Pre-Approval Access Program Value Proposition

By: Jane Reese-Coulbourne, MS ChE


  A Value Proposition that Increases Patient Access and Drives Real World Data Collection

Since its inception, pre-approval drug access (PAA) has broadened entry into clinical trials, allowing more critically ill patients access to experimental medicines.  The programs, also known as expanded access (EAP), and compassionate use (CU), have typically focused on access, not research. Very little formal data collection has been required by regulatory authorities.

As a panelist at the June 20th session “Modern Solutions for Pre-approval Expanded Access” at the BIO Annual Convention, I shared this vision for pre-approval programs and real world data collection:

In today’s environment, pre-approval access programs offer an opportunity to more systematically collect a variety of real world data that complement data collected in clinical trials. Pre-approval access populations are generally sicker and have more comorbidities, making them much more like “real world” patients who will first receive the medicine once approved by a regulatory authority.

More specifically, data collection on PAA outcomes could provide an opportunity to:

  • Better understand the safety and efficacy of new products, resulting in improved labeling and additional impact of approved indications
  • Support reimbursement, demonstrating patient value with real world data
  • Enable early identification of symptomatic toxicities and impact on adherence to avoid treatment discontinuation
  • Explore dose/side effects in conjunction with clinical trials before approval
  • Explore adaptive trial designs, e.g. I-SPY , Lung-Map

At the June 21-22 New York Academy of Sciences (NYAS)/New York University School of Medicine (NYUSM) meeting, “The Need to Accelerate Therapeutic Development – Must Randomized Controlled Trials Give Way?”  we explored a closely related topic: the ethical, legal, medical, scientific, safety and economic issues pertaining to the design of clinical trials, alternative trial designs and new ways of data collection. A group of diverse speakers focused on pitting randomized controlled trials against alternatives, resulting in a highly nuanced, ethically ambiguous discussion.  We considered the potentially profound effects these changes could have on public health and the regulation of pharmaceuticals.

To learn more about pre-approval access programs and real world data collection, download our latest FAQ, linked below. For more information, please contact Jane Reese Coulbourne, MS ChE, at jrcoulbourne@mkanda.com




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.

Back to Full Blog List