INTRODUCTION
Thank you for the opportunity to come here today and talk with you about how we make
Medicare coverage policy. Over the past 30 years, we have developed a coverage process that
assures access to medical advances for Medicare beneficiaries, while protecting them from
services whose effectiveness is unproven. This is one of HCFA's greatest challenges in
administering the Medicare program -- to maintain a dynamic decision-making process that
produces consistent coverage guidance in the face of rapid changes in medical technology and
health care delivery.
I am proud of the ways in which our coverage policy-making system is meeting that challenge.
For example, in recent years, we have approved ventricular assist devices to provide a bridge to a
transplant for our beneficiaries in Medicare certified heart transplant centers. More recently,
under our new program covering devices that are subject to an investigational device exemption
(IDE), we have begun to pay for a portable version of this device that will allow patients to leave
the hospital while they await their transplant.
In today's health care market, every insurer and health care purchaser must deal with coverage
policy. Private as well as public insurers, like Medicare, want to purchase high quality health
care for the best price. Health plans, whether public or private, managed care or traditional
indemnity sure the highest quality of care for the plans must control costs while still continuing
to assure the highest quality of care for their subscribers. This cannot be done without
authoritative evidence of the value of each individual service.
Medicare has emerged as a leader in the move toward such evidence-based decision making for
coverage policy. We rely on state-of-the-art technology assessment and on agencies such as: The
Agency for Health Care Policy and Research (AHCPR), The Food and Drug Administration
(FDA), the National Institutes of Health (NIH), the Department of Veterans Affairs (DVA), the
Department of Defense (DOD) as well as the advice of the medical community and private sector
studies. Our own extensive reimbursement data contain additional useful information for
assessing the effectiveness of all varieties of medical care. The experience from our program can
benefit the entire health care marketplace.
Furthermore, the sheer numbers of beneficiaries that we serve and the wealth of information that
we possess about them makes Medicare an important force in the market. We recognize that our
coverage process also controls services provided by managed care plans that serve Medicare
beneficiaries. Not only is the share of Medicare patients in managed care plans growing rapidly,
but plans must provide these enrollees with the same Part A and Part B benefits that Medicare
fee-for-service beneficiaries enjoy. As a result, managed care plans have an increasing interest in
our process being as scientific and thorough as possible.
Medicare coverage decisions probably also influence private plans' coverage decisions for their
non-Medicare patients, However, many private plans now have sophisticated technology
assessment and coverage processes of their own.
Many Medicare coverage decisions are made by our local carriers, based on the statutory
requirement to cover only services that are reasonable and necessary for the diagnosis and
treatment of illness or injury. In making their decisions, carriers depend upon the knowledge and
advice of local physicians and other local specialists and on local medical review policies that are
shared among carriers. HCFA has an overall interest in increasing the consistency of coverage
policy among carriers and making national policy for coverage issues that are significant.
THE COVERAGE POLICY PROCESS
Over the past 30 years, Medicare's coverage process has evolved from a relatively informal set
of procedures for deciding on payment of particular claims, to a sophisticated process that
includes extensive medical advice and state-of-the-art technology assessments.
The key to understanding Medicare coverage policy is its statutory foundation. The Social
Security Act lists 55 categories of benefits that are covered under the program. They include
physician services, inpatient hospital services, services in ambulatory surgical centers, and many
more. The Act also gives the Secretary of the Department of Health and Human Services broad
discretionary authority to go beyond these defined benefits to make coverage decisions on
individual items and services. The law says:
"Notwithstanding any other provisions of this title, no payment may be made... for items
or services which... are not reasonable and necessary for the diagnosis and treatment of
illness or injury or to improve the functioning of a malformed body member."
The interpretation of this provision, using authoritative evidence of whether a service is
Treasonable and necessary," gives us the ability to keep Medicare benefits current in the face of
changing medical practice.
The criteria used to make these decisions have evolved over time in response to our experience
and to public comments and suggestions. The criteria that now guide the coverage process are:
demonstrated effectiveness, appropriateness, and comparability to similar services. I will discuss
each of these in turn.
Demonstrated Effectiveness
Demonstrated effectiveness is the first criterion for determining that a service or health care
technology meets the Medicare coverage requirements. There must be authoritative evidence to
establish that the benefits of a service or technology to the patient outweigh its reasonably
anticipated risks. There must also be authoritative evidence to convince HCFA or its contractors
that use of the service or technology will result in improved health outcomes (such as decreased
morbidity or mortality or significantly increased quality of life, for beneficiaries or some groups
of beneficiaries). In addition, items or services that are regulated by FDA must have received
approval for marketing.
