What is MAPPs?
MAPPs (also formerly called Adaptive pathways or Adaptive Licensing) refers to a flexible development, registration and access pathway within the current regulatory framework in the EU, that maximizes the positive impact of new medicines on public health by balancing timely access with the need to provide evolving information on benefits and risks (B/R). Similar pathways are explored in the US and other parts f the world, but the EU context is clearly much more advanced from a policy and regulatory point of view.
Why is MAPPs important and for whom?
The regulatory environment is lagging behind evolving science; conventional R&D models are no longer financially viable and have become a major hurdle to efficient development and thus patient availability of new therapies. A more flexible pathway within the current EU regulatory and national reimbursement framework would not only accelerate access of crucial therapies but would also increase the probability of success as therapies would be oriented towards those patients deemed most likely to respond. The cost of development for industry and for healthcare providers could reduce
How should MAPPs work?
MAPPs will initially focus on targeted, stratified experimental molecules with clear biomarkers, well-defined populations, preferably an available diagnostic, and a potentially high efficacy/safety. It will offer an opportunity to obtain an initial (limited) EU marketing authorisation while simultaneously continuing further research to validate additional clinical endpoints, on the authorised or future indications/populations. After such initial authorisation, MAPPs will provide a means to generate confirmatory evidence from more observational data sources, while phase I/II trials continue for other indications. In this way, as more indications are validated, the targeted population will expand along with the evidence base.
The Author will also analyze the current challenges and the instruments available for MAPPs implementation.
Luk Maes graduated from the Brussels Free University with a PhD in Medicine. In 1986 he joined Bristol-Myers (before the merger with Squibb), and was active, for > 25 yrs, in the R&D developmental regulatory environment in gradually increasing responsibility roles and in multiple therapeutic areas. His experience with medicinal development and registration in Europe currently covers 17 centrally approved products. Since 2 yrs he is more active on the European “policy” front, working with EFPIA and IMI, and with EMA in numerous multi-stakeholder projects.