FDA issues final rule on ‘right to try’ reporting requirements

Regulatory NewsRegulatory News | 14 September 2022 |  By 

Drug firms that agree to supply investigational medicines under the Right to Try Act must submit an annual summary detailing each use to FDA under a final rule issued this week.
 
The final rule specifies that manufacturers include details of the number of doses supplied, the number of patients treated, the use for which the drug was made available and any known serious adverse events that take place.
 
The rule also establishes when manufacturers need to submit reports. FDA said it has “extended the submission date for the first annual summary report from 60 calendar days after the final rule becomes effective as proposed to a specific date of March 31, 2023.”
 
The agency added that “this rule specifies that submissions must be made electronically. Currently, this means attaching a PDF document to an email. In the future, FDA may move to electronic submission through other direct means.”
 
Right to Try
 
The Right to Try Act – which was signed into law in May 2018 – amended the Federal Food, Drug, and Cosmetic Act (FD&C Act) to establish an option for patients meeting certain criteria –people diagnosed with life-threatening diseases who have tried all approved treatments and cannot take part in trials – to request access to unapproved drugs.
 
The legislation is similar to the Expanded Access programme in principle, with a few key differences. Under the right to try legislation, FDA consent is not required whereas in the Expanded Access programme FDA must determine whether the drug is likely to be of benefit.
 
Also, experimental treatments provided through right to try do not need to be approved by an Institutional Review Board, which is required for medicines issued under the Expanded Access programme.
 
Separate reports
 
In the final rule FDA also shares details of comments, questions and suggestions it received from industry. Several urged the agency to consider combining the right to try reports with and Expanded Access reports or even as an addendum within investigational new drug (IND) annual reports.
 
FDA rejected the idea on the searching for right to try information within larger reports would be an inefficient use of resources.
 
“It would be extremely difficult and resource-intensive for FDA to examine all IND annual reports for the sole purpose of identifying those potentially few reports that have Right to Try information so that we can compile the annual summary required by section 561B(d)(2) of the FD&C Act.
 
“In addition, it is efficient to have separate reporting requirements for Right to Try Act and other investigational drug uses because section 561B(c) of the FD&C Act limits FDA’s use of clinical outcomes associated with the use of eligible investigational drugs under the Right to Try Act in ways that are not applicable to other uses of INDs,” FDA wrote.
 
FDA added that “For these reasons, it is more efficient to implement the annual reporting and summary requirements of the Right to Try Act by requiring the annual reports to be submitted as separate reports to FDA.”
 
Manufacturers not contractors
 
The Final Rule also makes clear that, under right to try, contract manufacturers are not expected to file annual reports.
 
FDA said, “A contract manufacturing organization that is not closely connected to the clinical investigation and approval process should not be considered a ‘manufacturer or sponsor’ under this rule, and therefore we have updated the regulatory text to specify that a contract manufacturer is not a ‘manufacturer or sponsor.’”
 
The agency explained that only those entities closely connected to the clinical investigation or approval process should submit annual summaries.
 
It added, “A manufacturer or sponsor is better positioned to have access to the relevant data required for the annual summary if their role is not merely to manufacture a drug to another entity’s specifications on behalf of the other entity.
 
“We expect that whenever a drug is distributed under Right to Try, there will be a manufacturer or sponsor with access to the relevant data who will submit the required annual summary.”

 

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