Welcome to our new website! If this is the first time you are logging in on the new site, you will need to reset your password. Please contact us at email@example.com if you need assistance.
Your membership opens the door to free learning resources on demand. Check out the Member Knowledge Center for free webcasts, publications and online courses.
The highly-anticipated fifth edition is here! Get the must-have resource for achieving compliance with medical device regulations.
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
The House Energy & Commerce Committee on Tuesday held a hearing to discuss a "Right-to-Try" bill passed in the Senate that the US Food and Drug Administration (FDA) is seeking to change.
The hearing discussed a bill that would seek to undercut FDA’s oversight of a program, known as expanded access, whereby terminally ill patients and their doctors can request access to an investigational drug outside of a clinical trial.
Right-to-Try laws have now passed in 37 states (and the US Senate) though proponents of the legislation, including the Goldwater Institute, are seeking to expand their initiative as a way to help terminally ill patients access drugs they might not be able to obtain. However, to date, only one doctor has used his state’s Right-to-Try law. Similarly, FDA has said only 30% of drugs accessed via expanded access go on to win approval.
Several House reps on Tuesday framed the debate over Right-to-Try as a case where patients with terminal or life-threatening diseases need a better system, or even one where FDA is not stepping in between doctors and patients.
But no provisions within the bill would require companies developing these investigational treatments to provide them to patients, and FDA has approved more than 99% of all expanded access requests it receives.
FDA Commissioner Scott Gottlieb, while acknowledging that the perception of FDA’s expanded access program "isn’t perfect," told the committee of a few changes the agency is working to address and that one of the primary reasons patients cannot obtain these investigational medicines is that a company lacks supply of the treatment.
Only a couple of situations have arisen where an expanded access program has had an impact on a drug’s development (one is this partial clinical hold for CytRx’s aldoxorubicin trial) and Gottlieb acknowledged that expanded access guidance in the form of a Q&A has been updated to address how the agency reviews adverse event data (as requested by GAO), "to clarify the IRB review requirements for individual patient expanded access treatment," and to reference the 21st Century Cures Act requirement that expanded access policies be publicly posted.
In addition to the updated guidance, the Reagan-Udall Foundation has put together an expanded access navigator to help patients identify potential treatments if their options are limited.
In his written testimony, Gottlieb also sought specific changes to the bill: "If enacted without changes, sponsors and other persons providing eligible investigational drugs to eligible patients under the specified conditions of the bill would not be subject to a number of sections of the FD&C Act and FDA regulations related to clinical trials, premarket approval, and labeling. The current language in S. 204 may therefore preclude FDA from taking enforcement actions based on those provisions."
And in both his written and verbal comments, Gottlieb recommended narrowing the eligibility of the bill from patients who face a "life-threatening disease or condition" to those facing "terminal illness."
"If the goal is to help terminal patients – the more conditions we sweep in, the more we risk undermining the whole venture to be tailored to those who don’t have good options," he said.
Gottlieb did not respond and said later: "The perception is that this legislation will pressure companies to supply more expanded access drugs but that remains to be seen."
Examining Patient Access to Investigational Drugs
Tags: Right-to-try, E&C Committee, Gottlieb, GAO, expanded access