FDA Issues Final Guidance on Expedited Access Program

Clinical evidence on devices designated under the U.S. FDA’s Expedited Access Program may include intermediate and surrogate endpoints, as long as the endpoints to predict a clear benefit to the patient, the agency says.

Companies should consider using these earlier endpoints if the traditional endpoint is rare or may be delayed, measurement of the endpoint is invasive or costly or the treatment effect is small enough that reaching a traditional endpoint would require an impractically large trial, the FDA says in final guidance on its EAP initiative released Thursday.

The guidance stresses that EAP devices may be approved based on a successful intermediate endpoint even if the result doesn’t lead to lower mortality. Sponsors whose devices are approved based on less than full-scale clinical trials will be required to gather additional data in a postmarket study. Postmarket data collection requirements will be set on a case-by-case basis and guided by separate guidance on balancing premarket and postmarket data requirements, also released Thursday (see related story).

Frequent Meetings With FDA Staff

The EAP, first proposed in April 2014 draft guidance, is meant to give sponsors of PMA and de novo devices that serve unmet needs in treating life-threatening or debilitating diseases a way to speed innovative technologies to patients.

Like the Center for Devices and Radiological Health’s Innovation Pathway for new technologies, the EAP relies heavily on early and frequent meetings between sponsors and FDA staff. It also requires sponsors to prepare a data development plan, including a description of clinical and nonclinical data the firm proposes to collect and a timeline for developing and marketing the device. The idea is to allow more data on device performance to be collected postmarket while maintaining a reasonable assurance of safety and effectiveness, CDRH Director Jeffrey Shuren says.

The EAP also allows the FDA to accept more uncertainty regarding how a device’s potential benefits outweigh its risks and whether this warrants earlier access. Decisions about allowing early access, however, should take into account the risk level of the device and the likelihood that postmarket surveillance will detect serious risks, the guidance says. There should also be strong indications that postmarket data collection will be completed in a timely manner, the FDA adds.

Examples of products that may qualify for EAP designation include a continuous glucose monitor that could replace blood glucose testing, a transcatheter heart valve that is delivered transcutaneously and an in vitro test for earlier diagnosis of preeclampsia, the guidance says.

Janet Trunzo, senior executive vice president for technology and regulatory affairs at AdvaMed, says the trade group welcomes the EAP and has proposed adding onto the EAP with an easier path to Medicare coverage for new technologies.

The EAP takes effect April 15. View the final guidance at www.fdanews.com/04-13-15-expedited.pdf. — Elizabeth Orr