CAR-T cell therapies remains one of the most exciting and actively pursued frontiers in cancer research today. While the emergence of these innovative therapies has generated immense interest, there is a pressing need to push beyond the hype to confront the very real challenges limiting their efficacy and accessibility, driving continuous innovation to enhance these potentially curative treatments.

In this exclusive interview, Deep Dive editor Eloise McLennan sat down (virtually) with Vittoria Biotherapeutics' CEO and co-founder, Nicholas Siciliano, to gain an insider's perspective on the state of the CAR-T field. Here, he shares his insights into the key challenges that must be addressed to propel this revolutionary modality forward into what he calls the “second inning” of CAR-T treatments. 

Eloise McLennan:

How did you get into the field of cell therapy?

cell therapy
Nicholas Siciliano:
For the last 15 years or so, I've had a front-row seat to all the advances in cell and gene therapy and the technologies being translated at Penn [University of Pennsylvania, Philadelphia]. It's been really exciting. 

I took a non-traditional path out of my doctoral degree. I co-founded a molecular diagnostic company in the greater Philadelphia area called Invisible Sentinel. It was with technology that I actually invented. We didn't spin it out of a university. Because of that, we had to take a “non-traditional” financing route. Long story short, [we] got it funded by a few high-net-worth individuals back in the 2008 market downturn, who thought it was less risky to invest in two first-time entrepreneurs than leave their money in the stock markets.

That was a great experience. It taught me a lot. It also plugged me into the network here in Philadelphia, of entrepreneurs and investors. On my travels, I met an individual by the name of Bruce Peacock – one of the premier life sciences executives, arguably in the country. He and I partnered about six or seven years ago on forming NewCos out of the greater Philadelphia ecosystem. It was through that endeavour that we came across the underlying technology for Vittoria.

Could you give us a brief overview of where we are at the moment in terms of CAR-T cell therapies?

baseball glove and bat
We're in the second inning of what I believe is a game that's going to go well into extra innings. With the initial translational work done at Penn with what is now Kymriah, the excitement level was really high. Then again, it took some time to really dig in and understand, how can we apply this to other indications? How can we make it better for B-cell malignancies? Then there was a lot of early excitement around allogeneic therapies. 

Unfortunately, some of the initial clinical results were not what everyone had aspired for. That's an interesting dynamic because I believe there's a place for both autologous and allogeneic therapies in the future of this modality. What I'm seeing now is this emergence of a third class that I like to term, “enabled autologous”. 

You may never get the same type of curative potential from an allogeneic that you would with autologous, but again, with autologous, there are still many drawbacks with logistics, and challenges. I think enabled autologous is trying to bridge that gap.

What are the most significant challenges currently facing CAR-T cell therapies in terms of efficacy and patient outcomes?

T cells visual
One of the challenges with the first-generation products is that we see complete responses in 30% to 40% of the patients. It'd be great to double that number. Obviously, we'd aspire for 100%. Increasing effectiveness is always top of mind.

Some drivers around that current shortfall are: Do the CAR-Ts expand for every patient? How can we confer enhanced proliferative properties? One of the strongest correlates so far is that early expansion of the CAR-Ts in the patient is important to achieve a complete response.

The second is the durability of the CAR-Ts. Often, what you see are the partial responses and then the patients relapse. Often, that's due to the T-cells becoming exhausted. Anything we can do to make them more resistant to exhaustion and have them be active longer in the patient will be important to increase that. Then, on the other side, there's the manufacturing and logistics piece, but I'll pause there to see if there's anything more you want to talk about therapeutically.

The high relapse rate is a significant concern in CAR-T therapies. What are the primary factors contributing to this issue, and what advancements are being made to address it?

time
One of the challenges with targeting a pan antigen – whether it be CD19 for B-cells or BCMA – is antigen escape. One of the things that drives that escape in general with cancer is selective pressure and time. Therapies that do a better job clearing tumours sooner rather than later, such as getting more exhaustive clearance early on, will help prevent things like antigen escape. 

There are unrealised checkpoint pathways. One that we work on at Vittoria is the CD5 signalling pathway, which can potentially unlock or harness the patient's natural immune response to the tumours.

