There is often a significant delay between approval by the US Food and Drug Administration (FDA) and reimbursement coverage by the Centers for Medicare and Medicaid Services (CMS), particularly for highly innovative technologies and therapies like focused ultrasound. During this time, patients do not have access to these critical therapies despite an FDA ruling that they are safe and effective. Fortunately, there have been several new FDA pathways introduced in recent years to address the significant barrier that this scenario poses for patients.
Parallel Review Program
In 2011, the FDA and CMS introduced the Parallel Review Program. This program was established to enable the FDA and CMS to simultaneously review medical device applications and clinical data, with the goal of reducing the lag time between FDA approval and a National Coverage Decision (NCD) by CMS. The program has two stages: (1) Both FDA and CMS meet with the applicant to provide feedback on clinical trial design for the pivotal clinical trial as part of their FDA pre-market approval (PMA) or De Novo application; (2) FDA and CMS concurrently and independently review the clinical trial results and determine whether the data meet their respective requirements and then discuss their findings with the manufacturer.
Unfortunately, since its inception in 2011, the parallel review program has only approved two products, and both were diagnostic devices approved in 2018. One criticism of Parallel Review is its requirement for CMS reimbursement through a National Coverage Determination (NCD). Many companies avoid the NCD process and prefer a Local Coverage Decision (LCD) through Medicare Administrative Contractors (MACs).
Breakthrough Devices Program
The Breakthrough Devices Program was established by the FDA in 2018 to allow fast-tracking of certain novel devices or device-led combination products (products that are therapeutic and/or diagnostic that combine drugs, devices, or biological products.) The goal of this program is to expedite patient access to devices that provide more effective treatment or diagnosis, through a more rapid assessment and review process, while preserving the FDA’s standards of approval. This program replaces the Expedited Access Pathway (EAP) and Priority Review for medical devices programs, because the EAP did not include 510(k) applications and Priority Review is now limited to drugs.
The Breakthrough Devices Program offers device manufacturers an opportunity to work directly with the FDA through the pre-market review stage of the approval process. Devices that are subject to PMA, 510(k), or requests for De Novo designation are eligible for breakthrough device designation if they meet two criteria. (1) The device must provide a more effective treatment or diagnosis for a life-threatening or debilitating human disease or condition than previous therapies. (2) The device must meet at least one of the following: a) represents breakthrough technology, b) no approved or cleared alternatives currently exist, c) provides a significant advantage over current cleared or approved alternatives, d) the device’s availability is in the best interest of patients.
To date, one focused ultrasound technology has been FDA authorized and designated a breakthrough device through the Breakthrough Device Program.
Payor Communication Task Force
In 2016, the FDA’s Center for Devices and Radiological Health (CDRH) established the Payor Communication Task Force to assist in the communication between device manufacturers and payers and potentially shorten the time between FDA approval and coverage decisions. By initiating communication earlier in the process, manufacturers may design their clinical trials to produce the data required for FDA approval and a positive coverage decision. Payers included in the task force are CMS as well as private payers such as Aetna, Cigna, and Blue Cross Blue Shield. Health technology assessment groups and other stakeholders who often provide input into the reimbursement process are also encouraged to join. This is a voluntary opportunity for medical device manufacturers to obtain payer input in clinical trial design or other strategies for gathering clinical evidence.