WASHINGTON — House Republicans easily passed a controversial bill aimed at helping some dying patients get new access to experimental therapies Wednesday, resurrecting legislation that suffered a major setback just a week ago.
Passage of the bill, on a largely party-line vote of 267-149, is a step forward for the”right-to-try” movement, after House Republicans failed to pass the legislation last Tuesday. Then, they had hoped to rely on an easier, quicker process typically reserved for noncontroversial bills, which requires a two-thirds vote. That day, the bill actually got more support than Wednesday, with a vote of 259-140.
It’s not clear what’s next for the legislation, which has been a priority for President Trump and Vice President Mike Pence. The Senate unanimously passed a broader right-to-try bill last fall, but did so largely to satisfy the demands of the bill’s sponsor, Sen. Ron Johnson (R-Wis.) as he held up another priority law. A single senator could similarly choose to block or hold up consideration of the now House-passed bill.
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Nice article. I am not sure what it will practically offer beyond the FDA’s current Expanded Access program which has been working well. Not likely the FDA will just expose dying patients to the kitchen sink of possible treatments. There will have to be some minimal checks and balances. You must remember, the risk a development company will be taking in providing an unproven drug to critically ill patients. Imagine the emotional response to accelerating a critically ill patients demise. The current program asks relevant questions to ensure basic alignment. As always, the devil will be in the details of deployment.
A great step in the right direction to allow the most critical ill patients gain access to not yet approved innovative drugs. The legislation ought to be amended to require drug companies to include a provision in phase III clinical studies to allow access to the experimental drug once the patients in control arm no longer benefits from the control treatment.
Thank you for passing this legislation and thank you STAT for covering the story.
the current process is too cumbersome. dr.’s don’t have the time to fill out IND trial applications and the FDA paperwork . The ” 99 % ” quote is illusory – 99 % of the very few applications that get to the FDA desk. 2500 or so approvals a year is pathetically low in a Country of 314 Million . The drugs being sought are usually late stage clinical trial drugs – remember ” Save Locky’s Dad ” ? he tried and failed to get Keytruda prior to approval. Remember ” Andis’ Army ” ? Andrea Sloan , She tried and failed to get Olaparib . Both drug are extending and saving lives .
I believe this topic may be suffering under the current weight of an environment of polarization. This is an option being proposed not an absolute..It’s implementation is similarly not binary.
Above all, there must be no reduction in Agency oversight. Ever. However, the continually cited 99% statistic cited for granting accelerated access requests addresses only those compounds that have sufficient preclinical data to be IND enabled.
There are cases, in extremis, where compounds that are less characterized (not yet under IND for any reason) have a scientific basis for use and may be a last-resort tool for the physician, for patients with no other option. This bill, by my understanding focuses on that small, defined segment of the patient population of potential benefit, where the benefit risk calculus could support trying a non IND treatment, where time does not allow generation of necessary preclinical support…responsibly.. considering clinical state of the patient, what is known about the compound, and the possible benefit versus the anticipated risk.
My thoughts only…fully admit these could be off point
The continually cited 99% statistic cited for granting accelerated access requests doesn’t mention whether a bureaucrat’s definition of “accelerated” meshes with a dying patient’s ie what percentage are approved while the patient is still alive?
Also, some doctor mentioned the paperwork burden to request approval is extremely burdensome. Some doctors may be dissuaded and not even apply.