That might even be a new approach that you see. We see some of that with engineered TIL [tumour-infiltrating lymphocyte] therapies now, but I think we can apply it to engineered T-cell therapies like CAR-T and TCR-T.

The high cost of CAR-T cell therapies is a significant barrier to widespread adoption. What steps are being taken within the industry to reduce these costs?

manufacturing
Some of that's going to come just generally with economies of scale as this becomes more of a common modality. The cost of the drug substances needed to make these products, the cost of the associated labour, all of that will benefit from some economy of scale. The big needle mover will be the automation of these processes, particularly autologous processes. 

By trimming days off the ex vivo manufacturing process, you do a few things. One is you reduce the labour costs, you reduce the time required in a GMP clean room facility. But interestingly, shorter manufacturing times for autologous cell therapies also confer a functional benefit to the resulting drug product. It actually makes the drug product more potent.

The less time a patient's cells spend outside of their body and in an artificial and external environment, the less exhausted they become and the better they do. By shortening the ex vivo manufacturing processes, you can also significantly lower the required dose in many cases – this is the case of Vittoria – because the expansion winds up happening in the patient, rather than in the manufacturing process. That's also important.

Beyond blood cancers, what new applications of CAR-T therapies are currently being explored by researchers and developers?

target
Some of the hottest indications or applications being talked about now are in immunology. Really exciting work is ongoing with lupus patients and other immunology applications, Graves' disease, for example, where B-cells are mediating the pathogenic effect.

At Vittoria, again, we've also taken an eye to that. Our second programme right now has a huge potential application in lupus. It also addresses one of the inherent safety issues with all of the first-generation B-cell-targeted CAR-Ts, in that it would specifically target the pathogenic B-cells and spare healthy B-cells in immunology applications.

In oncology, it would specifically target the malignant cells and spare the patient's healthy B-cells. That, I think, is a really interesting dynamic and one of the, again, areas where we'll start to see improvement in the space – call it on-target, off-tumour issues.

 

What are the key regulatory challenges facing CAR-T therapies? How can industry work with regulators to try and overcome them without impacting what patients are able to receive?

researcher
You know what? I’d like to take this opportunity to pay a compliment to the FDA. They’ve made a significant effort to be thoughtful about some of these newer game-changing modalities like cell and gene therapy and build into what has been historically a very rigid infrastructure. 

Now, the reality is, as a drug developer and just drug developers in general, the agency will never move fast enough because we want to be able to advance these technologies at lightning speed. But, over the last couple of years, I’ve seen a real effort to try to create pathways for accelerated approvals. One of the big challenges you have in this space, just again inherently, is that the technology is advancing so quickly that it becomes dynamic to manage the regulations. 

We all take a lot of responsibility playing in this space right now because it’s going to be up to us to, one, get it right and, two, avoid major pitfalls that would, again, push the agency to become more restrictive.

You’ve called this era of cell therapy the “second inning”, what innovations or research initiatives are addressing current limitations to push us into the third inning?

cell concept
One of the inherent challenges is that there are just so many targets. There’s an opportunity to get much more, I’ll call it, 'surgical' with antigenic targets. I think you’re seeing some of that with the TCR-T therapies, too, looking at epitopes in the context of MHC receptors.

There’s also this concept of cell therapy unlocking the patient’s naturally occurring anti-tumour T-cells and immune cells to create additional effector populations of cells in the patient. That’s something that we’re working on at Vittoria that we’re really excited about. That could be a real game-changer.

Finally, what excites you most about the future of CAR-T therapies, and what potential advancements or breakthroughs do you foresee in the next few years?

breakthrough
I think in the next couple of years, we’re going to see this modality solve for some of the “holy grails” in the space, such as solid tumour treatments and metastatic tumours.

I think we are moving in the right direction to start to make some progress there. Again, some of that comes with armouring technologies or the work we do at Vittoria with our SENZA5 platform to be able to overcome resistance and immunosuppression. 

Breaking into the solid tumour paradigm – that’s probably the next big near-term milestone to fall.

 

About the author

Eloise McLennan is the editor for pharmaphorum’s Deep Dive magazine. She has been a journalist and editor in the healthcare field for more than five years and has worked at several leading publications in the UK.

