By Vicki G. Norton and Sandra Stoneman
With Congress’s recent passage of the 21st Century Cures Act (the “Cures Act”) by an overwhelming majority, and President Obama’s anticipated signing of the bill, we expect that the Cures Act will soon become law. The Cures Act is intended to accelerate development and FDA approval of medical innovations such as cancer treatments, precision medicine and regenerative medicine.
The Cures Act also is designed to provide easier patient access to experimental therapies on a “compassionate use” or “expanded access” basis in response to patient demand for easier access to these therapies before they are FDA-approved. Companies engaged in clinical trials will need to adopt corporate expanded access policies and make them publicly available within the deadlines set by the Cures Act (described below).
If you have any questions about how to develop, implement or manage an expanded access policy or how any other aspect of the Cures Act will impact your company, please contact Vicki Norton, Sandra Stoneman, or any other member of the Duane Morris Life Sciences practice group.
The Cures Act
Although commentators have noted that many of the provisions of the Cures Act may take years to implement, one provision that will require compliance as soon as 60 days after enactment is the requirement that drug and device manufacturers make publicly available a policy on expanded access for patients seeking access to investigational drugs or devices for compassionate use. More specifically, Section 3032 of the Cures Act amends the Food, Drug and Cosmetic Act, to require that a manufacturer or distributor of an investigational drug for the diagnosis, monitoring or treatment of one or more serious diseases or conditions make “publicly and readily available” the manufacturer’s or distributor’s policy for evaluating and responding to requests under section 561(b) [21 U.S.C. § 360bbb] for expanded access to the drug. For drug manufacturers in Phase 2 or Phase 3 clinical trials, the policy must be available within 60 days of the date of enactment of the 21st Century Cures Act. For drug developers in Phase 1, the policy must be available on the date of the “first initiation” of a phase 2 or phase 3 study.
The Cures Act enumerates information that an expanded access policy must include, namely:
‘‘(1) contact information for the manufacturer or distributor to facilitate communication about requests;
‘‘(2) procedures for making such requests;
‘‘(3) the general criteria the manufacturer or distributor will use to evaluate such requests for individual patients, and for responses to such requests;
‘‘(4) the length of time the manufacturer or distributor anticipates will be necessary to acknowledge receipt of such requests; and
‘‘(5) a hyperlink or other reference to the clinical trial record containing information about the expanded access for such drug that is required under section 402(j)(2)(A)(ii)(II)(gg) of the Public Health Service Act.
In 2009 FDA published rules for “Expanded Access to Investigational Drugs for Treatment Use.” FDA has recently streamlined its procedure for patients to apply for expanded access in response to growing numbers of patient requests for expanded access, increasing pressure from patient advocacy groups, and enactment of state “right to try” laws. See our prior blog post here. Before the Cures Act, however, there were no requirements for drug and device manufacturers and distributors to adopt or make public corporate expanded access policies.
No Guarantee of Access
Although the Cures Act provides for greater transparency as to whether, and the circumstances under which, drug developers will provide expanded access to investigational drugs, the new statute also contains the caveat that “posting of policies by manufacturers and distributors” shall not serve as a guarantee of access to any specific investigational drug by any individual patient. Additionally, a manufacturer or distributor may also revise its policy at any time.