Notice2023-13544
Medicare Program; Transitional Coverage for Emerging Technologies
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
Published
June 27, 2023
Issuing agencies
Health and Human Services DepartmentCenters for Medicare & Medicaid Services
Abstract
This notice with comment period provides information to the public on the process we will use to provide transitional coverage for emerging technologies (TCET) through the national coverage determination (NCD) process under the Social Security Act (the Act). It also solicits public comment on the proposed TCET pathway.
Full Text
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<title>Federal Register, Volume 88 Issue 122 (Tuesday, June 27, 2023)</title>
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[Federal Register Volume 88, Number 122 (Tuesday, June 27, 2023)]
[Notices]
[Pages 41633-41644]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-13544]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Centers for Medicare & Medicaid Services
[CMS-3421-NC]
Medicare Program; Transitional Coverage for Emerging Technologies
AGENCY: Centers for Medicare & Medicaid Services (CMS), Department of
Health and Human Services (HHS).
ACTION: Notice with comment period.
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SUMMARY: This notice with comment period provides information to the
public on the process we will use to provide transitional coverage for
emerging technologies (TCET) through the national coverage
determination (NCD) process under the Social Security Act (the Act). It
also solicits public comment on the proposed TCET pathway.
DATES: To be assured consideration, comments must be received at one of
the addresses provided below, by 5 p.m. on August 28, 2023.
ADDRESSES: In commenting, refer to file code CMS-3421-NC.
Comments, including mass comment submissions, must be submitted in
one of the following three ways (please choose only one of the ways
listed):
1. Electronically. You may submit electronic comments on this
regulatory document to <a href="https://www.regulations.gov">https://www.regulations.gov</a>. Follow the ``Submit
a comment'' instructions.
2. By regular mail. You may mail written comments to the following
address ONLY: Centers for Medicare & Medicaid Services, Department of
Health and Human Services, Attention: CMS-3421-NC, P.O. Box 8013,
Baltimore, MD 21244-8013.
Please allow sufficient time for mailed comments to be received
before the close of the comment period.
3. By express or overnight mail. You may send written comments to
the following address ONLY: Centers for Medicare & Medicaid Services,
Department of Health and Human Services, Attention: CMS-3421-NC, Mail
Stop C4-26-05, 7500 Security Boulevard, Baltimore, MD 21244-1850.
FOR FURTHER INFORMATION CONTACT: Lori Ashby, (410) 786-6322.
SUPPLEMENTARY INFORMATION:
Inspection of Public Comments: All comments received before the
close of the comment period are available for viewing by the public,
including any personally identifiable or confidential business
information that is included in a comment. We post all comments
received before the close of the comment period on the following
website as soon as possible after they have been received: <a href="https://www.regulations.gov">https://www.regulations.gov</a>. Follow the search instructions on that website to
view public comments. CMS will not post on <a href="http://Regulations.gov">Regulations.gov</a> public
comments that make threats to individuals or institutions or suggest
that the individual will take actions to harm the individual. CMS
continues to encourage individuals not to submit duplicative comments.
We will post acceptable comments from multiple unique commenters even
if the content is identical or nearly identical to other comments.
[[Page 41634]]
I. Background
This notice describes the process we will use to provide
transitional coverage for emerging technologies (TCET) through the
national coverage determination (NCD) process. The TCET pathway is
designed to deliver transparent, predictable, and expedited national
coverage for certain eligible Breakthrough Devices that are Food and
Drug Administration (FDA) market authorized. It builds upon the Centers
for Medicare & Medicaid Services' (CMS') experience with the Parallel
Review program and the Coverage with Evidence Development (CED)
pathway. Additionally, the TCET pathway reflects the feedback received
from multiple stakeholder groups, including beneficiaries, patient
groups, medical professionals and societies, medical device
manufacturers, other Federal partners, and others involved in
developing innovative medical devices. This feedback was obtained from
informal and formal meetings, the comments we received as we conducted
rulemaking for the Medicare Coverage of Innovative Technologies (MCIT)
pathway (referenced later in this section) as well as during the
listening sessions that were held following the repeal of the MCIT/
Reasonable and Necessary (R&N) final rule (86 FR 62944, November 15,
2021). The TCET pathway described in this notice is intended to balance
multiple considerations when making coverage determinations: (1)
facilitating early, predictable and safe beneficiary access to new
technologies; (2) reducing uncertainty about coverage by evaluating
early the potential benefits and harms of technologies with innovators;
and (3) encouraging evidence development if notable evidence gaps exist
for coverage purposes. Further, the TCET pathway aims to coordinate
benefit category determination, coding, and payment reviews and to
allow any evidence gaps to be addressed through fit-for-purpose
studies.
The Medicare program serves over 62 million beneficiaries and is
the largest single health care purchaser in the U.S. Currently,
approximately 60 percent of the total Medicare beneficiary population,
or 36 million Medicare beneficiaries, receive coverage through Medicare
fee-for-service (FFS). More than 1.1 billion Medicare FFS claims were
processed in fiscal year (FY) 2021, comprised of approximately 221
million Part A claims (such as inpatient care in hospitals, skilled
nursing facility care, hospice care, and home health care) and 956
million Part B claims (such as doctor and other health care services
and outpatient care, durable medical equipment, and some preventive
services), providing approximately $424 billion in Medicare FFS
benefits.\1\
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\1\ <a href="https://www.cms.gov/Medicare/Medicare-Contracting/Medicare-Administrative-Contractors/What-is-a-MAC">https://www.cms.gov/Medicare/Medicare-Contracting/Medicare-Administrative-Contractors/What-is-a-MAC</a>.
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Medicare covers a wide range of items and services. In general, in
order for an item or service to be covered under Medicare, it must meet
the standard described in section 1862(a)(1)(A) of the Social Security
Act (the Act)--that is, it must be reasonable and necessary for the
diagnosis or treatment of illness or injury or to improve the
functioning of a malformed body member. CMS makes reasonable and
necessary coverage decisions through various pathways in order to
facilitate expeditious beneficiary access to items and services that
meet the statutory standard for coverage. We recognize that new
approaches are needed to make decisions on certain new items and
services, such as medical devices, more quickly to provide expedited
access to new and innovative medical technologies. On November 15, 2021
(86 FR 62944), CMS published a final rule that repealed an earlier rule
that never became legally effective and thus was not implemented.\2\ As
promised in the repeal, CMS has conducted additional opportunities to
engage with the public and stakeholders. We have incorporated that
input, along with input gathered in MCIT rulemaking, into our plans to
improve the Medicare coverage process when making decisions on certain
emerging technologies at the national level.
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\2\ <a href="https://www.govinfo.gov/content/pkg/FR-2021-11-15/pdf/2021-24916.pdf">https://www.govinfo.gov/content/pkg/FR-2021-11-15/pdf/2021-24916.pdf</a>.
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One of the issues identified in the prior rulemaking was that the
agency did not adequately address how certain steps, which are
necessary to implement national coverage determinations for a new item
or service, would be accomplished in a timely manner. Specifically,
under the Medicare program an item or service must fall within the
parameters of a benefit category that is within the scope of Part A or
Part B. Commenters have requested that CMS explain how benefit category
determinations (BCDs) will be made in connection with emerging
technology. CMS was also encouraged to align coding and payment
processes to facilitate coverage and payment for new or emerging
technologies.
Over the last several years, stakeholders have expressed support
for coverage process improvements and a new pathway that is more
flexible, transparent, predictable, and collaborative. Additionally,
stakeholders expressed that that they would like for CMS to develop a
more agile, iterative evidence review process that considers real world
evidence and fit-for-purpose evidence study designs. Further, we have
heard concerns from stakeholders that device coverage lags further
behind that of drugs and biologics and, devices are more in need of a
program like TCET. In light of the unique FDA criteria for Breakthrough
designation status (described later in this document), we are limiting
the TCET pathway to certain eligible FDA-designated Breakthrough
Devices, since we believe that this is the area with the most immediate
need.
We are committed to establishing an alternative coverage pathway
that better balances the needs of beneficiaries, patient groups,
medical professionals and societies, medical device manufacturers, and
others involved in developing innovative medical devices.
A. Current Medicare Coverage Mechanisms
Items and services, including medical devices, are currently
covered in Medicare in one of three ways, presented here for context.
