LETTER TO JOHN EISENBERG
REGARDING CRITERIA FOR COVERAGE OF CLINICAL TRIALS
(November 17, 2000)
November 17, 2000
John M. Eisenberg, M.D.
Director, Agency for Healthcare Research and Quality
2101 East Jefferson Street
Executive Office Center, Suite 600
Rockville, Maryland 20852
Dear Dr. Eisenberg:
The undersigned organizations advocate for responsible public policy positions concerning cancer treatment and research. Individually and collectively, we have long urged third-party payers, including Medicare, to agree that routine patient care costs should
be covered, regardless of whether such care is provided in the context
of standard therapy or a clinical trial. Our position was bolstered by
the fact that, at least for cancer patients, treatment in a clinical trial
is considered to be consistent with best available care. Thus, when the
President announced on June 7 a policy of Medicare reimbursement for routine
patient care costs associated with clinical trials, we believed we were
on the road to an enlightened model coverage policy.
Revisions of the coverage
proposal and decisions related to the implementation of the final coverage
decision have raised serious concerns in the cancer community. We strongly
recommend that: 1) the clinical trials self-certification process be simplified;
and 2) automatic Medicare coverage be extended to those trials that are
exempt from investigational new drug application (IND) requirements under
21 C.F.R. § 312.2(b)(1), or so-called IND-exempt trials.
The President's decision
to extend Medicare coverage to routine patient care costs in clinical
trials was explicitly based on a study and formal recommendations by a
prestigious expert panel of the Institute of Medicine (IOM), convened
pursuant to an Act of Congress. After reviewing all the facts and law
and hearing the views of numerous experts in clinical research and medicine,
the IOM issued recommendations, the first of which was that "Medicare
should reimburse routine care for patients in clinical trials in the same
way it reimburses routine care for patients not in clinical trials."
The IOM panel made
two recommendations which are relevant to the issues currently under consideration
by the Agency for Healthcare Research and Quality (AHRQ). IOM recommended:
- A broad definition
of clinical trials which should be eligible for reimbursement. Trials
of all phases and legitimate designs and sponsorship (government, industry,
or other) were included in the IOM recommendation. The basic standard
set by IOM required a trial to: (1) have a written protocol that describes
and scientifically sound study; and (2) be approved by all relevant
Institutional Review Boards (IRBs) before participants enroll.
- No special precertification
by HCFA, or any other administrative process, for researchers or physicians
participating in trials. IOM proposed that claims for care in a trial
should be submitted in the same manner as claims for care outside a
trial. According to the IOM panel, if there is an explicit reimbursement
policy for care in a clinical trial, there should be no requirement
that researchers adhere to a certification process.
We endorse the broad
coverage standard articulated by the IOM panel, which captures our longstanding
position that routine patient care costs for those participating in a
trial should be reimbursed in the same manner as costs incurred outside
trials. Imposing qualifying criteria on trial sponsors and requiring them
to certify their compliance may discourage sponsor, physician, and patient
participation in trials. A primary objective of the President's clinical
trials policy was to increase senior citizen participation in trials,
but critical implementation decisions may undermine the policy.
If the Medicare program
is intent on subjecting IND-exempt trials to a self-certification procedure,
then it is imperative that the process be simple, straightforward and
objective. There should be no opportunity for subjective second-guessing
by federal officials if we are to avoid a damaging chilling effect on
clinical cancer research. Thus, we strongly support the basic criteria
outlined in the IOM report. According to the IOM report, the criteria
for self-certification should be limited to (1) approval by an IRB and
full compliance with other relevant federal requirements, and (2) use
of a written protocol with defined end points. There should not be any
requirement for these trials to be conducted by investigators or institutions
with federal sponsorship or funding.
Coverage for IND-Exempt Trials
At the same time we
recommend that the criteria for coverage of IND-exempt trials be simple
and clear, we strongly urge the Health Care Financing Administration to
reconsider the requirement that IND-exempt trials be certified for reimbursement.
In contrast to the proposed coverage decision, the final version did not
extend "deemed," or automatic, coverage to clinical trials exempt
from the otherwise applicable requirements of "investigational new
drug" review by the Food and Drug Administration (FDA). Under 21
C.F.R. § 312.2(b)(1), an IND is not required for investigations involving
already approved drugs if certain criteria are met, notably if the investigation
complies with IRB standards and if the investigated usage does not significantly
increase risk to the patient. Essentially, these IND-exempt trials are
viewed, as a class, by FDA as not requiring hands-on review, largely because
they involve relatively minor deviations from the approved usage. Failing
to include these trials in the category of "deemed" coverage
will discourage investigator-initiated trials and undermine the original
intent of the President's June 7 executive memorandum. For the following
reasons, we request that the coverage decision be revised to extend "deemed" coverage to IND-exempt trials, at least those involved in cancer clinical research.
trials are an integral part of cancer care in the United States.
Thousands of cancer
patients are enrolled in investigator-initiated clinical trials that
are under the funding sponsorship of pharmaceutical companies or other
private entities. If, as we believe, the self-certification process
envisioned by Medicare for these trials acts to discourage them, then
patients will be deprived of investigational treatment options otherwise
available to them. While the differences between the approved usage
and the investigational approaches pursued in these trials are relatively
minor, such small differences can be significant in cancer therapy.
