EMA Develops Strategy on Adaptive Pathways

The European Medicines Agency (EMA) has published a final report about its pilot project on adaptive pathways, which it describes as a product development concept for medicines that address patients’ unmet medical needs.     The pilot project, which has now ended, showed that adaptive pathways can bring together regulators, health technology assessment (HTA) bodies, healthcare professionals and patients to agree on a prospective plan to generate data on a medicine across its lifespan in areas of unmet medical need.   Adaptive pathways can support medicine development in therapeutic areas where evidence generation is challenging, such as infectious diseases, Alzheimer’s disease, degenerative diseases, and rare cancers, according to EMA. These pathways can be defined as a planned, progressive approach to bringing a medicine to patients.   “It is not a new route of marketing authorization; it makes use of existing regulatory tools,” the agency stated. “Under this approach, the medicine will first be authorized in a small patient population that is likely to benefit most from the medicine. Then, additional evidence is gathered over time resulting in progressive licensing adaptations to extend or restrict the previously authorized indications of the medicine.”   The pilot began in March, 2014. The EMA received 62 applications, 18 of which were selected for in-depth, face-to-face meetings. At the end of the pilot, six of these applications had progressed to receive formal parallel advice by EMA and HTA bodies and one to benefit from simple scientific advice. Most proposals were considered not suitable for adaptive pathways, and the companies were advised to pursue traditional development routes.   The pilot helped to identify aspects for further reflection, including the need for increased involvement of patients to assist in the selection of candidates for adaptive pathways, the definition of methodologically sound strategies of real-world evidence collection to support the assessment of both efficacy and effectiveness, and the potential involvement of payers to provide input on pricing strategies.   EMA plans to explore adaptive pathways in the context of its parallel advice with HTA bodies, which provides a framework to include additional stakeholders (e.g., patients and, if relevant, payers). Medicine developers who are interested in following the adaptive pathways approach should submit a proposal to EMA. To gather the views and proposals from its stakeholders on the adaptive pathways approach, the agency will organize a workshop on December 8th, 2016.   Cooperation between stakeholders and a strong pharmacovigilance system are the basis for a systematic monitoring of the safety and the overall performance of a medicine in clinical practice; these are the two key elements underpinning the adaptive pathways concept, noted EMA.