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Regulatory News | 03 August 2017 | By Zachary Brennan
The Senate on Thursday passed via unanimous consent (meaning there was not actually a vote) a bill that claims to improve terminal patients' access to experimental drugs but, if passed in the House and signed into law, may further burden and undermine the US Food and Drug Administration (FDA).
The bill is part of what's known as the "Right-to-Try" movement, which has been sweeping across state legislatures (37 states now have such laws), and is part of a bid to increase access to experimental therapies for terminally ill patients while skirting around FDA, which regulates the use of investigational medical products outside of clinical trials under its expanded access program.
The libertarian Goldwater Institute, which praised the Senate bill's passage as protecting "doctors and pharmaceutical companies who come to patients' aid," has said that Houston-based oncologist Ebrahim Delpassand said he successfully treated 78 terminally ill cancer patients under Texas's Right-to-Try law, though he did not respond to a request for comment to verify. Delpassand is listed in Clinicaltrials.gov as sponsoring a Phase II trial, the status of which has not been updated in more than two years, offering 177Lu-DOTATATE therapy to treat lung, breast, gastrointestinal tract, skin and endocrine cancers.
Alison Bateman-House, an assistant professor in the Division of Medical Ethics at New York University Langone Medical Center, told Focus she also could not verify Delpassand's use of Right-to-Try or any other instances of the law enabling patients to access experimental treatments that they could not already access under FDA's expanded access program.
"The one benefit of the Right-to-Try [RTT] movement, as I see it, is that it made patients and doctors aware that they could seek access to investigational drugs outside of clinical trials in certain circumstances," Bateman-House said. "I think pharmaceutical companies will have a surge of requests. I think companies that already had expanded access experience/policies will treat handle RTT requests as always: via the expanded access pathway … So, I don't see RTT increasing patient access to drugs. If anything, I see it decreasing access because companies and hospitals (and other institutions where patients may seek to be treated with experimental drugs, like research centers) will be conservative if they fear doing something could harm them."
According to experts, the main barrier for patients trying to access such investigational treatments is the companies running the clinical trials for these investigational therapies, but "Right-to-Try" legislation does not address this barrier.
FDA, meanwhile, signs off on more than 99% of expanded access requests, as the agency allows such use after a company gives the OK.
At the heart of the revised Senate bill is a section on whether FDA can use clinical outcomes from trials in which the use of an investigational drug led to safety events that might delay or cause FDA to reject its approval.
The bill, a previous version of which barred FDA from even considering such data, now says that FDA can use the data if "that use of such clinical outcome is critical to determining the safety of the eligible investigational drug."
And if such a determination is made, an agency director at FDA would have to provide "written notice of such determination to the sponsor, including a public health justification for such determination."
The bill also makes clear companies, prescribers, dispensers and others will not be held liable with respect to "any alleged act or omission with respect to an eligible investigational drug provided to an eligible patient pursuant to section 561B of the Federal Food, Drug, and Cosmetic Act."
In addition, the bill would increase transparency around the use of investigational drugs as the manufacturer or sponsor of an eligible investigational drug shall submit "an annual summary of any use of such drug under this section. The summary shall include the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events."
FDA will then post an annual summary report of the use of this law on its website.
S. 204: Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017
Tags: Right-to-Try, right to try legislation, Senate right to try