The TCET pathway described in this notice will leverage the existing
NCD pathway, and CED in particular, to provide a streamlined coverage
pathway for emerging technologies. We note that the TCET pathway will
not alter the existing standards for these coverage mechanisms.
1. Claim-by-Claim Adjudication
In the absence of an NCD or a local coverage determination (LCD),
Medicare Administrative Contractors (MACs) make coverage decisions
under section 1862(a)(1)(A) of the Act and may cover items and services
on a claim-by-claim basis if the MAC determines them to be reasonable
and necessary for individual patients. Though claims may be denied if
they are not determined to be reasonable and necessary, the claim-by-
claim adjudication pathway remains the fastest path to potential
coverage. The majority of all Medicare Parts A and B claims have
coverage determined through the claim-by-claim adjudication process.
2. Local Coverage Determinations (LCDs)
MACs develop LCDs under section 1862(a)(1)(A) that apply only
within their geographic jurisdictions (see sections 1862(l)(6)(B) and
1869(f)(2)(B) of the Act). LCDs govern only the issuing MAC's claims
adjudication and
[[Page 41635]]
are not controlling authorities for qualified independent contractors
or administrative law judges in the claims adjudication process.
The MACs follow specific guidance for developing LCDs for Medicare
coverage as outlined in the CMS Program Integrity Manual (PIM), Chapter
13. LCDs generally take 9 to 12 months to develop. MACs usually
finalize proposed LCDs within 365 days from opening, per Chapter
13.5.1--Local Coverage of the PIM.\3\ That chapter will continue to be
used in making determinations under section 1862(a)(1)(A) of the Act
for items and services at the local level.
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\3\ CMS Program Integrity Manual, Chapter 13 Local Coverage
Determinations, available at <a href="https://www.cms.gov/Regulations-and-Guidance/Guidance/Manuals/downloads/pim83c13.pdf">https://www.cms.gov/Regulations-and-Guidance/Guidance/Manuals/downloads/pim83c13.pdf</a>.
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3. National Coverage Determinations (NCDs)
The term ``national coverage determination'' is defined in section
1862(l)(6)(A) of the Act and means a determination by the Secretary of
the Department of Health and Human Services (the Secretary) with
respect to whether or not a particular item or service is covered
nationally under Title XVIII of the Act. In general, NCDs are national
policy statements published to identify the circumstances under which a
particular item or service will be considered covered (or not covered)
by Medicare. NCDs serve as generally applicable rules to ensure that
similar claims for items or services are covered in the same manner.
Often an NCD is written in terms of defined clinical characteristics
that identify a population that may or may not receive Medicare
coverage for a particular item or service. Traditionally, CMS relies
heavily on health outcomes data to make NCDs.
Most NCDs have involved determinations under section 1862(a)(1)(A)
of the Act, but NCDs can be made based on other provisions of the Act,
such as section 1862(a)(1)(E) of the Act. Under section 1862(a)(1)(E)
of the Act, Medicare has provided coverage for certain promising
technologies with a limited evidence base on the condition that they
are furnished in the context of approved clinical studies or with the
collection of additional clinical data. CMS has used section
1862(a)(1)(E) of the Act to support the ``Coverage with Evidence
Development'' or ``CED'' policy since July 12, 2006, and the most
recent CED policy is described in our November 20, 2014 guidance
document.\4\ In general, CED enables providers and suppliers to perform
high quality studies that we expect will produce evidence that may lead
to positive national coverage determinations under section
1862(a)(1)(A) of the Act.
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\4\ The 2014 guidance document is available at <a href="https://www.cms.gov/medicare-coverage-database/view/medicare-coverage-document.aspx?MCDId=27">https://www.cms.gov/medicare-coverage-database/view/medicare-coverage-document.aspx?MCDId=27</a>.
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The Agency for Healthcare Research and Quality (AHRQ) reviews all
CED NCDs established under section 1862(a)(1)(E) of the Act. Consistent
with section 1142 of the Act, AHRQ collaborates with CMS to define
standards for the clinical research studies to address the CED
questions and meet the general standards for CED studies (<a href="https://www.cms.gov/Medicare/Coverage/Coverage-with-Evidence-Development">https://www.cms.gov/Medicare/Coverage/Coverage-with-Evidence-Development</a>).
NCDs also include a determination on whether the item or service
under consideration has a Medicare benefit category under Part A or
Part B,\5\ such as inpatient hospital services, physicians' services,
durable medical equipment, or others. All items and services coverable
by Medicare must fall within the scope of a statutory benefit category
and many of these specific terms are defined under section 1861 of the
Act and in implementing regulations. BCDs are made outside the Coverage
and Analysis Group. While they may often be completed within 3 months,
in some cases BCDs may take considerably longer. While CMS is working
to better align the coverage and BCD review processes, manufacturers
should be aware that in some cases benefit category reviews may not be
completed within the accelerated timeframes needed for the TCET
pathway. Moreover, in order to be covered, the item or service must not
be excluded from coverage by statute or our regulations at 42 CFR part
411, subpart A. The NCD pathway, which has statutorily prescribed
timeframes, generally takes 9 to 12 months to complete.\6\
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\5\ Note: Medicare does not develop NCDs for Part D.
\6\ Section 1869(f)(4) of the Act.
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In addition to these coverage pathways, CMS has established a
Clinical Trial Policy (CTP) NCD 310.1. The CTP policy is applied when
Medicare covers routine care items and services (but generally not the
technology under investigation) in a clinical study that is supported
by certain Federal agencies. The CTP coverage policy was developed in
2000.\7\ We note that coverage under CED and the CTP may not occur at
the same time. Additionally, this coverage policy has not generally
been utilized by device manufacturers because they usually seek
coverage of the device under investigation, which is not always
available under the CTP.
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\7\ CMS, National Coverage Determination for Routine Costs in
Clinical Trials available at <a href="https://www.cms.gov/medicare-coverage-database/details/ncd-details.aspx?NCDId=1&fromdb=true">https://www.cms.gov/medicare-coverage-database/details/ncd-details.aspx?NCDId=1&fromdb=true</a>.
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Lastly, CMS has established the Parallel Review program. In the
September 17, 2010 Federal Register (75 FR 57045), FDA and CMS
announced their intention to initiate a Parallel Review pilot program
in an effort to increase quality of patient health care by facilitating
earlier access to innovative medical technologies for Medicare
beneficiaries. In the October 24, 2016 Federal Register (81 FR 73113),
FDA and CMS published a joint notice that announced and described the
processes for the fully implemented Program for Parallel Review of
Medical Devices.
Parallel Review is a mechanism for FDA and CMS to simultaneously
review the clinical data submitted by a manufacturer about a medical
device in order to help decrease the time between FDA's approval of an
original or supplemental premarket approval (PMA) application or
granting of a de novo classification request (De Novo request) and the
subsequent CMS proposed NCD. Parallel Review has two stages: (1) FDA
and CMS meet with the manufacturer to provide feedback on the proposed
pivotal clinical trial; and (2) FDA and CMS concurrently review (``in
parallel'') the clinical trial results submitted in the PMA
application, or De Novo request. FDA and CMS independently review the
data to determine whether it meets their respective Agency's standards
and communicate with the manufacturer during their respective reviews.
This program relies upon a technology having a quality evidence base to
support the clinical analysis for the NCD.
B. Differences Between FDA and CMS Review
While FDA and CMS have a well-established history of collaboration
in review of evidence for emerging medical technologies, FDA and CMS
must consider different legal authorities and apply different statutory
standards when making marketing authorization and coverage decisions,
respectively, for medical devices. Generally, FDA makes marketing
authorization decisions based on whether the relevant statutory
standard for safety and effectiveness is met, while CMS generally makes
NCDs based on whether an item or service is reasonable and necessary
for the diagnosis or treatment of an illness or
[[Page 41636]]
injury for individuals in the Medicare population.
These two reviews are separate and are conducted independently by
the two agencies. At CMS, we respect the findings of our FDA colleagues
and appreciate the expertise they bring to the premarket review process
under the Federal Food, Drug, and Cosmetic Act (FD&C Act). The FDA
review of devices does not focus specifically on the Medicare
population.
Among other objectives, FDA conducts premarket review of certain
devices to evaluate their safety and effectiveness and determine if
they meet the applicable standard to be marketed in the United States.
