Expanded Access Expectations of 21st Century Cures Act

By: Jane Reese-Coulbourne, MS ChE

The 21st Century Cures legislation requires that the manufacturer of one or more investigational drugs for the diagnosis, monitoring, or treatment of serious diseases or conditions shall publicly disclose its policies for how and under what conditions it will or will not make available its investigational medical product(s). This is to be communicated by posting the company’s policy along with contact information on a publicly available website, beginning 60 calendar days after the December 13, 2016 enactment of the 21st Century Cures Act. Once contact information and policies are public, companies will likely be subject to an unprecedented amount of patient inquiries.

Before creating policies to post on a website, it is important to understand the current thinking regarding expanded access: ethical concerns; recent and proposed changes in the regulatory and legislative landscapes (state and federal); perspectives of disease specific patient advocacy and physician groups; effects of political movements such as the Goldwater Institute’s Right to Try (RTT) initiatives; influence of recent social media campaigns and other company experiences; program requirements and cost; as well as expert predictions of trends in expanded access.

Whether an organization, after thoughtful consideration, chooses to not get involved in any expanded access initiatives or chooses to create multiple programs, a communications strategy is important. If a company decides to embark on pre-approval access, it will need extensive guidance on setting up the program(s) and help in creating the outreach and engagement with key internal and external stakeholders: regulators, patient advocacy and professional groups, payers, and other players in this environment.

To discuss the factors surrounding this new policy and what it means for medical product manufacturers, Jane Reese-Coulbourne, MS ChE, senior consultant, MK&A, and Alison Bateman-House, PhD, MPH, MA, assistant professor, NYU School of Medicine, and co-chair of the Working Group on Compassionate Use and Pre-Approval Access, will offer a joint webinar on Tuesday, February 7, 2017 at 12:00 noon EST.

Click here to learn more and register for this free webinar.

About the Author:

Jane Reese-Coulbourne, MS ChE

Jane is a senior consultant at MK&A.  Jane brings comprehensive knowledge of evolving regulatory requirements and sensitivity to patients and sponsors’ needed in the conception and design of pre-approval drug access programs. She has helped shape policy and practice in this new paradigm of patient advocacy for early access to experimental treatments.

Most recently, Jane was the founding executive director of the Reagan-Udall Foundation for the FDA. Prior to that, she was an advisor to Dr. Richard Klausner, director of the National Cancer Institute. She served as executive vice president of the National Breast Cancer Coalition and as the board chair of the Lung Cancer Alliance.

Jane holds an MS in chemical engineering from the University of Virginia.

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