- The Food & Drug Administration (FDA) is making updates to its expanded access (EA) program to improve patient experiences in the program and streamline administrative hurdles.
The FDA’s expanded access program is intended for patients with a serious illness who cannot access a drug through a clinical trial. Through an application process, a patient can access experimental drugs that have not yet been approved for the market but have been proven safe.
“EA provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases and immediately life-threatening conditions outside of clinical trials when no comparable or satisfactory approved alternative therapy options are available,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “We’re taking new steps to improve this framework.”
Over the course of the past five years, FDA has approved 9,000 applications for expanded access. Emergency applications are usually completed immediately via telephone, while non-emergency applications are completed by mail within a few days.
Despite the program’s efficacy at granting treatment access to seriously ill patients, FDA is making improvements informed by stakeholder feedback and Congressional recommendations.
First, FDA has streamlined the supporting documents needed from a clinician alongside an EA application. These changes were set to reduce administrative burden for providers. With these streamlined updates, completing one of these documents should take a provider about 45 minutes, FDA says.
The agency has also streamlined the Institutional Review Board (IRB) review process. Previously, the entire board needed to come to a consensus to approve an application. Now, only the chair of the IRB or another appropriate board entity needs to approve an application, ideally cutting down on the time it takes to achieve approval.
Finally, FDA has updated how it gathers and reports patient safety data generated from the EA program. Previously, drug developers issued concerns about how this data is used and how it will impact their experiences on the markets.
Gottlieb also reviewed findings from independent reviews of the EA program. Although most industry stakeholders support the program and patients and providers alike hold generally positive view of the application process, there are areas for improvement.
For example, FDA’s public website does not display clear information about the different stakeholders involved in the EA program application process and how these stakeholders interact together. FDA recently announced an overhaul of its website to create a more user-friendly experience.
Additionally, FDA has established a network of experts to help patients navigate their programs.
By reorganizing the Office of the Commissioner, FDA has created a Patient Affairs Staff and Health Care Provider Affairs Program.
“This will enhance our engagement with these important external stakeholder groups,” Gottlieb said. “The Patient Affairs Staff is already in place and charged with serving as the ‘home base’ and primary point of entry for patients and physicians starting the EA process and navigating them through the steps.”
FDA has also created an agency-wide Expanded Access Coordinating Committee. This Committee will be in charge of organization inter-agency communication about patient treatment access.
“FDA is deeply committed to our Expanded Access program and facilitating access to medical products outside of clinical trials when no alternative therapy options are available to patients,” Gottlieb asserted. “And we are deeply committed to continuing to enhance this program going forward.”
FDA has long been dedicated to updating its EA program. Although most reports have concluded that the program effectively and efficiently connects patients with care options they otherwise would not have had access to, the agency has worked to perfect its processes.
For example, improvements in reporting patient safety data may allow FDA to offer more patients access to treatment.
Drug manufacturers have previously held concerns about whether patient safety data would hinder their experiences on the market. These doubts served as disincentives for program participation, according to a 2017 GAO report.
“Several stakeholders we spoke with, including the selected manufacturers we interviewed, raised concerns that FDA is not clear about how it uses expanded access adverse events data in its review of drugs being considered for sale and marketing in the United States,” GAO reported.
FDA maintains that it rarely uses that data during clinical trials because the expanded access program does not have the same controls as a typical clinical trial.
That fact notwithstanding, a review of communication materials between FDA and drug manufacturers showed that FDA’s guidance and transparency on drug data use is not sufficient. This can damage the intent of the program, GAO and stakeholders explained.
“These manufacturers noted that the lack of clear information can influence their decision whether to give patients access to their drugs, because of their concerns that an adverse event will result in FDA placing a clinical hold on their drug, which could delay its development,” GAO stated.
Creating a clearer and streamlined process for reporting patient data will ideally strengthen the program. Adding to that efforts to help patients and providers navigate the EA program will ideally help more patients access treatment.