The Biotechnology Industry Organization
(BIO) appreciates this opportunity to comment on
the Centers for Medicare and Medicaid Services’
(CMS) Tracking Sheet regarding the development of
a Clinical Research Policy (CRP) as a
reconsideration of its national coverage decision
(NCD) on Medicare coverage of clinical trials.
BIO is the largest trade organization to serve
and represent the biotechnology industry in the
United States and around the globe. BIO
represents more than 1,100 biotechnology
companies, academic institutions, state
biotechnology centers, and related organizations
in the United States. BIO members are involved
in the research and development of healthcare,
agricultural, industrial and environmental
biotechnology products. Our research initiatives
advance the understanding of disease pathology
and therapeutic mechanisms of action, clinical
effectiveness, health-related quality of life,
and health economic impacts of therapies in
addition to clinical safety and efficacy.
CMS is developing the CRP in conjunction
with the agency’s recent “Guidance for the
Public, Industry, and CMS Staff on NCDs with Data
Collection as a Condition of Coverage: Coverage
with Evidence Development (CED)” that was issued
on July 12, 2006. As BIO has stated in our past
comments regarding CED, we are committed to
increasing the body of evidence available
regarding diseases and their treatments. Our
members spend millions of dollars each year on
clinical studies, both before and after Food and
Drug Administration (FDA) approval of their
therapies, to produce high-quality clinical
evidence to support medical decision-making. We
also support the dissemination of this evidence
to further clinical knowledge and enhance and
improve clinical decision-making.
BIO also is committed to ensuring
beneficiary access to innovative biological
therapies. To that end, we support CMS’ efforts
to clarify its NCD on Medicare coverage of
clinical trials. We believe that a clarification
of Medicare’s coverage policy for clinical trials
has the potential to strengthen the ability of
biotechnology companies to develop and evaluate
innovative therapies that will specifically
benefit Medicare patients. BIO urges CMS to
consider its proposed CRP in light of our
continued concerns regarding the CED concept.
Additionally, we ask that CMS look at the broader
effect that these policies have on Medicare
coverage of clinical trials and beneficiary
access to them. We plan to comment separately on
the July 12, 2006 CED guidance and ask that those
comments be taken into account for purposes of
the CRP’s development too.
We urge CMS to clarify the NCD in a
manner that promotes Medicare beneficiary
enrollment into clinical trials and assures them
of coverage for their routine medical costs while
enrolled in these clinical trials. Specifically,
BIO requests that CMS expressly include clinical
trials exempt from the investigational new drug
application (IND) process, as well as establish a
mechanism for other research studies to qualify
for Medicare coverage. In addition, although BIO
strongly supports CMS’ goal of encouraging
greater participation of Medicare beneficiaries
in clinical trials, we urge CMS to carefully
develop any guidelines regarding the
representation of Medicare beneficiaries in
Medicare-covered trials in a manner that reflects
the challenges of enrolling Medicare
beneficiaries and fosters the goal of increasing
the participation of such patients rather than
limiting the number of trials available to this
population. Third, we continue to urge CMS to
set any data collection standards in a manner
that achieves CMS’ specific goals without
imposing undue burdens on patients, providers,
and clinical trial sponsors. Finally, we ask
that CMS clarify that a sponsor’s agreement to
make payment for uncovered expenses relating to
illness or injury resulting from the trial does
not make the sponsor a primary payer under the
Medicare Secondary Payer rules.
We have set forth these preliminary
comments more fully below, and we look forward to
the opportunity to comment on the proposed CRP.
I. Coverage of IND-Exempt Trials and Other
Research Studies
When CMS issued the NCD for clinical
trials in 2000, the agency included a list of
trials automatically “deemed” to be qualified as
covered by Medicare. This list included trials
conducted under an IND reviewed by FDA. CMS also
included as “deemed,” drug trials that are IND-
exempt only until other qualifying criteria were
developed. Criteria were not subsequently
published for the IND-exempt trials, and thus IND-
exempt trials have continued to operate
as “deemed” under a temporary status.