It is, after all, through these incremental steps that measurable progress
against cancer has been achieved.
In general, access
to clinical trials is considered integral to quality cancer care. Clinical
trials are so much a part of standard cancer care that 80% of cancer
physicians have participated in a clinical trial in recent months, according
to a survey of its members conducted by the American Society of Clinical
Oncology (ASCO), the leading medical society for physicians treating
cancer. One important category of these cancer clinical trials is investigator-initiated, IND-exempt trials that are disadvantaged under the final coverage decision.
trials are an important component of the overall clinical cancer research
effort and should not be deterred by Medicare coverage policy.
While, by definition,
IND-exempt trials are generally not intended to result in FDA-approved
labeling changes for the study drugs, company and other sponsors require
rigor in trial design, data collection and reporting of results. It
is a fact that some significant advances in treatmente.g., weekly
administration of taxolhave emerged through investigator-initiated
trials of the sort that are at issue here. Moreover, the results of
these trials have been published in distinguished peer-reviewed medical
journals such as the Journal of Clinical Oncology. It would be a loss
to clinical cancer research and would restrict patient treatment options
if the number and scope of these trials were reduced by virtue of Medicare
policy, particularly in a context where the President clearly intended
to support the research enterprise.
The evident intent
of both the President and the IOM was to ensure that Medicare beneficiaries
received all the care to which their premiums entitle them, including
access to clinical trials. It would be wrong for the Medicare program
to seek to use reimbursement policyultimately affecting quality
of cancer care for beneficiariesto exert leverage or control over
appropriate private sector research activities, especially when such
authority has specifically been given by Congress to the FDA.
3. The prospect
of retrospective audits and reimbursement recoupment will effectively
deter participation in clinical trials by physician investigators.
to the Health Care Financing Administration (HCFA) from various medical
associations made clear that the self-certification process will deter
physician participation in clinical trials. Oncologists are not exempt
from the same fears and concerns that motivate other specialties. Based
on the history of recent aggressive auditing and recoupment activity
by HCFA and the Inspector General, physicians will no doubt think twice
before making a specific, sworn representation that their activities
comport with criteria that may be vague, subjective and subject to revision
by federal officials.
4. Medicare coverage
of routine patient care costs is, and should be, presumed, and there
is no basis for overturning that presumption with respect to IND-exempt
It is important
to remember that the issue here revolves around "routine"
patient care costsitems and services like physician charges, hospital
stays and standard diagnostic procedures. These costs are ones to which
Medicare beneficiaries are undoubtedly entitled, and, if such costs
are not reimbursed, beneficiary premiums will have been paid in vain.
Thus, there is an effective presumption that such costs will be covered, and it
is only when the Medicare program takes affirmative action in the form
of a non-coverage decision that these costs are usually at risk of not
Here, the Medicare
program has made the decision that routine patient care costs should
be paid when incurred in connection with government-sponsored clinical
trials or clinical trials conducted under an IND. The program has thus
judged that these costs should be covered even when the context of treatment
is purely investigational, even involving largely unproved therapy such
as drugs or biologicals that have never been approved by FDA or any
other regulatory authority. It is therefore most anomalous that the
program would not extend the same degree of coverage to IND-exempt trials,
which involve already approved drugs being investigated in often slight
variations from the FDA approval. Indeed, it is difficult to articulate
a policy that would justify failing to extend coverage to IND-exempt
cancer trials when such coverage is available in trials conducted under
This approach can
only be viewed as a means of asserting Medicare control over purely
privately funded research, despite the fact that FDA, as the principal
federal regulatory authority, has made a considered policy judgment
that these trials do not require that sort of oversight. The rights
of Medicare beneficiaries to have their routine patient care costs covered
in this setting should not be placed at risk when a clinical trial is
otherwise in compliance with federal requirements.
HCFA should reissue
the national coverage decision, reverting to the original proposal, which
included IND-exempt trials in the "deemed" covered category.
If HCFA chooses not to do so across the board, it should at least do so
with respect to cancer clinical trials. If IND-exempt trials are not restored
to "deemed" status, the criteria for self-certification should
be simple, objective, and not subject to subjective second-guessing by
We appreciate the
opportunity to comment on implementation of this important policy.
Society of Clinical Oncology
Cancer Care, Inc.
Colorectal Cancer Network
Cure For Lymphoma Foundation
International Myeloma Foundation
Multiple Myeloma Research Foundation
National Coalition for Cancer Survivorship
National Patient Advocate Foundation
National Prostate Cancer Coalition
North American Brain Tumor Coalition
Oncology Nursing Society
Ovarian Cancer National Alliance
Pancreatic Cancer Action Network
The Susan G. Komen Breast Cancer Foundation
The Wellness Community
US-TOO International, Inc.
Y-ME National Breast Cancer Organization
The Honorable Donna Shalala
Secretary, Dept. of Health & Human Services
Michael Hash, Acting
Health Care Financing Administration
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