Following our joint conference with the MHRA, and continuing our regulatory theme on the BIA blog this month, today’s post takes a closer look at some of the European initiatives to increase early access to innovative medicines, including the adaptive pathways pilot.

The afternoon session started with an overview of recent activity at a European level, provided by Robert Hemmings, MHRA, who is a member of the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) and Chair of CHMP’s Scientific Advice Working Party.

RHemmingsRobert set out the challenges for regulators in this area, including the many stakeholders and competing demands agencies must balance. He said that early access must be sustainable as the cost of failure is high and it must be provided in a way that allows the collection of robust data after initial marketing authorisation.

Recognising that both national and EU-wide efforts are required, Robert highlighted the EU Medicines Agencies Network Strategy to 2020. This sets out joint key priorities and a high-level roadmap for how the regulatory agencies can work within their own country and with the EMA to achieve these and foster early access. Concluding his presentation, Robert said that there is a lot of activity ongoing and the challenge is putting all of the available tools for early access into a cohesive structure to help drug developers.

Adapting to adaptive pathways

Next, Francesca Cerreta, Senior Scientific Officer at the EMA, shared some learnings from her agency’s adaptive pathways pilot. The aim of the pilot was to engage in early dialogue with the relevant stakeholders to design a smart development programme that generates the relevant evidence base, using all data sources, so that decisions can be made quickly and seamlessly. A key challenge is bringing regulators, payers and health technology assessment (HTA) bodies together and aligning their different requirements in the programme design. The EMA also had a mixed experience with companies, with the information provided in applications often being “sketchy”, although SMEs have so far been the most creative when proposing solutions. Francesca said that although the adaptive pathways approach is valuable, it is applicable only in a limited number of cases where there is promise to fulfil unmet need and there are clear-cut, actionable endpoints.

FCerretta

Eliot Forster, CEO of Immunocore, provided an industry perspective on the EMA’s pilot. Immunocore have been engaged with the pilot for about a year and Eliot said he was very impressed so far. He believed it was the fastest way to get truly innovative therapies to patients. Eliot praised the practical approach taken by the EMA, adding that the conversations were timely and collegiate, with both sides learning throughout the process. He hoped this was a watershed moment for how regulators and developers could work together.

EForster

NICE’s Sarah Garner echoed Eliot’s view that the conversations as part of the adaptive pathways process had to be honest and focussed on coming up with solutions together. The adaptive approach provides NICE with the opportunity to inform the process so that the right real-world data can be collected, hopefully in the UK. She said there was political ambition in the UK for this to happen but sadly this is not true in all EU countries. The UK therefore has a real opportunity to capitalise on real-world data, she said, especially with the large number of registries we have and data-focussed institutes.

SGarner

The speakers from the session were joined on stage by Joy Duffen of the Cure Parkinson’s Trust and Christian Schneider from the National Institute for Biological Standards and Control (NIBSC) for a Q&A. Both Joy and Christian welcomed the adaptive pilot approach as being critically important for patients. In questions from the audience, the panel was asked whether there had been opportunities for treatment prices to be increased following successful data collection from the pilot. This hadn’t happened yet said Robert Hemmings, but a potentially-curative gene therapy might be the first chance for this in the coming year. Further discussion focussed on harmonising HTA processes across Europe but, as Sarah pointed out, healthcare systems vary massively across Member States making harmonisation very difficult. There will always be country-specific issues she thought.

Panel

For more information on upcoming BIA events go to the BIA website.