You’ve got a drug in the pipeline that’s showing great promise. Results from your clinical trials are making news. What happens when you start getting calls from physicians and desperately ill patients requesting access to the drug prior to final approval? Who takes the call? What is your response?
For patients who are terminally or seriously ill, have exhausted all available therapies, and cannot enter a clinical trial, access to an investigational drug/biologic outside the trial setting via an expanded-access program can represent a new treatment option.
Since the 1970s, FDA-sanctioned expanded-access programs have enabled patients, under specific circumstances, to access drugs or biologics that are still in development. These rules were recently clarified by FDA and new types of access for treatment use added to ensure “broad and equitable access to investigational drugs for treatment.”
The regulations now include all circumstances under which access to investigational drugs is permitted, including: individual patients in nonemergency and emergency settings; small groups of patients; and larger groups of patients under what is called a treatment IND.
While the new FDA rules clarify the types of access that are available, there remains flexibility as to exactly how and when these programs can be applied. This flexibility leads to what can be quite divergent and passionate opinions regarding access.