The event, which was attended by 85 people from industry, patient groups and others involved in medicines development, heard from speakers including Earl Howe, Parliamentary Under Secretary of State for Quality, Dr Hans-Georg Eichler from the European Medicines Agency (EMA), Dr Daniel O'Connor, Medicines and Healthcare products Regulatory Agency (MHRA), Dr Sarah Garner, National Institute of Health and Clinical Excellence (NICE) and Dr Tony Soteriou, Department of Health.
Lord Howe described adaptive licensing as a “golden opportunity” to demonstrate the strength of life sciences in the UK. He went on to say that if used successfully it will demonstrate that regulation is “not a noose which stifles but rather a safety mechanism which can release medicines to patients in need.”
To date, 14 companies have submitted applications for the adaptive licensing pilot, and the EMA will communicate shortly which of these will be taken forward. The opportunity to submit live assets for consideration remains open until further notice.
Key topics of discussion included the fact that this pilot involves a “coalition of the willing” including regulators, payors, industry and patients in a discussion in a safe harbour environment. There was great emphasis on the need for trust and open and honest dialogue between all involved. It was recognised that this approach now allows, an adaptive mindset, using multiple data sources to help patients. This recognises a shift from a blockbuster development strategy to one that considers smaller patient populations from the outset.
Various challenges were discussed including the capacity of EMA, MHRA and NICE to deal with potential additional workload, and it was confirmed that they are adequately resourced. Another concern was how adaptive licensing pricing will fit in a global product development strategy, and whether it is realistic for all European payors to accept greater uncertainty. A clear distinction was made between risk and uncertainty; this pilot is designed to allow the acceptance of greater uncertainty, not increased risk. Agreement on pricing and reimbursement will be fundamental to the success of the pilot, and going forward marketing communications will need to adapt to reflect products that are approved through adaptive pathways.
Bina Rawal, ABPI’s Research, Medical and Innovation Director, said:
“The ABPI has long called for a more agile and flexible licensing approach and we welcome the EMA pilot, which will explore the adaptive licensing approach with real medicines in development. This event has helped to explain how this step change will be implemented. Once we have addressed some of the outstanding challenges, the ABPI expects adaptive licensing to help support the development of medicines where there is high unmet need, such as novel antibiotics and medicines for dementias and rare diseases. Scientific progress and innovation are a continuum, requiring a progressive approach from medicines developers, regulators, NHS, NICE and patients in the best interests of all of us.”
Steve Bates, BIA Chief Executive Officer, said:
“The BIA has championed innovation in regulation for many years, and we are delighted to see that the reaction of the UK ecosystem as a whole to the EMA initiative has been first class. At today’s event we saw the commitment of our government and the expertise and professionalism of our regulatory and health technology assessment agencies, MHRA and NICE working alongside the EMA. We are delighted that a number of our member companies are already considering participating in the adaptive licensing pilot, and hope that by explaining the detail today more companies will fully understand the opportunities offered by this pathway. We hope as a result of this meeting, we will see more engagement from all stakeholders to speed the delivery of new innovative therapies to patients.”
Professor Richard Barker, Director of CASMI, said:
“We are now determined not only that effective adaptive development pilots are launched but also that the UK plays a major role in proving out the concept – as called for by the Prime Minister in his December 2012 life sciences speech. There are significant challenges in the trial design, informatics, reimbursement, ethics and public and patient communications which will be vital to success, and to which CASMI’s academic fellows are contributing. We are also working with UK trade bodies and other stakeholders, such as MHRA, NICE and the NHS to explore the potential in the UK.”
Notes to editor
About the ABPI
The ABPI represents innovative research-based biopharmaceutical companies, large, medium and small, leading an exciting new era of biosciences in the UK.
Our industry, a major contributor to the economy of the UK, brings life-saving and life-enhancing medicines to patients. Our members supply 90 per cent of all medicines used by the NHS, and are researching and developing over two-thirds of the current medicines pipeline, ensuring that the UK remains at the forefront of helping patients prevent and overcome diseases.
The ABPI is recognised by government as the industry body negotiating on behalf of the branded pharmaceutical industry, for statutory consultation requirements including the pricing scheme for medicines in the UK.
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