BIO urges CMS to permanently extend
deemed status to IND-exempt trials in the CRP.
This category of clinical trials is carefully
regulated. FDA permits a clinical investigation
of a drug product lawfully marketed in the United
States to be exempt from the IND process only if
certain requirements are met. This exemption is
intended to apply primarily to researchers “who
are beginning to explore new uses for marketed
drugs (i.e. not pivotal studies) or who are using
the drug as a research tool.” An IND-exempt
investigation also is permitted only where safety
is not an issue and the investigation is not
being conducted to support a labeling change such
as a new indication or a comparative safety
claim.
FDA expressly has encouraged use of this
IND-exempt process for qualifying trials. For
example, in 2004, FDA urged the oncology industry
not to submit INDs for all clinical research for
oncology products but instead to use the IND-
exempt process where possible. Clinical trials
operating under the IND-exempt process have been
influential in the post-approval development of
many important therapies, and this is
increasingly true as more companies seek to use
the IND-exempt process, at FDA’s urging.
Permanently adding IND-exempt trials to the list
of clinical trials “deemed” qualified for
Medicare coverage will reduce uncertainty among
patients and providers regarding Medicare
coverage for routine medical costs.
BIO also is concerned about CMS’
intention to remove the self-certification
process that never was implemented as part of the
2000 clinical trial NCD. We understand that an
inter-agency panel met and developed criteria for
the types of trials that should be covered under
this process. We encourage the agency to release
the panel’s findings to the public and to propose
an alternative qualifying process for those
research studies that are not deemed to be
qualifying clinical trials. This is necessary to
ensure that Medicare beneficiaries have access to
the full range of research studies being
conducted and are able to participate in the
studies that are most appropriate for their
conditions.
II. Regulation of IND-exempt Trials
BIO is concerned about CMS’ suggestion in
the Tracking Sheet that the CRP will attempt
to “[c]larify the scientific and technical roles
of Federal agencies in overseeing IND Exempt
trials.” As discussed above, IND-exempt trials
are regulated by FDA. FDA has established a
clear set of criteria for such trials as well as
issued guidances for industry to use in
determining which trials are appropriate for the
IND-exempt process. We believe that the
jurisdiction to regulate such trials clearly lies
with FDA, and we do not believe that CMS’
involvement in clarifying the scientific and
technical roles of Federal agencies in overseeing
these trials is a proper exercise of CMS’
authority. We note that CMS is a payer for
health services. Using this authority, CMS may
examine whether an item or service meets criteria
for coverage, and CMS may establish a list of
clinical trials and other research studies that
qualify for Medicare coverage of routine costs.
CMS is not tasked with regulating or overseeing
clinical trials, and we urge CMS not to assume
the responsibilities of other agencies by seeking
to participate in the oversight of clinical
trials. BIO urges CMS to work with FDA and the
criteria established by FDA for IND-exempt trials.
III. Increased Clinical Trial Participation by
Medicare Beneficiaries
BIO supports CMS’ goal of encouraging
more Medicare beneficiaries to participate in
research studies. We believe that CMS’ efforts
to clarify Medicare coverage of clinical trials
by developing a new CRP could have the effect of
making clinical trials more available to Medicare
beneficiaries. In the Tracking Sheet, CMS
proposes developing criteria to assure that any
Medicare covered clinical research study includes
a representative sample of Medicare beneficiaries
by demographic and clinical characteristics.
Although BIO supports the goal of increasing
Medicare beneficiary access to clinical trials,
BIO is concerned that setting specific criteria
requiring certain levels of Medicare enrollees in
a clinical trial could have the effect of
limiting beneficiary access to clinical trials.
As CMS no doubt is aware, many Medicare
beneficiaries are ineligible for clinical trials
due to age, comorbidities, or complications.
Others may choose not to participate if the trial
would require them to travel, change physicians,
or experience other substantial inconvenience.
