Patient Access to High-Risk Devices for Unmet Medical Needs

January 30, 2014 Meeting Summary

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The U.S. Food and Drug Administration, or FDA, promotes public health by ensuring that marketed medical devices are safe and effective, and by facilitating the delivery of cutting-edge products into patient care without unnecessary delay. Currently, FDA reviews all high-risk devices (with the exception of Humanitarian Device Exemptions) according to the same standard of safety and effectiveness.

Recently, FDA's Center for Devices and Radiological Health, or CDRH, decided to explore whether some data currently collected before device approval could be collected postmarket, to speed patient access to devices that fill an unmet medical need. The center's 2014 strategic priorities document proposed the data shift to “strike the right balance between premarket and postmarket data collection.” In an article in The Gray Sheet, CDRH director Jeff Shuren explained that the proposed change would incorporate aspects of the center's prior innovation pathway pilots.

On Jan. 30, 2014, The Pew Charitable Trusts held a meeting titled “Patient Access to High-Risk Devices for Unmet Medical Needs.” Stakeholders discussed FDA's current regulatory authority and whether new approaches were needed to facilitate patient access to high-risk medical devices for serious, unmet medical needs. The conference drew more than 100 participants representing manufacturers, providers, payors, patients, and other health stakeholders.

Shuren delivered the keynote address, followed by two panel discussions moderated by Allan Coukell, Pew's senior director of drug and medical device initiatives, and two panels moderated by Josh Rising, Pew's director of medical devices. To examine the potential shift of some premarket evidence collection to the postmarket setting, the panels focused on the following questions:

  • What steps can FDA take under current authority? What do stakeholders want the agency to do that would require new authority?
  • What effectiveness data should be collected prior to approval, and what could potentially be assessed in the postmarket setting?
  • If some evidence is shifted to the postmarket setting, what postmarket controls would ensure the ultimate collection of this data?
  • How should these products be covered and paid for, and how can reimbursement complement the regulatory process? 
Stakeholders at the meeting arrived at the following key findings:
 
  • The benefits and risks of a device are never known with 100 percent certainty at the time of approval. Patients
    with unmet needs might be willing to tolerate a higher level of uncertainty for devices when they have no
    other treatment options. However, patient preferences vary significantly, and more work must be done to
    understand these and to include patients in the decision-making process.
  • FDA intends to publish a guidance document clarifying its authority to shift some premarket evidence to the
    postmarket setting for devices that fill an unmet need;* this guidance will serve as a springboard for further
    discussion.
  • Ensuring the collection of postmarket data is essential in any effort that shifts premarket data to the
    postmarket setting. Other challenges include removing a device from the market that is ultimately found to be
    ineffective. Congress may have to approve new authorities for FDA to address these issues.
  • Early engagement between manufacturers and payors—including the development phase of a new device—