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YOUR GO-TO SOURCE FOR ANALYSIS OF ISSUES AFFECTING THE PHARMA & BIOTECH SECTORS

FDA recently signaled that it plans to be more involved in facilitating expanded access to investigational new drugs. This follows the agency’s announcement of its efforts to improve and clarify the expanded access program (EAP), as well as state and federal legislation intended to simplify the process to use investigational drugs for treatment purposes.

In an unconventional step, Commissioner Scott Gottlieb announced this move in a December 14, 2018, BioCentury article, which he subsequently tweeted, stating that FDA’s “goal is to facilitate and streamline the patient experience with our expanded access program; to create concierge-like service for providers and patients who are trying to get access to drugs. . . .” Part of this “concierge-like service” is a proposed program in which agency staff will field patient and physician EAP calls and complete single-patient EAP request paperwork. According to BioCentury, FDA will then send the forms to the physician for signature, forward the expanded access request to the product manufacturer, and ensure that the request is sent to an IRB (though how this will be done is not clear).

Perhaps most interestingly, based on the article, under the proposed plan, there will be an expectation that manufacturers respond to EAP requests within a yet to be determined timeframe. And, while companies will still have the discretion to provide or not provide the investigational product for EAP use, Richard Pazdur, the director of FDA’s Oncology Center of Excellence, which is where the program will be piloted, was quoted saying that companies will “have to give the reason for denying access.” Commissioner Gottlieb was further quoted saying that companies have an “obligation to consider expanded access, especially in areas of unmet medical need” and “[t]here are advantages for patients for FDA contacting the sponsor . . . . We can have a different conversation [with a drug company] than an individual patient or physician.”

Finally, through this process, BioCentury reported that the agency will collect data on expanded access requests and outcomes. According to the article, if the agency determines that a company is receiving multiple expanded access requests for a particular drug, it may recommend that the company open an EAP protocol.

These initiatives appear to be a clear and strong response to both a 2018 EAP assessment and recent Right to Try laws. The assessment found that paperwork is a “key hurdle” to the EAP. Stakeholder and IRB coordination was also reported to be difficult, especially for first-time applicants. Thus, through these steps, FDA aims to facilitate EAP use of investigational drugs, potentially making the EAP more appealing to patients and physicians considering Right to Try access. If ultimately implemented, FDA’s proposal would update the EAP process to be more aligned with the goals of the Right to Try laws. The timeline for implementation of these programs, however, remains unclear given limited resources at the agency, especially during the government shutdown.

At the end of the day, what should stakeholders do? As we have said before, stakeholders should continue to track the EAP and Right to Try, as changes continue to develop. According to the BioCentury article, the agency plans to hold a public meeting about the proposed program and conduct a cancer pilot program in the first half of 2019. This will provide stakeholders an opportunity to provide FDA feedback on its proposal. Investigational drug sponsors, and especially ones developing oncology products, should further assess their internal EAP policies and procedures to ensure that they are clear. Sponsors and their personnel should also be prepared to handle EAP requests, including requests that may soon be coming from FDA.