Patient Advocacy Organizations Outline Joint Position on Expanded Access to Experimental Drugs for Seriously Ill Patients

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WASHINGTON, D.C., May 2, 2017–Seven patient advocacy organizations today laid out a joint set of principles to guide any efforts that seek to change the process of accessing unapproved therapies outside of a clinical trial, also known as compassionate use or expanded access. The patient advocacy organizations include: Alliance for Aging Research, American Cancer Society Cancer Action Network, Friends of Cancer Research, Leukemia and Lymphoma Society, National Organization for Rare Disorders, Parent Project Muscular Dystrophy and St. Baldrick’s Foundation.

The organizations issued the following statement:

“Since the 1980s, patients with serious diseases that have no options for effective treatments and who do not qualify for clinical trials have been able to access unapproved therapies through a process known as expanded access or compassionate use. We support this process and are committed to making it as efficient as possible.

“Unapproved therapies have not been shown to be safe and effective, which is why there is a strong set of patient protections in place for clinical research to ensure safe and ethical use of these therapies.

As policymakers consider changes to the compassionate use process, it is imperative that patients seeking expanded access to unapproved therapies be treated with the same ethical standards and protections as patients taking part in clinical trials.

“Ultimately, the goal is to get safe and effective therapies on the market and to patients as soon as possible. Well-designed clinical trials are the most expedited way to demonstrate a therapy’s safety and efficacy, thereby achieving approval by the U.S. Food and Drug Administration (FDA).

“We urge drug sponsors to be as inclusive as possible with their clinical trials to reduce the need for patients to seek access to unapproved therapies outside of clinical trials. When trial participation is not an option for seriously ill patients, and expanded access is sought, review by an institutional review board and the FDA is essential to allow an informed third-party to ensure that use of the unapproved therapy is ethically and scientifically sound.

“The goal of these principles is to help keep the focus on patients and their needs, and we look forward to working with Congress and the administration to ensure that an efficient expanded access process continues to provide options for patients with serious or life-threatening conditions and their families.”