SMU: BIO Comments on Creating an Alternative Approval Pathway for Certain Drugs to Address Unmet Medical Need


The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit written comments on the proposal to create an alternative approval pathway for certain drugs intended to address unmet medical need, also referred to as a Special Medical Use (SMU) designation. 

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. 

BIO applauds the Administration and the FDA for seeking input on proposals focused on approaches to stimulate innovation and expedite access to medicines that will improve the quality of life for patients that need them the most. BIO has a long-standing position of supporting modern approaches to clinical development and effective review that will invigorate research, development, and availability of drugs to patients.  This is consistent with FDA’s dual role in both protecting and promoting the public health. 

Both the President’s Council of Advisors for Science and Technology (PCAST) 2012 report, “Propelling Innovation in Drug Discovery, Development, and Evaluation”, and the FDA’s January 15th public hearing notice underscore the importance of efficiently and effectively developing new therapies intended to treat targeted subpopulations, rare diseases, infectious diseases, and serious manifestations of a more common condition.  We understand that the PCAST, FDA, and Infectious Diseases Society of America (IDSA) proposals are intended to facilitate more narrow, targeted, and expeditious clinical development programs in such cases.  We also understand that the intent is for products approved through this new pathway to utilize an additional communication tool in the form of a special designation and logo that would inform healthcare providers and patients that the therapy was studied in specific subpopulations only, without interfering with the practice of medicine. 

Following discussions of the SMU designation concept with BIO’s member companies and consideration of the February 4th testimony of other stakeholders, BIO supports implementation of the SMU pathway for a broad range of potential diseases and conditions as scientifically appropriateif it can be structured in such a manner that it will promote biomedical innovation and contribute to improved health outcomes for patients suffering from serious, debilitating and life-threatening diseases.  We are pleased to provide the following recommendations on how the SMU pathway can be designed to achieve this common goal.  BIO looks forward to collaborating with the FDA, PCAST, the Administration, and other stakeholders to work through the details of this important proposal and any questions regarding its establishment and implementation.  We would also look forward to the opportunity to comment further as FDA develops specific proposals.

In this regard, BIO notes that FDA currently has broad and flexible authority under its authorizing statute and accompanying regulations to develop streamlined approaches for the development and review of important new therapies, with appropriate labeling regarding the risks and benefits thereof.  Thus, depending on the specifics of the SMU pathway, new legislative authority may not be necessary.  Nonetheless, BIO stands ready to work with the Administration and Congressional leaders on any such legislation, as appropriate.

Key Elements of a Special Medical Use Pathway:

BIO believes that the following seven key factors are fundamental elements of a potentially successful SMU pathway:

  1. Voluntary for Sponsors
  2. Prospective and Available Early in Drug Development
  3. Broad Eligibility for All Relevant Indications
  4. Clear Eligibility Criteria
  5. Clarity on Expedited Clinical Development Approaches
  6. Focuses Clinical Research on Relevant Subpopulations  
  7. Does Not Infringe on the Practice of Medicine

We are pleased to provide more detailed comments on each of these elements below.

Read the full comments (PDF)