An FDA-regulated product must receive marketing authorization \8\
(unless exempt from FDA premarket review) for at least one indication
to be eligible for consideration of Medicare coverage (except in
specific circumstances). However, FDA approval or clearance alone does
not entitle that technology to Medicare coverage, given Medicare
statutory coverage requirements. While FDA reviews devices to ensure
they meet applicable safety and effectiveness standards, there is often
limited evidence regarding whether the device is clinically beneficial
for Medicare patients specifically because of the lack of evidence
concerning individuals in the Medicare population. This is an important
consideration for manufacturers and other interested parties who are
seeking the most appropriate coverage pathway under Medicare. Where
there is limited evidence on the health outcomes for individuals in the
Medicare population, there may be insufficient evidence to support a
fully favorable Medicare national coverage determination under section
1862(a)(1)(A) of the Act. In these instances, it is difficult to make a
prospective national reasonable and necessary determination as to
whether Medicare should cover the device with evidence development or
should limit the NCD to coverage for only individuals with certain
conditions or procedures performed by certain practitioners or health
care facilities with expertise necessary to safely treat the individual
with the new technology.
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\8\ Additional information on FDA marketing authorization,
specifically device approvals, denials and clearances can be
accessed here: <a href="https://www.fda.gov/medical-devices/products-and-medical-procedures/device-approvals-denials-and-clearances">https://www.fda.gov/medical-devices/products-and-medical-procedures/device-approvals-denials-and-clearances</a>.
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In general, as discussed, under the Medicare statute (section
1862(a)(1)(A) of the Act), Congress required CMS to determine whether
items and services are reasonable and necessary to diagnose or treat an
illness or injury or to improve the functioning of a malformed body
member for an individual with Medicare. For CMS, the evidence base
underlying FDA's decision to approve or clear a device for particular
indications for use has often been crucial for determining Medicare
coverage through the NCD process. CMS looks to the evidence supporting
FDA market authorization and the device's approved or cleared
indications for use for evidence generalizable to the Medicare
population, data on improvement in health outcomes, and durability of
those outcomes. If there are no data on those elements in the Medicare
population, it is difficult for CMS to make an evidence-based decision
whether the device is reasonable and necessary for the Medicare
population.
Because Medicare beneficiaries are often older, with multiple
comorbidities, and are often underrepresented or not represented in
many clinical studies, CMS considers whether the evidence shows that
the item or service will improve the health of Medicare patients.\9\
According to a recent study,<SUP>10 11</SUP> approximately 50 percent
of Medicare patients have two or more diseases. Clinical studies that
are conducted in order to gain FDA market authorization are not
necessarily required to include participants with similar demographics
and characteristics of the Medicare population. A potential reason
there may not be a strong evidence base specific to the Medicare
population could include the desire by device manufacturers to
demonstrate the safety and effectiveness of a device as clearly as
possible. To achieve this aim, many studies impose stringent exclusion
criteria that disqualify individuals with certain characteristics, such
as comorbidities and concomitant treatment, that might make the effect
of the investigational device more difficult to determine.
Consequently, the potential benefits and harms of a device for older
patients with more comorbidities may not be well understood at the time
of FDA market authorization.
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\9\ Davide L Vetrano, MD, Katie Palmer, Ph.D., Alessandra
Marengoni, MD, Ph.D., Emanuele Marzetti, MD, Ph.D., Fabrizia
Lattanzio, MD, Ph.D., Regina Roller-Wirnsberger, MD, MME, Luz Lopez
Samaniego, Ph.D., Leocadio Rodr[iacute]guez-Ma[ntilde]as, MD, Ph.D.,
Roberto Bernabei, MD, Graziano Onder, MD, Ph.D., Frailty and
Multimorbidity: A Systematic Review and Meta-analysis, The Journals
of Gerontology: Series A, Volume 74, Issue 5, May 2019, Pages 659-
666, <a href="https://doi.org/10.1093/gerona/gly110">https://doi.org/10.1093/gerona/gly110</a>.
\10\ Tan, Y.Y., Papez, V., Chang, W.H., Mueller, S.H., Denaxas,
S., & Lai, A.G. (2022). Comparing clinical trial population
representativeness to real-world populations: an external validity
analysis encompassing 43 895 trials and 5 685 738 individuals across
989 unique drugs and 286 conditions in England. The Lancet Healthy
Longevity, 3(10), e674-e689.
\11\ Varma T, Mello M, Ross JS, et al Metrics, baseline scores,
and a tool to improve sponsor performance on clinical trial
diversity: retrospective cross sectional study BMJ Medicine
2023;2:e000395. doi: 10.1136/bmjmed-2022-000395.
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C. FDA Breakthrough Devices Program
Under the TCET coverage pathway, CMS will coordinate with FDA and
manufacturers of Breakthrough Devices as those devices move through the
FDA premarket review processes to ensure timely Medicare coverage
decisions following any FDA market authorization, as described in
detail later in this section. The Breakthrough Devices Program is an
evolution of the Expedited Access Pathway Program and the Priority
Review Program. See section 515B of the FD&C Act, 21 U.S.C. 360e-3; see
also final guidance for industry entitled, ``Breakthrough Devices
Program.'' \12\
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\12\ <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents/breakthrough-devices-program">https://www.fda.gov/regulatory-information/search-fda-guidance-documents/breakthrough-devices-program</a>.
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FDA's Breakthrough Devices Program is not for all new medical
devices; rather, it is only for those that FDA determines meet the
standards for Breakthrough Device designation. In accordance with
section 515B of the FD&C Act (21 U.S.C. 360e-3), the Breakthrough
Devices Program is for medical devices and device-led combination
products \13\ that meet two criteria. The first criterion is that the
device provides for more effective treatment or diagnosis of life-
threatening or irreversibly debilitating human disease or conditions.
The second criterion is that the device must satisfy one of the
following elements: It represents a breakthrough technology; no
approved or cleared alternatives exist; it offers significant
advantages over existing approved or cleared alternatives, including
the potential, compared to existing approved alternatives, to reduce or
eliminate the need for hospitalization, improve patient quality of
life, facilitate patients' ability to manage their own care (such as
through self-directed personal assistance), or establish long-term
clinical efficiencies; or device availability is in the best interest
of patients (for more information see 21 U.S.C. 360e-3(b)(2)). These
criteria make Breakthrough designated devices unique. Devices meeting
these criteria
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are also likely to be highly relevant to the needs of the Medicare
population, if the item or service falls within a Medicare benefit
category.
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\13\ Information on device-led combination products can be
accessed here: <a href="https://www.fda.gov/media/119958/download">https://www.fda.gov/media/119958/download</a>.
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II. Provisions of the Notice With Comment Period
This notice proposes to create the TCET pathway. Since the TCET
pathway relies on our existing authorities, we believe that
establishing TCET through a procedural notice rather than rulemaking
has the advantages that it is faster to implement and can be more
easily modified as we gain experience with the approach. We also
describe the procedures for how stakeholders and the public at large
may engage with CMS to facilitate the TCET pathway. The topics
addressed in the notice include the following: (1) TCET general
principles; (2) appropriate candidates for the TCET pathway; (3)
procedures for the TCET pathway; and (4) general roles.
We continue to pursue our efforts to work with various sectors of
the scientific and medical community to develop and publish guidance
documents on our website that describe our approach when analyzing
scientific and clinical evidence to develop an NCD. In response to
stakeholder feedback, our proposed CED and Evidence Review guidance
documents propose to incorporate robust fit-for-purpose evidence
development where manufacturers may use fit-for-purpose studies to
close any evidence gaps. Fit-for-purpose studies are those where the
study design, analysis plan, and study data can credibly answer the
research question. Additionally, CMS intends to publish a series of
guidance documents that review health outcomes and their clinically
meaningful differences within priority therapeutic areas. The public
will have an opportunity to provide comments on these guidance
documents which will be available on the CMS coverage website which can
be accessed at <a href="https://www.cms.gov/Medicare/Coverage/CoverageGenInfo/index.html">https://www.cms.gov/Medicare/Coverage/CoverageGenInfo/index.html</a>.