This may be particularly true for patients in
rural areas, minorities, and women, who
traditionally have been under-represented in
clinical trials. BIO urges CMS to develop a
policy that recognizes the many impediments to
enrolling Medicare beneficiaries in clinical
trials. In order to ensure that Medicare
coverage is available to those beneficiaries who
do qualify for and choose to enroll in clinical
trials, it is critical that CMS not impose
stringent criteria that in fact hinder
beneficiary participation in clinical trials.
BIO also encourages CMS to take
additional steps to make Medicare beneficiaries
more aware of ongoing clinical trials for which
they may be eligible. BIO supports increased use
of the NIH clinical trials registry as one means
of educating Medicare beneficiaries about
available clinical trials, and BIO urges CMS to
consider other methods of more broadly
disseminating this information as well.
IV. CED and New Data Collection Requirements
As mentioned briefly above, BIO currently
is drafting comments to the July 12, 2006 CED
guidance, and these comments also will be
relevant to the CRP. We ask that CMS take our
CED comments into account when developing the CRP
proposed decision memorandum. Overall, BIO
supports a rigorous evidence development process
that encompasses all aspects of a disease. We
greatly appreciate the agency’s recent
clarifications regarding the potential
application of CED, particularly which it will be
used infrequently and generally will be used to
expand access to technologies and treatments for
Medicare beneficiaries. Nonetheless, we continue
to have some concerns regarding the potential
application of CED to drugs and biological
products. Most relevant to the CRP, we are
concerned about the imposition of data collection
requirements in addition to those required by
FDA. To the extent that CMS sets forth any data
collection requirements in the CRP, we urge CMS
to set data collection standards that can achieve
its specific goals while imposing minimal burdens
for patients, providers, and clinical trial
sponsors.
The data collection required by CMS, when
in addition to any FDA-required data, adds to the
costs of a clinical trial. We urge CMS to take
every effort to minimize these costs and to pay
particular attention to the costs imposed on
beneficiaries and providers. Beneficiaries’ cost
of care should not increase as the result of
increased data collection requirements. If
beneficiaries are forced to incur greater costs
for receiving care in Medicare-covered clinical
trials they will choose other, potentially less
appropriate, care options. CMS also must
minimize physicians’ costs in operating clinical
trials. Physicians who participate in clinical
trials often donate considerable amounts of time
and resources to evaluating patients’ eligibility
for trials, data collection, and drug
administration services that frequently are not
reimbursed by trial sponsors.
In determining whether additional data
collection is necessary for Medicare covered
trials, we urge CMS to carefully balance the
value of the information gathered against the
burden of collecting it, align any data
collection requirements with FDA’s clinical study
requirements and with other research priorities
to ensure that our research resources are used
efficiently, and require that data collection
continue only as long as important questions
remain and the effort and resources required to
collect this data are justified by the potential
value of the information to be collected. We
believe it is critical that data collection needs
be determined at the outset so that the study
will produce the data needed to satisfy CMS’
needs and to ensure that any coverage decisions
relying in part on such data will be made in an
efficient and timely manner. We also urge CMS to
consider ways to compensate physicians more
appropriately for the data collection activities
they undertake, as well as services they provide
relating to evaluating patient eligibility and
drug administration.
In the Tracking Sheet, CMS notes that the
CRP will attempt to “[c]larify how
items /services that do not meet the requirements
of 1862(a)(1)(A) but are of potential benefit can
be covered in clinical research studies as an
outcome of the National Coverage Determination
process.” We believe this inquiry more
appropriately belongs in the CED policy, and we
look forward to commenting on this issue as part
of our comments on the CED guidance.
V. Medicare Secondary Payer (MSP) Issues
In developing the CRP, BIO urges CMS to
clarify that when a clinical trial sponsor, study
site, or investigator assures a study subject
that he or she will not be responsible for out-of-
pocket payments for medical services resulting
from a trial-related illness or injury, that
assurance will not turn the sponsor, site, or
investigator into a primary payer, and render
Medicare a secondary payer.
