Responding to Real-World Data Guidance, Industry Calls for Flexibility and Further Clarity
Commenters praised the FDA’s recent draft guidance on using electronic health records (EHRs) and medical claims data in trials, but they hope the agency will acknowledge issues inherent to real-world data (RWD), offer clarity in certain areas and be flexible as sponsors and sites adjust to the new recommendations.
In its 24 pages of comments, PhRMA, for example, expressed concerns about the September 2021 guidance’s recommendation that sponsors ensure the agency is able to access RWD owned by others, such as data vendors, registries and insurance providers.
The comment deadline, which ended Jan. 24, drew a flood of comments (57 at time of publication), with feedback coming in from health data organizations, healthcare technology companies, nonprofits, trade groups and a slew of others, including big name companies like IQVIA, Janssen, Regeneron and EMD Serono.
“PhRMA recommends that the guidance acknowledge that sponsors may be working with various partners that own this information and that challenges/mitigations related to information access may exist and could be part of the sponsor’s discussions with the review division,” the trade group said.
The Biotechnology Innovation Organization’s (BIO) comments were similar but even longer, spanning 59 pages and centering on three key recommendations: implement appropriate flexibility, pinpoint effective, efficient FDA-sponsor communication methods and identify best practices for curating, processing and handling data.
For example, BIO wants to see case studies on how EHRs and claims data have been used successfully and unsuccessfully in efficacy studies, or at least some theoretical instances beyond the conceptual examples included in the draft guidance. The group feels that while the guidance provides helpful information on selecting data sources, it doesn’t detail how the agency has used submitted data from EHRs and claims data in its regulatory decisions.
BIO also feels that the protocol and statistical analysis plan submission process described in the draft guidance may take sponsors too long — possibly several months to years per research project, it said. The group expressed hope that the agency’s RWE Pilot Program and Type D meeting that are under PDUFA VII will help streamline scientific discussions with the agency, but recommended that it also work with sponsors and sites to identify efficient communication methods that enable quicker but fruitful discussions on RWE.
The Duke Margolis Center for Health Policy also stressed the importance of facilitating conversations on RWD, specifically requesting the agency hold talks with sites and sponsors on what appropriate data quality standards and benchmarks might look like.
Read the comments here: https://bit.ly/34kMZf7.