Medical device manufacturers may soon benefit from an expedited review pathway now only afforded to "breakthrough" pharmaceutical products if a bipartisan trio of US legislators have their way.
On 24 April 2015, Sens. Richard Burr (R-NC), Michael Bennet (D-CO) and Orrin Hatch (R-UT) introduced the Advancing Breakthrough Devices for Patients Act (ABDP Act). As written, the law is heavily based on the "breakthrough therapies" section (Section 902) of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) which created the breakthrough therapies designation now being used by dozens of companies.
In short, breakthrough therapy designation affords a company wider access to US Food and Drug Administration (FDA) regulators earlier on in the product's lifecycle. The hope is that the earlier advice and access will "expedite the development and review of drugs for serious or life-threatening conditions."
Only a certain subset of products is eligible for breakthrough therapy designation—namely, drugs intended to treat serious or life-threatening conditions that are supported by preliminary clinical evidence indicating the drug "may demonstrate substantial improvement on a clinically significant endpoint over available therapies."
To date, FDA has granted 74 products "breakthrough therapy" designation.
|Breakthrough Therapy Requests|
| ||Requests Received||Granted||Denied|
*As of 17 April 2015
Breakthrough Status for Medical Devices?
Now the legislators behind the original FDASIA legislation are eyeing an expansion of the breakthrough therapy program in the hopes of expediting access to life-saving medical devices.
The ABDP Act would apply to all medical devices which:
- represent "breakthrough technologies"
- are intended for conditions "for which no approved alternatives exist"
- offer "significant advantages over existing approved or cleared alternatives
- should be made available based on "the best interest of patients"
That's similar to—though not the same as—the eligibility criteria for breakthrough pharmaceutical and biological products. The statutory requirements for a breakthrough therapy in FDASIA apply to any drug that:
is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
A notable difference in the new ABDP Act relative to FDASIA is the inclusion of language that a breakthrough device may be so designated based on a "best interest of the patient" standard. It's unclear how this standard would be implemented by FDA, and it could subject the agency to a certain amount of political pressure—by companies, patients or legislators.
In practice, the breakthrough devices bill would work in much the same way as its pharmaceuticals counterpart. Companies would need to specifically request breakthrough designation from FDA's Center for Devices and Radiological Health (CDRH), which would need to either grant or deny the request within 60 calendar days.
Upon designation, FDA would "assign a team of staff, including a team leader with appropriate subject matter expertise and experience, for each device" and "provide for interactive and timely communication with the sponsor of the device during the development program and review process." FDA would also need to expedite the review of a company's manufacturing facility. Problems with a company's regulatory compliance with FDA's Quality System Regulation (QSR) can often result in approval delays for a medical device.
And, perhaps most importantly, FDA would be tasked with facilitating, "when scientifically appropriate, expedited and efficient development and review of the device through utilization of timely post-market data collection." In plain terms: Figuring out which data is absolutely necessary to secure pre-market approval, and which data can be collected at a later date as part of a post-marketing approval commitment by the sponsor.
These similarities, however, could also prove problematic for FDA. As with the original FDASIA legislation, the ABDP Act does not authorize additional funding for FDA to conduct its reviews. The resource-intense breakthrough therapy reviews have taxed FDA regulators, and without additional funding, non-breakthrough devices may find their devices being reviewed more slowly.
The bill also calls for FDA to issue a guidance document on the Breakthrough Devices designation within one year of the law being passed.
The legislation should be familiar to keen watchers of FDA-related legislation. Subtitle E, Title I of the recent draft of the 21st Century Cures Actcontains a nearly identical section, titled "Priority Review for Breakthrough Devices."
An updated draft to that 21st Century Cures Act is expected to be released in late April 2015.
Advancing Breakthrough Devices for Patients Act (ABDP Act)