To find evidence that we consider authoritative in demonstrating the effectiveness of a service
on patient outcomes, we examine:
- published articles based on controlled clinical trials, other controlled studies, and case
series- 5
- formal technology assessments from recognized government and private entities, which
examine both published and unpublished data;
- evaluations or studies initiated by Medicare contractors; and
- authoritative approvals of other agencies, such as FDA, that a service has therapeutic or
diagnostic benefit for patients.
Sometimes these assessments are made by HCFA staff and our advisors. But with increasing
frequency we are obtaining formal technology assessments from the AHCPR and other
government and private organizations.
Assessments of health care technology have at least two features that are important to HCFA.
First, rather than evaluating services in an investigational setting, they are focused on the
effectiveness of technologies in general medical practice and on patient outcomes including
morbidity, mortality, health status and health-related quality of life. Second, they use systematic
methods for evaluating the quality of the scientific evidence available rather than relying on the
less formal examination of medical opinion or consensus. They also grade the quality of the
evidence based on the study methods. We have adopted these evidence-based methods as our
primary means for making Medicare coverage decisions.
The evidence-based environment increases the importance of giving the public access to HCFA
while we are in the process of making coverage decisions. We have expanded the opportunities
for interested parties to provide information and expert advice both before and after decisions are
made. To ensure that our process for determining coverage is evidence-based, we consult
extensively with interest groups and receive expert medical advice from both government and
private clinicians. Our Internet Home Page is another means by which we hope to increase
participation by interested parties. In addition, we are continually improving our communication
methods with our contractors, who are often the first to become aware of the use of new health
care technologies.
Appropriateness
Appropriateness means, to HCFA, that a service is suitable for, but not in excess of, the
beneficiary's medical needs and condition. The qualifications of those providing the service and
the setting where the service is provided may be a part of this criterion. The service must be
furnished by personnel who are qualified by training, experience and licensure to provide that
service and, in some instances, who meet HCFA-established training levels. The service must be
furnished in a setting that is suitable for, but not in excess of, the beneficiary's medical needs and
condition and must meet any facility requirements established by HCFA. For example, cardiac
catheterization may be covered for some patients in a freestanding facility, but for patients with a
high risk of complications, a hospital may be the only appropriate setting for receiving this
service.
Comparison with Similar Technologies
It is apparent that cost-effectiveness analysis is extremely controversial for beneficiaries,
providers and suppliers. It raises fears of rationing based on cost. HCFA has not and does not
intend to make coverage decisions based solely on cost-effectiveness, and we will not refuse to
cover services merely because they are costly. Moreover, manufacturers or providers do not
need to submit formal cost-effectiveness analyses to HCFA in order to have a service considered
for coverage.
Nevertheless, in order to become prudent purchasers of health care, third party payers, including
HCFA, must consider the full value of any medical service they are considering for coverage.
Cost-effectiveness analyses, when available, can be used in this regard. These analyses allow us
to consider the full range of present and future costs and benefits of a service. Without it, payers
and providers often focus on the present costs of expensive technologies rather than on the full
value of the service.
Under specific and limited conditions, relative costs and effectiveness will be factors considered
in determining Medicare payment policy. That is, if a service is more expensive but equally
effective, it would still be covered and paid at the rate of the lower cost alternative. If, however,
the service is found to be more effective for a specific group of patients, it could be paid at the
higher rate. This criterion assures value for the Medicare program and its beneficiaries.
LOCAL AND NATIONAL COVERAGE DECISIONS
The Medicare coverage process is sometimes confusing to the public because of the coexistence
of national coverage decisions with those made locally by Medicare contractors. National
coverage decisions are issued by HCFA after a thorough assessment of the statutory and clinical
issues and analysis of the data. These decisions are binding on Medicare contractors. In the
absence of national decisions for particular services, contractors have the discretion to issue local
medical review policies.
Most Medicare coverage decisions are made locally, by HCFA contractors, according to the
criteria that I just described and any applicable national coverage policy. Medicare contractors
can issue local medical review policies to describe the circumstances under which an item or
service will be covered. In developing such policies, our contractors are required to seek formal
comment from the medical community who provide the item or service. Following the comment
process, our contractors must publish final policies 30 days prior to implementation.
In addition, there are Carrier Medical Director (CMD) workgroups, national and regional
meetings between the CMDs and HCFA, collaboration between carriers and the Peer Review
Organization (PRO), and Carrier Advisory Committees (CACs), which represent medical
physician specialties, beneficiaries and HMOs. All of these activities aid in the process of
developing, revising, and gauging the impacts of the development of local policy. All local
medical review policies generated by a contractor are published; frequently the contractors
conduct seminars, generate special bulletins, or use other methods to ensure that the medical
community is aware of new policies. Though well planned and highly examined, such local
policies have at times led to geographic variation in Medicare coverage that concerns our
beneficiaries and providers.
HCFA cannot guarantee the consistency of Medicare coverage by directing local policy.