A. TCET Pathway--An Opportunity To Accelerate Patient Access to
Beneficial Medical Products While Generating Evidence
Since CMS started covering technology in the context of clinical
studies almost two decades ago, the timing of evidence development and
the stages of the technology development lifecycle have evolved. Over
the past few years, innovative technologies have come on the market
earlier in the technology development lifecycle and reached the market
with limited or developing evidence for coverage purposes. CMS has
received inquiries for coverage of new technologies that are early in
the product lifecycle, which means the clinical evidence is just
starting to accumulate. For new technologies, it is rare that there is
sufficient clinical evidence to support broad national coverage at this
point.
In general, CMS relies heavily on health outcomes data, including
but not limited to health outcomes data as it relates to the Medicare
population, before proposing an NCD. Early in the product lifecycle,
there is usually evidence about whether the product is safe and may
produce the intended result: for example, a laboratory measurement,
radiographic image, physical sign or other measure that is believed to
predict clinical benefit, but is not itself a measure of clinical
benefit. However, there is often little evidence in the early stages of
the product lifestyle regarding health outcomes (for example,
mortality, disease progression, quality of life). When premarket,
pivotal clinical study data is collected to support an application to
FDA for market authorization, it provides clinical evidence for a
defined population enrolled in the study.
If there is health outcome evidence for a new technology, it may
not be generalizable to the Medicare population if Medicare
beneficiaries are insufficiently represented in pivotal clinical
studies. Medicare beneficiaries have been historically underrepresented
in pivotal studies due to age, access, multiple comorbidities, and
concurrent treatments. When there is little or limited evidence, CMS
may not have enough information to make a favorable NCD due to gaps in
research about health outcomes, including potential safety risks to the
Medicare population.
While CMS has attempted to streamline the NCD process with the
Parallel Review program, we recognize that most emerging technologies
are likely to have limited or developing bodies of clinical evidence
that may not have included the Medicare population (that is,
individuals over age 65, people with disabilities, and those with end
stage renal disease). Many Medicare beneficiaries have comorbid medical
conditions, and those factors may have limited their participation in
certain clinical trials. Additionally, we recognize the importance that
applicable clinical trials reflect the demographic and clinical
diversity among the Medicare beneficiaries who are the intended users
of the intervention. At a minimum, this includes attention to the
intended users' racial and ethnic backgrounds, sex and gender, age,
disabilities, important comorbidities, and depends on data being
available on these characteristics and relevant social determinants of
health. We believe that the TCET pathway can support manufacturers that
are interested in working with CMS to generate additional evidence that
is appropriate for Medicare beneficiaries and that may demonstrate
improved health outcomes in the Medicare population to support more
expeditious national Medicare coverage. While we believe that
leveraging the statutorily established NCD process will allow us to
responsibly cover new, innovative technologies with limited or
developing evidence, it is important that we provide an evidence
generation framework that, when appropriate, not only develops reliable
evidence for patients and their physicians but also provides safeguards
to ensure that Medicare beneficiaries are protected and continue to
receive high quality care.
Specifically, CED has been used to support evidence development for
certain innovative technologies that are likely to show benefit for the
Medicare population when the available evidence is not sufficient to
demonstrate that the technologies are reasonable and necessary for the
diagnosis or treatment of illness or injury or to improve the
functioning of a malformed body member under section 1862(a)(1)(A) of
the Act. In instances where there is limited evidence, CED may be an
option for Medicare beneficiaries seeking earlier access to promising
technologies. CED has been a pathway whereby, after a CMS and AHRQ
review, Medicare covers items and services on the condition that they
are furnished in the context of approved clinical studies or with the
collection of additional clinical data. Participation in a CED trial is
voluntary, but beneficiaries are protected by separate regulations
including those at 45 CFR part 46 related to the protection of human
research subjects.
CMS has issued a total of 26 NCDs requiring CEDs over the last two
decades to provide Medicare beneficiary access to promising items and
services that could not otherwise be covered under section
1862(a)(1)(A) of the Act. CMS has approved 109 CED studies and five
national registries to facilitate evidence development for these CED
NCDs. Forty-two of these studies have generated evidence across 14
topics covered under CED. Three CED NCD topics have had the CED
requirement removed following an NCD reconsideration and have received
national coverage.
With respect to evidence generation, the TCET pathway would build
upon
[[Page 41638]]
CMS and AHRQ's ongoing collaboration on the CED NCD process. We
anticipate that many of the NCDs conducted under the TCET pathway will
result in CED decisions, and AHRQ will continue to review all CED NCDs
consistent with current practice. Additionally, AHRQ will collaborate
with CMS as resources allow on evidence development activities
conducted to support Medicare coverage under the TCET pathway and will
have opportunities to offer feedback throughout the process that will
be shared with manufacturers. Approvals related to evidence development
will be a joint CMS-AHRQ decision. CMS and AHRQ have made iterative
refinements to the CED coverage pathway over time, and while we believe
CED has reduced barriers to innovation and expanded beneficiary access
to new technologies and therapies, our experience over the last several
years indicates that further improvements can be made to the CED
process. We believe that certain coverage decisions--in particular,
those involving innovative devices--would benefit from a more
systematic framework for CED that establishes a more predictable and
transparent approach for the public when facilitating evidence
development.
Working in conjunction with AHRQ, our goal is to improve CED so
that it fulfills its potential as a mechanism that simultaneously
reduces barriers for innovation and enables CMS to make better informed
decisions on coverage for medical devices that improve health outcomes
for Medicare beneficiaries. CMS believes that public input should
inform this effort, and we will continue to provide numerous
opportunities for stakeholders to engage with us as we convene future
Medicare Evidence Development & Coverage Advisory Committee (MEDCAC)
meetings and update specific aspects of the CED paradigm.
For example, CMS has been actively collaborating with AHRQ on
potential revisions to the general criteria for CED studies, originally
described in 2014, to ensure the criteria are up to date and continue
to maintain rigorous evidentiary standards. In November 2022, in order
to better inform the CED process, AHRQ released a final report on ``The
Analysis of Requirements for Coverage with Evidence Development
(CED).'' \14\ The AHRQ report was first released in draft form in
September 2022 and the public had an opportunity to provide comment on
the draft report. The AHRQ report served as the basis for discussion at
the February 13-14, 2023 MEDCAC meeting. CMS convened the MEDCAC to
examine the general requirements for clinical studies submitted for CMS
coverage under CED. The MEDCAC panel consisted of a variety of experts
on the topic and included an industry representative and patient
advocate. MEDCAC guest panel members included representatives from FDA,
AHRQ, and National Institutes of Health (NIH). Specifically, the MEDCAC
evaluated the CED criteria to assure that studies informing CED are
assessed using consistent, feasible, transparent and methodologically
rigorous criteria. The MEDCAC advised CMS on whether the criteria are
appropriate to ensure that studies approved to inform CED decisions
will produce informative evidence that CMS can rely on when making
future reasonable and necessary determinations.\15\ AHRQ and CMS
collaboratively evaluated the information discussed at the MEDCAC
meeting as well as the MEDCAC panel scores and are considering
corresponding refinements to the proposed new criteria. CMS is
proposing updated criteria in a proposed CED guidance document and the
public will have an opportunity to provide comment on that document.
With respect to beneficiary safeguards, the NCD process allows for
coverage with appropriate safeguards for Medicare beneficiaries
including coverage criteria based on evidence regarding eligibility,
frequency, provider experience, site of service or availability of
supporting services. Specifically, CMS develops clinician and
institutional requirements after careful review of expert physicians'
specialty society guidelines and clinical study results. These
guidelines and recommendations are often part of NCDs. Unless these
coverage criteria are established within coverage determinations,
devices could be provided by unqualified individuals, offered at
inappropriate facilities, and utilized by patients who may be unlikely
to benefit.
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\14\ <a href="https://effectivehealthcare.ahrq.gov/products/coverage-evidence-development/research-report">https://effectivehealthcare.ahrq.gov/products/coverage-evidence-development/research-report</a>.
\15\ Additional information on the MEDCAC can be found at
<a href="https://www.cms.gov/medicare-coverage-database/view/medcac-meeting.aspx?medcacid=79&year=all&sortBy=meetingdate&bc=15">https://www.cms.gov/medicare-coverage-database/view/medcac-meeting.aspx?medcacid=79&year=all&sortBy=meetingdate&bc=15</a>.
