Introduction
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.
Current Law
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.
Disclaimer: This Alert has been prepared and published for informational purposes only and is not offered, nor should be construed, as legal advice. For more information, please see the firm's full disclaimer.