17 September 2015
A new publication from a European biopharmaceutical body has set out key challenges to be overcome in the path towards personalised medicine, saying that a paradigm shift is necessary if the promises of personalised medicine are to be realised.
European Biopharmaceutical Enterprises (EBE) is a specialised group within the European Federation of Pharmaceutical Industries and Associations (EFPIA), established in 2000. The new EBE White Paper on Personalised Medicine calls for a number of measures to speed up delivery of patient benefits, whilst emphasising that many of these ‘do not require a change in the law, so much as a change in mind-set’.
The need for collaboration
In addressing the monumental challenge of creating a new understanding of the molecular basis of disease, the paper calls for ongoing research funding and an international, cross-sector collaboration, as well as the incorporation of personalised medicine into education for healthcare professionals, patients and the wider public.
The rapidly expanding knowledge associated with the advance of personalised medicine is noted to necessitate new infrastructure and training in order to deliver the biobanks, e-health records and databases needed, and bioinformatics tools to use these crucial resources, as well as standardisation of protocols and standards. The paper calls for urgent development of data privacy and protection laws that ‘effectively protect patients while at the same time making pan-European research initiatives possible’ as well as consistent approaches to consent procedures to facilitate pan-European data sharing.
The need for improved European regulation to support better, faster clinical trials is also noted, in particular the imperative for greater flexibility in trial design. Adaptive licencing pathway approaches are advocated as being particularly suited to personalised medicine; it is suggested that consistent datasets and interoperable e-health record systems would support this. The report also calls for new incentives for the development of new personalised medicines.
Focusing more specifically on the development of novel diagnostics, including companion diagnostics and clinical decision support systems, the white paper calls for a similarly flexible and adaptive regulatory framework to ensure consistency of performance, and for ‘efficient co-ordination’ between the European Medicines Agency (EMA) and ‘future diagnostics competent bodies’ for co-development of new therapeutics and diagnostics.
A flexible approach
Overall, the white paper calls for prompt action in the various areas considered in order to deliver speedy patient benefits. EBE Personalised Medicine Working Group Chair Tom Lillie of company Amgen observed: “we need more flexibility in the way we invent, develop, approve, prescribe, reimburse and use medicines. This demands an unprecedented level of collaboration of everyone involved“.
Transformation of health services: what will it take?
The new EBE report takes a lead in looking ahead and anticipating key challenges for personalised medicine and their potential solutions. The pharmaceutical and biotechnology industries do of course have clearly vested interests in the effective development and delivery of personalised medicine. However, this does not invalidate their perspectives on how it can be achieved; their voice in this issue is as important as that of health professionals, patients, policy makers, regulators and the many others involved.
The realisation that achieving a genuine transformation of healthcare on the basis of genomic research is going to take more than research, however ground-breaking, is an important one. The scale of the challenge posed by implementation of personalised medicine in the English National Health Service (NHS) at least has been recognised to the tune of £300 million government funding for the 100,000 Genomes Project, which aims to lay the groundwork for embedding genomic medicine within the health service.
The need for anticipatory action
The paper also notes that technologies ‘that may have significant budget impact or other challenges to implementation’ are a particular priority for action, allowing health care systems in Europe to adapt in anticipation of their introduction. This is a factor that is all too frequently overlooked, perhaps because in the UK at least there is limited oversight of emerging and potentially transformational technologies for healthcare. Incentives for basic and translational research exist, and are growing as their critical importance gains widespread recognition.
However, in general terms there is no body charged with spotting potentially disruptive or transformational healthcare technologies on the horizon, nor in performing the work needed to anticipate their potential impact on clinical services and creating a receptive environment for their prompt adoption. It seems incredible that policy development work of the kind so often provided by the PHG Foundation should be the preserve of an independent, charitable organisation – and that there are no supporting structures or incentives for health professionals to play their vital role in this process.
Investing in active uptake
The new governmental Accelerated Access Review offers a new opportunity to tackle the problem, aiming to speed up access to transformative health technology (including diagnostics). Efforts to embed ‘adoption and diffusion’ of innovations in the NHS is one area of focus; this is promising, but danger lies in any belief that this is a passive and naturally occurring process. The transition from bench to bedside is now recognised as a crucial one, but similar attention is needed to move innovations from use only by early adopters and enthusiasts, to equitable national access via any relevant health professional.
The recent PHG Foundation response to the interim consultation questions in the process underlined the need to ‘bridge the gap between extremely complex science and policy-makers, clinicians and commissioners – to explain the potential of the innovation, but also the supporting mechanisms that would be required for health service implementation (such as the necessity for new standards or guidelines, complexity of regulation, or specific training)’. If we want to push personalised medicine from research into practice – and from small sub-groups to all relevant patients – then very small investments in these areas could yield significant returns.