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Bridging the gap: Mitigating disparities in prostate cancer outcomes

Bridging the gap: Mitigating disparities in prostate cancer outcomes

While overall cancer survival rates have increased over the past several decades, the widespread impact of progress in cancer research and treatment is hampered by persistent disparities in time to diagnosis, access to care, and health outcomes. This sobering fact is particularly evident in prostate cancer. 

The disease is the second most common cancer diagnosis and cause of cancer death in men, accounting for 29% of new cancer diagnoses each year, but mortality rates vary substantially by race and socioeconomic status. A complex interplay of biological, environmental, and social factors drives these disparities, making it anything but simple to develop solutions. However, improving patient access to simple, efficient, and specific screening methods is a foundational step in bridging the gaps in prostate cancer outcomes. 

Notable disparities in prostate cancer incidence & outcomes

Racial differences in prostate cancer represent the largest cancer health disparity in the United States. Despite a lower incidence of cancer overall, prostate cancer mortality rates among Black men are roughly double that of all other races. Black men diagnosed with prostate cancer also tend to be younger and are more likely to have more advanced cancer at the time of initial biopsy. These disparities are influenced by a complex interplay of social, environmental, and biological variables. Evidence suggests that biological factors, such as epigenetic alterations and comorbidities, may play a role in driving racial disparities in prostate cancer. However, because Black men are underrepresented in prostate cancer clinical trials relative to their disease incidence, clinical research may fail to capture key data on differential health outcomes in the population.

Socioeconomic status (SES) and other social determinants of health are also significant sources of disparity in prostate cancer diagnosis and treatment. While prostate cancer diagnosis rates are positively associated with socioeconomic status, lower SES tends to correlate with worse outcomes. Men with lower SES tend to be diagnosed at later stages of the disease, are more likely to have metastases at the time of diagnosis, and have decreased survival rates. Overcoming these disparities requires a multifaceted approach that includes increasing awareness and education about prostate cancer risk, addressing social determinants of health, and, perhaps most critically, making screening and treatment more accessible and affordable for all patients. 

 

Challenges and limitations in prostate cancer screening

The timing of prostate cancer diagnosis can have a significant impact on patient outcomes. Cancers detected in early stages may be addressed with less invasive treatment approaches and are often associated with improved survival rates; prostate cancer detected prior to metastasis has a five-year survival rate of nearly 100%. Racial and socioeconomic disparities are evident in the timing of diagnosis, as both Black men and men from lower socioeconomic backgrounds are more likely to be diagnosed with advanced disease. 

Early cancer detection requires effective, accessible, and actionable screening approaches. The prostate-specific antigen (PSA) test is the current standard for prostate cancer screening. Elevated levels of PSA in the blood are indicative of prostate disease, warranting follow-up testing, such as a biopsy, to establish a cancer diagnosis. However, the PSA test is not a perfect cancer screening tool. Because an elevated PSA result can be caused by non-cancerous conditions, such as an enlarged prostate, prostatitis, or even riding a bicycle, high PSA can essentially be a false positive that results in further unnecessary testing. Men with an elevated PSA level have a roughly 1 in 4 chance of having prostate cancer, but not all cases constitute high-grade disease that warrants intervention. 

The limitations of PSA testing are further exacerbated by the discomfort and risk associated with prostate biopsy. In addition to being stressful and unpleasant for most patients, the procedure can result in infection and even life-threatening sepsis. When screening methods fail to yield clear results and leave patients to face unnecessary follow-up testing, there is little incentive to actively pursue screening. This only widens the disparities in timely prostate cancer diagnosis associated with distrust, embarrassment, financial strain, and limited access to healthcare. 

Overcoming disparities with accessible & accurate screening methods

While public health research provides clear evidence of the need for increased education and communication in communities disproportionately affected by prostate cancer, overcoming disparities in disease burden will require concrete steps to increase access to timely diagnosis and treatment. Reimagining our approach to prostate cancer screening is a key first step. According to new guidelines published by the Prostate Cancer Foundation, Black men are now encouraged to consider baseline PSA testing between the ages of 40 and 45, rather than the established recommendation of ages 55 to 69, followed by screening at regular intervals until age 70. However, if current methods are inconclusive, requiring inconvenient, unpleasant, and often unnecessary follow-up testing to yield actionable results, is it realistic to expect patients to assume the personal and financial burden of regular screening? 

