GAO Report on the FDA’s Steps to Improve Expanded Access Program

November 3, 2017 -

The General Accountability Office (GAO) has released a report concerning the Food and Drug Administration’s (FDA) expanded access program that assists patients with serious or life threatening ailments where there exists no other comparable medical options. This special access allows these patients to obtain access to investigational drugs outside of a clinical trial for investigational drugs with the consent of the pharmaceutical company.


Of the nearly 5,800 expanded access requests that were submitted to FDA from fiscal year 2012 through 2015, FDA allowed 99 percent to proceed. Almost 96 percent of these requests were for single patients (either emergency or non-emergency). FDA’s review process for expanded access requests is designed such that all requests are either allowed or not allowed to proceed within 30 days of receiving each request. FDA typically responded to emergency single-patient requests within hours and other types of requests within the allotted 30 days.


FDA and other stakeholders, such as drug manufacturers, have taken steps to improve the expanded access process and patient access to drugs. For example, in response to concerns that the process to request expanded access to drugs was complex and cumbersome, FDA simplified its website, guidance, and the forms required. Other streamlining efforts have included a project by which institutional review boards approve treatment plans for expanded access drug use and the development of a pilot advisory group to help a drug manufacturer manage expanded access requests.


Sates have also enacted “Right-to-Try” laws to facilitate patient access to investigational drugs, which provide liability and licensing protections for manufacturers and providers under state law if an adverse event. Nonetheless, stakeholders have said that these laws may not help patients access drugs because do not compel a manufacturer to provide access. Drug manufacturers are reluctant because of the lack of clear information from FDA as to how its decision could be influenced if a patient has an adverse event with an investigational drug.  It is not clear whether the FDA would use this information to place a clinical hold on their drug, which could delay its development for serious or life-threatening diseases.


The entire GAO report can be downloaded at this site:

Disclaimer: The opinions expressed within this article are the views of the writer and do not necessarily reflect the views and opinions of ASME.

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