However, local policies are shared among carriers and guide other carriers' local medical review
policies. To increase the consistency of coverage, HCFA began a system of Carrier Medical
Director workgroups to examine coverage issues. These groups pool resources and develop
model policies, which operate to guide local policies. The model policy process is one way to
increase consistency in coverage across the country for services that are not the subject of
national policies. HCFA has also begun to issue a greater number of national coverage decisions
to increase uniformity and to make our coverage policy more timely and flexible in response to
available medical evidence. Issues are reviewed for national coverage for a number of reasons
including high potential for the service to diffuse rapidly on a national basis; considerable local
variation in coverage; or the availability of substantial amounts of medical evidence.
When we, at HCFA, decide to review a service for national coverage, we may perform an
internal review of policy or consult with our technology advisory committee (TAC) consisting of
ten carrier medical directors, including a managed care medical director, and representatives of
NIH, AHCPR, VA, FDA and CHAMPUS. The TAC provides interchange between local and
national policy and considers when an issue becomes of such prominence that it warrants a
national policy. HCFA develops the agenda that the TAC will follow to evaluate and make its
recommendations.
The TAC could recommend that HCFA: issue a national coverage policy, refer the issue for
assessment by the Public Health Service or other qualified assessment organization, postpone the
decision until there is more information, or decline to establish a new policy, HCFA can then
accept or reject the TAC's recommendation.
Although TAC proceedings are closed to the public, thficantly reducing our ability to design the type of controlled
study needed to provide good medical evidence.
To address these critical issues in coverage policy-making, Medicare has undertaken one of its
most important efforts to date - "coverage with conditions." I will describe some of the recent
steps we have taken.
Coverage with Conditions
Our recent decision to pay for certain lung volume reduction surgery procedures in conjunction
with a clinical trial is an example of "coverage with conditions." This type of coverage may be
used when existing data suggest a potential benefit to some patients, but there is not sufficient
information to predict the effect of generalized use of the technology or its long term effects or
appropriate patient selection criteria.
Coverage with conditions may include limitations of use, data collection, or the provision of the
service under protocol conditions. These efforts may be implemented by making payments only
to providers involved in a study sponsored by the National Institutes of Health or other research
organizations. The objective of coverage with conditions is to advance medical knowledge
about the effectiveness of the services before they are diffused to general practice and to assure
that Medicare beneficiaries are not put at undue risk from their use.
Only services that meet all of the following criteria will be considered for this coverage:
- the service is of substantial interest for Medicare coverage because it would potficantly reducing our ability to design the type of controlled
study needed to provide good medical evidence.
To address these critical issues in coverage policy-making, Medicare has undertaken one of its
most important efforts to date - "coverage with conditions." I will describe some of the recent
steps we have taken.
Coverage with Conditions
Our recent decision to pay for certain lung volume reduction surgery procedures in conjunction
with a clinical trial is an example of "coverage with conditions." This type of coverage may be
used when existing data suggest a potential benefit to some patients, but there is not sufficient
information to predict the effect of generalized use of the technology or its long term effects or
appropriate patient selection criteria.
Coverage with conditions may include limitations of use, data collection, or the provision of the
service under protocol conditions. These efforts may be implemented by making payments only
to providers involved in a study sponsored by the National Institutes of Health or other research
organizations. The objective of coverage with conditions is to advance medical knowledge
about the effectiveness of the services before they are diffused to general practice and to assure
that Medicare beneficiaries are not put at undue risk from their use.
Only services that meet all of the following criteria will be considered for this coverage:
- the service is of substantial interest for Medicare coverage because it would potentially
affect a large number of beneficiaries or provide an important new benefit to some;
- enough information exists about the service for HCFA to judge the service reasonable and
necessary under particular conditions;
- the service is unproven with regard to questions remaining about risks and benefits that must
be answered for a final coverage decision;
- the information necessary for HCFA to make a national coverage decision is unlikely to be
developed by researchers unless coverage with conditions is offered; and
- any regulated services used with the service in question are approved for marketing by the
Food and Drug Administration (FDA).
It is Medicare's long standing policy that, in general, services covered in clinical trials cannot be
reimbursed because there is insufficient evidence of their benefits and risks. The above criteria
differentiate coverage with conditions from clinical trials.
Lung Volume Reduction Surgery
An example of coverage with conditions is Medicare's recent agreement with the National Heart,
Lung, and Blood Institute (NHLBI) regarding Lung Volume Reduction Surgery (LVRS). LVRS
encompasses procedures intended to improve lung function and relieve the debilitating
symptoms for emphysema patients. Despite the very limited evidence of its efficacy, this surgery
diffused rapidly from 1993-1996.
