What are expanded access programs and why is it important to understand this FDA policy?
Expanded access programs - also known as early access, compassionate use or named patient programs - have been around for years. As myeloma patients we all know the story of Thalidomide. As a result of the birth defects caused by this drug, the Kefauver Harris Amendment of 1962 established a FDA regulated drug approval process. This amendment’s intent was to ensure both SAFETY and EFFICACY during drug development.
Although this regulatory process was established with patient safety at mind, there were concerns that the newly designed drug approval process would hinder timely and equitable access to potentially life-saving treatment. Consequently the FDA developed a policy to allow individuals with serious or life-threatening illness to access drugs in development outside of a clinical trial when there are no other treatment options available. This is the expanded access program.
Over the years, revisions to the original expanded access program made it easier to put in a request to try an investigational agent. Most recently, the 21st Century Cures Act of 2016 was passed. As part of this legislation drug developers are required to disclose their expanded access policy on the clinicaltrials.gov website as well as publish their policy on their own website.
Now patients can openly search for available options when in need. It’s important to note that companies are never required to provide their investigational products to patients outside of the clinical trial. It is their choice.
The eligibility criteria are usually less stringent for an expanded access program than they are for a clinical trial, but there are still strict rules involving administration and any adverse event reported during the expanded access program must be presented to the FDA. If patients are interested in seeking access to an investigational drug they should work closely with their doctor. The expanded access process cannot proceed without a licensed physician willing to act on a patient’s behalf.
For a current example, the bispecific antibody teclistamab is now available as part of an expanded access program. Teclistamab, an off the shelf bispecific antibody, has shown very promising results in highly treated relapsed/refractory myeloma. Janssen, the makers of this drug, has opened an expanded access program for this agent.
To find information about this program you can refer to the Pre-approval Access Single Patient Request for Teclisamab on the government‘s Clinical Trials website.
You can view a guest lecture by Dr. Lee, a myeloma specialist at MD Anderson Cancer Center, on Teclistmab in Health Tree University.
about the author
Cynthia Chmielewski is a professional educator and myeloma advocate. As a former teacher, she now teaches myeloma patients how to advocate for themselves as the Director of HealthTree University.