CLC COMMENTS ON PROPOSED COVERAGE CRITERIA
(July 17, 2000)
July 17, 2000
Health Care Financing
Department of Health & Human Services
200 Independence Avenue, S.W.
Room 443-G - HHH Bldg.
Washington, D.C. 20201
Re: Notice of
Intent to Publish a Proposed Rule, 65 Fed. Reg. 31124 (May 16, 2000)
The undersigned groups
submit these comments on behalf of people with cancer, their caregivers
and cancer research organizations. We appreciate the opportunity to provide
advance comment regarding the agency's plans to institute new coverage
criteria for items and services provided to Medicare beneficiaries. As
our comments below indicate, we have serious reservations about, and objections
to, these plans, but expect to comment more extensively should the agency
proceed with publication of a proposed rule.
Attributes of Modern
Our concerns about
the proposal are based in significant part on our perception that it is
inconsistent with the way quality cancer care is practiced today. Therefore,
we think it appropriate to review briefly some of the characteristics
of modern cancer therapy, which we believe must be taken into account
before establishing new, more restrictive coverage criteria.
of cancer is highly individualized.
Cancer is not one
but more than a hundred different diseases. Even among very specific
cancer types, there may be great heterogeneity in different patients.
Cellular characteristics of each patient's cancer may vary significantly,
and treatment may correspondingly vary. In such circumstances, formulaic
approaches to coverage determinations should be suspect to the extent
that such determinations limit the treatment options available to individual
patients with their potentially very different diseases.
Cancer research is well organized and integrated into standard patient care.
Cancer is unique among diseases in that it alone benefits from an extensive
government-sponsored research network that provides patients, even in
remote locations, access to new therapies through clinical trial participation.
The research structures in cancer, under the auspices of the National
Cancer Institute and otherwise, provide monitoring, evaluation and eventual
publication of clinical trials results, ensuring a degree of oversight
and quality perhaps not so consistently found elsewhere. The research
orientation of cancer care helps to drive cancer therapies toward gradual
improvements in morbidity and mortality rates, as recent national statistics
have shown, and cancer advocates do not favor relinquishing decisionmaking
to entities that are not involved in the cancer research enterprise.
in cancer is incremental but relentless.
One seldom sees
dramatic or immediate improvements in cancer therapy, but progress occurs
nevertheless, as the data show. This progress depends mostly on small
refinements in treatment discovered through the painstaking process
of many clinical trials, testing new hypotheses and eventually establishing
new standards of care, albeit often not markedly different from before.
Cancer is thus a therapeutic area that must value incremental steps
and decline to embrace dramatic developments as the standard for coverage.
cancer therapy can change rapidly on the basis of research reports.
Even though progress
against cancer is usually incremental, small changes in therapy can
be incorporated quite quickly as research results become available to
oncologists. Historically, the pace of change in cancer care has been
such that the review processes of the Food and Drug Administration (FDA)
lag behind, as do specific coverage decisions by third-party payers.
Accordingly, it is well established in cancer that so-called "off-label," or not specifically approved, uses of FDA-approved drugs are appropriate
for cancer therapy and should be eligible for reimbursement. Such special
circumstances in cancer calls for coverage rules tailored to its particular
While any proposed
coverage criteria should consider the special circumstances of cancer
therapy, they must also be guided by any pertinent statutory restrictions.
There are at least two such restrictions in the Medicare authorizing legislation,
one very specific and the other general but nonetheless important.
for anti-cancer chemotherapy are set by statute and may not be altered
During the 1980's
and early 1990's, cancer patients experienced many denials of coverage
for medically appropriate cancer care because their therapy involved
"off-label" uses of approved drugs and such uses simply were
not recognized as valid by carriers and intermediaries. In response
to concerns expressed by cancer patients and their physicians, Congress
mandated the Medicare program to cover all medically appropriate uses
of FDA-approved drugs employed in an anti-cancer chemotherapy regimen,
with medical appropriateness to be determined by reference to independent
expert sources specified in the statute. See 42 U.S.C. 1395x(t)(2),
enacted in the Omnibus Budget Reconciliation Act of 1993. Given this
explicit statutory directive, the agency has no latitude to apply different
or more restrictive coverage criteria with respect to cancer therapy
involving drugs or biologicals.
statute does not authorize "cost-effectiveness" as a basis
for coverage decisions.
The Notice assumes,
without discussion or justification, that the Medicare program is authorized
to make coverage determinations on the basis of cost-effectiveness or
cost comparisons among different technologies. It is by no means clear
that this approach was intended by Congress. Certainly, if the views
of those charged with implementing the statute at the outset were taken
into account, there would be no reason to believe that cost should be
a consideration in coverage decisions, and in fact for most of the life
of the Medicare program cost has not been a factor. Only in the failed
Notice of Proposed Rulemaking in January 198925 years after the
initiation of the programdid agency officials suggest that cost-effectiveness might be considered, and of course that proposal sat unfinalized for
more than a decade and was ultimately withdrawn in April 1999. The assumption
that cost-effectiveness is inherent in the statutory standard requiring
coverage of items and services "reasonable and necessary"
for medical care will not go unchallenged. Many would argueand
the agency's own history of interpreting the statute would supportthat,
so long as financial resources are available (as they clearly are in
an entitlement program), the reasonableness of treatment for a life-threatening
disease like cancer should not depend on its cost. If the agency insists
on retaining cost-effectiveness as a coverage criterion for life-threatening
diseases when and if it publishes a proposed rule, we will have much
more to say then with respect to the appropriateness of this approach.
