USC Policy Center Holds Summit on Coverage with Evidence Development (Video, Summary)

Thanks to Jason Shafrin for pointing to USC's recent 3-hour symposium on Coverage with Evidence Development.   Find the home page here, with links to a YouTube video of the whole summit:

https://healthpolicy.usc.edu/events/the-future-of-medicares-coverage-with-evidence-development-policy/

The program was co-supported by the Tufts Center for Evaluation of Value and Risk in Health and the Stanford Mussallem Center for Biodesign.  It was September 25.

Here's the program summary online:

Medicare is considering changes to an important program meant to provide beneficiaries with access to newly approved drugs and devices while collecting data on how they work in the real world. The nearly 20-year-old program, known as Coverage with Evidence Development (CED), has recently faced questions about whether it’s working as intended. 

The USC Schaeffer Center, in partnership with the Tufts Center for Evaluation of Value and Risk in Health and the Stanford Mussallem Center for Biodesign, hosted a conversation on the future of CED at USC’s Capital Campus in Washington, D.C. Attendees heard from the Centers for Medicare and Medicaid Services (CMS) official overseeing updated guidance, learned about the latest research and shared ideas on how CED could be improved.

Below, I include a news article generated by Chat GPT from an autotranscript.  I also include, further below, a similar news article generated by (free) Google Notebook LM.

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CHAT GPT 4o:

A Critical Juncture for Medicare’s Coverage with Evidence Development (CED): 

Calls for Change and New Thinking

The USC Schaeffer Center’s symposium on Medicare’s Coverage with Evidence Development (CED) policy, held on September 25, 2024, provided a unique forum for discussing the evolving challenges, limitations, and future direction of this two-decade-old policy. While CED has historically offered a pathway for Medicare to conditionally cover emerging technologies while gathering additional evidence, panelists at the event highlighted growing concerns about whether the policy is truly meeting its goals and what course corrections are necessary.

Among the panelists were policymakers, academic experts, and industry leaders, including Dr. Josh Makower, the head of Stanford Biodesign Institute. The symposium emphasized the increasing need for better coordination between the design of new technologies and the regulatory landscape they must navigate. “We need to rethink how evidence is generated,” Makower said during a panel discussion. “The current approach often leaves developers scrambling to meet Medicare’s standards after the fact, when the focus should be on integration from the very beginning.”

CED: A Framework Under Pressure

As CMS continues to rely on CED as a tool to allow access to innovative therapies, many experts at the symposium voiced concerns that the policy is no longer well-suited for the rapidly changing landscape of medical technology. Tamara Syrek Jensen, Director of CMS’s Coverage and Analysis Group, acknowledged this tension, admitting that CED, while valuable, can sometimes act as a “qualified yes,” rather than providing the full coverage patients need. “Yes is a much easier place to be than no, but we are often saying yes with conditions, which can leave patients in a kind of limbo,” said Jensen.

The concern is not just theoretical. Many stakeholders have pointed to real-world examples where CED’s processes have limited access to necessary treatments. A case in point, raised by Dr. Barry Lynn, Director of Public Policy at USC Schaeffer, was the experience of patients needing advanced diagnostic tests for cancer, who faced delays because of slow evidence collection processes under CED. “When time is of the essence, particularly with progressive diseases, even a short delay in coverage can have lasting consequences for patients,” Lynn emphasized.

Panelist Critiques: A Call for Reform

Many of the panelists argued that CED, as it currently stands, requires significant reform. Dr. Josh Makower took a critical stance on the rigidity of CED’s evidence requirements, stressing that it often places undue burdens on manufacturers to provide data that goes beyond what the FDA requires. “We’ve seen a disconnect between FDA approval, which focuses on safety and efficacy, and Medicare’s coverage determinations, which ask for an entirely different set of data focused on reasonable and necessary criteria,” said Makower. “This creates a bottleneck for innovation and discourages smaller companies from pursuing Medicare coverage.”

Moreover, as Jensen explained, the CED process is resource-intensive for CMS itself. The agency’s staff is stretched thin, with only 38 employees in her group, and fewer than half of those work on national coverage determinations. This shortage of resources creates a backlog of technologies waiting for CED approval. “We have an NCD dashboard where we track these requests, and there are currently nine technologies waiting for review,” Jensen said. “We simply don’t have the resources to keep up.”