Mitigating prostate cancer disparities through earlier detection will require affordable and accurate testing that provides specific, actionable results and is easy to implement in virtually any community healthcare setting. Novel diagnostic technologies have largely shifted to a focus on biomarkers at the level of the genome or transcriptome, but the data gleaned from these approaches represents molecular instructions that may not directly translate into protein- or disease-relevant alterations. While their insights can be valuable for guiding the selection of molecularly targeted therapies, the relatively high cost and complex interpretation of RNA- and DNA-focused diagnostics limits their applicability as screening tools. 

Alternatively, by turning to simpler, more cost-effective methods that emphasise efficiency and accuracy, we can create screening tools that overcome the limitations of PSA testing while broadening patient access. Innovative approaches, such as tools that assess alterations in PSA structure, rather than concentration, can help identify patients at greatest risk for prostate cancer without requiring specialised equipment or advanced interpretation. More specific insights can, in turn, reduce the number of unnecessary biopsies and mitigate the stress and costs associated with excessive diagnostic procedures.

Pursuing improved prostate cancer outcomes for all patients

Decades of research have built a deeper understanding of cancer than ever before, enabling scientists and clinicians to identify valuable biomarkers, develop targeted therapies, and improve outcomes. Despite this progress, significant disparities remain in prostate cancer incidence and outcomes across race and socioeconomic status. While completely overcoming inequities in diagnosis and care will require significant social and financial investment, creating accessible, accurate, and patient-friendly methods for early prostate cancer screening will be a foundational contribution in closing these gaps.

About the author

Dr Arnon Chait is the chief executive officer of Cleveland Diagnostics. An entrepreneur, scientist, and educator, Dr Chait’s extensive multidisciplinary background spans physics, engineering, and bioscience. As CEO, he applies his vast experience commercialising innovative science to drive Cleveland Diagnostics’ vision of bringing early cancer detection into everyday practice with advanced testing that is more simple, accurate, and cost-effective than current options.

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Heard on the pipeline: ASCO 2024

Heard on the pipeline: ASCO 2024

Every year, the American Society of Clinical Oncology (ASCO) annual meeting brings together a worldwide community of innovators dedicated to advancing the frontiers of cancer research.

Amidst a wealth of knowledge and experience, pharmaphorum editor-in-chief, Jonah Comstock, was presented with a profound opportunity on the ASCO floor: the passing of the torch to the next generation of oncology experts.

As seasoned professionals reflect on their journeys, they share invaluable insights, lighting the way for those ready to carry the baton in the fight against cancer.

“What advice would you give to a young professional entering the field of oncology today?”

Warner Biddle

Head of commercial affairs, KITE, a Gilead Company

This is a conversation that I’m actually having with my daughter. She's in second year right now at STSU taking a biology and chemistry degree and she's very interested in coming into the field.

And so we're having this conversation right now about what we do in this industry and all the passion that we have, as well as the terrific science. And to your point, just the incredible progress that we've made, but also how much potential there's still left in the oncology field.

So I think there's a lot of motivation, a lot of hunger and desire from a lot of these young scientists to come forward and make a career in oncology. And we do need more support and more help to treat these patients and bring these transformative therapies through the clinical development programme so that we can get them to patients.

Watch the full interview with Warner Biddle here.

 

Andree Amelsberg

SVP global and US oncology medical affairs, Eisai

What is exciting in oncology is when I think back ten years ago, 20 years ago, 30 years ago and you compare how we can treat patients today with more effective and safer drugs. That's a tremendous progress that we as the medical community, the manufacturers, and others have made on really making an impact and making life better for our patients.

And I think that is what I would tell a younger person entering the oncology space right now: it might be hard work right now, but there's continuous progress over time.

Watch the full interview with Andree Amelsberg here.

