Future of prospective collection and further research

BOUVY Jacoline, O’ROURKE Diana, JONSSON Pall, XOXI Entela

National Institute for Health and Care Excellence, Manchester, United Kingdom

Regulatory and health technology assessment (HTA) engagement has been a key component in ROADMAP – an Innovative Medicines Initiative project in Alzheimer’s disease (AD). To facilitate this an expert advisory group (EXAG), consisting of regulatory and HTA experts was established to provide guidance on the use of real-world evidence in AD from a regulatory and HTA perspective.

During the course of the ROADMAP project, EXAG discussions have explored the challenges associated with preparing Europe’s healthcare systems for a disease-modifying drug for AD. Because disease-modifying drugs will likely target earlier disease stages of the disease than currently licensed treatments, it is not clear what type of evidence base might be required for these products to go through regulatory and HTA procedures.

Many of the challenges that the EXAG identified could be solved by generating better real-world data in AD. Recommendations from the EXAG included the need for the use of real-world evidence to support disease progression modelling assumptions, the need for consensus on relevant outcomes in early AD, and to provide the required information to accommodate for differences between national and regional settings as part of a pharmacoeconomic model.

This presentation and discussion will provide an overview of the lessons learned from ROADMAP Work Package 6 (regulatory and HTA engagement) and the ROADMAP EXAG, as well as reflecting on the practical implications and challenges of using real world data in HTA and regulatory decision making.