CASMI has played a key role in the development of Adaptive Licensing, since the publication of the Cooksey Review by our Advisory Board Chairman, Sir David Cooksey. Through the initial efforts of the Athenaeum Group, convened by our Director, and working together with the NEWDIGS initiative, in which Associate Fellow Dr Sarah Garner took an active part, CASMI has championed Adaptive Pathways.
Following the EMA’s announcement of their approval of the concept, 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 still significant challenges in the design, reimbursement, ethics and communications to which CASMI’s academic fellows are contributing. We are also working with UK trade bodies and other stakeholders to explore the potential in the UK.
We held a joint meeting with the ABPI and BIA in June 2014, giving medicines developers across the life sciences sector the opportunity to further enhance their understanding of how they can engage with this important initiative.
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 benefits for the UK in taking a strong lead in this process are recognised in the Life Sciences Strategy and can be summarised for each stakeholder as follows:
- Patients: earlier access to promising new medicines, with close clinical supervision of the results
- Clinicians: ability to treat unmet medical need with the most advanced medicines available
- Companies: ability to reach the market and earn revenues without the major investment normally required for Phase 3 trials (particularly valuable for SMEs)
- Regulators: greater flexibility in dealing with high priority medicines that are urgently needed
- Reimbursement agencies: an ability to see real world effectiveness data before final evaluation of price (NB this implies a provisional price at the point of initial conditional approval)
Active UK participation in these developments will also help in the collective efforts by NIHR and industry bodies to reestablish the UK as a leading European market for clinical trials and life sciences investment.