Carolyn Sousa

Commercial lead, solid tumours, Johnson & Johnson

Oncologists must remember the patient behind the treatment that they're providing, and that the treatment is much more than just the drug. They need help from a dietary standpoint, they need help with physical therapy, with mental health. 

It's really important that they not only stay curious, but also work collaboratively with all of the different allied health professionals that can help make patients' experience of treatment as good as it possibly can be.

Watch the full interview with Carolyn Sousa here.

Paul Burton

Chief medical officer, Amgen

Get strongly scientifically trained. Don't be in a rush to move quickly through a career pathway. Really deeply learn the science, really deeply learn the clinical medicine that you want to go and treat. And the other thing I would say is keep patients always at the centre of everything you do.

This is a huge meeting. There's so much going on here for all of us. The North Star is to help patients live longer, better lives and to help them and their families; that's fundamental to what young physicians should do today.

Watch the full interview with Paul Burton here.

Chris Haqq

Chief medical officer, Elicio Biosciences

The answer that I would give is to not trust only what you see in mice. Of course, we have to look to the animal models to give us the confidence that we need to take an approach now into human patients.

But I always tell my colleagues that they will be the ones who tell me if I gave them the right project or not, because it's the human data that is really the most important. And I think it's very important to remember that mice and humans have some differences and that there can be big surprises.

I've seen before in my career that drugs that looked a little bit too toxic in mice, ended up being quite effective and well tolerated in the human trials, for example, abiraterone, which I worked on in the past. So there's a lot to be said for doing the translational work to take that first step in the clinic.

And I would encourage everyone to take molecules forward that have a sound scientific hypothesis and to ask the human patient experience to tell us the best value and the best place to use those therapies.

It's a great area with a lot of need. It's a great career, really satisfying. So I'd encourage every young investigator to get involved in clinical trials. They're really important to inform us about how we can move the whole field forward.

Michael Petroutsas

US president and head of commercial, Astellas Pharma

I would say be curious. A lot of times we get into oncology because we want to help serve and support patients, but also we fall in love with our programmes. You work in your organisation, I work in mine, and we fall in love with what we have and what we're creating. 

But come to ASCO and be curious. See the possibility. I met with a physician earlier today and we talked about the complexities of targeted therapy versus chemo and how do you get to a place where you no longer have to use chemo? And I really turned back and said to myself, look at all the biases I bring to think, is that even possible? Because it's been the standard of care for years. And it just reminded me, you have to be curious.

You have to come to ASCO, listen to the science, be curious, and learn to see how we cannot individually help patients, but only collectively. And that's where great partnerships come into play.

Watch the full interview with Michael Petroutsas here.

Thanos Zomas

Global medical lead for Lymphoma, Takeda

I think there couldn't be a better timing for someone to enter the oncology field. And this is because there's so much hope, so many different treatment solutions and options.

People like myself or even older than me, when we were getting into oncology, we had to face a lot of challenges, a lot of disappointments. And honestly, we had to experience dramatic stories with specific patients. This is not so much the case.

Now we are able to deliver much more curable, efficient, tolerable treatments. And we can see in front of us this hope blooming. So, for example, we can cure more than 90% of childhood leukaemia. Now, that’s a dramatic change. We have a large number of physicians who have been treated for acute leukaemia or even Hodgkin's lymphoma in their teenage years or their childhood.

So this hope is going to continue and I think the new generation of oncologists will be in the best place to serve patients in with cancer now.

Watch the full interview with Thanos Zomas here.

About the authors

Eloise McLennan is the editor for pharmaphorum’s Deep Dive magazine. She has been a journalist and editor in the healthcare field for more than five years and has worked at several leading publications in the UK.

Jonah Comstock is a veteran health tech and digital health reporter. In addition to covering the industry for nearly a decade through articles and podcasts, he is also an oft-seen face at digital health events and on digital health Twitter.

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How AI and predictive analytics are set to transform patient care

How AI and predictive analytics are set to transform patient care

The healthcare industry is on the cusp of change, with artificial intelligence (AI) and predictive analytics paving the way for unprecedented advancements in patient care and operational efficiency. This evolution is being driven by the burgeoning amount of healthcare data that is being collected and made available through electronic patient records (EPRs), alongside the urgent need for systems that can analyse and leverage this information in order to improve patient outcomes and resource management.

