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European Pharmaceutical Contractor

Common Cause

In order to get a new drug to market, clinical drug development has traditionally been focused on fulfilling the requirements put forward within regulatory agency guidelines or scientific advice procedures. In the EU, this has concentrated on study design, selection of endpoints and suitable comparators. This process has worked well for many years and – in most cases – has enabled pharmaceutical firms to reach the market through marketing authorisation.

Consumer Power

But the world has changed: marketing authorisation is no longer enough to gain access to patients. In recent years, many companies have realised that the payers now have the final say on whether or not a product will gain access to the market. We have seen products being refused reimbursement, or prices being cut to meet payers’ expectations.

This begs the question: do the costs of these new drugs match the value gained for the patient and society? In many circumstances, industry is ill-prepared to provide an answer. Following the traditional route of obtaining regulatory approval first, clinical studies have endeavoured to do so, at the expense of payers’ expectations with regard to duration, endpoints and comparator.

In other words, there exists a mismatch between the regulatory requirements and the expectations set by health technology assessment (HTA) bodies. So far, the industry has tried to fill this gap by setting up post-authorisation studies, either as randomised trials, cohort or more ‘real-life’ studies. The result has been an unsatisfactory one: an extended delay in patients getting access to new medicines. This process has to be revised for the sake of everybody’s interest, and the potential solution is a simple one.

Working in Unison

To improve the chances of good medicines reaching patients faster, we really need a more integrated approach, where regulators and HTA bodies engage in mutual discussion, striving to reach a consensus and, in doing so, bring payers’ and regulators’ demands together. Not only would this benefit the industry, but ultimately patients: driving a constructive dialogue which takes into consideration the shared requirements of both parties.

If this new approach is to be realised, it is imperative that the structural set-up of the industry is addressed. The aforementioned groups must look to break down the silo – distinct departments with disparate objectives, and incentives based on individual achievements – working in a more uniform fashion instead. Regulatory affairs, market access and HTA experts must be engaged at an early stage during drug candidate development, not only to discuss labelling and claims, but to create a cohesive drug development programme encompassing the needs of both regulators and HTAs.

Route Forward

For some years now, the industry in Europe has had access to joint scientific and HTA advice offered collaboratively by the EU agencies (Committee for Medicinal Products for Human Use and European Network for Health Technology Assessment). The process has worked as a sequential consultation, beginning with regulatory review, followed by input from the relevant HTA bodies.

Owing to its inclusion in the EU’s current pilot of an adaptive pathway, this procedure has now been taken one stage further – a clear step in the right direction. Although regulators and HTA experts may agree and look to guide the industry in the direction of a more aligned drug-development process, we still need to see payers accepting this approach.

With its greater, and perhaps more relevant indications – from a payer and societal perspective – the adaptive pathway is considered to be less rigorous, offering more reliable assurances for the financing of further development. Moreover, this pathway is an EU-centralised approach, whereas pricing and reimbursement is a purely national policy decision – creating further potential for discrepant views. If these obstacles are not overcome, the adaptive pathway will not be able to succeed in gaining patients faster access to new medicines.

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Steffen Thirstrup is a medical doctor and specialist in clinical pharmacology and therapeutics. He holds a PhD in Pharmacology and has a long background in clinical internal medicine, with special emphasis on adult respiratory medicine. Steffen worked at the Danish Health and Medicines Authority, first as the Danish member of the Committee for Medicinal Products for Human Use at the EMA, followed by a short period as head of the Danish Institute for Rational Pharmacotherapy, dealing with HTA and best practice guidelines for primary care. In March 2013, he joined the NDA Group as a full-time medical advisor on its advisory board, and soon after was appointed its director.
Steffen Thirstrup
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