Concerns of the
In light of the above
background information on cancer care and the above-discussed statutory
limitations, we urge the agency to consider the following before publishing
a proposed rule implementing a new coverage process.
Abandon cost-effectiveness as a coverage criterion, at least for life-threatening diseases like cancer.
Given the lack
of apparent statutory authority for application of this proposed coverage
criterion and the impact it could have on development of, and access
to, potentially life-extending new therapies, we urge the agency not
to proceed until such time as the Congress has specified the role, if
any, that cost-effectiveness should have in making coverage decisions
for the Medicare program.
the proposed coverage criteria will not apply to anti-cancer chemotherapy.
In light of specific statutory guidelines on Medicare coverage of drugs and biologicals used in an anti-cancer chemotherapy regimen, the proposed coverage criteria should explicitly exempt items and services associated with this statutorily mandated coverage
the role of informed physician decision-making in highly individualized
diseases like cancer.
In diseases with
great individual variability and thus some degree of uncertainty in
diagnosis and treatment, rigid coverage formulas may inhibit best care.
In the treatment of cancer, the individual oncologist must retain flexibility
to pursue the therapy that, in his or her well-informed judgment, is
necessary for the particular patient.
Define "breakthrough" technology to include incremental advances in life-threatening diseases like cancer.
As noted above,
cancer treatment is characterized by small advances in outcome through
often rather minor revisions to standard care determined through many
clinical trials. For people with life-threatening cancer, no advance
is too small to be considered a "breakthrough" as described
in the agency's proposal. In this and other aspects of its adoption
of new coverage criteria, the agency should remain mindful of the special
circumstances that may apply in the case of potentially life-threatening
diseases like cancer.
reviews to correspond to the needs of the individual disease and other
particular circumstances, including the need for rapid and efficient
review of new therapies.
The cancer community
is strongly supportive of reliance on sound clinical evidence in the
determination of medical practice. However, third-party payers like
Medicare must resist the temptation to use evidence review as a means
of inhibiting dissemination of new technologies. Moreover, the process
for determining coverage must be efficient enough to avoid delays in
access to potentially beneficial therapy for people with life-threatening
diseases like cancer. In general, the agency should seek to rely on
existing independent resources rather than creating its own new bureaucracy
for the purpose of reviewing clinical data. At least in cancer, the
leadership of the National Cancer Institute and various other evidence-evaluating
and guideline-setting organizations provides ample opportunity for the
Medicare program to seek outside guidance regarding the safety, efficacy
and medical appropriateness of new therapies. By using these resources,
the program can make decisions that are informed by the best medical
judgment in each particular medical specialty in the most direct and
efficient manner and without the delay and expense of establishing an
elaborate new review process within the government.
Do not penalize
the Medicare population for lack of age-specific data.
The Notice suggests
that the agency might require clinical trial evidence specific to the
Medicare population in making coverage determinations. We believe this
would be an inappropriate burden on beneficiaries' access to new technologies.
It is well known, certainly in the cancer community, and even supported
by a study of the Southwest Oncology Group, that the Medicare population
is seriously under-represented in cancer clinical trials. One reason
for this situation, however, is almost certainly the position of the
agency with respect to coverage of routine patient care costs in clinical
trials. If the agency adopts the position articulated by the President
in his June 7 Executive Memorandum with respect to coverage of patient
care costs in clinical trials, this particular impediment will be removed.
But there will still be many reasons why Medicare beneficiaries are
not adequately represented in clinical trials, including restrictive
eligibility criteria. Beneficiaries, however, should not be deprived
of access to new therapiesparticularly in the case of life-threatening
diseases like cancersolely because of lack of age-specific data
and in the absence of any reason grounded in safety or efficacy to doubt
that such therapies would be appropriate in elderly patients.
The cancer community
strongly supports the practice of evidence-based medicine, in oncology
and elsewhere. We also recognize, however, that a single formula for determining
access to new technologies may not be appropriate, especially for disease
like cancer that have many unique attributes, as discussed above. Therefore,
we urge that any new coverage process be sufficiently flexible to account
for the particular circumstances of different diseases and different patient
populations, as Congress has already indicated with regard to anti-cancer
chemotherapy coverage. In addition, any new coverage process must be efficient
and timely, especially where new technologies under review are for the
treatment of life-threatening diseases.
Finally, any new process
should proceed with caution in employing cost-effectiveness as a means
of denying coverage. The "science" of cost-effectiveness analysis
is not well-defined, and the Medicare program has no meaningful experience
in applying such criteria to coverage decisions. Use of cost-effectiveness
as a coverage criterion should await specific authorization and guidance
for Lung Cancer Advocacy, Support, and Education
American Society of Clinical Oncology
American Society for Therapeutic Radiology & Oncology, Inc.
Association of American Cancer Institutes
The Children's Cause, Inc.
Coalition of National Cancer Cooperative Groups
Colorectal Cancer Network
Cure For Lymphoma Foundation
International Myeloma Foundation
Kidney Cancer Association
Multiple Myeloma Research Foundation
National Coalition for Cancer Survivorship
National Patient Advocate Foundation
North American Brain Tumor Coalition
Oncology Nursing Society
Ovarian Cancer National Alliance
Pancreatic Cancer Action Network
The Susan G. Komen Breast Cancer Foundation
US-TOO International, Inc.
Y-ME National Breast Cancer Organization
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