September 5, 2018
By: Scott Gottlieb, M.D.
FDA Commissioner Scott Gottlieb, MD
Advancing the public health means helping to make sure patients have access to safe, effective medical products. Access is a matter of public health. And our commitments to patients don’t stop at the time of a product’s approval. We take many steps to make sure products can be safely accessed in the post-market setting, and that new innovations continue to deliver their anticipated benefits. We actively monitor for safety findings in the post-approval period. We have updated labeling to provide modern guidance to providers and patients. And as part of our dedication to the promotion of public health, we also look for opportunities to help advance wider access to safe and effective innovations.
Patients may not have access to an FDA approved or cleared device in the absence of adequate coverage. So, the FDA recognizes the importance of working collaboratively with the payor community to streamline the path from FDA market authorization to payor coverage and reimbursement.
Over the past few years, we’ve sought creative solutions to decreasing the gap between the FDA’s decision to advance a product to the market and the time it takes both public (e.g., Medicare, Medicaid) and private payors to determine whether and how they will provide coverage.
Today, we’re announcing some new steps to help narrow this chasm by expanding our work with private payors. First, some history on the efforts that we’ve undertaken to date:
Previously, one of our early and most successful efforts was the creation of the Parallel Review Program. This program was piloted in 2011 with the Centers for Medicare and Medicaid Services (CMS). It was fully adopted in 2016 after proving to be a valuable mechanism for decreasing the time between the FDA’s approval of a pre-market medical device application and CMS national coverage determination.
The Parallel Review Program enables the FDA and CMS to review, at the same time, the clinical evidence that supports both the approval and coverage of a new medical device. Traditionally, the FDA reviews the relevant non-clinical and clinical evidence first and then issues a regulatory decision. Then, after the FDA approves the device, payor organizations like CMS would begin reviewing the relevant clinical evidence. As one might expect, a parallel review process is much more efficient than a serial review process.
Through the Parallel Review Program, we saw that earlier opportunities for collaboration during the process were critical for success. Manufacturers who design and conduct the clinical trials benefit from early feedback from the FDA and CMS as they develop their plans to conduct clinical investigations to gather valuable clinical evidence. Including CMS in the Pre-Submission meeting can allow manufacturers to obtain feedback from both the FDA and CMS while planning a clinical trial. Manufacturers can then factor this information into their design of a pivotal clinical trial. The development program can be structured to meet the needs of both regulators like the FDA and payors. Such an approach has many advantages in addition to timelier access, and lower costs. For example, if medical device manufacturers have a better understanding of the clinical evidence needed by both agencies, then manufacturers might consider ways to collect all the necessary evidence through clinical programs that are more efficient. They may be able to reduce the size and duration of trials, and expose fewer patients to an experimental product.
To date, the FDA has received more than 75 inquiries into the process and 36 formal applications to participate in Parallel Review. The FDA treats these inquiries and applications as confidential. We don’t disclose a manufacturer’s participation in the program. However, the company may choose to make its participation public.
While CMS covers a significant number of Americans, private payor organizations also play an important role in evaluating clinical evidence and making coverage decisions for patients in the U.S. Building on the lessons learned through the Parallel Review Program, in 2016 the FDA launched a new program aimed at engaging private payors and Health Technology Assessment groups through the Private Payor Program.
The Private Payor Program is a voluntary opportunity for manufacturers to receive feedback from the FDA and other non-governmental health technology assessors/payors on what’s needed in order to develop a more efficient evidence generation strategy during the FDA pre-submission meeting.
Today, we are announcing that CareFirst BlueCross BlueShield and United Health Group have joined the FDA’s growing list of private payors available to participate in medical device manufacturer pre-submission meetings.
In addition to these two new participants, there are currently six other payor/health technology assessment organizations participating in the program, including BlueCross BlueShield Association, Duke Evidence Synthesis Group, ECRI Institute, Humana, Kaiser Permanente, and the National Institute for Health and Care Excellence (NICE).
By facilitating communications between device makers and payors, the FDA hopes to shorten the time between FDA approval and coverage decisions. This can be particularly beneficial for manufacturers creating new and innovative devices who also need to secure coverage of their devices by payors.
The process of acquiring clearance or approval from the FDA, and then coverage from a payor, can take several years to achieve. This delay can be challenging for patients, particularly those with limited alternatives for diagnosing and treating potentially serious medical conditions. In some cases, the long timeframe between those decisions may also have an adverse consequence on smaller medical device companies, which may be unable to sustain themselves for an extended duration of time with limited revenue streams. These challenges are significant. And they’re driving our continued efforts to identify new ways to improve the speed and efficiency of our regulatory review process and coverage decisions.
To date, there have been 10 engagements between private payors or Health Technology Assessment (HTA) groups and manufacturers. This includes four pre-submission meetings.
The program continues to gain momentum. We’re receiving new inquiries weekly.
The FDA believes that early engagement with manufacturers and payors during the planning phase of medical device clinical trials will expedite patient access to high quality, safe, and effective medical devices. We appreciate the complexity of the process of achieving patient access to new medical devices. And we recognize that a variety of stakeholders have important roles in that process.
In streamlining the process, our goal is to improve coordination so that access to approved products can be achieved without unnecessary delay. To that end, we continue to support and encourage the collection of clinical evidence that’ll not only support regulatory decisions but also inform coverage decisions — ultimately providing an opportunity for more timely patient access to new technologies.
We’re all beneficiaries of this collaborative model to ensure the availability of safe, timely, effective and affordable health care options. For patients, it can mean earlier access to safe, effective, innovative technologies. And for payors, it can mean learning about new technologies beyond their current horizon scanning, and being able to provide suggestions about what data and analyses would be useful for evaluation, gaining insight and increased understanding of the FDA review process.
For manufacturers, it’s early engagement of payors in discussions about evidentiary needs, being able to consider and address coverage-related issues earlier in the process, and gaining the potential for earlier reimbursement through earlier engagement with payors. For the FDA, it’s improving public health by facilitating more efficient patient access to innovative, safe and effectively medical devices.
As our work continues to grow in scope and complexity, so must our efforts to collaborate on innovative and streamlined approaches to finding the best ways to serve the American public.
I hope that by sharing this insight and vision, we inspire other organizations and manufacturers to come together early in the process so we can more effectively identify opportunities, challenges and approaches to access, and maximize our collective resources to get beneficial devices to patients.
Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration