Expanded access programs, overseen by the U.S. Food and Drug Administration (FDA) as a historic pathway to allow patients with serious conditions to access investigational drugs that may help them, are now used as a means of collecting efficacy and safety data contributing to submissions to regulatory agencies as contribution to real-world data. Ultimately, such data can be used to inform the public health use of a specific drug.
What are Expanded Access Programs?
Also known as “Compassionate Use,” this program represents the use of an unapproved drug or medical device (or diagnostic technology) under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by people with serious or life-threatening conditions who do not meet the enrollment criteria for any clinical trials in progress. It is essentially a channel to get seriously ill patients access to experimental drugs that are not approved.
These programs started in the late 1970s and a formal program was established in 1987 in response to HIV/AIDS patients requesting access to experimental drugs not yet approved by the FDA. A influential milestone, the Abigail Alliance v. von Eschenbach established that there is no legal right to experimental drugs (the Supreme Court opted not to hear the case). TrialSite News breaks down some trends recently discussed by Ms. Christine Brennan with Cancer Therapy Advisor.
Why are Drug Sponsors starting to collect data from Expanded Use scenarios?
The drug sponsors are increasingly using expanded access scenarios to systemically and methodically collect data about efficacy and safety. According to experts, such as Alison Bateman-House, PhD, MPH, MA, assistant professor in the division of medical ethics at NYU Langone Health, and co-chair of the Working Group on Compassionate Use and Preapproval Access (recently interviewed with Cancer Therapy Advisor) the collection of this Expanded Use data represents “an ongoing trend that companies collect.”
Cancer Therapy Advisor reports that Erika Segear, PhD, RAC, associate director of regulatory affairs notes big changes are now in motion as industry sponsors seek to “capture these data so that they can be used to inform the broader public health use of that product.”
Drug companies invest heavily in new research and development. If they are able to garner more valuable insight, such as supplementing clinical trial data in regulatory review, informing off-label use and driving label expansions. Moreover, with the agency embrace of real-world evidence drug sponsors now have the opportunity to more systemically and methodically capitalize on expanded use data, reports Christina Bennett writing for Cancer Therapy Advisor.
FDA Convenes Public Meeting on Expanded Use Data Collection
That the FDA is alert to his should be understood by all involved in clinical Expanded Use programs. Last Nov. 19, 2018 the regulatory body hosted a public meeting concerning the “leveraging” of such data. See a report from this meeting.
A New Way of Augmenting Clinical Trial Data?
Drug sponsors may increasingly use expanded access program data if it contributes to a positive outcome. Drug sponsors are reporting on such data at medical meetings. For example, Ms. Brennan reports that at the 2019 American Association for Cancer Research (AACR) Annual Meeting (Atlanta, GA) that sponsor Bayer presented data not only from the actual clinical trial but also the the company reported on data from 11 patients who received selitrectinib (aka LOXO-195) under the FDA expanded access single-patient protocol.
Ms. Brennan introduces another example: Atara Biotherapeutics’ expanded access study reveals data from patients who received their tabelecleucel at the 2020 Transplantation & Cellular Therapy Meetings, Orlando, FL.
The Working Group of Compassionate Use and Preapproval Access
As mentioned, Alison Bateman-Houes, PhD, MPH, MA, assistant professor in the division of medical ethics at NYU Langone Health, also co-chairs the Working Group on Compassionate Use and Preapproval Access. Led by NYU Langone, the group seeks to offer the public information about this process. See the list of members.
Call to Action: Are you interested in learning more about expanded access programs? The members of the NYU Langone managed “Working Group” represent some of the top experts in America. Consider networking with this group—or at least participating in a meeting.