The Medicare statute requires payment for
items and services that are reasonable and
necessary for the treatment of illness or
injury. It is clear that medically necessary
services provided to treat complications arising
in the course of a clinical trial are intended to
be covered by Medicare. Indeed, CMS regulations
specifically authorize Medicare payment for
complications arising from clinical trials
involving the use of medical devices. In
addition, the current NCD itself calls for
coverage by defining routine costs in qualifying
clinical trials to include items and services for
the treatment of complications.
The Medicare Secondary Payer (MSP) statute
provides that Medicare payment “may not be made…
with respect to any item or service to the extent
that payment has been made or can reasonably be
expected to be made” under a “primary plan.”
The statute defines “primary plan” to include (1)
a group health plan or large group health plan
and (2) a worker’s compensation law or plan or
automobile or liability insurance policy or plan
(including a self-insured plan) or no fault
insurance. Nothing in the MSP statute or its
legislative history suggests that Congress
intended to expand the reach of the MSP
provisions to preclude Medicare payment for
covered items and services when the sponsor of a
clinical trial offers in an informed consent
document and related clinical trial agreement to
make payment for uncovered expenses relating to
illness or injury resulting from the trial. In
effect, such an interpretation of the MSP statute
would turn clinical trial sponsors into primary
health care insurers -- a result surely not
intended by Congress, and one that runs contrary
to the policy of encouraging the participation of
Medicare beneficiaries in clinical trials.
Accordingly, BIO urges that the CRP explicitly
clarify that a promise by a clinical trial
sponsor or study site to pay for uncovered trial-
related illness or injury will not result in the
sponsor being viewed as a “primary plan,” or
render the sponsor, site, or investigator
a “primary payer,” under the MSP provisions. CMS
should assure beneficiaries that they will not be
denied coverage merely because they have
volunteered to participate in a clinical trial.
In addition, we ask that CMS clarify that neither
the MSP statute, nor the exclusion from Medicare
coverage for items or services for which a person
has no legal obligation to pay operate to
eliminate Medicare coverage for otherwise covered
items where the sponsor has agreed to cover those
clinical care costs that would not, in any event,
have been recognized as an expense covered by
insurance (e.g., the costs of care for uninsured
trial participants). CMS should make clear that
beneficiaries may not be denied coverage for
otherwise covered items or services as a result
of having volunteered to participate in a
clinical trial whose sponsor has agreed to cover
those clinical care costs that are not, for any
particular patient, normally (i.e., absent the
trial) covered by insurance.
VI. Conclusion
BIO appreciates this opportunity to
comment on CMS’ efforts to developed a proposed
CRP, and we look forward to commenting on the
proposed CRP once it is issued. We hope that our
recommendations are useful to CMS in developing a
proposed CRP that establishes Medicare coverage
of clinical trials in a predictable manner that
ensures beneficiary access to innovative drugs
and biologicals. Specifically, we urge CMS to:
• Expressly designate clinical trials
exempt from the IND process as “deemed” as well
as establish a mechanism for other research
studies to qualify for Medicare coverage;
• Carefully develop any guidelines
regarding the representation of Medicare
beneficiaries in Medicare-covered trials in a
manner that reflects the challenges of enrolling
Medicare beneficiaries and fosters the goal of
increasing the participation of such patients
rather than limiting the number of trials
available to this population;
• Set any data collection standards in a
manner that achieve CMS’ specific goals without
imposing undue burdens on patients, providers,
and clinical trial sponsors; and
• Clarify that Medicare coverage of a
clinical trial is not conditioned on the clinical
trial sponsor serving as a primary payer for
certain medical costs that may be associated with
the trial.
We look forward to working with CMS to
encourage increased Medicare beneficiary access
to and participation in clinical trials. As this
is an important policy for BIO and its members,
we would be pleased to have the opportunity to
discuss our comments with you in greater detail.
If you have any questions regarding our comments,
please contact me at 202-312-9273. Thank you for
your attention to this very important matter.
Sincerely,
/s/
> Jayson Slotnik
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