Industry group BIO and biopharma companies Allergan and Roche are calling for the FDA to be flexible in its approach for using patient experience data (PED) and in allowing the use of such data in labeling.
BIO said that it “strongly believes” that in order for a broad adoption of PED, any upcoming guidance documents on patient-focused drug development “should emphasize the FDA’s willingness to exercise regulatory flexibility and acceptance of innovative designs and approaches for collecting PED.”
For example, the draft guidance on gathering patient input, according to Danielle Friend, director of science and regulatory affairs at BIO, is “highly focused on statistical requirements for collection of subjective measures and although representation of the population is paramount, there is also a need to balance such representativeness with statistical validity, as the requirement of many strata for representation could have a negative impact on statistical power.”
Similar to BIO, Allergan urges the FDA to consider the need for flexibility with respect to the methodological requirements and opportunities for engagement to provide sponsors with clarity to inform evidence generation in a timely manner.
BIO also calls for flexibility on sampling methods and in collecting data for smaller patient populations, such as with rare diseases. The industry group additionally requests that the FDA delineate between collecting PED to inform clinical studies (e.g., development of a clinical outcome assessment tool or to inform clinical study endpoints) and PED collected within a clinical study meant to inform a regulatory decision.
Roche’s Genentech, meanwhile, calls on the agency to further clarify when PED can be label-enabling.
“As different types of patient experience data serve different purposes, we urge the FDA to describe reasonable standards that enable the inclusion of fit-for-purpose patient experience data within different sections of drug label,” Janet Jenkins-Showalter, head of US regulatory policy at Genentech, writes.
Allergan also calls on the FDA to substantiate the differences in evidentiary standards for PED for regulatory-decision making vs. labeling.
Jenkins-Showalter adds: “We would also highlight the importance of credentialing and sharing patient experience data deemed of sufficient quality to inform drug development program design (e.g., burden of disease, burden of existing treatments) that is collected outside the context of an IND program. It is important that these regulatory quality data are shared with the broader community of Sponsors, measurement scientists, patients, and regulators such that clinical science is able to advance towards the development of outcomes that are meaningful to patients.”
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email firstname.lastname@example.org for more information.
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