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.
Patient access to investigational drugs was the topic of a recent forum held in Washington, D.C., where representatives from FDA, industry, advocacy groups, the medical profession, and the bioethics community gathered to share their perspectives. It was clear from the forum that each situation in which expanded access may be leveraged has its own unique dynamic and requires thoughtful consideration of patient needs, the company’s situation, and regulatory guidelines. And while the new FDA regulations provide an effective framework for access, many questions remain:
- When should access be allowed—after Phase I trials, when safety has been established, or later in the trial process when data on efficacy is available?
- Who should get access? Must a patient be terminally ill? How do we define “terminally ill”?
- In the case of a cancer drug, must the patient seeking access have the type of cancer the drug is being tested against in trials? What is considered fair when choosing which patients can get access and which cannot?
- How do we appropriately balance the needs of desperately ill patients with safety concerns?
- How do we ensure expanded access does not put clinical trial enrollment at risk?
Ultimately, the choice to offer expanded access, or not, is left up to the drug developer. FDA regulations do not force companies to offer access to their investigational drugs. When considering this option, however, companies must undertake a thorough evaluation of important questions such as when to offer access and for which patients.