FDA Proposes New Right to Try Requirements Including Annual Reports from Life Science Companies

In a recently proposed rule, the FDA proposed Right to Try law requirements. The agency will require drug manufacturers to issue annual reports including the number of doses supplied and their use, the number of patients treated, and any serious adverse effects from drug use. The agency has previously stated it lacks necessary transparency around the law’s effectiveness in expanding access to experimental treatment. The FDA would also post online an annual summary report based on information acquired. The FDA will accept comments on the proposed rule for 60 days after it is published in the federal register.

Proposal

 In a release with the proposal, Anand Shah, M.D., FDA’s Deputy Commissioner for Medical and Scientific Affairs said the agency “is dedicated to achieving the goals that Congress set forth in the Right to Try Act, so that patients facing terminal conditions have another avenue to access investigational medicines. Today’s proposed rule builds on the FDA’s long-standing dedication to enhancing access for patients who are facing life-threatening diseases or conditions and our continued commitment to transparency.”

The Right to Try Act, or the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act of 2017, provides a pathway for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial, to access certain unapproved treatments. Ultimately, the sponsor or manufacturer who is developing the drug or biologic, not the FDA, is responsible for determining whether to make their product available to patients who qualify for access under the Right to Try Act.

The proposed rule implements a statutory requirement for sponsors and drug manufacturers to provide an annual summary to the FDA for any eligible investigational drug they provide to eligible patients under the Right to Try Act. The Summary Reporting Requirements Under the Right to Try Act are intended to provide more transparency around how patients are using experimental drugs through annual reporting requirements. The requirements also are designed to help FDA assess the effectiveness of the Right to Try pathway at opening access to innovative drugs. Since last fall, there have only been two publicly reported cases of the Right to Try Act authority. The FDA has stated that it lacked necessary information on the number of patients that had accessed or requested pharmaceuticals under the law.