Following President Trump’s decision to sign new Right to Try Legislation which bypasses the FDA into law, the regulator is making changes to its own Expanded Access program for compassionate use of drugs. Among these improvements is a shift to a simplified documentation process and a streamlined process for Institutional Review Board (IRB) evaluation of each application which could allow patients to be granted access to experimental new therapies faster.
The FDA’s Expanded Access program is designed to help patients who are terminally ill and have exhausted all available treatment options gain access to investigational new drugs outside of a clinical trial setting. Despite the criticisms this program has faced – largely from supporters of the new Right to Try rules – the FDA estimates that 99 percent of all Expanded Access requests are approved, with over 9,000 applications being authorized in the last five years alone.
“As a cancer survivor, I understand, on a very personal level, that patients who are fighting serious or life-threatening diseases want the flexibility to try new therapeutic approaches, including investigational medical products,” said FDA Commissioner Scott Gottlieb. “This is especially relevant when there’s no other FDA-approved treatment option available to a patient.”
While the program has had its successes, Gottlieb agrees that improvements could be made. Taking suggestions from Congress and other stakeholders, the agency has already put a few of these program updates into effect.
For one, the FDA has reduced the amount of supporting documentation that needs to be completed and submitted by the patient’s treating physician as part of the Expanded Access request. As a result, the patient application form can be completed in less than an hour, in a move that Gottlieb says reduces the administrative burden on the physician.
What’s more, the new program no longer requires the entire IRB to agree that the patient should be allowed to access the experimental therapy; instead, the go-ahead from just one IRB member will be sufficient.
Since the Expanded Access program has implications for the drugmakers themselves, the FDA has also made provisions to ensure that the compassionate use of a drug – and any safety data that might generate – does not interfere with the drug’s clinical development. While adverse events that occur when a patient is provided with expanded access to a drug will still need to be reported to the FDA, the regulator has never made the decision not to approve a drug based on this safety data alone. However, since the FDA cannot force a pharmaceutical company to provide expanded access to a drug, the hope is that these clarifications will be enough to help drugmakers make the decision that’s best for the patient as to whether to provide the drug.
While Gottlieb was previously skeptical of the new Right to Try act, he seems to be willing to support the legislation now that it has been signed into law.
“It’s important to note that while expanded access represents FDA’s primary avenue for facilitating access for certain patients to unapproved, investigational treatments, the ‘Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017,’ recently signed into law by the President, represents a separate and distinct pathway,” said Gottlieb. “The FDA has established a work group to consider what steps may be required to implement this legislation in a way that advances Congress’ intent to promote access and protect patients. As a first step, today we are launching a Right to Try webpage that will assist patients in understanding this alternative pathway.”