Medicines Adaptive Pathways to Patients’ (MAPPs), the new EMA term for adaptive licensing, is a proposed life-cycle approach to evaluation and licensing of medicines. The ultimate aim of MAPPs is to enable quicker access for patients whilst providing adequate and evolving evidence as to the clinical benefits and harms. MAPPS are defined by the EMA as ‘prospectively planned, adaptive approach to bringing drugs to market.’ The idea is that products, in areas with high patient unmet need, will initially get an authorised niche indication. Once achieved, iterative phases of evidence gathering and progressive licensing adaptations will provide further evidence to support the initial indication and obtain potential further indications.
The vision of achieving faster regulatory approval for products in areas of high unmet needs is a great initiative, however, consideration must be given to how these products can achieve price and reimbursement. Current HTA processes are likely to limit or reject reimbursement at a price that manufacturers would consider to be acceptable for these MAPP products due to the lack of clinical data available at the time of HTA submission. The HTA process would also delay patient access (a typical NICE evaluation takes 21.4 months), defeating the aim of the MAPP initiative.
To ensure EMA’s aim for MAPPs can be achieved a project known as ADAPT SMART (Accelerated Development of Appropriate Patient Therapies: a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes), involving key stakeholders, such as payers, patient groups, industry and academics has been initiated. The objective of ADAPT SMART is to ensure any issues that may delay patient access can be addressed through the development of a MAPP framework that is responsive to the needs of all stakeholders. Throughout its endeavour ADAPT SMART will need to address a myriad of questions as to, amongst others: what products qualify for MAPP; the patient groups that benefit; the required evidence base; the impact on healthcare and social security systems; alternative pricing and reimbursement models; pragmatic trial designs and the re-evaluation of medicines, from a regulatory and reimbursement perspective, based on real world evidence.
NICE has agreed to be an active partner of the ADAPT SMART project and hopes that the process can identify activities that make the development and regulation of medicines more efficient. Sarah Garner, Associate Director for Science Policy at NICE said: “MAPPs represents a more considered and integrated approach to the development and assessment of new drugs … the project will explore the feasibility of alternative reimbursement models and more pragmatic trial designs which are needed in the adaptive landscape”. Finn Borlum Kristensen, Secretariat Director, EUnetHTA, Danish Health and Medicines Authority, also added: “ADAPT SMART will act as a catalyst with an expectation that, as a multi-stakeholder project, it will make a meaningful contribution to efforts aiming at seamless introduction and faster patient access to effective health technologies within sustainable health systems.”
This ADAPT SMART initiative illustrates that there is a real desire for both regulators and HTA bodies to engage in a collaborative project to ensure that patients, in areas of high unmet need, can have earlier access to innovative treatments. The mention of alternative reimbursement models from NICE and EUnetHTA gives rise to the possibility that a ‘Value Based Pricing’ approach may be resurrected. This could result in the initial price of a product approved through the MAPP process being adjusted, upwards or downwards, as new real world evidence for the product becomes available. It is clear that NICE are willing to consider such new alternative reimbursement models, the questions remains as to whether other major EU countries are willing to consider new approaches to pricing and reimbursement of innovative treatments.