Makower and others argued that this backlog is a symptom of a larger issue: the need for better infrastructure to handle the increasing volume of emerging technologies. “This isn’t just a resource issue—it’s a structural problem,” Makower contended. “The entire process needs streamlining. From early engagement with FDA and CMS to better alignment of evidence requirements, we have to create a system that fosters innovation rather than stifling it.”

The Role of T-SET: A Step Toward Integration

One of the most widely discussed initiatives during the symposium was CMS’s Transitional Coverage of Emerging Technologies (T-CET) program. Designed to address some of the bottlenecks and delays associated with CED, T-CET aims to bring CMS and manufacturers into alignment earlier in the FDA approval process. Jensen noted that the goal is to start conversations about evidence requirements up to a year before FDA market authorization, so that by the time a technology is approved, CMS has already gathered enough data to make a coverage determination.

“T-SET is about creating a seamless transition from FDA approval to Medicare coverage,” Jensen explained. “Ideally, by the time the FDA grants approval, the evidence base will already be established for Medicare coverage, and we won’t need to invoke CED.”

Panelists were cautiously optimistic about T-SET but pointed out that its success depends on meaningful coordination between CMS, the FDA, and industry stakeholders. “T-SET is a step in the right direction, but it will only work if CMS can truly engage early with manufacturers and provide clear guidance on what evidence is needed,” Makower noted. “Without that, we’ll just see more delays.”

Debates on Real-World Evidence: A Path Forward or a Compromise?

A key point of contention was the role of real-world evidence (RWE) in the CED process. Jensen emphasized that CMS has been shifting away from demanding randomized controlled trials (RCTs) for all new technologies, instead allowing for observational studies and RWE to inform coverage decisions. “RCTs are difficult to implement in Medicare’s claim systems, and they’re not always necessary,” Jensen explained. “Real-world evidence, especially in the form of registries, can give us the data we need without imposing an undue burden on patients or manufacturers.”

However, several panelists, including experts from Tufts University, voiced concerns that relying too heavily on RWE might compromise the quality of evidence. “We need to be careful not to lower the bar too much,” warned Dr. Peter Neumann, a health economist at Tufts. “Yes, RWE is valuable, but it must be held to high standards, especially when we’re talking about the health of older, more vulnerable populations.”

The discussion on RWE also underscored disparities in access to clinical trials, which can skew the data. Jensen herself acknowledged that many clinical studies do not reflect the diversity of the Medicare population, which is older, more likely to have multiple comorbidities, and more racially diverse than the general population. “We need to ensure that our evidence base reflects the real-world complexity of Medicare beneficiaries,” Jensen said. “That means better representation of women, minorities, and patients with multiple chronic conditions.”

CED in Practice: Successes and Failures

One example frequently cited at the symposium was Medicare’s use of CED for Transcatheter Aortic Valve Replacement (TAVR) procedures. While TAVR has been successfully covered under CED and has become a standard procedure for high-risk patients, the process of getting it approved took years and required extensive data collection through national registries. “TAVR is a success story for CED,” said Dr. Lynn. “But for every TAVR, there are countless other technologies that languish in the CED process, waiting for data to be collected.”

Conversely, the recent controversy surrounding the coverage of monoclonal antibodies for Alzheimer’s disease demonstrated the limitations of CED. CMS issued a decision requiring further evidence for this class of drugs, but few clinical trials met the agency’s standards, leading CMS to create its own registry to collect data. As Jensen pointed out, “We had to take matters into our own hands because we didn’t get enough industry engagement.”

Looking Ahead: The Need for Systemic Change

The panelists agreed that CED remains an essential tool for Medicare, but they were united in their call for reform. Whether through T-CET, better alignment between CMS and FDA, or greater reliance on real-world evidence, the consensus was clear: without systemic changes, CED risks becoming an outdated policy that hinders, rather than facilitates, innovation.

“We need to stop thinking of CED as a policy of last resort,” Makower concluded. “It should be part of a broader strategy that includes early engagement, flexible evidence requirements, and real-time data collection. If we can get there, we’ll have a system that not only benefits patients but also encourages the development of cutting-edge technologies.”

As the symposium wrapped up, the future of CED remained uncertain, but the calls for reform had been made clear. If CMS, the FDA, and industry leaders can come together to reimagine the policy, Medicare beneficiaries could see faster access to life-saving technologies without compromising the quality of evidence that underpins their care.