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More specifically, coverage under a CED NCD can expedite earlier
beneficiary access for individuals who volunteer to participate in the
clinical studies of innovative technology while ensuring that
systematic patient safeguards, including assurance that the technology
is provided to clinically appropriate patients, are in place to reduce
the potential risks of new technologies, or to new applications of
older technologies. CMS' current CED guidance document contains
specific criteria that details patient protections under CED. As we
note earlier, we are proposing updated criteria that reflects the
feedback received on the November 2022 AHRQ report and February 2023
MEDCAC in a proposed CED guidance document. Because the TCET pathway
described in this document would utilize the existing CED NCD process,
all of these safeguards would apply if finalized.
Stakeholder input is important to CMS and we are particularly
interested in engagement with patient advocacy organizations and
medical specialty societies as they have valuable expertise and first-
hand experience in the field that will help CMS develop Medicare
coverage policies. Because the TCET pathway would utilize the current
NCD process, these opportunities for stakeholder engagement would also
be available in TCET.
B. TCET General Principles
CMS is committed to ensuring Medicare beneficiaries have access to
emerging technologies. CMS' goal is to finalize an NCD for technologies
accepted into and continuing in the TCET pathway, within 6 months after
FDA market authorization. The TCET pathway builds off of prior
initiatives, including CED. The TCET pathway will meet the following
principles:
<bullet> Medicare coverage under the TCET pathway is limited to
certain Breakthrough Devices that receive market authorization for one
or more indications for use covered by the Breakthrough Device
designation when used according to those indications for use.
Manufacturers of FDA-designated Breakthrough Devices that fall within a
Medicare benefit category may self-nominate to participate in the TCET
pathway on a voluntary basis. We note that many Breakthrough Devices
are currently coverable without the TCET pathway because they are not
separately payable (that is, the device may be furnished under a
bundled payment, such as payment for a hospital stay) or they are
addressed by an existing NCD. Others are not indicated for use in a
population that includes Medicare beneficiaries (for example, those
devices that are targeted toward a pediatric population).
<bullet> CMS may conduct an early evidence review (Evidence
Preview, more details in section II.D.1.g. of this notice with comment
period) before FDA decides on marketing authorization for the device
and discuss with the manufacturer the best available coverage
[[Page 41639]]
pathways depending on the strength of the evidence.
<bullet> Prior to FDA marketing authorization, CMS may initiate
discussions with manufacturers to discuss any evidence gaps for
coverage purposes and the types of studies that may need to be
completed to address the gaps, which could include the manufacturer
developing an evidence development plan and confirming that there are
appropriate safeguards for Medicare beneficiaries.
<bullet> If CMS determines that further evidence development (that
is, CED) is the best coverage pathway, CMS will work with the
manufacturers to reduce the burden on manufacturers, clinicians and
patients while maintaining rigorous evidence requirements. CMS will
work to ensure we are not requiring duplicative or conflicting evidence
development with any FDA post-market requirements for the device.
<bullet> CMS does not believe that an NCD that requires CED as a
condition of coverage should last indefinitely, including under the
TCET pathway. If the evidence supports a favorable coverage decision
under CED, coverage will be time-limited to facilitate the timely
generation of sufficient evidence to inform patient and clinician
decision making and to support a Medicare coverage determination under
section 1862(a)(1)(A) of the Act.
<bullet> Manufacturers and CMS have the option to withdraw from the
pathway up until CMS opens the NCD by posting a tracking sheet. CMS
will not publicly disclose participation of a manufacturer in the TCET
pathway prior to CMS' posting of an NCD tracking sheet, unless the
manufacturer consents or has already made this information public or
disclosure is required by law. If a manufacturer does not wish the
information that would be revealed by the posting of the NCD tracking
sheet to become public, it should withdraw from the TCET pathway prior
to this point. CMS requests that a manufacturer who wishes to withdraw
from the TCET pathway notify CMS by email at <a href="/cdn-cgi/l/email-protection#336770766773505e401d5b5b401d545c45"><span class="__cf_email__" data-cfemail="174354524357747a64397f7f6439707861">[email protected]</span></a>.
C. Appropriate Candidates
Appropriate candidates for the TCET pathway would include those
devices that are--
<bullet> FDA-designated Breakthrough Devices;
<bullet> Determined to be within a Medicare benefit category; \16\
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\16\ For more information on benefit category determinations see
the CMS Innovator's Guide to Navigating Medicare (<a href="https://www.cms.gov/medicare/coverage/councilontechinnov/downloads/innovators-guide-master-7-23-15.pdf">https://www.cms.gov/medicare/coverage/councilontechinnov/downloads/innovators-guide-master-7-23-15.pdf</a>). Please note that an updated
version of the Innovators' Guide is forthcoming. The updated guide
will reflect a new name, the CMS Guide for Medical Technology
Companies and Other Interested Parties, which can be found here upon
release (the URL we have requested for this is: <a href="https://www.cms.gov/cms-guide-medical-tech-companies-other-parties">https://www.cms.gov/cms-guide-medical-tech-companies-other-parties</a>).
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<bullet> Not already the subject of an existing Medicare NCD; and
<bullet> Not otherwise excluded from coverage through law or
regulation.\17\
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\17\ Information on coverage exclusions can be accessed here:
<a href="https://www.cms.gov/Regulations-and-Guidance/Guidance/Manuals/Downloads/bp102c16.pdf">https://www.cms.gov/Regulations-and-Guidance/Guidance/Manuals/Downloads/bp102c16.pdf</a>.
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In section 201(h)(1) of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 321(h)(1), the definition of device includes diagnostic
laboratory tests. Diagnostic lab tests are a highly specific area of
coverage policy development, and CMS has historically delegated review
of many of these tests to specialized MACs. We believe that the
majority of coverage determinations for diagnostic tests granted
Breakthrough Designation should continue to be determined by the MAC
through existing pathways.
D. Procedures for the TCET Pathway
The TCET pathway has three stages: (1) premarket; (2) coverage
under the TCET pathway; and (3) transition to post-TCET coverage.
1. Premarket
a. Nominations for the TCET Pathway
The appropriate timeframe for manufacturers to submit TCET pathway
nominations to CMS is approximately 12 months prior to anticipated FDA
decision on a submission as determined by the manufacturer.
Manufacturers are generally aware of when they intend to submit their
application, and the FDA has agreed to review time goals as part of its
device user fee program.\18\ CMS encourages manufacturers not to delay
submitting nominations to facilitate alignment among CMS benefit
category determination, and coverage, coding and payment
considerations.
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\18\ For more information on the specific review time goals that
apply to different types of device premarket submissions, see MDUFA
Performance Goals and Procedures, Fiscal Years 2023 Through 2027
(<a href="https://www.fda.gov/media/158308/download">https://www.fda.gov/media/158308/download</a>).
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The manufacturer may submit a nomination for the TCET pathway by
sending an email to <a href="/cdn-cgi/l/email-protection#f0a4b3b5a4b0939d83de989883de979f86"><span class="__cf_email__" data-cfemail="025641475642616f712c6a6a712c656d74">[email protected]</span></a>, which indicates their interest in
the pathway. CMS will acknowledge receipt of nominations by email. The
following information will assist CMS in processing and responding to
nominations:
<bullet> Name of the manufacturer and relevant contact information.
<bullet> Name of the product.
<bullet> Succinct description of the technology and disease or
condition the device is intended to diagnose or treat.
<bullet> State of development of the technology (that is, in pre-
clinical testing, in clinical trials, currently undergoing premarket
review by FDA). The submission of a copy of FDA's letter granting
Breakthrough Designation and the PMA application, De Novo request or
premarket notification (510(k)) submission, if available, is preferred.
<bullet> A comprehensive list of peer-reviewed, English-language
publications that support the nominated Breakthrough Device as
applicable/available.
<bullet> A statement that the medical device is not excluded by
statute from Part A or Part B Medicare coverage or both, and a list of
Part A or Part B or both Medicare benefit categories, as applicable,
into which the manufacturer believes the medical device falls.
Additionally, manufacturers are encouraged to provide additional
specific information to help to facilitate benefit category and coding
determinations.
