- The FDA has successfully enacted its expanded patient medication access program, which allows patients with serious or life-threatening illnesses access to medications that have not yet moved past the clinical trial period, according to a report from the Government Accountability Office (GAO).
The program targets patients with serious illnesses who have no other comparable medication or drug option. FDA offers medications that have gone through the clinical trial process but have not yet undergone other testing. Both the drug manufacturer and FDA must approve a request for the expanded access program.
Between fiscal years 2012 and 2015, the FDA approved 99 percent of the 5,800 expanded access requests, GAO found. Ninety-six percent of these requests were made by individual patients as opposed to a group of patients with the same illness or ailment.
The FDA has also been successful in approving expanded access requests in a timely manner, a notable finding considering the wait time issues plaguing the healthcare industry.
Per program regulations, the FDA must respond to expanded access requests within a 30-day window. GAO reported that the FDA typically responded to these requests within hours, but in the case that responses did take longer, they have always fallen within the required timeframe.
In addition to successfully carrying out the program, the FDA and its affiliated stakeholders have also worked to make the process simpler and more accessible for patients. The agency and its partners have obtained public patient comments on the request website and implemented changes to make the interface more navigable for patients.
“Efforts by other stakeholders include a project to educate and streamline the process by which institutional review boards approve treatment plans for expanded access drug use and a pilot advisory group to help a drug manufacturer manage expanded access requests,” GAO reported.
Additionally, some states have created “Right to Try” laws, which allow terminally ill patients access to investigational drugs or medications. These state laws create a liability structure for drug manufacturers in the case of an adverse drug reaction.
“However, some stakeholders GAO interviewed cited concerns that these [Right to Try] laws may not help patients access drugs, in part because they do not compel a manufacturer to provide access,” the agency explained.
Despite these successes, GAO did find some areas for improvement.
As a part of the FDA expanded access program, the agency must collect patient safety data from drug manufacturers. This data should stem from the clinical trials that the medication has already undergone before it has been offered as a part of the expanded access program. Data should also stem from drug use in the expanded access program.
FDA reported that it does use this data during the final drug approval process, but not extensively.
“FDA reported using these data from expanded access use in a few cases during the drug approval process but not more widely, because its use does not have the same controls as clinical trials,” the agency said. “FDA officials reported that they communicate with manufacturers on how they will use expanded access adverse events data.”
However, GAO found that the communication documents between the FDA and drug manufacturers contained scant information about how to use the adverse drug event data. The manufacturers confirmed these findings with GAO, reporting that there is little guidance as to how they or the FDA should use this data.
The limited data use between the FDA and drug manufacturers may hinder patient access to drugs or medications down the line, according to GAO.
“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 explained.
“This could impact FDA’s goal of facilitating expanded access to drugs for treatment use by patients with serious or life-threatening diseases or conditions, when appropriate,” the agency continued.
GAO recommended that FDA develop clearer and more detailed guidelines on how to use adverse events data to improve the drug approval and eventual drug access process. FDA and its overseeing agency HHS reportedly agreed with the recommendations.
“HHS noted that while there have only been two instances in which adverse event data have contributed to decisions to temporarily put development of investigational drugs on partial clinical holds, additional clarity on how FDA uses such data from expanded access use may allay manufacturers concerns,” GAO concluded.