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 firstname.lastname@example.org 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.
Our new book is a comprehensive look at a vital part of medicines development and regulatory affairs. Grab your copy today!
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
Aside from Thursday’s summit on President Barack Obama’s Precision Medicine Initiative (which can be viewed in its entirety here), the Senate and House held two other hearings focused on the inner workings of the US Food and Drug Administration (FDA).
At the Senate Homeland Security and Government Affairs Committee hearing, senators discussed FDA’s compassionate use program with the Goldwater Institute’s Dary Olsen, who’s been behind all of the Right-to-Try state legislation; the mother of a son who has Duchenne Muscular Dystrophy (DMD), a rare and fatal disease with no approved treatments, who called on FDA to approve Sarepta's DMD drug so the next drug in the company's pipeline can be approved and help her son; a young man who had to move with his family to Europe to access the osteosarcoma drug mifamurtide, which is not approved by FDA; Nancy Goodman, a woman who lost a son to pediatric brain cancer and is executive director of Kids v Cancer; and Dr. Joseph Gulfo, Executive Director, Rothman Institute of Innovation and Entrepreneurship, at Fairleigh Dickinson University.
Notably absent at the hearing was anyone from FDA or elsewhere who could defend the agency’s compassionate use program.
More than a year ago, FDA put forth a draft policy to simplify compassionate use and it took a lot of slack at Thursday’s hearing for failing to finalize the draft. And although that expedited process has yet to be finalized, witnesses at the hearing recognized that the majority of issues linked to compassionate use are related to companies that deny or are reluctant to grant compassionate use requests because others in the past have had clinical trial programs halted after complications.
Olsen was quick to note that FDA’s shortened process for completing passionate use requests won’t solve the problem of the “disincentives that companies face” to grant such requests.
She also explained to senators that 24 states have now adopted Right-to-Try legislation as a way to help patients access investigational drugs more quickly, though a Goldwater spokeswoman told Focus that it’s only aware of one doctor in the 24 states that is actually treating patients under a Right-to-Try law, and “he and his patients are not yet ready to be public.” Olsen noted in the hearing that that doctor is treating 28 patients though offered no further details.
Sen. Tom Carper (D-DE) defended FDA, noting the agency “has given an extraordinary level of attention to the requests of patients with life-threatening conditions. In fact, they’ve approved more than 99 percent of requests for emergency treatments. Despite these high approval rates, I understand that the FDA believes more can be done and is continuing to work to improve patient access to these experimental medical treatments.”
Olsen also discussed Sen. Ted Cruz’s (R-TX) bill, which Sen. Ron Johnson (R-WI) called a "non-brainer," to allow drugs on the US market that are approved elsewhere (though as it’s been noted by Focus and the Harvard Bill of Health blog, there are very few drugs on the market in the EU that aren’t in the US and most of those kept off the US market are for safety reasons, and the idea that Congress could override FDA presents a number of concerns).
Gulfo, who has called for FDA’s role to be more limited in imposing standards requiring the demonstration of clinical utility for approval, also discussed the realities of pediatric cancers and the difficulty for pediatric patients in meeting entry criteria for trials.
On the House side, the Appropriations Agriculture Subcommittee grilled former acting FDA commissioner Stephen Ostroff mostly on food safety issues and the FY2017 budget request, though the conversation did veer into talk of drug and device regulations.
When asked about FDA finalizing a new generic drug labeling rule, which is expected to come in July, Ostroff noted that FDA’s primary consideration is not related to litigation but the “need to make sure the labels are focused on safety.”
He added that the labeling issue is complicated because for many products “there are multiple generics so we believe it’s important to be consistent across all of the various forms of a drug. One manufacturer might have access to information that others don’t. Or the reference product might not be marketed anymore. Moving information between companies can be challenging.”
Rep. Rosa DeLauro (D-CT) also criticized Ostroff for FDA’s investigation of the birth control device Essure, which is due to be completed by the end of this month. DeLauro said Essure needs to be pulled from the market while the investigation is ongoing as thousands of women have had specific incidents.
And near the end of the hearing, Rep. Sam Farr (D-CA) questioned the impact a provision in the omnibus spending package that prevents FDA from reviewing applications related to genome-editing tools to modify the DNA of human embryos “until serious and unquantifiable safety and ethical issues can be resolved.”
Ostroff said the situation is a sensitive one and benefits and risks need to be weighed, which is why FDA commissioned the recent report from the Institute of Medicine on mitochondrial replacement therapy.
Farr said the provision was intended “to stop designer babies” though he thought the actual language of it went too far and would mean the US will lose research opportunities.
House Subcommittee FDA Budget Hearing
Tags: Essure, compassionate use, right-to-try, Ostroff, Ted Cruz, genetic manipulation