Two good sources of information to facilitate the development of
nomination submissions are the CMS Coverage website at <a href="https://www.cms.gov/Center/Special-Topic/Medicare-Coverage-Center">https://www.cms.gov/Center/Special-Topic/Medicare-Coverage-Center</a> and the CMS
Innovators' Guide to Navigating Medicare at <a href="https://www.cms.gov/medicare/coverage/councilontechinnov/downloads/innovators-guide-master-7-23-15.pdf">https://www.cms.gov/medicare/coverage/councilontechinnov/downloads/innovators-guide-master-7-23-15.pdf</a>, which provides information that may facilitate durable
medical equipment, prosthetics, orthotics, and supplies (DMEPOS) BCDs,
along with coverage, coding and payment processes, and considerations.
We note that an updated version of the Innovators' Guide is
forthcoming. The updated guide will reflect a new name, the CMS Guide
for Medical Technology Companies and Other Interested Parties, which
can be found at the URL we have requested for this upon release:
<a href="https://www.cms.gov/cms-guide-medical-tech-companies-other-parties">https://www.cms.gov/cms-guide-medical-tech-companies-other-parties</a>.
<bullet> A statement describing how the medical device addresses
the health needs of the Medicare population.
<bullet> A brief statement explaining why the device is an
appropriate candidate for the TCET pathway as described under the
section II.C. of this document (``B. Appropriate Candidates'').
CMS will contact the manufacturer by email to confirm that a
submitted nomination appears to be complete and is under review by CMS.
This email will include the date that CMS initiated the review of the
complete nomination. If
[[Page 41640]]
the nomination is not complete, CMS will contact the manufacturer for
more information.
b. CMS Consideration
CMS may contact the manufacturer to request supplemental
information to ensure a timely review of the nomination. CMS commits to
making at least a preliminary decision to provisionally accept or
decline a nomination within 30 business days following the date noted
in CMS' email to manufacturer as described previously and will
communicate this information to the manufacturer by email. The process
for determining whether or not the technology falls within a benefit
category may take longer and, in those instances, CMS will send a
subsequent email to the manufacturer communicating a final decision on
the nomination when the benefit category review is completed.
c. Intake Meeting
Following the submission of a complete TCET nomination, CMS will
offer an initial meeting with the manufacturer to review the nomination
within 20 business days of receipt of a complete nomination. In this
initial meeting, the manufacturer is expected to describe the device,
its intended application, place of service, a high-level summary of the
evidence supporting its use, and the anticipated timeframe for FDA
review. CMS will answer any questions about the TCET process. CMS
intends for these meetings to be held remotely to reduce travel burden
on manufacturers and expeditiously meet these timeframes. These
meetings will have a duration of 30 minutes. If a manufacturer declines
to meet or if there is difficulty finding a mutually convenient time
for the meeting, then CMS action on the nomination may be delayed.
d. Coordination With FDA
After CMS initiates review of a complete, formal nomination,
representatives from CMS will meet with their counterparts at FDA to
learn more information about the technology in the nomination to the
extent the Agencies have not already done so. These discussions may
help CMS gain a better understanding of the device and potential FDA
review timing.
As noted in the Memorandum of Understanding \19\ between FDA and
CMS, FDA and CMS recognize that the following types of information
transmitted between them in any medium and from any source must be
protected from unauthorized disclosure: (1) trade secret and other
confidential commercial information that would be protected from public
disclosure pursuant to Exemption 4 of the Freedom of Information Act
(FOIA); (2) personal privacy information, such as the information that
would be protected from public disclosure pursuant to Exemption 6 or
7(c) of the FOIA; or (3) information that is otherwise protected from
public disclosure by Federal statutes and their implementing
regulations (for example, the Trade Secrets Act (18 U.S.C. 1905), the
Privacy Act (5 U.S.C. 552a), the Freedom of Information Act (5 U.S.C.
552), the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et seq.),
and the Health Insurance Portability and Accountability Act (HIPAA),
Public Law 104-191).
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\19\ <a href="https://www.fda.gov/about-fda/domestic-mous/mou-225-10-0010">https://www.fda.gov/about-fda/domestic-mous/mou-225-10-0010</a>.
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e. Benefit Category Review
Following discussions with FDA, CMS may initiate a benefit category
review if all other pathway criteria have been met. Emerging devices
may fit within a Medicare benefit category but that does not mean that
all medical devices will fall within a benefit category. If CMS
believes that the device, prior to a decision on its approval or
clearance by FDA, is likely to be coverable through one or more benefit
categories, the device may be accepted into the TCET pathway. This is
an interim step that is subject to change upon FDA's decision regarding
approval or clearance of the device by FDA. Acceptance into TCET should
not be viewed as a final determination that a device fits within a
benefit category. However, if it appears that a device, prior to a
decision on its approval or clearance by FDA, will not fall under an
existing benefit category, the TCET nomination will be denied and this
rationale will be discussed in the denial letter. CMS will likely not
assess every submitted application for a benefit category review, as
the TCET pathway is limited in its size per the discussion that follows
in section II.G. of this document.
f. Manufacturer Notification
As noted previously, upon completion of CMS' review of the
nomination, including the initial meeting with the manufacturer,
discussions with FDA, and benefit category determination, CMS will
notify the manufacturer by email whether the product is an appropriate
candidate for the TCET pathway at this time. In instances where CMS
does not accept a nomination, CMS will offer a virtual meeting with the
manufacturer to answer any questions and discuss other potential
coverage pathways.
g. Evidence Preview
Following CMS' determination that the product is an appropriate
candidate, CMS will initiate an Evidence Preview, which is a systematic
literature review that would provide early feedback on the strengths
and weaknesses of the publicly available evidence for a specific item
or service. The Evidence Preview will be a focused, but not necessarily
exhaustive, review that will help CMS to identify any material evidence
shortfalls. We believe the review conducted for the Evidence Preview
will offer greater efficiency, predictability and transparency to
manufacturers and CMS on the state of the evidence and any notable
evidence gaps for coverage purposes. It is intended to inform judgments
by CMS and manufacturers about the best available existing coverage
options for an item or service. CMS intends for the Evidence Preview to
be conducted by a contractor using standardized evidence grading, risk
of bias assessment, and applicability assessment according to a
protocol initially developed in collaboration with AHRQ in 2020. In
order to initiate an Evidence, Preview, CMS will request written
permission from the manufacturer to share any confidential commercial
information (CCI) included in the nomination submission with the
contractor. CMS anticipates that the Evidence Preview will take
approximately 12 weeks to complete once the review is initiated,
following acknowledgement of an accepted nomination in the TCET
pathway. More time may be needed to complete the review in the event
the product is novel, has conflicting evidence or other unanticipated
issues arise.
h. Evidence Preview Meeting
CMS will share the Evidence Preview with the manufacturer via email
and will offer a meeting to discuss it. The Evidence Preview will have
been previously shared with AHRQ and may also be shared with FDA to
obtain their feedback, as relevant. Representatives from those Agencies
may participate in the Evidence Preview meeting. Manufacturers will
have an opportunity to propose corrections to any errors and raise any
important concerns with the Evidence Preview.
CMS will review the manufacturer feedback on the Evidence Preview
and work with our contractor to revise the draft, as appropriate, prior
to finalization. Upon finalizing the
[[Page 41641]]
Evidence Preview, manufacturers may request a meeting to discuss the
strengths and weaknesses of the evidence and discuss the available
coverage pathways (examples include an NCD, which could include CED, or
seeking coverage decisions made by a MAC). These meetings to discuss
the Evidence Preview may be conducted virtually or in person and will
be scheduled for 60 minutes.
For those manufacturers who withdraw from the TCET pathway
following the completion of an Evidence Preview, there will be no
publicly posted tracking sheet and no public notification that an
Evidence Preview was completed. However, we believe it is in the best
interests of patients and the Medicare program to share the Evidence
Preview with the MACs to aid them in their decision making since the
development of an Evidence Preview represents a substantial investment
of public resources in a thorough evidence review for pre-market
devices. We solicit public comment on this approach.
i. Manufacturer's Decision to Continue or Discontinue With the TCET
Pathway
Upon finalization of the Evidence Preview, the manufacturer may
decide to pursue national coverage under the TCET pathway or to
discontinue with the pathway. If the manufacturer decides to continue,
the next step would include a manufacturer's submission of a formal NCD
letter expressing the manufacturer's desire for CMS to open a TCET NCD
analysis. Most, if not all, of the information needed to begin the TCET
NCD would be included in the initial TCET pathway nomination, however,
CMS invites the manufacturer to submit any additional materials the
manufacturer believes would support the TCET NCD request.
j. Evidence Development Plan (EDP)
If evidence gaps are identified by CMS and/or AHRQ during the
Evidence Preview, the manufacturer should also submit an evidence
development plan (EDP) to CMS that sufficiently addresses the evidence
gaps identified in the Evidence Preview. The EDP should be submitted to
CMS at the same time as the formal NCD request cover letter. The EDP
may include traditional clinical study designs or fit-for-purpose study
designs or both, including those that rely on secondary use of real-
world data, provided that those study designs follow all applicable CMS
guidance documents. Additional information can be found here: <a href="https://www.cms.gov/Medicare/Coverage/DeterminationProcess/Medicare-Coverage-Guidance-Documents-">https://www.cms.gov/Medicare/Coverage/DeterminationProcess/Medicare-Coverage-Guidance-Documents-</a>.
