Working with other stakeholders, CASMI is leading an evaluation of the UK as a potential test site for a European adaptive licensing pilot that the European Medicines Agency (EMA) is proposing.
CASMI is part of the Expert Group working on the regulatory innovation challenge set by the Strategy for UK Life Sciences. The Prime Minister committed to supporting an Adaptive Licensing pilot in the UK in the recent One Year On report.
Current regulatory approaches are based on a standardised process particularly with respect to the types and quantity of evidence that it required. This has led to concerns of unnecessarily high R&D costs and delays to market, resulting in high prices. These issues are exacerbated if the regulatory trials do not meet the needs of HTA agencies.
Adaptive approaches aim to streamline the research, licensing and market access processes by involvement of all stakeholders including the sponsor, regulator, payers/providers and the research community. A drug-specific development plan is agreed that provides sufficient information on risk versus benefit to enable prompt authorization in a defined group of patients and/or treatment settings. This followed by monitoring of ‘real-life’ effectiveness and safety and leads to further license adaptation. AL may use existing regulatory pathways or explore novel methodologies.
Adaptive approaches are not suitable for all products and will not replace current mechanisms. It could be used for initial indications where early data suggests a positive risk-benefit profile and there is a compelling case for supported market entry, for example a demonstrated unmet need. Alternatively it could be used for second and subsequent indications, where there is already regulatory data, to inform a judgement of risk vs. benefit.
The CASMI team are working with other international groups who are interested in adaptive approaches including:
This project is funded by the Wellcome Trust