In the past, FDA has been criticized for failing to clearly explain in regulations or guidance the range of IND mechanisms by which access could be obtained. Some concerns were that the lack of clarity resulted in disparate access for different types of patients, and access that was primarily limited to patients with certain diseases (i.e., cancers and HIV infection). To address these concerns, FDA revised its expanded access regulations in 2009. The revised regulations are intended to increase awareness and knowledge about expanded access programs and the procedures for obtaining investigational drugs for treatment use.
The documents discuss questions including:
- When should an access protocol submission be used?
- Who can make a submission for individual patient expanded access when there is an existing IND for the drug?
- When might it be appropriate to deny a request for individual patient access when previous requests for the same drug for the same or a similar use has been permitted?
- Is Institutional Review Board (IRB) review and approval required for individual patient access uses?
- In general, how does FDA determine that authorizing expanded access to a drug will not interfere with clinical trials or drug development?
- Can FDA require a company to provide expanded access to its drug if FDA authorizes the expanded access?
- Under 21 CFR 312.8, who must request authorization from FDA to charge for an investigational drug for use under an IND?
- How can a sponsor charge for its investigational drug in a blinded, controlled, clinical trial without compromising the blind and, therefore, the integrity of the clinical data generated from the trial?
- Does a sponsor need FDA authorization to charge for the costs of drug delivery, including the costs associated with formulation, packaging, instrumentation, monitoring, disposables, setup, and nursing care?