Over the last several years, and most recently during the two
stakeholder listening sessions we held on February 17, and March 31,
2022, we heard from stakeholders that they would like for CMS to
utilize a more agile, iterative evidence review process that considers
fit-for-purpose (FFP) study designs, including those that make
secondary use of real-world data. An FFP study is one where the study
design, analysis plan, and study data are appropriate for the question
the study aims to answer. FFP study designs scale sample size,
duration, and study type, etc., based off of the utilization and risk
profile of the item or service. We are partnering with AHRQ to consider
how to incorporate greater flexibility into the CED paradigm by
allowing FFP evidence study designs that meet rigorous CMS evidence
requirements. Any updates will be communicated in guidance documents
and potential rulemaking as applicable and will include an opportunity
for public comment. We believe that FFP study designs will be less
burdensome for manufacturers. We also believe that by incorporating FFP
study designs, we will address one of the public's concerns that CED
should be time-limited to facilitate the timely generation of evidence
that can inform patient and clinician decision making and lead to
predictable Medicare coverage.
Postmarket FFP study proposals, particularly those that rely on
real world data, have the potential to generate evidence that
complements tightly controlled premarket traditional clinical trials by
demonstrating external validity. Nonetheless, manufacturers should be
aware that these studies require considerable planning in data
validation, linkage, and transformation; specification of the study
protocol; data analysis; and reporting. The study design, patient
inclusion criteria, primary and secondary endpoints, treatment setting,
analytic approaches, timing of outcome assessment, and data sources
should be fully pre-specified in the submitted protocol. When writing
EDPs, manufacturers should propose clinically meaningful benchmarks for
each study outcome and provide supporting evidence.
Manufacturers should conceive a continued access study that
maintains market access between the period when the primary EDP is
complete, the evidence review is refreshed, and a decision regarding
post-TCET coverage is finalized. The continued access study may rely on
a claims analysis, with a focus on device utilization, geographic
variations in care, and access disparities for traditionally
underserved populations.
k. EDP Submission Timing
Because of the tight timeframes that are needed to effectuate CMS'
goal of finalizing a TCET NCD within 6 months after FDA market
authorization, manufacturers are strongly encouraged to begin
developing a rigorous proposed EDP as soon as possible after receiving
the finalized Evidence Preview. To meet the goal of having a finalized
EDP approximately 90 business days after FDA market authorization, the
manufacturer is encouraged to submit an EDP to CMS as soon as possible
after FDA market authorization.
l. EDP Meeting and Finalization of the EDP
Once CMS receives the EDP from the manufacturer, it will share the
document with AHRQ. CMS will have 30 business days to review the
proposed EDP and provide written feedback to the manufacturer. During
this time, CMS will collaborate with AHRQ to evaluate the EDP to ensure
that it meets established standards of scientific integrity and
relevance to the Medicare population. CMS will incorporate AHRQ's
feedback on the EDP and will share the consolidated feedback with the
manufacturer by email. Soon after providing written feedback, CMS will
schedule a meeting with the manufacturer, which may also include AHRQ,
to discuss any recommended refinements and address any questions.
In the EDP meetings, the manufacturer should be prepared to
demonstrate: (1) a compelling rationale for its evidence development
plan; (2) the study design, analysis plan, and data are all fit for
purpose; and (3) the study sufficiently addresses threats to internal
validity. The EDP should include clear enrollment, follow-up, study
completion dates, and the timing and content of scheduled updates to
CMS on study progress. Manufacturers should present and justify their
study outcomes and performance benchmarks.
Following the EDP meeting, the manufacturer and CMS will have
another 60 business days from the date of the EDP meeting to make any
adjustments to the EDP. We recognize that, in some instances,
manufacturers may require additional time to develop and refine their
EDP. In these instances, CMS may provide additional time to
manufacturers but we note that delays in submitting and revising an EDP
may substantially impact the overall timeline for providing coverage
under the TCET pathway. Elements of the CMS and
[[Page 41642]]
AHRQ approved EDPs, specifically the non-proprietary information, will
be made publicly available on the CMS website upon posting of the
proposed TCET NCD. In instances where the manufacturer's EDP is
insufficient to meet CMS' and AHRQ's established standards and is
therefore not able to be approved, CMS may exercise its option to
withdraw participation from the TCET pathway as noted in II.B. of this
document. We anticipate this will be a rare occurrence as CMS will make
every effort to provide flexibility and information to manufacturers to
facilitate the development of EDPs.
2. Coverage Under the TCET Pathway
CMS follows the statutory requirements, which includes an open and
transparent process, when developing coverage policy at the national
level. Though some elements of coverage review can be accelerated,
gathering and reviewing meaningful public comment takes time. When CMS
undertakes an NCD, we draw upon our analysis of the available evidence
to identify the specific beneficiaries and conditions of coverage that
are appropriate for the item or service. CMS also strongly considers
information from patient advocacy organizations, specialty society
guidance, expert consensus and recommendations for beneficiary
selection, provider training and certification requirements, and
facility requirements.
a. CMS NCD Review and Timing
If a device that is accepted into the TCET pathway receives FDA
marketing authorization, CMS will initiate the NCD process by posting a
tracking sheet following FDA market authorization (that is, the date
the device receives PMA approval; 510(k) clearance; or the granting of
a De Novo request) pending a CMS and AHRQ-approved Evidence Development
Plan (in cases where there are evidence gaps as identified in the
Evidence Preview). The manufacturer may also request that their device
be withdrawn from the TCET pathway at this stage in the process, in
which case CMS would not proceed with the NCD review described in this
section. As previously noted, the goal is to have a finalized EDP no
later than 90 business days after FDA market authorization.
The process for Medicare coverage under the TCET pathway would
follow the NCD statutory timeframes in section 1862(l) of the Act. CMS
would start the process by posting a tracking sheet and elements of the
finalized Evidence Preview, specifically the non-proprietary
information, which would initiate the start of a 30-day public comment
period. Following further CMS review and analysis of public comments,
CMS would issue a proposed TCET NCD and EDP within 6 months of opening
the NCD. There would be a 30-day public comment period on the proposed
TCET NCD and EDP and a final TCET NCD would be due within 90 days of
the release of the proposed TCET NCD. Our goal is to release the
proposed and final NCD in advance of the statutory deadline that
applies to all NCDs. More information on the NCD process is set forth
in the August 7, 2013 Federal Register notice (78 FR 48164).
b. Request for Specific Stakeholder Input on the Evidence Base and
Conditions of Coverage
Since the evidence base for these emerging technologies will likely
be incomplete and practice standards not yet established, we believe
that feedback from the relevant specialty societies and patient
advocacy organizations, in particular their expert input and
recommended conditions of coverage (with special attention to
appropriate beneficiary safeguards), is especially important for
technologies covered through the TCET pathway.
Upon the opening of an NCD analysis, CMS strongly encourages these
organizations to provide specific feedback on the state of the evidence
and their suggested approaches to best practices for the emerging
technologies under review. While CMS prefers to have this information
during the initial public comment period upon opening the NCD, we
realize that in many cases it may take longer for these organizations
to provide their collective perspectives to CMS since these
technologies will have only recently received FDA market authorization.