In the past, clinicians primarily used healthcare data as a decision support resource to pull in basic information on the number of medication errors that had occurred over a given time period, for example, and to use that basic intelligence to shape the clinical choices that were made. 

Today, the technological capabilities available to healthcare teams have ramped up several levels. No longer just theoretical concepts in a healthcare context, AI and predictive analytics are now proving themselves to be critically important in tackling some of the most pressing healthcare challenges. From reducing medication errors through advanced decision support systems to predicting antibiotic resistance with sophisticated AI algorithms, these technologies are setting new standards for care quality and safety. 

Moreover, their application in public health surveillance and chronic disease management is showing promise in pre-empting health crises, and improving long-term health management.

The benefits of these technologies are increasingly tangible. For example, initiatives that bring together vast volumes of patient data across different healthcare providers have demonstrated significant improvements in operational efficiency, and patient engagement. Equally, they have proven to be beneficial in terms of healthcare coordination. Insights about a particular patient can be made available quickly to help inform a 360-degree treatment plan for the patient, with care and social workers, dietitians, GPs, and hospital doctors all coming to together to discuss the evolution of the plan.

Analytics can also play a key role in patient surveillance, helping to ensure that a patient diagnosed with hypertension, for example, is taking their medication as prescribed, and rapidly analysing the blood pressure readings they take at home to ensure that no dangerous patterns are emerging.  

AI in oncology: Enhancing care across the board

No area is perhaps more illustrative of these multiple benefits than oncology, the branch of medicine that deals with the prevention, diagnosis, and treatment of cancer.

AI and predictive analytics are crucial in oncology due to their ability to manage and address the vast complexity and variability inherent in cancer treatment and tumour biology.

Enhancing diagnostic precision

The enhancement of diagnostic precision is a standout application of AI in oncology. AI-powered machine learning models are adept at parsing complex imaging data, identifying subtle patterns that are beyond human visual detection, and pinpointing early signs of malignancy. 

This has notably improved the accuracy of diagnostic procedures, such as mammograms in breast cancer screening, significantly reducing both false positive and false negative results. Early detection facilitated by AI not only increases the likelihood of successful treatment, but also substantially improves the survival rates among cancer patients.

Tailored treatment with predictive insights 

One of the primary benefits of AI in oncology is its ability to analyse large volumes of data quickly and with high precision. EPRs, which contain detailed patient histories, treatment outcomes, and genetic information, produce an extensive dataset that, when analysed on its own or through AI algorithms, can reveal patterns and correlations that may not be visible to human analysts. This capacity for deep data analysis helps in understanding the unique progression of cancer in each patient and forecasting their response to different therapeutic strategies.

Tailored

Beyond diagnostics, AI can help in identifying the most effective treatment plans based on historical data from similar cases. This is especially beneficial in oncology, where the effectiveness of treatments can vary widely among patients due to the genetic differences in tumours. By predicting which treatments are likely to be most effective for a specific patient, AI not only enhances the likelihood of successful outcomes, but also minimises the risk and severity of potential side effects.

Furthermore, predictive analytics can anticipate patient responses to certain medications or treatment regimes, based on their genetic makeup, lifestyle, and previous health records, which allows for adjustments to be made before adverse reactions occur.

Improving operational efficiency

AI also boosts operational efficiency in oncology departments. By optimising scheduling and resource allocation through predictive analytics, hospitals can ensure that their operations run more smoothly, reducing patient wait times and increasing satisfaction. This is particularly key with cancer care, where patients will frequently visit multiple different healthcare settings for their treatment; from oncology management to different chemotherapy and radiotherapy sessions that they need to carry out.  

Moreover, AI enables continuous monitoring of oncology patients, providing healthcare professionals with real-time updates on patient conditions. This prompt response capability is particularly critical for oncology patients, who may experience rapid changes in health status.

Learning from clinical trials 

Another notable application of AI in oncology involves clinical research and trials. AI models expedite the identification of suitable clinical trial candidates and monitor patient responses during trials, allowing for quicker adaptations and potentially speeding up the approval of new cancer treatments. Additionally, AI systems help manage vast amounts of research data, aiding in the discovery of new oncological treatments and therapies.

