What are Medicines Adaptive Pathways to Patients (MAPPs)?
MAPPs refer to flexible development and access pathways within the current regulatory framework that balance early patient access, public health and societal benefits. MAPPs start with an early authorisation of a product focused on a well-defined and targeted population with a clear safety and efficacy profile
The target population is adjusted as the evidence base expands. MAPPs may integrate adaptive clinical trial design, patient centric benefit/risk assessments and continuous re-evaluation as new evidence becomes available. MAPPs, therefore, relate to the entire life cycle of a medicine from development, through licensing to patient access (reimbursement and healthcare delivery).
However, a pre-requisite for the success of the implementation of MAPPs lies in the full and common understanding of its value, not just for industry, but across the entire innovation life cycle: for regulators, HTAs, payers, governments, clinicians and, most importantly, patients.
- MAPPs will initially focus on targeted, stratified medicines with clear biomarkers, well-defined populations, preferably an available diagnostic, and high level of efficacy and safety
- MAPPs imply an initial marketing authorisation for a limited patient population upon demonstration of a positive benefit/risk balance
- It will generate confirmatory evidence from observational data sources while randomised clinical trials continue in support to the original or additional indications
- As more indications are validated, the population approved for the new medication will adapt to the evolving evidence base, as will the initial pricing and value assessment.
The evidence generation plan would be established in advance through joint Scientific Advice involving at least regulators and HTA/payers, or using other mechanisms for collaborative development or market access conversations. This process would include:
- Setting the data requirements for early regulatory approval based on initial evidence
- Iterative benefit-risk evaluations at pre-agreed milestones to:
– Confirm initial findings
– Consider expansion of indicated population using evidence from real world and parallel clinical development
– Consider re-assessment of value as longer-term effectiveness and outcomes are understood
- Agreement on evidence package and methodologies for re-assessment
- The early, collaborative agreement by stakeholders (patients, regulators, HTA/payers, practitioners, industry) on the evidence package required for early regulatory approval and access for patients
- The willingness of patients, payers and industry to operate with increased uncertainty
- The IT infrastructure needed to provide the real world evidence base
- IMI multi-stakeholder coordination and stakeholder dialogue initiatives
- IMI research projects to develop missing enablers of MAPPs (tools and methodologies including for modelling impact on all parties)
- EMA Adaptive Licensing Pilot project (‘Safe Harbour’) – a regulator led forum to simulate adaptive regulatory pathways for example live assets.