Ensuring access: how to help patients seeking experimental treatments
James Shannon, Ronald Brus and Dennis Akkaya discuss the impact of the debate on the Right-to-Try laws in the US and what needs to be done to improve access to innovative drugs.
The debate around terminally ill patients' access to experimental treatments appears to be coming to a head in the US, with growing discussion about federalising the Right-to-Try (RTT) laws. The Senate has passed a version of the law, and the House has held its public hearing. A vote, up or down, is looming.
(left to right) James Shannon, Ronald Brus and Dennis Akkaya
The many constituents in the RTT discussion have attracted much media attention. Perhaps without their efforts, the pre-approval access issue may have remained subdued. However, rather than debating whether the FDA is the problem or the solution, it would be of greater benefit to focus on the patients, and address the issues with more pragmatism. Equipping the lawmakers with actionable solutions would help steer the discussion into the right direction.
The RTT campaign asserts, correctly, that the ultimate form of patient empowerment is the ability to access all possible treatment options. To achieve this in the most effective way would require a system that is impartial and independent, while at the same time satisfying all stakeholder demands within the current regulatory framework. Patients (and their advocates), as well as regulators, would be better served by considering improved ways to align interests, incentivise pharmaceutical and biotech companies to actively participate in pre-approval access programmes, and to reallocate resources to actually increase the number of patients treated in the safest way, while also enhancing the current clinical trial process.
The solution is a pre-approval access platform for medicines that serves physicians and their patients with unmet medical needs. Physicians and patients need to be (and are not currently) fully informed about the choices available and to have access to treatment options whenever they exist. We seek to collaborate with drug manufacturers, physicians and all relevant stakeholders, including regulatory authorities, to make development-stage drugs available for treatment without compromising essential safety oversight or clinical research principles during the process.
This initiative promotes enrolment in both pre-approval access protocols and randomised clinical trials, because ineligibility for the latter is a requirement to recommend the former. This system facilitates freedom of choice for the individual as well as accelerated access for the broader population through a marketing approval/drug development process that will be supplemented by valuable real-world data – a win-win result for all parties in this complex debate. This model also improves transparency, assures adequate oversight to protect patient safety, and embraces the essential role of the FDA in the 21st century.
Regardless of what anyone thinks about RTT, as new FDA Commissioner Scott Gottlieb testified before Congress, the neglected and more important issue here is getting companies to make their developmental drugs available for pre-approval access programmes. Congress should address this.
We have been facilitating pre-approval access overseas for the past three years, and our experience offers valuable lessons on its use and what can facilitate 'actual' access to investigational drugs for those facing unmet medical needs. In fact, our work suggests that the fundamental impediment keeping pre-approval access programmes from flourishing in the US is the lack of reimbursement by commercial and governmental payers for these important treatment efforts.
Being active in 17 countries across Europe and South America, we see that reimbursement, of both drug manufacturers and the providers who administer pre-approval access treatment, is one of the most important factors to 'grease the squeaky wheel' to encourage companies to participate in supplyingpre-approval access drugs to the patients who need them. While both federal and commercial payers in the US reflexively reject coverage as 'experimental', the fact is that these are treatments, even if they are end-of-life treatments, and should be treated as such. It should not be considered unreasonable to allow these companies, often considered 'innovators', and predominantly small in size, to seek an economic return in order to participate in a sustainable way. To keep pre-approval access from being only for a few, reimbursement needs to be built in to the system from the start. Similarly, treating physicians should be compensated for their time and effort when dealing with pre-approval medications. Why not learn from successful global examples, where incentives for drug companies, patients, payers, and health care providers co-exist and have created participation and enhanced access?
It is no secret that both physicians and pharmaceutical companies are facing an ever-more-arduous drug development ecosystem as even safe and effective drugs struggle to gain regulatory approval and reimbursement market access. All stakeholders suffer under this now unsustainable system. Inevitably a new paradigm must arise, most probably by increasing the significance of the phase 2 drug-development phase supplemented by real-world data. In addition, this should also become the basis for additional stakeholder value creation. Phase 2 is also the stage when most pre-approval access requests start. This creates a unique opportunity to address the current shortcomings of the drug development system by concurrently offering treatment options to more heterogeneous and excluded groups of patients who share the same problems, while not disrupting the definitive clinical trial process.
The polarised nature of the discussion over RTT does little to help address the real failings in the system. We believe it would be prudent, now, to acknowledge that the current drug development system has flaws and needs improvement. We have decided to stay neutral in the RTT vote/debate and continue to focus on helping physicians, patients and companies to find ways to increase the number of treatments available to those facing unmet medical needs. It is our strong belief that a critically important and enabling factor is creating clear incentives that will allow companies to participate in this process. Actual experience and common sense dictate that market place solutions will be needed.
Interestingly, the current administrations’ rhetoric seems to lean towards the idea of a better functioning ‘market place’ that would create more dynamism, competition and treatments for patients, while keeping a meaningful role for the FDA. We welcome many transparency and patient-friendly initiatives and applaud the determined trajectory that Gottlieb has chosen.
The RTT debate has inflamed passions and created uncertainty for those on all sides of the issue; but there is now also a unique opportunity to make real change in the system. We should regulate compassionate use in a truly more compassionate way by finding a more effective way to increase the number of patients who actually receive treatments. We need to acknowledge the fact that offering choice to patients will simultaneously require and facilitate the creation of value for all other stakeholders as well.
About the authors:
Dr James Shannon is trained in Medicine and Cardiology. He received his undergraduate and postgraduate degrees at Queen’s University of Belfast and is a Member of the Royal College of Physicians (UK).
He entered the pharmaceutical industry in 1987, joining Sterling Winthrop. Following the acquisition of Sterling in 1994 he joined Sandoz in Basel as head of Drug Regulatory Affairs. He held a number of significant positions in Novartis, rising to become Global Head Pharma Development in 2005.
He retired from Novartis in 2008 and subsequently served on the boards of various pharma companies until he joined GlaxoSmithKline in 2012 as Chief Medical Officer until retiring in April 2015.
He currently serves on the boards of Mannkind Corporation, Immodulon and Horizon Pharma.
Dr Ronald Brus is the Founder/CEO of myTomorrows, launched in 2013 to offer patients early access to medicines and to speed up procedures for the testing and approval of medicines.
With over 25 years of experience in biotechnology at Crucell (CEO) and Galapagos (co-founder and board member), Brus is committed to improving the bureaucracy after clinical trials.
He holds a medical degree from the University of Groningen (The Netherlands).
myTomorrows supports physicians and patients with high unmet medical need that are excluded from clinical trials, bringing access to innovative drugs in development.
Its internet-based platform collects and offers global early access programmes and ensures rapid deployment of knowledge, infrastructure and technology to optimise access for physicians and their patients, in addition to collecting real-world data.
Dennis Akkaya serves as corporate development at myTomorrows.
In his previous role at a life sciences boutique bank, he held various financial advisory roles. In his different advisory capacities for a wide range of biotech and pharma clients, he was responsible for handling IPOs and raising capital through various means of financing.
He holds a financial management degree from the Business University of Nyenrode.
