Public Comments

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MEMORANDUM
To:

From:
Date:
Re:

Hon. Robert F. Kennedy, Jr., Secretary of Health and Human Services
Hon. Mehmet Oz, MD, Administrator, Centers for Medicare and Medicaid
Services
Andrew Langer, President, Main Street Foundation
August 10, 2026
Comments on the Department of Health and Human Services Centers for
Medicare and Medicaid Services Request, “Collection Requirements for
Compendia for Determination of Medically-accepted Indications for Offlabel Uses of Drugs and Biologicals in an Anti-cancer Chemotherapeutic
Regimen (CMS-10302),” Docket #CMS-2026-2180, Published June 10, 2026

Below are comments of the Main Street Foundation’s Center for Regulatory Analysis and
Engagement (CRAE) in response to the Department of Health and Human Services Centers for
Medicare and Medicaid Services Request, “Collection Requirements for Compendia for
Determination of Medically-accepted Indications for Off-label Uses of Drugs and Biologicals in
an Anti-cancer Chemotherapeutic Regimen (CMS-10302),” Docket #CMS-2026-2180,
published June 10, 2026
CRAE is a project of the Main Street Foundation, a recently-formed non-profit, non-partisan
501(c)(3) research and education foundation. Our mission is to bring a disciplined, commonsense perspective to the regulatory process, one grounded in real-world experience, sound
science, and rigorous economic analysis. We work to ensure that the costs, risks, and benefits of
regulatory proposals are evaluated transparently and accurately, and that the voices, interests,
and freedoms of Americans, particularly small businesses and working families, are
meaningfully represented in regulatory debates. Above all, we focus on outcomes: regulations
should address real problems, function effectively in practice, and improve conditions on the
ground—not exacerbate the challenges they are intended to solve.

