FDA, Regulatory

Industry calls for changes in two REMS guidances

Industry groups representing the pharmaceutical, biotechnology and generic drug industries are calling for changes to two recent FDA draft guidances on REMS.

The draft guidances, released for comment in January, detail a framework for companies to develop an assessment plan for their REMS programs and provide recommendations for surveying patient and health care provider knowledge of REMS information.

While PhRMA says it commends the FDA for providing recommendations for developing REMS assessments, the group argues that the guidance should be prospective and not apply to existing REMS that already have assessments in place to minimize disruption to those programs.

PhRMA says it is concerned that new metrics for assessing a REMS introduced in the draft guidance may pose challenges to sponsors with existing REMS. “Such changes may significantly modify the REMS program and increase stakeholder burden through changes to data collection requirements,” PhRMA writes.

Instead, PhRMA asks the FDA to weigh the utility of new metrics against how efficiently they can measure the effectiveness of the REMS.

PhRMA also asks the FDA for additional details on how metrics on patient access and burden to the healthcare system should be incorporated into assessment plans. “PhRMA believes that if a pre-specified threshold is not met, there should be an opportunity to revise and improve the REMS goals,” PhRMA writes.

Similarly, BIO calls for the FDA to clarify how the guidance will apply to existing and well-established REMS and encourages the FDA to exempt existing REMS from evaluating patient access and burden on the healthcare system.

BIO points out that there are different challenges to assessing a REMS required as part of a product’s approval and one required after approval due to a post-market safety issue. “In the case of REMS that are required as part of an initial approval, there is no comparator group of patients who had access to the product without REMS. Without that comparator group, it is very difficult to measure the effect of the REMS program on patient access,” BIO writes.

Representing the generic drug industry, AAM raises concerns about how the guidance will apply to shared REMS systems. “The recommendations in the guidance are easier to implement in a brand REMS context and will be much more difficult to employ for single, shared system REMS or separate shared REMS that have multiple ANDA participants,” AAM writes.

In its comments on the survey methodologies guidance, PhRMA requests that the FDA expand the guidance to include recommendations for surveying patient and healthcare practitioner attitudes in addition to surveying their knowledge.

The draft guidance specifies that patients should either be currently taking the drug or have taken it within a “reasonable time,” which PhRMA says should be flexible depending on the characteristics of the drug and its dosing schedule.

The draft guidance also says that survey participants should not have previously participated in a REMS survey for the same product. “PhMRA believes this will be challenging for sponsors and other stakeholders … where the drug has been on the market for a long time, or for a rare disease product used in a limited patient population and prescribed by a smaller number of specialized prescribers,” PhRMA writes.

Additionally, PhRMA asks for more details from the FDA on the utility of open-ended questions in the surveys. While the guidance recommends surveys include a variety of question types, PhRMA says it believes the utility of responses to open-ended questions in this context would be limited.


First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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