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Google Notebook LM

Coverage with Evidence Development: A Policy in Need of an Overhaul?

Coverage with Evidence Development (CED) is a Medicare policy intended to provide beneficiaries access to promising new technologies while simultaneously generating evidence to assess their value. However, as the policy enters its third decade, experts are raising concerns about its effectiveness and calling for significant reforms.

One of the primary criticisms of CED is that it has a mixed record of success. While there have been instances where the policy has successfully facilitated early patient access and generated valuable data, these successes are often overshadowed by concerns about the program's complexity, cost, and limited impact on coverage decisions.

Dr. Darius Lakdawalla, Chief Scientific Officer of the Schaeffer Center for Health Policy and Economics at USC, notes that CED is often successful in expediting patient access to therapies that might not otherwise be covered. However, he also highlights cases where CED can inadvertently restrict access for certain patient populations, potentially exacerbating existing healthcare disparities.

A key challenge identified by experts is the lack of clarity surrounding the criteria for requiring CED. The concept of “reasonable and necessary,” which underpins Medicare coverage decisions, is subject to interpretation and can be difficult to define in a transparent and consistent manner, particularly when economic considerations are excluded. This ambiguity creates uncertainty for innovators, particularly small startups, who may be reluctant to pursue CED due to the risk of a non-coverage decision, which could be fatal to their technology.

Another concern is the burden placed on providers and patients involved in CED evidence generation activities. Registries and trials associated with CED often involve extensive data collection requirements, which can be time-consuming and complex. For example, the TVT registry for transcatheter valves has over 276 fields and can take clinicians up to four hours to complete. This burden can lead to attrition from studies and potentially compromise data quality. Additionally, the Centers of Excellence model, which often requires patients to travel to specialized facilities for treatment, can impose significant travel burdens, particularly on those in rural areas.

Dr. Lakdawalla's research indicates that the Centers of Excellence model can unintentionally exacerbate access issues for rural and socioeconomically disadvantaged populations. His analysis of discharge statistics reveals that hospitals eligible for CED participation tend to serve a significantly smaller proportion of rural patients and patients from socioeconomically disadvantaged areas compared to other hospitals.

The unclear process for reconsidering or ending CED requirements is another area ripe for improvement. The absence of fixed timelines or clear guidance on what constitutes sufficient evidence to lift CED requirements can prolong the evidence development process and delay final coverage decisions.

Several experts suggest that the time is ripe for a comprehensive overhaul of the CED program. Dr. Peter Neuman, Director of the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, argues that CED “debuted in a period of blackberries and DVDs” and is in dire need of modernization to leverage advancements in analytics and data collection. He advocates for a more robust investment in data infrastructure and a shift away from the current one-off approach to evidence generation, proposing a “national evidence generation ecosystem” that transcends CED but incorporates it as a key component.

There is also a growing consensus that CMS needs significantly more resources to effectively manage and improve the CED program. While the $10 million allocated in the proposed HR 1691 bill is a step in the right direction, experts argue that a more substantial investment is necessary to address the program's backlog, expand staffing, and enhance data collection infrastructure. Some experts have even suggested exploring user fees, similar to the model employed by the FDA, as a potential funding mechanism, although this idea has faced resistance from industry groups.

The debate over whether different types of technologies, such as drugs and devices, require distinct CED pathways is another point of contention. Greg Daniel, Vice President of Global Public Policy at Eli Lilly and Company, argues that the different regulatory frameworks, evidentiary requirements, and practice settings for drugs and devices necessitate tailored approaches to CED. He points to the experience with Alzheimer's disease drugs, where the application of CED designed for medical devices has resulted in unintended consequences, such as limited patient access despite traditional FDA approvals based on robust clinical endpoints.

Commercial payers have also expressed interest in expanding CED into the private sector. Bob McDonough, Executive Director for Clinical Policy and Research at Aetna, highlights the potential for private payers to play a more active role in evidence development. However, he acknowledges that there are open questions regarding the feasibility and benefits of private payer participation, as well as the willingness of plan sponsors to support such initiatives.

Ultimately, the future of CED hinges on the ability of policymakers, CMS officials, and stakeholders to address these challenges and forge a path toward a more efficient, effective, and equitable system for evaluating and covering new medical technologies.