As many of you are aware, the 21st Century Cures Act (Pub. L. 114-255) (“Cures Act”) was signed into law by President Barack Obama on December 13, 2016.

Under Section 3032 of the Cures Act (“Section 3032”), pharmaceutical companies are generally required to have publicly available policies explaining how the company evaluates and responds to patient requests for certain experimental drugs outside of clinical trials. The deadline for adopting these policies with respect to a particular investigational drug is the later of February 12, 2017 or the first initiation of a phase 2 or 3 study of the investigational drug. For those of you who have investigational drugs that are already in phase 2 or 3, the February 12, 2017 deadline is approaching fast.

Section 3032 requires manufacturers or distributors of investigational drugs for diagnosing, monitoring, or treating serious diseases or conditions to develop and make publicly available (e.g., on the company’s website) the manufacturer’s policy for responding to requests for so-called “expanded access” use of the drugs. The policy must include the following information:

  • contact information for the manufacturer or distributor for expanded access requests, in order to facilitate communication regarding such requests;
  • procedures for making expanded access requests;
  • the manufacturer’s or distributor’s general criteria for reviewing and responding to expanded access requests for individual patients;
  • the anticipated length of time for the manufacturer or distributor to acknowledge receipt of expanded access requests; and
  • a link or reference to the clinical trial record included in the clinical trial registry data bank maintained by the National Institutes of Health pursuant to section 402(j) of the Public Health Service Act, which includes a field specifying whether or not there is expanded access to an investigational drug and, if so, how to obtain information about such access.

While manufactures and distributors of investigational drugs must make their expanded access policy publically available, nothing in Section 3032 (or in the Cures Act more broadly) requires these manufacturers and distributors to grant an individual patient’s request for expanded access to an investigational drug. Accordingly, an individual patient’s expanded access request can be denied for any reason, provided such denial is consistent with the published policy. Additionally, under Section 3032, manufacturers and distributors of investigational drugs are entitled to revise their expanded access policies at any time.