Since CMS may consider any information provided that is in the public
domain while undertaking an NCD, CMS encourages these organizations to
publicly post on their website any additional feedback, including
relevant practice guidelines, within 90 days of CMS' opening of the
NCD. These organizations are encouraged to notify CMS when
recommendations have been posted. All information considered by CMS to
develop the proposed TCET NCD will become part of the NCD record and
will be reflected in the bibliography as is typical for NCDs.
c. Coverage of Similar Devices
FDA market-authorized Breakthrough Devices are often followed by
similar devices that other manufacturers develop. We believe that it is
important to let physicians and their patients make decisions about the
best available treatment depending upon the patient's individual
situation. Rather than extending privileged coverage status only to the
first device that achieves FDA market authorization, we are seeking
comments on whether coverage of similar devices using CED would
establish a level playing field and avoid delays in access that would
occur if a separate NCD were required to ensure coverage. To be
eligible for coverage under a TCET NCD, similar devices will be subject
to the same coverage conditions, including a requirement to propose an
EDP. Elements of the approved EDPs for similar devices, specifically
the non-proprietary information, will be posted on the CMS website. In
some cases, studies under the EDP may continue beyond the pre-specified
NCD reconsideration date. In this case, CMS strongly encourages
manufacturers to complete these studies even if further evidence
development is voluntary. CMS seeks public comments on its approach for
providing coverage for similar devices under the TCET pathway.
d. Duration of Coverage Under the TCET Pathway
The duration of transitional coverage through the TCET pathway will
be tied to the CMS and AHRQ approved EDP. The review date specified in
the EDP will provide one additional year after study completion to
allow manufacturers to complete their analysis, draft one or more
reports, and submit them for peer-reviewed publication. Given the short
timeframes in the TCET pathway, an unpublished publication draft that a
journal has accepted may also be acceptable. In general, we anticipate
this transitional coverage period would last for a period of 3 to 5
years as evidence is generated to address evidence gaps identified in
the Evidence Preview. However, CMS retains the right to reconsider an
NCD at any point in time.
3. Transition to Post-TCET Coverage
TCET provides time-limited coverage for devices with the potential
to deliver improved outcomes to the Medicare population but do not yet
meet the reasonable and necessary standard for coverage under section
1862(a)(1)(A) of the Act. Consequently, TCET coverage is conditioned on
further evidence development as agreed in a CMS and AHRQ approved EDP.
a. Updated Evidence Review
CMS intends to conduct an updated evidence review within 6 calendar
months of the review date specified in
[[Page 41643]]
the EDP. To conduct the review, CMS intends to engage a third-party
contractor to conduct a systematic literature review using detailed
requirements that CMS developed in collaboration with AHRQ. The
contractor will then perform a qualitative evidence synthesis and
compare those findings against the benchmarks for each outcome
specified in the original NCD. After conducting quality assurance on
the contractor review, CMS will assess whether the evidence is
sufficient to reach the reasonable and necessary standard. CMS will
also review applicable practice guidelines and consensus statements and
consider whether the conditions of coverage remain appropriate. CMS
will collaborate with AHRQ and FDA as appropriate as the updated
Evidence Review is conducted and will share the updated review with
them.
b. NCD Reconsideration
Based upon the updated evidence review and consideration of any
applicable practice guidelines, CMS, when appropriate, will open an NCD
reconsideration by posting a proposed decision which proposes one of
the following outcomes: (1) an NCD without evidence development
requirements; (2) an NCD with continued evidence development
requirements; (3) a non-coverage NCD; or (4) permitting local MAC
discretion to make a decision under section 1862(a)(1)(A) of the Act.
Neither an FDA market authorization nor a CMS approval of an Evidence
Development Plan guarantees a favorable coverage decision. Standard NCD
processes and timelines will continue to apply, and following a 30-day
public comment period, CMS will have 60 days to finalize the NCD
reconsideration.
The steps previously described for the TCET process follows with
the applicable estimated timelines for obtaining a CMS coverage
determination are illustrated in the diagram:
[GRAPHIC] [TIFF OMITTED] TN27JN23.013
E. Roles
CMS has outlined the general roles of each participant in the TCET
pathway.
1. Manufacturer
The manufacturer initiates consideration for TCET by voluntarily
submitting a complete nomination as outlined previously under ``1.
Nomination,'' of section II.D of this document entitled ``Procedures
for the TCET Pathway.'' In the interest of expediting CMS decision
making, the manufacturer should be prepared to quickly and completely
respond to all issues and requests for information raised by the CMS
reviewers. If CMS does not receive information from manufacturers in a
timely fashion, CMS review timelines will be lengthened, potentially
significantly. Manufacturers are encouraged to submit any materials
they plan to present during meetings with CMS at least 7 days in
advance of the scheduled meeting. Manufacturers should be prepared with
the resources and skills to successfully develop, conduct, and complete
the studies included in the EDP.
2. CMS
CMS will provide a secure and confidential nomination and review
process as outlined previously in section II.C. of this document. CMS
will initiate review of nominations for the TCET pathway by retrieving
applications from the secure mailbox, and communicating with FDA
regarding Breakthrough Devices seeking coverage under the TCET pathway.
Throughout all stages of the TCET pathway, CMS intends to maintain open
communication channels with FDA, AHRQ and the relevant manufacturer and
fulfill its statutory obligations concerning the NCD process.
3. FDA
FDA will keep open lines of communication with CMS on Breakthrough
Devices seeking coverage under the TCET pathway as resources permit.
Participation in the TCET pathway does not change the review standards
for FDA market authorization of a device, which are separate and
distinct from the standards governing a CMS NCD.
4. AHRQ
Currently, AHRQ reviews all CED NCDs established under section
[[Page 41644]]
1862(a)(1)(E) of the Act. Consistent with section 1142 of the Act, AHRQ
collaborates with CMS to define standards for clinical research studies
to address the CED questions and meet the general standards for CED
studies (<a href="https://www.cms.gov/Medicare/Coverage/Coverage-with-Evidence-Development">https://www.cms.gov/Medicare/Coverage/Coverage-with-Evidence-Development</a>). Since we anticipate that many of the NCDs conducted under
the TCET pathway will result in CED decisions, AHRQ will continue to
review all CED NCDs consistent with current practice. Additionally,
AHRQ will collaborate with CMS as resources allow to evaluate the
Evidence Preview and EDP and will have opportunities to offer feedback
throughout the process that will be shared with manufacturers. AHRQ
will be a partner with CMS as the Evidence Preview and EDP are being
developed and approvals for these documents will be a joint CMS-AHRQ
decision.
F. TCET and Parallel Review
While the TCET pathway will be limited to Breakthrough Devices,
other potential expedited coverage mechanisms, such as Parallel Review,
remain available. Eligibility for the Parallel Review program is
broader than for the TCET pathway and could facilitate expedited CMS
review of non-Breakthrough Devices. To achieve greater efficiency and
to simplify the coverage process generally, CMS intends to work with
FDA to consider updates to the Parallel Review program and other
initiatives to align procedures, as appropriate.
G. Prioritizing Requests
CMS intends to review TCET pathway nominations and respond within
30 days after receipt of the email. At present, CMS anticipates
accepting up to five TCET candidates annually due to CMS resource
constraints. CMS intends to prioritize innovative medical devices that,
as determined by CMS, have the potential to benefit the greatest number
of individuals with Medicare.
III. Collection of Information Requirements
Based on our initial assessment of Breakthrough Devices applying
the characteristics we list in II.C. of this notice with comment period
regarding appropriate candidates for the TCET pathway, we anticipate
that we will receive approximately eight nominations for the TCET
pathway per year. Due to current CMS resource constraints, we do not
anticipate the TCET pathway will accept more than five candidates per
year. Since we estimate fewer than 10 respondents, the information
collection requirements are exempt in accordance with the implementing
regulations of the Paperwork Reduction Act (PRA) at 5 CFR 1320.3(c). As
we gain experience with the TCET pathway, if we receive a higher number
of respondents than anticipated, we will provide an updated analysis.
IV. Response to Comments
Because of the large number of public comments, we normally receive
on Federal Register documents, we are not able to acknowledge or
respond to them individually. We will consider all comments we receive
by the date and time specified in the DATES section of this notice,
and, when we proceed with a subsequent document, we will respond to the
comments in that document.
Chiquita Brooks-LaSure, Administrator of the Centers for Medicare &
Medicaid Services, approved this document on June 20, 2023.
Dated: June 21, 2023.
Xavier Becerra,
Secretary, Department of Health and Human Services.
[FR Doc. 2023-13544 Filed 6-22-23; 4:15 pm]
BILLING CODE 4120-01-P
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