In today’s prepublication of the Federal Register appears the final rule entitled Annual Summary Reporting Requirements Under the Right to Try Act (here). The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina, Right to Try Act of 2017 (Right to Try Act), was signed into law on May 30, 2018. This Act provides an alternative option to patients “who meet certain criteria to request access to certain unapproved drug products and for sponsors and manufacturers who agree to provide those certain unapproved drug products, other than through FDA’s expanded access program.”

The FR notice indicates that “[T[his law provides a new pathway for patients to request and manufacturers or sponsors to choose to provide access to certain unapproved, investigational drugs, including biological products, for patients diagnosed with life-threatening diseases or conditions as defined in § 312.81 (21 CFR 312.81) who, as certified by a physician, have exhausted approved treatment options and who are unable to participate in a clinical trial involving the investigational drug.”

“This rule law does not require that physician determinations be submitted to FDA”, however, it does require the submission of certain data to the FDA on an annual basis which include:

  • the number of doses supplied of an eligible investigational drug
  • the number of patients treated
  • the uses for which the drug was made available
  • and any known serious adverse events.

 

In addition, the law requires FDA to specify through regulation the deadline for submitting the annual summary.  That date is being changed from 60 days from the rule being published to a specific date of March 31, 2023, but the first report should include all data from the passage date of the act through December 31, 2022.  FDA notes that “[A]fter this annual report, the manufacturer or sponsor must submit a report that covers every January 1 through December 31 annual period by no later than March of the following year”. Quite interestingly, the summary must be submitted in electronic form, but because the FDA does not yet have an established system for receiving those reports the FR notice indicated that “[C]urrently, this means attaching a PDF document to an email.” Also noting that in the future the FDA may move to electronic submission through other means.

The FDA received fewer than 50 comments on the proposed rule and they exhaustively go through those responses and the Agency’s reasoning for acceptance or rejection of the comments. The rule is very self-explanatory and provides a reasoned thinking on requirements for industry.