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INTRODUCTION
The Main Street Foundation’s Center for Regulatory Analysis and Engagement (“CRAE”)
appreciates the opportunity to comment on the Centers for Medicare & Medicaid Services’
proposed extension of this information collection. Transparent, evidence-based evaluation of offlabel anticancer therapies serves patients, physicians, taxpayers, and the Medicare program.
Appropriate disclosure and management of material conflicts of interest can strengthen
confidence in the compendia on which Medicare coverage decisions depend.
Drug compendia are not merely informational publications. Their evaluations can materially
affect whether an off-label use of a drug or biological qualifies as a medically accepted
indication and, consequently, whether a Medicare beneficiary can obtain coverage. Timely and
reliable evaluations are especially important for patients with rare cancers, molecularly defined
conditions, or diseases for which labeled therapies have proven ineffective.
CMS proposes an extension of an existing information collection rather than a substantive
change to Medicare coverage policy. CRAE supports continuing the collection. A routine
extension nevertheless should involve more than carrying forward historical assumptions. CMS
should determine whether the information collected remains necessary, useful, appropriately
tailored, and sufficient to evaluate the operation of a system with significant consequences for
patient access.
CMS should modernize the collection to provide meaningful information about the timeliness,
transparency, consistency, and reconsideration procedures of recognized compendia. It should
also revalidate its burden estimates, preserve methodological flexibility, and avoid requirements
that could unnecessarily delay evidence evaluation or discourage qualified compendia from
participating.
I. Compendium Processes Have Direct Consequences for Patient Access
Drug compendia are professionally maintained references that evaluate drugs, biological
products, clinical evidence, and medically accepted uses. In the context of anticancer treatment, a
supportive citation in a CMS-recognized compendium can help establish that an off-label use is
medically accepted. The physician’s authority to prescribe a therapy off label and the patient’s
practical ability to obtain Medicare reimbursement are distinct questions. Compendium treatment
of an indication can help bridge that divide.
Off-label use may allow an existing therapy to reach patients without waiting for a manufacturer
to obtain FDA approval for an additional indication. This pathway is especially valuable for rare
cancers, small patient populations, and molecularly targeted uses for which pursuing
supplemental approval may not be commercially practical. Repurposing an existing therapy can
also provide an alternative to developing an entirely new product and may enable patients to
benefit more quickly from credible emerging evidence.
The compendia therefore exercise substantial gatekeeping influence. Transparent and timely
evaluation can facilitate access while preserving evidence-based safeguards. Slow, opaque,
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inconsistent, or outdated evaluations can instead become barriers to appropriate care. CMS
should assess the proposed collection in light of these real-world consequences and not treat the
collection exclusively as an exercise in paperwork compliance.
II. CMS Should Collect Information That Measures Whether the System Functions
Effectively
CMS should determine whether recognized compendia evaluate material evidence within
reasonable periods. The collection should address the frequency of updates, the ordinary interval
between receipt of significant evidence and a determination, and the availability of expedited
consideration when new evidence may materially affect patient care. It should also explain how
compendia evaluate emerging evidence involving rare diseases, small populations, or other
circumstances in which conventional large-scale studies may be difficult to conduct.
Transparency should extend beyond conflict disclosures. Stakeholders should be able to
understand the evidentiary standards, classification methods, and review procedures that produce
supportive or unfavorable determinations. The collection should identify procedures for
submitting evidence, requesting reconsideration, correcting outdated information, and disclosing
material methodological changes. It should also establish whether compendia provide sufficient
explanations for their conclusions.
CMS need not require every compendium to employ an identical methodology or classification
system. Methodological diversity may contribute useful perspectives and allow evidence to be
assessed in different ways. CMS should instead ensure that each methodology is sufficiently
clear to permit patients, physicians, researchers, manufacturers, and Medicare contractors to
understand how evidence was considered and how a determination may be revisited.
CRAE supports meaningful disclosure and management of material conflicts of interest. Those
requirements should remain focused on relationships relevant to the therapy under review and
should distinguish material interests from remote or attenuated connections. Standards should not
automatically exclude qualified clinicians and researchers merely because their expertise
includes appropriate work with pharmaceutical manufacturers. Transparent disclosure,
proportionate safeguards, and reasoned management of conflicts are preferable to categorical
rules that may deprive reviews of valuable expertise.
III. CMS Should Reevaluate Its Burden Estimates and Minimize Unnecessary Costs
CMS estimates that the collection will involve 845 respondents, 900 annual responses, and 5,135
annual hours. The same figures appeared in CMS’s 2023 extension. CMS should explain
whether it independently validated these estimates for the present request, identify the data
supporting them, and account for changes in the number and activities of affected participants.
The environment in which compendia operate continues to evolve. Precision medicine, targeted
biological products, rapidly developing oncology evidence, electronic publication, automated
research tools, and modern conflict-disclosure practices may have changed both the nature and
cost of compliance. Some technological developments may reduce burdens, while greater
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evidentiary volume and complexity may increase them. CMS should evaluate these competing
effects rather than automatically carrying forward historical figures.
Information requirements should remain limited to material that CMS needs and uses. When
required information is already publicly available in a reliable form, CMS should permit
incorporation by reference rather than require duplicative submissions. Standardized electronic
reporting and interoperable disclosure formats may further reduce burden while improving
clarity and comparison.
CMS should also consider the competitive effects of compliance costs. Excessive fixed costs
may discourage qualified organizations from seeking or retaining recognition as compendia. That
outcome could concentrate gatekeeping authority among a small number of institutions and
reduce methodological diversity. A proportionate collection should protect transparency and
integrity without creating unnecessary barriers to participation.
IV. CMS Should Connect the Collection to Measurable Outcomes
The Paperwork Reduction Act directs agencies to consider the practical utility of collected
information. CMS should therefore identify how it uses the information to determine whether
compendia remain current, complete reviews within reasonable periods, explain their
methodologies, provide workable reconsideration procedures, and manage material conflicts.
CMS should also assess whether deficiencies revealed through the collection are associated with
avoidable delays in access to medically supported treatments.
Such evaluation should respect the proper institutional role of CMS. The Agency should not use
an information collection to dictate particular clinical judgments, prescribe a uniform
methodology, or displace the expert evaluation entrusted to the compendia. Its responsibility is to
ensure that recognized sources satisfy statutory standards and operate through processes that are
transparent, reliable, and capable of producing timely decisions.
CMS should periodically review the collection and the operation of the compendium-recognition
system. That review should examine whether the information collected has produced identifiable
administrative, evidentiary, or patient-access benefits. Future extensions should rely on current
data, describe material changes in compendium operations, and explain how CMS has used the
collected information since the preceding approval.
CONCLUSION
CRAE supports extending this information collection and maintaining appropriate transparency
and conflict-of-interest safeguards. Compendium determinations can influence whether Medicare
beneficiaries obtain access to medically accepted off-label cancer therapies. That consequence
makes the quality and utility of this collection important even though CMS is not presently
proposing a substantive change to Medicare coverage policy.
CMS should modernize the collection to address review timeliness, evidentiary transparency,
reconsideration procedures, material conflicts, and measurable outcomes. It should
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independently revalidate its burden estimates and eliminate duplicative or unnecessary
requirements. A focused, proportionate, and current collection can protect the integrity of
Medicare coverage determinations without allowing administrative processes or opaque private
gatekeeping to delay access to innovative and medically supported care.
Sincerely,

Andrew M. Langer
President
Main Street Foundation

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