Expanding the horizon – The future of AI in oncology

The potential of AI and predictive analytics extends beyond current applications, promising to bring about even more sophisticated tools for diagnosis, treatment planning, and patient management in oncology. These tools are expected to increasingly aid in the detection of early-stage cancers, predict disease progression, and personalise therapy to an unprecedented degree.

Tailored

The integration of AI and predictive analytics into oncology represents a major leap forward in the quest for precision medicine. These technologies offer the promise of significantly improving how cancer care is delivered by enabling more accurate diagnoses, personalised treatments, and efficient resource management. 

As healthcare continues to evolve, the role of AI and predictive analytics will only grow, underscoring the need for ongoing investment in these technologies. In light of these ongoing developments, the future of oncology care looks increasingly positive, promising better outcomes for patients worldwide. Through such advancements, the medical community is set to transform the landscape of cancer treatment, making it more effective, efficient, and patient-centred.

About the author

Rami Riman is director of clinical and business improvements at InterSystems. Prior to joining the company, he gained a decade of clinical experience across multiple hospitals in the US, UK, Lebanon, and UAE. 

Riman’s passion remains in increasing patient engagement to improve community’s health and awareness. He holds a BA from the American University of Beirut in Biology, a Doctorate in Medicine from St. George’s University School of Medicine and a Master’s degree in infectious diseases from the University of London. He is also board certified in Internal Medicine and Medical Oncology.

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Inside the open access movement: Unleashing the potential of oncology research

Inside the open access movement: Unleashing the potential of oncology research

In the hallowed halls of scientific publishing, a revolution is quietly unfolding – one that, if delivered upon, promises to fundamentally reshape the landscape of clinical research. At its core lies a simple yet profound idea: the practise of exchanging knowledge and ideas, building upon discoveries of the past, and sharing further findings with the wider community, should be freely accessible and unencumbered by the cultural and financial barriers that have long impeded its flow. This is the essence of the open access movement.

The origins of open access can be traced back to the late 1990s, when the Internet was rapidly becoming the great democratiser of information. As researchers grappled with the transformative potential of this new and exciting publishing medium, a growing chorus of voices began to question the traditional model of subscription-based access to scientific literature. Why, they wondered, should the dissemination of knowledge be constrained by paywalls and restricted access when the marginal cost of sharing it with an ever-growing audience was negligible?

“In an era of print, journals were printed and distributed to people who were interested. Each time you added a reader, you added cost, so you charged them to subscribe to the journal,” explains Theodora Bloom, an executive editor for The British Medical Journal (BMJ). "Once journals are online, the cost is mainly associated with producing the article and getting it online. The additional cost of having more readers is almost nil. There is an opportunity to allow everyone to read, research, and use it and to put the charges where the costs are incurred, which is at the point of producing the articles.”

The answer, or so it seemed, lay in a radical reimagining of publishing models – where the costs of publication would be borne upfront, with the resulting research made freely available to all.

The promise and challenges of open access in oncology

While the idea of a paper being open or closed may seem relatively black and white, there are actually multiple different tiers of open access publishing, each offering varying levels of accessibility and reuse rights. 

Gold open access represents the most comprehensive form, where articles are immediately and freely available upon publication, often under a Creative Commons license that allows liberal reuse and distribution. Other options include green tier open access where authors self-archive their work in institutional or subject-specific repositories, sometimes after an embargo period set by the publisher, and hybrid tier, where subscription-based journals offer authors the option to make individual articles openly accessible for a fee. Lastly, diamond or platinum open access describes journals that publish open access content without charging authors or readers, typically funded by institutions or consortia.

“Subscription access to journals has usually been managed by libraries, the large institutional systems for payment, whereas, if you're collecting an individual article processing charge for each article that's published, that's a transaction the author has to get involved in,” says Bloom. “Increasingly, institutions are taking out big agreements that cover open access fees, but, for some authors, finding out whether your grant covers that payment and handling the payment, that's just a logistical challenge. In an area like oncology, research is very largely funded, people are being given grants and other means of payment to do the research.”

