Right to Try One Year Later: Limited Patient Involvement but More FDA Clarity Coming

Regulatory NewsRegulatory News | 30 May 2019 |  By 

The Right to Try Act has officially been in place for one year and although just two patients have publicly announced that they have used the law to gain access to experimental therapies, hundreds more may follow.

The US Food and Drug Administration (FDA) on Thursday offered a new website to explain what patients and what investigational drugs are eligible under the law. The website includes eight questions and answers on the law, clarifying that companies are not required to provide access to their investigational treatments under the law, and that a drug under a clinical hold is not an eligible investigational drug and cannot be provided under the Right to Try Act.

In addition, a proposed FDA rulemaking is expected in September and would require an annual summary from sponsors and manufacturers who use Right to Try to provide an eligible investigational drug for use by an eligible patient. FDA also said it will post online a consolidated annual summary report of Right to Try Act use.

As for the two examples of patients accessing investigational drugs via Right to Try — the first involved a California patient diagnosed with an aggressive form of brain cancer, while the second patient gained access to an ALS drug from a company, known as Brainstorm, which later said it will not offer the drug to other patients under Right to Try or FDA’s expanded access program.

But the Goldwater Institute, a libertarian organization behind the new law from the beginning, told Focus that other patients are accessing investigational drugs under the law but have not been publicly revealed.

“While there is a legal requirement to report instances of treatment under Right to Try to the federal government, and we are aware of other patients who are currently receiving treatment under the law, the question of who has been treated under the law can only be answered by the government,” Goldwater said. “We look forward to the Secretary's promulgation of the Right to Try reporting rule which will provide clear directions for those companies that are moving forward with treatment under the Right to Try law.”

And plans for more patients to use the law are gearing up.

Richard Garr, CEO of Beacon of Hope, a niche contract research organization dedicated exclusively to Right to Try treatment programs, told Focus via email that he’s spent the past six months building a network of national and regional clinics, hospital chains and physician practices that will treat patients under Right to Try.

“They run the gamut from various oncology indications to ALS and Alzheimer's and even some psychiatric diseases that can lead to suicide and thus are potentially fatal,” he said. 

And by the end of June, Garr expects to launch a website and patient-facing portal. Patients will begin to be treated for several indications under the law later this summer, he said.

“The first to come on-line will be an ALS program and I expect to treat between 200 and 300 patients for this particular program over the next 12 months in the Southeast. The sponsor may expand the program nationally,” he added. “Companies are doing this in exchange for data which provide pre-approval insights to guide their traditional clinical trial programs. And as we are doing this at scale, I expect it will impact large numbers of patients.”

But he did not offer any specifics on companies offering treatments, explaining all programs are "confidential, and only the sponsors can make their information public if they so chose."  

Alison Bateman-House, assistant professor in the Department of Population Health at New York University, also explained to Focus that companies may be hesitant to use the new law because they do not want to cut FDA out of the process.

“If patients die or are seriously injured, they can say FDA was on board and it wasn’t just us being cowboys. I have yet to encounter a company saying they don’t need FDA involvement,” she said.

And companies are continuing to use FDA’s expanded access program in much larger numbers than the Right to Try law. For instance, in Fiscal Year 2018, more than 1,000 individuals gained access to investigational drugs through FDA’s expanded access program.

Bateman-House said she does not foresee Right to Try ever approaching the number of patients who access investigational drugs via expanded access. But the main benefit of the new law, she added, is that more people are now aware that they can access investigational drugs under FDA’s expanded access program.


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