Department of Health Grant: Improving Access to Investigational Therapies (R01)

National Academy of Sciences, Engineering, and Medicine (the Academies)

• Research and examine the landscape and develop a framework to guide the improvement of the U.S. system for expanded access to investigational therapies, taking into consideration ethical, scientific, regulatory, financial, and infrastructural issues. Articulate guiding principles and an ethical framework for expanded access to investigational therapies.

• Research and address scientific considerations and implications for medical product clinical development programs when the investigational product is available through an expanded access program.

• Research and address regulatory considerations and the role of FDA, including providing guidance for the collection and evaluation of both safety and efficacy data relating to investigational products provided through expanded access programs.

• Research and address business and financial considerations relating to offering investigational products through expanded access programs, including consideration of:

o Financial considerations for both small and large companies, and business models for manufacturing and furnishing the products outside of the context of a development program;
o Liability and related legal considerations relating to providing unapproved therapies to patients outside of the context of a research protocol; and
o Implications for continued access after approval if the product is not affordable to a patient who had been granted expanded access.
o Identify characteristics of the optimal structure and governance of a system for expanded access, including making recommendations relating to:
o Guidance to and structure of ethics review committees;
o Systems for reporting and accountability of companies providing investigational products through an expanded access mechanism; and
o Improvement of information sharing among the multiple decision making parties (provider, sponsor, and FDA) to facilitate patient access to investigational therapies through the most appropriate means (e.g., clinical trials, expanded access programs, individual access requests).