The benefits of this approach are manifold, particularly in fields like oncology, where timely access to the latest research is crucial. By embracing open access, researchers can not only accelerate the pace of discovery, but also foster unprecedented levels of collaboration and knowledge-sharing, transcending geographical and institutional boundaries.

"Open access is not just about reading research for free," says Bloom. "It's about the ability to download, text mine, and use computational methods that are becoming increasingly important. The idea is that you pay for the publishing upfront, and then you have a fully reusable resource in perpetuity.

Yet, despite its promise, the journey towards open access in oncology research has not been without its challenges. Several significant hurdles stand in the way of widespread adoption. The deeply ingrained culture of prestige associated with publishing in traditional, closed-access journals can be difficult to dislodge, even in the face of compelling arguments for openness. Transitioning an industry that has operated under a subscription model for centuries requires careful navigation of funding mechanisms, payment models, and author fees.

And then there is the issue of content.

Addressing reproducibility issues in cancer research

One of the most compelling arguments for open access in oncology research is its potential to address the ongoing reproducibility crisis in science. A substantial proportion of published work cannot be independently validated or replicated, a problem that has significant implications for the progress of cancer research and the development of new treatments.

Tim Errington, senior director of research at the Center for Open Science, found this out the hard way during his time working on the Reproducibility Project: Cancer Biology, an eight-year effort to replicate experiments from high-impact cancer biology papers published between 2010 and 2012. 

Errington and his team were tasked with preparing replications of 193 experiments from 53 papers, a seemingly straightforward process - or so he thought. 

“It was really, really hard. There were a lot of challenges, more even than I was prepared for,” he says. 

Only 2% of the designated 193 experiments had open data. Zero contained completely described protocols. Looking to navigate the missing information challenge, the team reached out to the authors behind the original papers. Some (41%) of those original authors were more than happy to assist, others (32%) were not helpful (or unresponsive). But even with additional information, the results highlighted a real issue in reproducibility.

“Of the findings that we could replicate, that the average effect size – so the mean difference – was 85% smaller than the original,” he explains. “That's like an animal study showing a 20-day difference in survival in the original finding from placebo to some intervention, which is pretty standard, to us finding the same effect, but it only is three days.”

So, how does open access help to address this issue? For Bloom, there is one clear advantage.

“Once papers are open, you get more and more people reading them and trying to reuse them and saying, ‘Wait a minute, I haven't got all the information I need, all the underlying data to be able to build on this study’,” says Bloom. “In a way, papers have become more bloated, they have more figures and tables and supplementary files, but still not necessarily providing good access for reuse by other people.”

Of course, as Errington and his team found, being able to access the research paper through open access publishing is just one step in the journey towards open science.

“Open access is crucial, but it's not enough,” he says. “You need to be able to expose the rest of that process downstream, which this project illustrated. It's not enough just to publish a paper. You have to give us access to everything behind that paper. Open access is the first step, which is, 'Can I see the paper?', without having to have a paywall or institutional access, which is essentially somebody paying for it. I still need more past that.”

Final thoughts 

The ethical implications of open access in oncology research cannot be overstated. By promoting data transparency, enabling replication studies, and facilitating collaborative efforts, open access principles align with the ethical imperative of ensuring that cancer research, which directly impacts human lives, is held to the highest standards of reproducibility and scientific integrity. This transparency can foster a culture of accountability and rigorous peer review, reducing duplication of efforts, and accelerating the pace of discovery in oncology.

As we look to the future, the imperative for open access in oncology research only grows stronger. In a world where knowledge is power, and where the stakes are quite literally life and death, the choice seems clear: to embrace openness, to tear down the barriers that impede progress, and to unleash the full potential of human ingenuity in the fight against cancer.

As Bloom eloquently reminds us: "We don't need to argue any longer about whether it's a good idea to share the results with everyone. That case has been made. We simply have to make it happen." And happen it must, for the sake of patients, researchers, and the boundless frontiers of medical knowledge that are waiting to be explored.

About the author

Eloise McLennan is the editor for pharmaphorum’s Deep Dive magazine. She has been a journalist and editor in the healthcare field for more than five years and has worked at several leading publications in the UK.

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