I appreciate the opportunity to comment on the
proposed changes to Medicare’s guidance document
and National Coverage Decision which provide
coverage and payment for certain costs for
Medicare beneficiaries in routine clinical
trials. CMS has listed three overarching goals
and in addressing these goals CMS should consider
the following points.
By providing payment for services as Medicare
benefits for patients in clinical trials, access
to such services may improve because many
clinical trials rely on third party reimbursement
for clinical care. Coverage of clinical costs is
also a way to encourage the ongoing development
of evidence to improve clinical decision making
and health policy formulation. However, CMS
needs to be mindful that by defining clinical
trials as Medicare covered services (even in
part) imposes a 20% copayment on the
beneficiaries. While the clinical trial may have
important goals in terms of advancing scientific
knowledge, the trial and its results also
benefits future Medicare beneficiaries, future
policymakers, and the sponsoring industry. The
current Medicare beneficiary may support these
same goals, but being an experimental subject
should be free of any additional financial
burden. The only inherent limitations to
Medicare beneficiary participation in clinical
studies are study design attributes that CMS
urges investigators to adopt: 1) randomization,
2) placebo control, 3) exclusion of patients with
factors which introduce bias such as co-
morbidities that generally accompany advancing
age.
Rather than insist on clinical trial validation
for every treatment with every possible
combination of factors and co-morbidities, it
might be equally useful for CMS to investigate
how well physicians use basic controlled clinical
trial results in caring for patients with
complicating factors. The Medicare claims
database coupled with the rational use of a “Pay
for Performance” strategy is more likely to
produce information about how new technologies
effect health outcomes in real clinical
settings. This approach would require CMS to use
coding and payment policies that provide
differentiation of treatment and results while
imposing minimal burden on the beneficiary.
There are also serious questions of burdens
placed on beneficiaries and physicians by these
covered clinical trials. CMS has declared that
manufactures and institutions should bear the
cost of administrative overhead and database
maintenance. The ultimate source of the clinical
trial data is the physician. Even if
manufacturers or institutions are willing to
compensate physicians for this activity, such a
practice has serious ethical and legal (anti-
kickback) concerns. Because CMS has stated that
data collection and reporting in the context of
clinical trials by physicians would not be
considered “reasonable and necessary,” CMS will
not pay for the additional time and effort by
physicians or the additional cost imposed on
beneficiaries if the physician bills the
beneficiary for these services as a non-covered
services. CMS needs to recognize that physicians
are providing a valuable and medically necessary
service and that physicians should not be forced
to decide if they will perform it for free or
pass on the cost to the beneficiary. A change in
coverage, coding and payment policy should
recognize the burden and responsibility of the
treating physician. Without recognition of this
important role by treating physicians,
investigation of the quality and completion of
data will be comprised.
In the event that CMS determines that the
evidence of effectiveness for a particular
procedure is insufficient but that further
research should be encouraged, it is worthwhile
to facilitate that further research. CMS in
collaboration with NIH launched a scientific and
well conceived examination of lung volume
reduction surgery and obtained results that might
never have been obtained otherwise. The
regulation developed by CMS and FDA to allow
payment for category B IDE devices is precisely
the situation enumerated in overarching goal 3,
where FDA has been satisfied about relative
safety but has not yet drawn final conclusions
about the effectiveness. This regulation has led
to sound policy and access without increased cost
for beneficiaries and no uncompensated burden for
investigators or institutions. What does remain
as a problem is the antiquated HCPCS coding
system maintained and used by CMS that makes it
impossible to extract useful outcomes information
that could be used by CMS and FDA.
As CMS reconsiders its coverage policy for the
routine costs of clinical trials, it should
consider these particular points:
1) CMS should insure that study
participation does not result in additional
financial burdens on beneficiaries. If CMS’s
legal authority does not allow payment under §
1862(a)(1)(A), then CMS should use demonstration
authority or further explore whether § 1862(a)(1)
(E) might apply. Ultimately, Medicare
beneficiaries in clinical trials should not be
burdened with added costs, providers should not
be required to perform uncompensated works and
sponsors should not be given the “Hobson’s
choice” of either accepting compromised data or
making potentially unethical and illegal payments
to providers.
2) Coordination with NIH should go beyond
inclusion in the clinical trials database. The
successful lung volume reduction (NETT) trial
demonstrates the value of inter-agency
collaboration. CMS should develop this policy
with an MOU between NIH, FDA, AHRQ and CMS
remembering the useful and beneficial result of
an MOU between FDA and CMS leading to IDE device
payment.
3) Criteria to encourage broad participation
in clinical trials will recognize the need to
treat physician investigators equitably for their
effort by providing coverage, codes for
reporting, and adequate reimbursement for their
effort.
4) In a trial within a clinical setting, as
opposed to feasibility or early trials, a patient
has the right be benefit from any and all
activities. Even tests which might not
ordinarily be done will provide information
directing care. While test results not available
to the treating physician are not ordinarily of
clinical benefit, CMS seems to be taking the
position that because results and data are
available to formulate future policy they are
within CMS’s authority to require them. Anything
required by CMS for payment should be
considered “reasonable and necessary” and
therefore covered and reimbursed.
5) Any attempt to make policy regarding IND
and IND exempt trials should be done at the HHS
level or in joint decision-making with FDA
through notice and comment rulemaking.
6) It is unclear how an item or service that
is of potential benefit to a patient would not
meet the requirements of § 1862(a)(1)
(A). “Reasonable and necessary” or as stated in
the statute “not reasonable and necessary” does
not describe something that is of potential
benefit. Few treatments in medicine are
delivered with absolute certainty that they will
benefit a particular patient but are delivered
supported by a rationale that they are likely to
be of benefit in a particular situation. Because
CMS has abandoned any development of regulations,
further defining the meaning of § 1862(a)(1)(A)
is left to the guidance issued by CMS. It does
not appear that certainty of outcome is currently
required, therefore a potential benefit from a
treatment would be reasonable under the
reasonable and necessary standard.
7) Because some clinical trials are
described and covered by an NCD, it is a simple
matter for that NCD to categorically provide
coverage for item or services otherwise not
covered by an existing NCD.
8) Humanitarian devices are provided in the
belief that they present a potential benefit in a
circumstance where no other option is
reasonable. It is hard to imagine a policy that
would deny potential benefits in this situation
as “not reasonable and necessary”.
CMS has taken important steps in the integration
of evidence-based medicine into health policy.
The success of prior efforts should be recognized
and built upon, such as the NETT trial, FDA-CMS
collaboration in promulgating a regulation for
IDE device payment, and creation of the standards
to be met by self-certified clinical trials. In
expanding coverage and promotion of trials, CMS
must at the same time recognize the roles and
responsibilities of investigators, providers. and
Medicare beneficiaries rather than focus only on
manufacturers and industry.