REMS: BIO Comments on Standardizing and Evaluating Risk Evaluation and Mitigation Strategies

Dear Sir/Madam:

The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments in response to its 2-day public meeting “Standardizing and Evaluating Risk Evaluation and Mitigation Strategies.” 

BIO represents more than 1,100 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, thereby expanding the boundaries of science to benefit humanity by providing better healthcare, enhanced agriculture, and a cleaner and safer environment. 


As mentioned in our testimony at the public meeting (see Appendix A), BIO supports FDA’s ongoing PDUFA V initiatives to identify potential projects that may help standardize Evaluating Risk Evaluation and Mitigation Strategies (REMS) and integrate them into the health care delivery system.  BIO has long advocated for a holistic approach to drug safety, and the PDUFA V framework demonstrates industry’s commitment to a lifecycle approach to product evaluation by strengthening FDA’s post-market surveillance and benefit-risk management capacity.  Drug safety is not absolute, but rather a matter of balancing a drug or biologic’s predicted benefits against known risks.  A product is considered safe if it has an appropriate benefit-risk balance for the intended population and use, and a REMS program can play an important role in minimizing risk to maximize the drug’s potential benefit-risk profile.  Effective risk management approaches, including REMS, can help facilitate appropriate patient access to efficacious therapies with known safety issues that may not otherwise receive FDA approval.

BIO’s testimony highlighted four main principles: 

  1. FDA and Sponsors should communicate about REMS and risk management strategies as early as possible in the review cycle;
  2. Comprehensive REMS implementation efforts should be reserved for REMS with elements to assure safe use (ETASU) programs;
  3. Standardization should include establishing a standard set of best practice principles regarding the design, development, testing, implementation, evaluation, modification and termination of REMS tools; and
  4. REMS program effectiveness assessments should evaluate the totality of the REMS program. 

Below BIO provides responses and suggests projects relating to FDA’s questions posed in the Federal Register Notice and the above stated BIO principles.