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European Biopharmaceutical Review

Tailored Medicine

The promise of personalised medicine (PM) is clear: safer, more effective medicines, with a compelling value proposition to payers. However, many hurdles remain before reaching these goals. Regulators are taking a proactive and evolving stance to PM, as evidenced by the recent proposed update to the European In Vitro Diagnostic (IVD) Directive from the European Medicines Agency (EMA). However there is a continuing need for clarity and transparency in some cases – for example, around laboratory-developed tests (LDTs) (in-house testing) and their oversight. From a fi nancial point of view, device companies frequently argue that diagnostics do not receive ‘value-based’ reimbursement, with little incentive therefore for innovation. In addition, pricing for targeted drugs generally, and biologics in particular, remains a topic of discussion.

Growing Push to PM

The age of PM has been here for some time now, and indeed the historical precedents stretch back at least two decades. Within the oncology industry, there are now an increasing number of therapies that require patient selection based on the use of a pathway-specific companion diagnostic prior to the initiation of that therapy. Vemurafenib with a BRAF V600E mutation test, and crizotinib with ALK FISH testing, are recent examples. Oncotype and Mammoprint also represent examples of tests which provide prognostic information, thereby facilitating decisions on when and how aggressively to treat early breast cancer. Other therapeutic areas – notably infectious disease – can boast of similar successes in identifying the right drug for the right patient.

Clearly, in order to fully deliver on the promise of PM, many different stakeholders will need to align around a common vision. As local and regional health authorities have ultimate responsibility for ensuring the safety and efficacy of new, innovative therapies for their citizens, regulators’ views of and expectations for PM – as delineated in published guidelines – remain a prime focus for the pharmaceutical and IVD industries. A number of health authorities, including the EMA, have published guidelines and reflection papers on many aspects of PM, usually preceded by a consultation period, with the overall aim of achieving a suitable (fit for purpose), robust, transparent and sustainable framework for regulatory approval.

Regulations and Guidelines

On 26th September 2012, the European Commission adopted a proposal for a Regulation of the European Parliament and of the Council on IVD medical devices which will replace the existing IVD Directive, adopted in June 2000 (1). The main drivers for the change were a need for simplification of the current Directive, an approach that could accommodate rapid technological advances, and a desire to converge to the extent possible on the Global Harmonisation Task Force for Medical Devices (GHTF) model, which influences, among other things, risk categorisation of devices and performance requirements.

Two important and impactful changes to the Directive are worth highlighting as both relate to the new classification system proposed. The first is the requirement on many manufacturers who hitherto were able to apply self certification, to now involve the review of a notified body. This is an important difference, yet a justified one on the grounds of safety. The second involves broadening the scope of the existing Directive to include LDTs or so-called in-house testing. Such devices are not covered by the Directive, as it states the “devices are manufactured and used only within the same health institution and on the premises in the immediate vicinity without having been transferred to another legal entity”.

Considering companion diagnostics in particular – an increasingly important class of diagnostic in the oncology area and elsewhere – in the existing directive, IVDs that are used as companion diagnostics are not listed in Annex II, and are therefore self-certified by the manufacturer. In the revised directive, companion diagnostics by default will fall within a classification requiring notified body review. Again, from a safety and efficacy perspective, this is eminently sensible and, based on feedback during public consultation, has overwhelming support from stakeholders.

Financial Implications

Increasing the monetary burdens on IVD manufacturers resulting from tighter and more encompassing regulation are easy to recognise. So too are the challenges to innovation in an environment where test reimbursement remains lower than many IVD manufacturers consider reasonable, given the perceived value that personalisation of healthcare can and does bring to society.

Unlike medical device regulation, there is no pan-European process for medical device reimbursement. Most countries use a system of diagnosis-related groups to set a price for a particular medical procedure, including any products that will be used in the specific procedure. However, the decision on which medical devices will ultimately qualify for reimbursement, and at what price, is defined by each local government healthcare policy.

A fundamental tenet of PM, especially for predictive tests, is that the value proposition to payers will be persuasive: the right group of patients receiving the right drug with clear, unequivocal benefit. In principle, this can and should justify a price of a medicine that reflects the innovation and expense of product development, which is currently estimated for an oncology biological product to be around €750 million (2). However is it justified? Ultimately, the answer to this question is societal. Payers, whether single and public (such as Canada or the UK) or multiple and private (for example, the US), or a blend of the two (as in France), cannot pay for every available health technology, and so rationing – implicit or explicit – is inevitable.

The total cost of prescription drugs (including any associated companion diagnostic test, typically a very small fraction of the cost of the drug itself ) as a proportion of total direct healthcare spend is estimated at between five per cent and nine per cent for most Western European countries (3). Yet this cost is highly visible and highly amenable to constraint, in ways that inpatient care costs are not.

Conclusion

Personalised medicine will continue to deliver better and more precise ways to tailor treatments in the coming years. The advent of next generation sequencing, and recent advances in informatics (without which understanding this tidal wave of genetic information becomes impossible), means that this process can only accelerate. Being able to show more persuasive benefit risk improvements, and a corresponding value proposition to payers, is arguably already a reality. The decision, however, on what constitutes ‘persuasive’ and ‘value’ remains the remit of society and its elected representatives. In the meantime, industry and academia will continue to strive to develop newer, better and more bespoke treatments.

References

1. Visit: http://ec.europa.eu/health/medical-devices/documents/revision/index_en.htm

2. Vernon et al, Health Econ, 2010

3. Khayat, Cancer, 2011


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Vincent O'Neill is a UK trained Medical Oncologist with over 10 years' industry experience. He received his MD from the University of Glasgow, UK, where he was a clinical lecturer, and trained at the Beatson Oncology Centre. His drug development experience spans Phase 1 to Phase 4, with a particular interest in personalised medicine and novel clinical trial design. He has worked in Europe, the Far East and the US, for a number of global pharmaceutical companies, and currently heads the Personalised Medicine and Companion Diagnostics group at Sanofi.

Debasish Roychowdhury received his medical training and Doctorate of Medicine from All India Institute of Medical Sciences, New Delhi and the University of Illinois, Chicago. He received his fellowship training in haematology/oncology from the University of California, San Francisco, where he was also a scholar in the clinical research programme. After nearly five years as a member of the faculty at the University of Cincinnati, he joined Eli Lilly in 1999 and subsequently, GlaxoSmithKline in 2005 and Sanofi in 2009. He has vast expertise in drug development and in particular clinical development and regulatory affairs. Debasish is currently Senior Vice President and Head of the Global Oncology Division, spanning R&D to commercial.


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