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

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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AI CORNER

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Note.  The first Chat GPT draft was concise with a couple quotes.  I asked for a longer, more detailed version.  I also asked for quote from Dr Makower, since Stanford was a co-sponsor.  It made a better, longer article, and, surprised me with insertion of 4 Makower quotes (!).  I've left the text as generated.

CHAT GPT 4o:

Improving Medicare’s Coverage with Evidence Development (CED): 

A Policy at the Crossroads

The recent symposium at USC's Schaeffer Center, held on October 22, 2024, brought together experts from government agencies, academia, and industry to address the challenges and opportunities surrounding Medicare’s Coverage with Evidence Development (CED) policy. The event illuminated the evolving role of CED in bridging the gap between emerging medical technologies and the evidence needed to justify their coverage under Medicare.

The Legacy of CED: A Policy Born Out of Necessity

CED has been a tool in Medicare’s arsenal for over 20 years, designed to provide conditional coverage for innovative technologies while collecting additional data to confirm their effectiveness. As Tamara Syrek Jensen, Director of the Coverage and Analysis Group at CMS, explained during the event, CED allows Medicare to avoid saying "no" when the evidence supporting a technology is incomplete, but its potential clinical benefit is promising. It provides a pathway for patients to access new therapies while data collection continues, reducing the risk that a potentially life-saving or life-enhancing treatment is denied coverage prematurely.

However, Jensen also acknowledged that CED has not always worked smoothly. The process, while valuable, often encounters delays, and there are persistent challenges in aligning evidence development timelines with regulatory and reimbursement requirements.

Challenges in CED Implementation

One of the key challenges discussed was the complexity of coordinating parallel review processes between the FDA and CMS. FDA approval is based on safety and efficacy, while CMS coverage decisions hinge on whether a technology is "reasonable and necessary" for the Medicare population. This statutory difference in focus creates inherent friction in how quickly technologies can move from approval to coverage.

Jensen highlighted that the small size of CMS's Coverage and Analysis Group, which includes only about 38 staff members, is a bottleneck for processing National Coverage Determinations (NCDs) and managing CED programs. In addition, while CED allows for coverage during the evidence development phase, the burden of generating high-quality evidence—without the resources for randomized controlled trials (RCTs) in every case—poses a significant obstacle for sponsors.

The Role of Sub-Populations and Representation in Evidence Development

A pivotal issue raised at the symposium was the need for better representation of Medicare beneficiaries in clinical studies. Medicare patients tend to be older and have multiple comorbidities, which distinguishes them from the general population often used in FDA trials. Jensen stressed the importance of ensuring that studies reflect the diversity of the Medicare population, not just in terms of age and health conditions but also race and gender. This represents a shift towards requiring more robust, real-world evidence that can capture the outcomes relevant to the complex, multimorbid Medicare population.

The Push for Transitional Coverage of Emerging Technologies (T-CET)

In response to these challenges, CMS is piloting a new initiative known as Transitional Coverage of Emerging Technologies (T-CET). T-CET aims to engage with manufacturers earlier in the development process, ideally a year before FDA approval, to align evidence generation requirements and facilitate faster coverage decisions. The goal, as Jensen described, is to avoid unnecessary delays by ensuring that the evidence needed for CMS decisions is already being collected during the FDA trial phase, potentially bypassing the need for CED altogether in some cases.

Balancing Speed with Quality

While the aspirations behind CED and T-CET are clear, the execution remains a balancing act. There is a desire to move faster—both in terms of approving coverage for promising technologies and in expediting the evidence collection process. But as Jensen pointed out, the statutory requirements around public comment periods and the need for thorough data review create a natural tension between speed and thoroughness.

The introduction of T-CET marks an attempt to address some of these concerns, especially the resource constraints at CMS. By encouraging early collaboration between CMS, FDA, and manufacturers, the hope is that evidence generation can be better streamlined, reducing the time to market for innovative therapies.

Looking Forward: A Call for Greater Resources and Streamlined Processes

Despite the clear benefits that CED offers—particularly in avoiding outright denials for new technologies—the backlog of NCDs waiting for review and the limited resources available for processing them pose significant barriers. Jensen was candid about the need for more resources, not only to clear this backlog but to proactively engage in CED and T-CET initiatives. The wish list, according to Jensen, includes expanding CMS's capacity to handle both the increasing volume of NCD requests and the burgeoning complexity of evidence requirements.

The event underscored that while CED remains a critical tool in ensuring Medicare patients have access to cutting-edge treatments, it is a policy in need of refinement. Greater efficiency in evidence development, more robust representation of Medicare populations in trials, and early engagement through programs like T-SET are seen as essential to the future of Medicare coverage policy.

As the healthcare landscape evolves with new technologies and treatments, so too must the policies that govern their coverage. CED has helped to navigate the uncertainty surrounding new technologies, but it will require continued innovation and support from policymakers to fully realize its potential.

The challenge moving forward will be to maintain the integrity of Medicare’s coverage determinations while accelerating access to new therapies—striking the right balance between speed and evidence quality.

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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.