EFPIA statement on the EMA report "Adaptive pathways: key learnings and next steps"

EFPIA welcomes the report's statement that adaptive pathways can foster multi-stakeholder dialogue, bringing regulators, interested HTAs, healthcare professionals and patients. We also note the project's findings that agreement between stakeholders can be reached on a prospective approach to evidence generation. EFPIA supports the Increased participation of patients in the adaptive pathways model, as a means of accelerating access to medicines for those candidates who would most benefit from this.

The challenges to creating methodologically sound strategies of real-world evidence collection are clear. These include: data not always generated to the same standards, and therefore not comparable; e-health systems not always compatible between hospitals or countries; patients not having a single, electronic health record that stores data in one place; and data being collected for different purposes, which can’t always be linked together, with the added problem that regulation sometimes stops data transfer between systems.

EFPIA Director General Richard Bergström said: "Prior to the December workshop EFPIA will carry out its own evaluation of members' experience. But we can already now say that we agree with the EMA that new methodologies are needed for better patient involvement in the choice of development strategies. There is also an immediate need to agree standards and approaches to use real world evidence in addition to randomised clinical trials. Combination therapies, and medicines for children and in multi-morbid elderly patients are often better studied in real-life than in RCTs. The involvement of payers in early dialogue is already under way but must be scaled-up and broadened to involve more countries.”