US: Right to Try one year later - limited patient involvement but mre FDA clarity coming


US: Right to Try one year later - limited patient involvement but mre FDA clarity coming

(Source: RAPS 2019-06-04)

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 Focusthat 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.

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