Monday, June 3, 2019

FDA to help some terminally-ill patients access unapproved drugs


Move comes as advocates push for broader reforms that help patients with other life-threatening illnesses



The Food and Drug Administration will provide “concierge service” to some doctors seeking access to unapproved cancer drugs, the agency announced Monday.

Patients, caretakers, & legislative
leaders pose for a photo at the Right 
To Try signing ceremony in 2018.
The goal is to remove hurdles for physicians who want to use the agency’s “expanded access” program, said Richard Pazdur, director of the agency’s Oncology Center for Excellence. The pilot program includes a new call center that will serve as a single point of contact for oncologists submitting requests. 

The FDA has worked to streamline its antiquated access program in recent years in response to criticism by patient rights advocates, but the process is still too burdensome for most doctors to complete.

“The FDA taking this step is an admission of a big problem with the expanded access process,” said Matt Bellina, a Navy veteran and ALS patient who is receiving treatment under the new federal Right To Try law. “Unfortunately this project doesn't address the real barriers to patient access and inexplicably ignores every disease outside of cancer.”

At the urging of patients and advocacy groups, last year Congress passed the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act, which allows doctors and patients to completely bypass the outdated compassionate use program. 

Alison Bateman-House, assistant professor in the Department of Population Health at New York University, explains that some pharmaceutical companies still prefer the old program because it allows them to shirk responsibility when patients have adverse reactions.

“If patients die or are seriously injured, they can say FDA was on board and it wasn’t just us being cowboys,” she said last week.

The FDA’s new pilot project was announced at the annual meeting of the American Society of Clinical Oncology in Chicago.