Personalised medicines: challenges and opportunities
In our personalised medicine themed month, Duncan McHale discusses the barriers to adoption and the benefits of personalised medicine.
Personalised medicines have the potential to revolutionise the experience of healthcare for many patients and clinicians, and to dramatically improve treatment outcomes. However, current systems of regulation and reimbursement for medicines across Europe, combined with a traditionally conservative and change adverse clinical environment, could cause a lag between the approval and wide-spread adoption of these new medicines. It is important that healthcare and regulatory systems respond to keep pace with the new discoveries being made in order to ensure rapid adoption of truly innovative advances for patients and to enable appropriate incentives for pharmaceutical companies to develop medicines and accompanying diagnostics to deliver on these opportunities.
Our changing understanding about the development and mechanisms of diseases is revealing a new classification of disease and related treatment options. As the pharmaceutical industry progresses from the era of one size fits all 'blockbuster' therapies, and develops a greater understanding of the complexity and heterogeneity of disease, we have entered an exciting era where personalised medicines are starting to enable us to target therapies to subpopulations of individuals at risk of developing diseases before they have symptoms or sub-populations of patients most likely to respond to therapeutic interventions. This has the potential to result in better health due to more effective medicines, fewer side effects and greater cost efficiencies.
Take for example the field of oncology, a therapeutic area where personalised medicine is already making significant headway. Solid tumour types had, until relatively recently, been considered to be a single type of disease, classified according to the organ affected (breast, lung, bowel etc.), histological type, their size and whether they had spread beyond the presenting organ. As knowledge of cancer genetics has progressed, and the practice of genetic sequencing has become more accessible to researchers, our understanding about different types of cancer, their causes and the most effective way of treating them has, in many respects been revolutionised.
There are numerous other disease areas where better molecular profiling and targeted, personalised medicines, offers the potential to yield promising new therapeutic opportunities. Take neurodegenerative conditions, such as Alzheimer's and Parkinson's, for example. With an aging population and ballooning costs associated with providing care and treatment to this rapidly growing patient population, being able to identify early on, perhaps even before symptoms present, who will be affected by these conditions will allow the development of targeted treatments to prevent or modulate disease progression, rather than treating the symptoms of the condition once the disease presents. This approach has the potential to benefit a number of other chronic health conditions such as diabetes or epilepsy, for which treatment has historically been focused on 'symptom management' rather than targeting the underlying cause of the disease.
"This has the potential to result in better health due to more effective medicines, fewer side effects and greater cost efficiencies."
As pharmaceutical companies and researchers seek to deepen their knowledge about the molecular causes of a disease, to more accurately predict responses to particular therapies amongst patients, regulatory processes and reimbursement systems will need to adapt.
In order to accurately identify which patients could benefit from personalised medicines diagnostic tests must be developed early in the drug development process. In practice, it is often difficult to develop these tests until the phase II trials of a medicine as it is necessary to use patient response data to identify the biomarkers predicting response. The current FDA regulatory requirement for large scale, prospectively stratified studies before the approval of a diagnostic test, often creates a lag between the commercial availability of a new personalised medicine and an accessible method of diagnosing / identifying patients within clinical practice. Healthcare systems need to become more forward looking and start anticipating the issues this could cause in the development of personalised medicines as it disincentivises the innovator because of the additional costs involved and the lag time to gaining a return in investment. Regulatory, payer and clinical systems need to incentivise and encourage companies to develop, commercialise and deliver faster and more efficient diagnostics.
Reimbursement bodies and systems (such as NICE) may also need to rethink their approach to personalised medicines, and any accompanying diagnostics testing. These tests tend currently to be reimbursed at a rate related to their consumables, which does not take into account the R&D costs likely to be required in developing these innovative and technical measurements and more importantly the value they bring to the healthcare systems in terms of health and gained and overall healthcare costs reduced.
Furthermore, for personalised medicines to fulfil their true potential, the way in which diseases are currently classified needs to expand and deepen, becoming more diverse and based on molecular aetiology rather than phenotypic features. This will have the effect of making patient populations for each individually identified 'disease' much smaller, but will create much more homogenous disease groups with the potential of massively increasing the chances of identifying novel successful treatments. A reimbursement approach which focused on value rather than medicine cost, would recognise the higher efficacy of the medicines and the reduction in unnecessary treatment costs associated with non-personalised medicines. This approach could also offer pharmaceutical companies tangible incentives to further develop innovative personalised approaches to medicine even after launch. The system will also enable the development of medicines for rare diseases which can also suffer from a lack of ability to recoup a fair return on R&D investment.
Clinical attitudes also need to change if personalised medicines are to reach the patients who need them. Clinical conservatism and a lack of experience of using more complex tests can limit the implementation of diagnostics into practise. To address this support, information and training must be added to medical education so that doctors take on board a new and more detailed approach to classifying disease and mapping personalised treatment.
Delivering personalised medicines will bring a new age of prevention of ill health and medical intervention for serious diseases, but realising the potential requires a re-thinking of many of the frameworks that underpin our healthcare systems. Government and regulators must create the right incentives to drive forward and accelerate the innovation already happening in the pharmaceutical and diagnostics industries.
About the interviewee:
Duncan McHale is the Vice President of Global Exploratory Development at UCB.
Duncan has a long history in personalised medicines. He helped to set up the pharmacogenomics programme at Pfizer, where they orchestrated biomarker technologies to predict drug responses. He then headed up the clinical personalised medicines department at AstraZeneca for four years before moving to UCB. He is also the ABPI stratified medicines lead and was previously the EFPIA pharmacogenomics lead.
About UCB:
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 8,000 people in about 40 countries, the company generated revenue of EUR 3.2 billion in 2011. UCB is listed on Euronext Brussels (symbol: UCB).
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