April 2, 2019
Dockets Management Branch (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852
Re: Docket No. FDA-2018-D-4628-0001: REMS Assessment: Planning and Reporting
The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments to the Draft Guidance on REMS Assessment: Planning and Reporting.
BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial, and environmental biotechnology products.
BIO applauds the FDA on the work done to develop this Draft Guidance - it offers clear, practical information to sponsors and other stakeholders who are involved in the design and effectiveness evaluation of REMS programs. This important Draft Guidance is well written and provides instrumental recommendations particularly on how the REMS program goals, objectives and REMS design may impact the selection of metrics and data sources, which will be used to assess whether the program is meeting its risk mitigation goals.
BIO appreciates the Agency’s recognition that the “data sources, methodologies, and metrics used to assess the effectiveness of REMS continue to evolve” and that there are limitations to Sponsors’ ability to accurately evaluate safety-related health outcomes as part of REMS assessment.1 BIO suggests the Agency continue to keep the challenges of isolating the effect of REMS on health outcomes in mind when Sponsors are proposing metrics in the assessment category, “Health Outcomes and/or Surrogates of Health Outcomes.” BIO would welcome the opportunity to partner with the Agency to define improved metrics to evaluate safety-related health outcomes.
Of note, it is not clear from the Draft Guidance how existing/well-established REMS for existing drugs will be addressed. BIO encourages the FDA clarify that existing REMS would be grandfathered, particularly regarding evaluation of burden on healthcare system and patient access...