ISPOR Panel explains the CMS path to cover new technologies

In March 2017, CMS issued its new rule for coverage of continuous glucose monitoring (CGM) for Medicare beneficiaries with diabetes, and what should have been a moment of celebration after years of advocacy turned into a collective cry. .

The notice stated that anyone seeking CGM reimbursement could only use the receiver that came with the system — and not the smartphone app that could display glucose data on a user’s phone or share it with a caregiver. The rule not only stripped the technology of an important feature, but also forced Dexcom to disable app compatibility on any CGM sent to Medicare enrollees.

For beneficiaries, the smartphone episode became an example of everything that was wrong with CMS’s approach to technology. But it may have helped advocate a new path forward, one that allows Medicare to adopt new tools and pay for them, while also gathering the evidence needed to ensure the technology is safe and appropriate for an older population.

The Transitional Coverage Path for Emerging Technologies (TCET), proposed in June 2023 and discussed in a Viewpoint article published in JAMA Health Forum,¹ was featured in “Revolutionizing Regulatory Pathways: Unleashing the Power of Real-World Evidence, Adaptive Trials, and Synergistic Collaboration for Accelerated FDA Device Approval, Breakthrough Designation, and CMS Reimbursement,” a panel discussion that took place Tuesday at ISPOR— The Professional Society for Health Economics and Outcomes Research Meeting in Atlanta, Georgia.

The discussion, moderated by Renee Arnold, PharmD, RPh, of the National Institutes of Health, also covered reforms to improve technological success rates at the FDA, the use of real-world evidence in appropriate trials, and the emergence of CMS as a assessment of health technologies.

More coordination at FDA

In 2020, speaker Douglas Kelly, MD, came to the FDA as deputy director and chief scientist of the agency’s devices, diagnostics, and digital health branch, after 30 years of trying to bring devices to market and seeing the struggle on the other side.

Kelly explained that for decades, his agency failed to recognize the key differences between biotechnology and medical technology – medicines can reach the public comparatively quickly and with much lower risks than devices; thus, the failure rate for medical devices is 90%. As many noted during the conference, Kelly said the FDA’s “safe and effective” standard and Medicare’s “reasonable, necessary, and appropriate” standard are not always aligned.

With medical devices, he said, “no single stakeholder owns the process,” and coverage is far from guaranteed even if the FDA approves the product; Therefore, it is very difficult to obtain financing for medical device ventures. At the same time, since his time at the FDA, he has been shocked by how many potential candidates propose ideas without ever having spoken to a patient outside of their own office.

The solution is TAP, which stands for Total Product Life Cycle (TPLC) Consulting Program.

“We believe that by bringing everyone together early on, we can eliminate a lot of late-stage failures,” Kelly said. A group of TAP advisors coordinates discussions among stakeholders under the principle: “We don’t tell doctors how to practice medicine; we listen to doctors about how they practice medicine and use that to help us decide where the real risks lie in the device and its use.”

TAP has 2 phases. The diagnosis phase assesses the status of the project and which elements are missing; The second phase is a process and policy phase that presents options across the FDA. TAP team members have diverse backgrounds, Kelly said. “If you have a problem, you can find someone who has solved it or has dealt with it before.”

The goal is to avoid pitfalls and reduce the failure rate. “We want to help innovators plan ahead for stakeholder risks.” It doesn’t make sense, he said, “to start your clinical trial before you’ve really figured out what questions you need to ask and answer to all your stakeholders.”

Of course, patient concerns are the FDA’s bottom line. “It is ethically dubious to subject a group of patients to a clinical trial when the chances of it reaching commercialization are very low,” he said.

TCET allows time to gather evidence

When CMS issued its infamous rule prohibiting CGM coverage if seniors used the technology with a smartphone, its reasoning was that a smartphone did not meet the definition of durable medical equipment. An attorney who successfully sued Medicare to force MCG coverage called the restriction “absurd.”

But as Steven Farmer, MD, director of coverage strategy at CMS, explained, it’s the very nature of innovation that can make advances in technology challenging to fit into Medicare benefit categories that were created decades ago, before One would imagine some of the devices that seem like legitimate candidates for coverage today.

Medicare’s need to show that there is evidence that a technology is “reasonable, necessary, and appropriate” for its distinct population can also be challenging, as studies may leave out older patients or patients with comorbidities. “We believe that establishing high standards of evidence is essential to ensure we are providing the highest quality care to our patients,” said Farmer. “CMS is able to extend coverage to an item or service if it meets the reasonable and necessary standard.”

