Monday, July 1, 2024

New in JAMA! Lee Fleisher, Robert Califf, Others, on "Payers and Evidence Generation"

Update - This is one of a pair of articles this summer in JAMA by FDA on clinical evidence.

This paper, this blog, is Abbasi et al., with Lee Fleisher, on payers and evidence.

There was also a slightly earlier paper by Abbasi, Curtis, Califf, "Why should FDA focus on pragmatic clinical research?"  In print June 3, 2024, 332:103-104.  

JAMA also links these two FDA op eds to a consensus essay by Angus et al., "Integration of clinical trials with the practice of medicine."  June 3, 2024, 332:153-162.


 Have you wondered when CMS will finalize 2023's proposed rule on new technologies, the TCET rule (here)?  

Here's a joint article in JAMA, July 1, authored by the head of FDA (Califf) and the prior Chief Medical Officer of CMS (Fleisher).   

Not Much of a Link to TCET

I was interested from a TCET perspective, because a publication like this might suggest that the final version of TCET is about to appear.  But on further review, it's not so clear.  The article is primarily by FDA authors, taking an FDA perspective (albeit on medical insurance).     

The TCET proposal is mentioned but only tangentially.  Had TCET been carefully summarized and praised in this essay, I would have tagged that, as a sign that TCET's finalization was imminent.  See also an earlier, 8/2023 article in JAMA Health Forum by Fleisher and colleagues on TCET, here.

FDA Authors Take on Payers

So take the FDA-and-payers article on its own.   FDA reports several ways in which private payers are, or could be, pivotal to post-FDA-approval evidence generation, including the simple fact that they hold all the claims data.   See the FDA's detailed website about its collaborations with payors here.

CMS CMMI and the "ACE"

The FDA article mentions TCET, coverage with evidence development (CED), and a proposal from the CMMI innovation center called "ACE, Accelerating Clinical Evidence," which would be related to different payments for drugs that have and have not completed post-approval trials.  New to me; see also here, page 3.


Nerd note - the FDA article says that CMS first got the authority to cover routine costs of devices in clinical trials in 2003, in the Medicare Modernization Act, but it actually goes back to the Clinton-era NCD (2000) on clinical trials.  And the even earlier 1995 joint CMS-FDA regulation on Medicare coverage of IDE Category B trials (60 FR 48417) which itself developed at least in part from a legal case dating to 1994 (2nd Circuit, Yale New Haven Hosp v Leavitt, 11/16/2006).

Unlike many policy articles, this one DOES have an abstract, which I clip below.


Importance  The US leads the world in bringing new medical products to market, but the ability to generate evidence to inform clinical practice in postmarket settings needs improvement. Although a diverse group of stakeholders is working to improve postmarket evidence generation, the role of private payers has been underappreciated.

Observations  Payers are crucial allies in improving evidence generation because better data would better inform coverage decisions, their policies and practices influence the conduct of care and research, and their claims data are a source of real-world evidence used in medical product evaluation. In addition, payers have a stake in improving evidence generation because the kinds of evidence needed to inform health care and coverage decisions are often not available when a product enters the market and may not be generated without their involvement. Here, we describe several key steps payers could take to improve evidence generation, including participating in efforts to reduce administrative and financial barriers to the conduct of clinical trials, directly incentivizing evidence generation on high-priority questions by funding potential cost-saving trials, increasing engagement with the medical products industry on evidentiary needs for coverage decisions, and improving usability of claims data by reducing data lags and routinely recording unique device identifiers. Broad payer engagement with US Food and Drug Administration recommendations regarding evidence generation will ensure that the opportunities to participate in clinical research are extended to all communities and that evidence needed to inform care is generated in trials and surveillance systems that reflect the clinical reality across the US.

Conclusions and Relevance  Increasing payer involvement in evidence generation can benefit all participants in the medical innovation ecosystem. The importance of payers in these efforts will continue to grow in response to imperatives to increase integration of care and research, engage a diverse set of communities in clinical research, and move toward alternative payment models.


