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Legislation removes FDA involvement when patients seek experimental medications. Here’s what critics and proponents say.
Some patients with life-threatening diseases will now be able to receive experimental treatments without receiving approval for their use from the FDA first. That’s because President Trump, on May 30, signed the Right-to-Try Act into law. The signing came shortly after the House passed the legislation May 22. The Senate passed the legislation in August 2017. Under the legislation, patients can access experimental treatments if they are unable to participate in any clinical trial involving an investigational drug and if they have exhausted all other approved treatment options. The bill requires that any unapproved drug must have an active application and not be subject to clinical hold. In addition, the government may not use a clinical outcome associated with use under the legislation to delay or adversely affect a product’s review or approval, unless it is critical to determining its safety or the sponsor requests use of the outcome. Proponents and critics, who we spoke to prior to the president’s actions, have a lot to say about the potential effects.
Right to Try vs. Expanded Access: Under the FDA’s Expanded Access Program, patients under certain circumstances can take experiential drugs that have not yet been FDA approved; however, the FDA must sanction their use. The agency says it approves 99% of the more than 1,000 annual requests for experimental drugs requested through the program.
What proponents say
Starlee Coleman, senior policy advisor, Goldwater Institute, says the FDA is the ultimate arbiter of which drugs should be made available. Still, she believes that the FDA approval requirement under the Expanded Access Program is too time consuming (it requires doctor/patient application to the FDA for a single patient investigational new drug treatment and approval from an independent review board), especially for terminal patients.
“We don’t think Americans should have to apply to the government to save their lives,” she says. “The government shouldn’t be able to say ‘no’ if a manufacturer and doctor approve. Right to Try provides access to potentially life-saving treatment for patients without other options who cannot get into a trial.”
Coleman says drugs available through Right to Try have passed phase 1 trials and continue to be tested, making them safe enough for terminal patients while offering possible benefits. “Right to Try could be particularly beneficial if a trial ends with positive results, but a drug has not yet been approved,” she says.
She also emphasizes that Right to Try is voluntary for patients, doctors, and manufacturers.
Next: What opponents say
What opponents say
“The most virulent opponents of federal Right-to-Try legislation say that it is not significantly different from expanded use but is simply part of an effort to reduce the regulatory scope of the FDA and the federal government in general,” says Harold Bishop, senior legal analyst, Health Law Group, Wolters Kluwer, a global information company.
Other criticisms, according to Bishop include:
A January article in the New England Journal of Medicine, outlines other objections, including:
Arthur Caplan, director, Division of Medical Ethics, New York University’s Langone Medical Center, says proponents of Right to Try believe the drug approval process is long and slow and if a patient is dying, there is no time for patients to enter a clinical trial because they might not qualify if they are too sick.
He isn’t convinced, however, that the FDA is preventing patient access to experimental drugs, as proponents assert. “It’s drug manufacturers who decide who gets a drug, but instead of doing anything to help persuade companies to make their products available to patients, which is the key for access, they (proponents) set their sights on the FDA and accuse it of keeping patients from promising experimental medicines,” Caplan wrote in a recent Forbes article.
Amy K. Dow, member of law firm Epstein Becker Green, agrees. “Proponents of Right-to-Try legislation seem to overlook the fact that the FDA is not, in general, a significant barrier to access to products. Rather, it approves the vast majority of expanded access requests it receives,” says Dow.
The efforts by supporters of Right-to-Try legislation to remove FDA from the process of providing patients with access to investigational therapies eliminates an important element of oversight provided by the agency but is unlikely to significantly impact the number of patients obtaining access to these products, she adds.
She says limitations on supplies of investigational products and burdens imposed on manufacturers in responding to drug requests are not addressed by the legislation.
Under Expanded Access and Right to Try, manufacturers may choose whether to make an investigational drug available. Brookings, a public policy organization based in Washington, D.C., suggests that drug manufacturers are hesitant to provide preapproval access because of a limited supply-only enough available for a clinical trial-and if a patient suffers adverse effects after taking a drug, that might deter other patients from participation in clinical trials or from purchasing the product after FDA approval.
The Pharmaceutical Researchers and Manufacturers of America, a trade group representing pharma companies, has not taken a formal position on Right-to-Try at press time. Still it has created criteria for determining when to grant access to an experimental treatment outside of a clinical trial, including if a patient has a serious or life-threatening illness and is ineligible for, or otherwise unable to participate in, a clinical trial.