Medicare also looks at current standards of care when evaluating emerging technologies, and sometimes “there may not be an established standard.”

The TCET pathway seeks to respond to these challenges, Farmer said. The idea is to provide coverage for a limited time to create opportunities for evidence collection. He explained the guidance documents:

• Evidence review: Farmer said this explains how CMS looks at the body of evidence when deciding whether an item or service is “reasonable” or “necessary.”
• Coverage with evidence development (CED): describes the criteria applied to coverage determination (CD) studies.
• Clinical outcomes guidance series: seeks to define what outcomes are available in a therapeutic area? Which endpoints are relevant to the Medicare population? How can we define a clinically significant difference? How long is the follow-up required?

“The idea is that by providing greater clarity about what our expectations are, manufacturers far upstream can be more effective in incorporating this data into their critical clinical studies,” and will be prepared to meet CMS’ expectations when arrive at the market, said the farmer.

The following is fit-for-purpose study guidance. “In the near-market phase, the TCET pathway seeks to accelerate our coverage reviews by engaging with manufacturers well in advance of FDA marketing authorization,” he said. “In the proposed notice, we set an expectation of engagement with manufacturers approximately 1 year prior to FDA marketing authorization.”

As with the FDA’s TAP initiative, the idea is to think through the details, such as coding issues, in a collaborative way that proactively finds any evidence gaps and develops a plan to address them in the post-market period, through from a CED study. The transitional coverage period is then limited in time, with a clear set of expectations for monitoring safety signals and completing all studies.

He referred to the need for a study “fit for purpose”, which was the topic of the ISPOR 2024 meeting; This approach uses data to ensure that a system or program is operationalized in a way that meets patient needs in a given environment or context.

“Whether something is fit for purpose really depends on the question and the context in which the question is being asked,” Farmer said. “But we strongly encourage manufacturers to think about how to design this fit-for-purpose study and what data sources are available.”

Evolution of CMS as an HTA organization?

Peter Neumann, ScD, of Tufts Medical Center, presented insights from a paper he co-authored with former FDA and CMS official Sean Tunis, MD, MSc, now director of Rubix Health. The article, “Transforming CMS into a Health Technology Assessment Organization,”^two took the position that “CMS is being asked to evaluate drugs and devices in an increasingly complicated world,” Neumann said. This is especially true because there is no central health technology assessment (HTA) in the federal government, he said.

Certainly, CMS has evolved from its origins in paying claims – and expressly not interfering with the way care was delivered. Of course, Neumann said, there was an interest in ensuring that public funds paid for things that worked and that things evolved. A change occurred in 2001 with the news that CMS would evaluate new technologies, and in 2006 came the creation of coverage with evidence development.

After the FDA began granting breakthrough status to certain technologies, there was a need to think about covering devices in Medicare “when faced with substantial uncertainty about benefits and risks,” Neumann said.

“The growing number of promising but expensive devices and drugs has sparked new questions about how Medicare will evaluate and pay for them,” he said.

The 2022 controversy over aducanumab coverage following FDA approval has raised new questions, and now, the Reducing Inflation Act shifts CMS’ evaluation role into an entirely new realm. “Negotiating prices inevitably means evaluating the clinical evidence underlying medicines,” he said.

“So again, you see CMS taking on more and more this role as a body similar to the HTA, and it’s very different from what we see overseas in other places, where HTA organizations evaluate the evidence, often at drug launches, and this is evaluating the evidence, after The drugs have been on the market for some time. However, that’s the idea.”

There are workforce challenges, ongoing needs for greater coordination with the FDA, a need to improve the coverage determination process with new legislative authority, Neumann said. “Ideally, this is not easy to do and will be challenging in any environment, but certainly in this kind of polarized political environment. But the idea is that the legislative authority would reinforce the ability to make CD, which is sometimes questioned”, and should be clarified.

“We argue that, once again, whether we like it or not, CMS is becoming a kind of ATS body. They should embrace the role in our opinion and do as good a job as [they] he can.”

References

1. Farmer SA, Fleisher LA, Blum JD. The Path to Transitional Coverage for Emerging Technologies – enhancing innovation and establishing safeguards for patients. JAMA Health Forum. 2023;4(8):e232780. doi:10.1001/jamahealthforum.2023.2780.
2. Neumann PJ, Tunis SR. Transforming CMS into a health technology assessment organization. N Engl J Med. 2023;389(8):682-684. doi:10.1056/NEJMp2305280