FDAs Arguments, Payers, Data, and MolDx

Just like a famous criminal said he robbed banks, "Because that's where the money is," FDA looks to payers for real world evidence, because that's where the claims are.  CMS has tons of Medicare data, from claims forms, some free, some available on payment for data runs.   But in diagnostics, more and more claims fall under the MolDx unlisted code 81479, plus a Z code.   (81479 is virtually unused outside of MolDx MACs).   But if you buy that data, you won't know the Z codes, and can't do public health research.  Even if CMS is able to sell you the claim field containing the Z code, the Z code is confidential, so you won't know what test, or category of test, was used for large proportions of molecular test claims.


AI Corner

I also pulled an AI-generated summary (Chat GPT 4).


Summary of the Article: "Why Evidence Generation Should Matter to Payers and How They Can Help"

Authors: Ali B. Abbasi, MD; Lesley H. Curtis, PhD; Lee A. Fleisher, MD; Robert M. Califf, MD

Main Points:

  1. Current State of Evidence Generation:

    • The US excels in bringing new medical products to market but lags in generating postmarket evidence that informs clinical practice and policy.
    • Postmarket evidence is crucial for understanding comparative effectiveness, cost-effectiveness, and optimal sequencing of treatments.
  2. Role of the FDA:

    • The FDA is working on enhancing real-world evidence (RWE) in regulatory decisions, promoting pragmatic clinical trials, improving trial diversity, and integrating care with research.
    • The agency collaborates with federal and external partners but sees an underappreciated role for private payers in supporting evidence generation.
  3. Importance of Payers:

    • Private payers are critical for generating evidence to improve health outcomes and guide coverage decisions.
    • They influence care and research through their policies and practices, and their claims data is a valuable source of RWE.
  4. Proposed Steps for Payers:

    • Reduce Barriers: Streamline administrative and financial barriers to conducting clinical trials.
    • Incentivize Evidence Generation: Fund trials addressing high-priority questions and cost-saving interventions.
    • Engage with Industry: Collaborate with medical product developers on evidentiary needs for coverage decisions.
    • Improve Claims Data: Enhance usability of claims data by reducing lags and recording unique device identifiers.
  5. Benefits of Payer Involvement:

    • Increased payer involvement can lead to better evidence generation, ultimately benefiting all stakeholders in the medical innovation ecosystem.
    • This involvement aligns with broader goals of integrating care and research, engaging diverse communities in clinical trials, and transitioning to alternative payment models.
  6. Case Studies and Examples:

    • Historical precedents and current initiatives illustrate the potential for payer-supported trials to provide valuable evidence and cost savings.
    • Examples include CMS’s coverage with evidence development policy and the Enhancing Oncology Model, which emphasize the importance of payer engagement in research.
  7. Operational Considerations:

    • Improving claims data quality can enhance postmarket surveillance and support the efficient conduct of clinical trials.
    • Initiatives like the FDA’s Sentinel Initiative and the Biologics Effectiveness and Safety System demonstrate the utility of high-quality claims data in regulatory decisions.
  8. Conclusion:

    • Increasing payer engagement in evidence generation is essential for the future of medical innovation and healthcare delivery.
    • Collaboration among stakeholders, including payers, is necessary to ensure that evidence generation is integrated with clinical practice and addresses public health priorities.

Author Information:

  • Ali B. Abbasi, MD: Affiliated with the US Food and Drug Administration (FDA).
  • Lesley H. Curtis, PhD: Affiliated with the US Food and Drug Administration (FDA).
  • Lee A. Fleisher, MD: Affiliated with the University of Pennsylvania, Philadelphia.
  • Robert M. Califf, MD: Commissioner of the US Food and Drug Administration (FDA), based at the White Oak Campus in Silver Spring, Maryland.

Article Citation:

  • JAMA. doi:10.1001/jama.2024.7616
  • Published online July 1, 2024.

This article emphasizes the pivotal role of private payers in generating postmarket evidence to improve healthcare outcomes and support effective medical innovation. The authors argue for a collaborative approach involving payers, the FDA, and other stakeholders to address gaps in evidence and enhance the quality and applicability of clinical research.