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Analysis

FDA Launches Pilot Program for Unapproved Cancer Therapies

By John Commins  
   June 03, 2019

Expanded Access provides patients with life-threatening diseases access to experimental medical products, including drugs, and biologic or medical devices.

The Food and Drug Administration on Monday announced a new initiative to help oncologists expedite unapproved and experimental therapies for cancer patients who've "exhausted available treatments."

The new Project Facilitate will provide a single point of contact between the FDA's Oncology Center of Excellence and oncologists in the field who submit Expanded Access requests for patients, FDA said.

"The first option for patients who have exhausted available treatments is to enroll in a clinical trial," Acting FDA Commissioner Ned Sharpless, MD, said in a media release. "But when that is not an option, we support Expanded Access and are exploring ways to make it easier for patients, their families and health care professionals to understand the process and how to access investigational therapies."

Expanded Access provides patients with life-threatening diseases access to experimental medical products, including drugs, and biologic or medical devices for treatment outside of clinical trials when there are no comparable or satisfactory alternative therapy options available, FDA said.

Because the treatments are not FDA approved, the agency's role in Expanded Access will be to weigh the potential benefit of an experimental treatment against the potential risks.

Under the program, physicians will ask the drug or device maker to provide the unapproved product, which the company can either approve or deny.

Related: FDA Eases Paperwork To Help Some Patients Get Experimental Drugs

While the FDA said it authorizes "the vast majority" of these requests, Sharpless said that "for many patients or healthcare professionals, especially those not familiar with the Expanded Access program, the process may appear confusing or burdensome."

"Ultimately, a patient cannot submit an application for an investigational medical product; only a qualified physician is able to officially make the request," said Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence.

"Through this pilot program, experienced FDA oncology staff will be available to support physicians and other healthcare professionals with their questions, assist in filling out the appropriate paperwork and acting as a facilitator for the process," Pazdur said.   

“Experienced FDA oncology staff will be available to support physicians and other healthcare professionals with their questions, assist in filling out the appropriate paperwork and acting as a facilitator for the process”

John Commins is a content specialist and online news editor for HealthLeaders, a Simplify Compliance brand.


KEY TAKEAWAYS

Expanded Access facilitates treatment outside of clinical trials when there are no alternative therapy options.

Because the treatments are not FDA approved, the agency will weigh the potential benefit of an experimental treatment against the potential risks.

The pilot is the latest initiative in FDA's ongoing efforts to streamline an otherwise complex and confusing process.


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