CDRH Releases Final Guidance for Expedited Access to Devices for Unmet Medical Needs

By Allyson B. Mullen –

On April 13, CDRH released the final guidance document for “Expedited Access for Premarket Approval and De Novo Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions.”  We previously blogged on the draft guidance here.  The goal of the program is to facilitate access to novel devices through more interactive communications between FDA and manufacturers during the development process and more interactive reviews of premarket applications (e.g., IDEs, PMAs and de novo requests) and by focusing on post-market data. 

This final guidance was issued merely a year after issuance of the draft, which is a short period of time for FDA.  This swift release of the final guidance is presumably due in part to the modest changes that were made to the guidance from the draft and lack of controversy.  These minor changes include:

• The scope of the program was expanded from PMA devices only to PMA devices and de novo devices.  510(k)s, however, continue to be excluded. 
• The guidance was generally revised to include an increased focus on patient benefit.
• The final guidance clarified that interactive review, senior management involvement, and case manager assignment will be provided in the program to the extent resources are available.  It is still unclear, however, how availability will be determined and if any resources will be specifically allocated for this program.   
• The expedited access program (EAP) designation process was clarified, including, in Appendix 1 where it indicates that CDRH plans to review a Pre-Submission requesting EAP designation within 30 days of receipt.  The guidance also clarifies that granting of EAP designation does not mean that CDRH agrees with the applicant’s draft Data Development Plan or that any proposed study design will support a subsequent marketing application. 
• Additional examples have been added to facilitate better understanding of cases in which the EAP may be the appropriate pathway to device approval or de novo clearance.
• The final guidance indicates that it may be possible for applicants to use registry data to satisfy post-approval study requirements. 

Finally, the guidance includes a new Program Evaluation through which CDRH will publish statistics regarding the program, including the number of designation requests received and granted, and corresponding pre-market submissions approved or cleared following designation.  This evaluation will begin one year after issuance of the guidance and will continue for three years thereafter, after which CDRH will consider whether changes to the program are necessary. 

This evaluation may be an important addition to the guidance because CDRH has attempted other priority review programs in the past, including the 510(k) Priority Review Program, and these programs have generally been unsuccessful.  In fact, since 1995, only 23 510(k)s have been designated for priority review, and between 2008 and 2012, a mere five 510(k)s were designated for expedited review.  These five 510(k)s had an only marginally better review time than the average time for all other 510(k)s during that same time period.  Perhaps CDRH’s commitment to track performance data will help spur better results than similar prior programs.