FDA Streamlines Expanded Access Application for Patients to Get Investigational Drugs

Nick Bryant

July 2016, Vol 7, No 6 - Health Policy


The Individual Patient Expanded Access Application, which is designed to streamline the application procedure for individual patients who apply for expanded access to investigational therapeutics, including expanded access to drugs that are not in clinical trials, was recently updated by the FDA. Form FDA 3926 authorizes expanded access to investigational drugs for patients with serious or life-threatening conditions who have no therapeutic options.

“Access to investigational treatments requires the active cooperation of the FDA, industry, and health care professionals in order to be successful,” said FDA Commissioner Robert Califf, MD, in a statement. “But we know that navigating that process can be challenging and time consuming, and we are committed to reducing the procedural burdens on physicians and patients whenever possible.”

The FDA anticipates that the new application will take 45 minutes to complete, instead of the approximate 100 hours that the previous form required.

Along with the new application, the FDA released step-by-step instructions detailing how to complete the application, as well as a final guidance document outlining when and how to request expanded access. In addition, a document explaining how drug sponsors or manufacturers can charge when investigational drugs are used indicated that they will not be able to charge for indirect costs; indirect costs include those associated with developing treatment protocol, reporting to the FDA, or corresponding with the Institutional Review Board (IRB) or the drug manufacturer.

Over the years, the FDA has been supportive of patients receiving expanded access medications. Between 2010 and 2014, the FDA granted 5962 of 5995 requests for expanded access, which is a 99% approval rate.

Although the vast majority of expanded access requests are approved, the FDA is often criticized for how long it takes to move applications through the process. Although the updated form will mitigate some of these criticisms, there are still additional FDA requirements in place that may delay or prevent patients from receiving time-­sensitive treatments.

Before patients can apply for expanded access, their physician must first obtain an authorization letter from the drug manufacturer. However, drug manufacturers are not required to offer expanded access to their drugs that are in clinical trials, and possible lawsuits may dissuade them from allowing access to these and other medications with a Risk Evaluation and Mitigation Strategies program.

Once an application is submitted, the FDA must first determine that there is no “comparable” or “satisfactory alternative” therapy to grant expanded access. Next, the FDA mandates that a patient’s prospective administration of a medication be reviewed by an IRB to confirm that the risks posed by the medication are commensurate with its possible benefits. Last, the FDA’s expanded access program allows pharmaceutical manufacturers to charge patients for their drugs. The latter FDA proviso could represent a significant barrier for patients, especially if third-party payers decline to reimburse patients for experimental medications.

The Individual Patient Expanded Access Application comes in the wake of several states drafting “Right to Try” guidelines, which are intended to circumvent FDA restrictions and provide terminally ill patients with access to experimental treatments. Opponents of Right to Try, including the American Society of Clinical Oncology, have posited that Right to Try exposes patients to possibly toxic drugs that have an unproven medical benefit and have the potential to hinder patient enrollment in clinical trials.

Unlike Right to Try, the FDA expanded access criteria are more restrictive concerning the drugs a patient may receive, and the FDA also requires an IRB to oversee the expanded access process. Consequently, the FDA will continue to offer safer guidelines than the states’ Right to Try initiatives, but the Individual Patient Expanded Access Application will concomitantly expedite patients’ requests for experimental program.