On December 13, 2016, the Breakthrough Devices provisions were added to the Food, Drug, and Cosmetic Act through section 3051 of the 21st Century Cures Act. This program is intended to help patients have more timely access to devices and breakthrough technologies that provide for more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases.
On October 25, 2017, FDA released a new critical draft guidance designed to make the device development process for certain medical devices more efficient and predictable. The Draft Guidance For Industry, Breakthrough Device Pathwayreflects FDA’s proposal regarding the process by which a sponsor may seek a designation as a breakthrough device, assigning a team of staff to review devices designated for expedited development and priority review; and information about the criteria that will be used in evaluating a request for designation (the “Breakthrough Devices Program”). This new Breakthrough Devices Program will supersede the current Expedited Access Pathway (EAP), which FDA launched in 2015. It will also supersede FDA’s Priority Review Program, which implemented statutory criteria for granting priority review to premarket submissions for medical devices. Breakthrough device innovations that are highly innovative can also be more complex to assess. Thus, earlier, more regular interaction, “sprint” discussions, and feedback between the FDA and manufacturers should allow manufacturers to make the best use of their resources to bring novel medical technologies to the market faster.
As an initial matter, the Draft Guidance clarifies that FDA will approve Breakthrough Devices subject to a Premarket Approval Application (PMA), only if it determines there are reasonable assurances of safety and effectiveness. But it also signals that FDA may accept a greater degree of uncertainty of the benefit-risk profile for Breakthrough Devices so long as that uncertainty is sufficiently balanced by other factors, including the probable benefits for patients to have earlier access to the device and adequate postmarketing controls. Furthermore, sponsors can still look to FDA’s “Balancing Premarket and Postmarket Data Collection for Devices Subject to Premarket Approval”for guidance on FDA’s policy regarding balancing premarket and postmarket data collection during PMA review.
The BDP features the important role of obtaining agreement on a data Development Plan and for clinical trials in the process. In the guidance, FDA says it will work interactively with sponsors who choose to pursue a clinical protocol agreement. Upon reaching agreement, FDA will issue a letter documenting that agreement. An agreement in writing for clinical protocols will be considered binding on both FDA and the sponsor, subject to the following:
- Any changes to the previously agreed-upon protocol are agreed upon in writing by both FDA and the sponsor; or
- The director of the Office responsible for reviewing the device submission determines that a substantial scientific issue essential to determining the safety or effectiveness of the device exists. In this case, the director’s decision must be provided in writing and can be made only after FDA has provided an opportunity to the sponsor to meet and discuss the substantial scientific issue(s). Such a meeting would need to include the Office director and clearly document the substantial scientific issue(s) discussed.
In taking “steps to ensure that the design of clinical trials is as efficient and flexible as practicable, when scientifically appropriate”, the FDA provides a list of points to consider including:
- Prespecified endpoints regarding the minimum clinically meaningful effect;
- Intermediate and surrogate endpoints where evidence is provided to support the endpoint as reasonably likely to predict the clinical benefit of a device;
- Composite endpoints with an explicit rationale for the meaningful effect size; and
- Phased study design.
More information is contained within the Guidance document. You can submit comments and suggestions to FDA on the Breakthrough Guidance by December 26, 2017 to the Regulations.gov site.