Stakeholder Suggestions for FDA Registries RWD Guidance

Pharmaceutical Research and Manufacturers of America (PhRMA) says using registries as part of real-world data (RWD) to support drug and biologic regulatory decision-making “leverages unique, available, and important RWD to understand more about patients and their medical history and can characterize potential benefits and risks in diverse patient populations.” Commenting on an FDA draft guidance on the topic, PhRMA says that while the guidance is part of a set of four relating to RWD, common elements from the other guidances are not always aligned with those in this document, which it says may be confusing.

“Similar approaches should be applicable across many RWD sources and the guidances should be aligned accordingly,” PhRMA says. The association also gives line-by-line specific comments.

In its comment letter, Janssen says the guidance is “an important step in ensuring that sponsors understand FDA’s expectations as they relate to leveraging RWD for regulatory decision making. Importantly, this guidance also provides sponsors and non-sponsor stakeholders with important information on how to design registries so that the registry data can be leveraged for regulatory decisions.”

Janssen gives recommendations on contributions of real-world evidence stakeholders, secondary use of existing regulatory data, and linking registry data to benefit/risk. It also gives line-by-line specific comments.

The Duke Margolis Center for Health Policy says more clarity from FDA would be beneficial when it addresses delineating existing registries and purpose-built registries, facilitating and prioritizing the collection of outcomes data that are important to patients, clarifying the roles and responsibilities of sponsors and registry managers, developing consistent definitions for key terms or noting where consistency is lacking, and building multi-stakeholder support for well-funded, multi-purpose registries.

The center suggests that FDA and other relevant stakeholders act in the near term on these steps as the guidance is revised and implemented: formalizing and advancing patient-level or -reported endpoints that are capturable in well-designed registries; continuing to develop and refine consistent definitions for key terms, concepts, and endpoints; and engaging the conceptualization and development of multi-stakeholder registries that would appeal to diverse healthcare stakeholders with a range of disease interests, like cell and gene, Alzheimer’s disease, and rare disease therapies.

The Professional Society for Health Economics and Outcomes Research says clearer definitions, particularly regarding demonstration of reliability and a delineation between registry data types, with substantive differences, would be of great benefit. It gives general comments on relevance and reliability, data linkage and integration, registry definitions, and regulatory considerations. It also gives detailed line-by-line comments.