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  • Antero Pharma Consultant

Expanded Access Program - overview and notes



What is an Expanded Access program?

Expanded access or Compassionate use is the FDA program by which a doctor or registered physician can use an investigational drug (unapproved drug) on a patient suffering from a severe or immediately life-threatening disease. The patient care team can leverage this pathway as the last option to save the patient’s life by using an investigational drug since all the approved FDA medications have been tried and no comparable or satisfactory therapeutic alternative is available.


Industry and FDA’s perspective on this program

Manufacturers or Industry sponsors have shown reluctance to use their drug in expanded access for several reasons that may jeopardize their goal of bringing this drug into the market, such as inadequacy of safety and efficacy data, financial and resource investment in drug development, potential risk of profound side effects caused by the drug. Ultimately, the drug is still in the development phase. The data needed to make sense of side effects at this stage is limited.

Similarly, the FDA is also concerned with the emergency use of an investigational drug but has always been committed to raising awareness about expanded access while protecting the public's health and rights. FDA suggests that, wherever possible, the patient must be part of a clinical trial; however, sometimes conditions may vary, such as when there are no ongoing clinical trials for a life-threatening indication, the patient does not have access to a clinical trial due to distance from the clinical trial site or may not be eligible for the trial. In such cases, expanded access is an appropriate pathway by which any patient with a serious condition can be referred to an IND investigational drug by a licensed physician. The FDA, however, states that even if the patient meets the FDA's laws and regulations, it is mandatory that the patient, their doctor, the institutional review board, and the drug company or sponsor mutually agree to decide on expanded access.


Procedure to apply for expanded access program

Physicians can submit an initial application for an unapproved investigational drug for single or multiple patients. Once the drug manufacturer or drug sponsor agrees to provide the use of their unapproved investigational drug, registered physicians can request via a call or email to the FDA office. Form 3926 and a letter of authorization from the manufacturer must be submitted electronically. The FDA evaluates the request and recommends a "hold" or approves it. For most emergencies, the FDA approves 99.9% of expanded access requests.

Health Canada and European agencies have similar kinds of programs with different terminology called Special Access Program (SAP) and Compassionate Use, respectively, to bridge the unavailability of marketed medicines for life-threatening severe, long-lasting, or seriously debilitating illnesses.

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