In September 1995, a workshop on LVRS, sponsored by the National Heart, Lung, and Blood
Institute (NHLBI) called for controlled studies of this surgery. A few months later, HCFA issued
a policy of noncoverage for all LVRS based on the lack of medical evidence and the potential for
morbidity and mortality among Medicare beneficiaries from LVRS. At the same time, we
requested that the Agency for Health Care Policy and Research (AHCPR) perform an
assessment of LVRS procedures.
AHCPR's assessment report in April 1996, said that the objective data did not permit a scientific
conclusion about the risks and benefits of LVRS. AHCPR further recommended that the
procedure only be covered within the context of a controlled clinical study. Later that month,
HCFA and NHLBI agreed that NHLBI would design and fund such a study, now known as the
National Emphysema Treatment Trial (NETT), and HCFA would pay for the medical care
provided to Medicare beneficiaries participating in the study.
In its FYI 997 Appropriations Conference Report, Congress required HCFA, with the advice and
technical assistance of AHCPR, to report to Congress on the treatment of end-stage emphysema
and chronic obstructive pulmonary disease and on unilateral and bilateral lung volume reduction
surgery (LVRS). HCFA was asked to review all available studies and, based on this review,
make a recommendation as to the appropriateness and conditions of Medicare coverage of such a
procedures.
In the preliminary summary that accompanies this testimony, we present the results of our review
of 22 recently published articles on LVRS and our findings from the Medicare databases. Based
on this review, we believe that the conclusions of the AHCPR assessment are still valid, and their
implications for Medicare policy the same. That is, LVRS should be covered only in the context
of a controlled study. As described in the attached summary of our forthcoming report to
Congress regarding LVRS, many of the weaknesses found in the data by AHCPR still exist.
These weaknesses include study design problems, very brief follow-up periods and inconsistent
measurement of important patient outcomes. Moreover, results may be biased as a result of
many patients that were not followed after surgery.
Our analysis of Medicare claims data raised further concerns. We examined 711 Medicare
claims for LVRS between October 1995 and January 1996. Of these patients, 26 percent had
died by January 1997. Forty percent of the patients were hospitalized during the approximately
12 months following surgery. These patients averaged 2.1 acute care hospitalizations for a total
of 20 hospital days. Sixteen percent of the patients had either a long term care hospital
admission (averaging 30 days) or a rehabilitation hospital admission (averaging 24 days) The
controlled study will allow us to identify and distinguish between LVRS' impact on these
patients' mortality and morbidities requiring hospital stays and the impact of the disease itself.
This year, 18 clinical centers including 21 hospitals will begin to provide this service to
Medicare patients and receive Medicare reimbursement. The study will include patients
undergoing LVRS via median sternotomy or via video assisted thoracoscopy along with maximal
medical therapy and patients receiving only maximal medical therapy in the non-surgical arm of
the study. The study is scheduled to be completed in five years. The final study protocol is now
being determined by the Executive Steering Committee for the study and will be completed by
the end of May 1997.
Medicare payment for LVRS will be made only for patients who are participating in NHLBI's
controlled clinical study of this new procedure. However, we are committed to modifying this
policy quickly whenever new and scientifically conclusive data regarding the surgery confirm
that it meets our criteria for coverage. For example, if the study's Data Safety Monitoring Board
were to recommend and NHLBI to agree that some group of patients were achieving a clear
benefit from LVRS, we would extend coverage to them at once. Likewise, a finding of clear
ineffectiveness or harm for some patients would result in a noncoverage policy for that group.
Coverage of Category B Investigational Devices
Another type of coverage with conditions involves certain devices and drugs subject to FDA
approval. It has long been our policy that items cannot be covered under Medicare until they
receive approval for marketing from FDA. In September 1995, an agreement between Medicare
and FDA regarding medical devices subject to investigational device exemptions (IDES) led to a
significant coverage policy change.
FDA agreed to classify devices with IDEs into two categories:
- Category A IDEs are experimental\investigational devices, meaning that significant risk
is being investigated.
- Category B IDEs are non-experimental\investigational devices, meaning that incremental
risk is being investigated, and that the fundamental issues of safety and effectiveness have
already been resolved.
Those IDEs that were labeled "Category B," based on their substantial equivalence to existing
devices, were made eligible for Medicare coverage while they are being studied for incremental
risk. A condition of this coverage is that the service is being provided under the FDA protocol.
THE PRESIDENT'S ADVISORY COMMISSION ON CONSUMER PROTECTION AND
QUALITY IN THE HEALTH CARE INDUSTRY
We expect the recently created President's Advisory Commission on Consumer Protection and
Quality in the Health Care Industry to provide a forum for better understanding of the changes in
the health care financing and delivery system and to provide valuable information to further
inform our evidence-based coverage decision making. This high level commission, chaired by
the Secretaries of Health and Human Services and Labor, includes representatives from the
health care professions, other health care workers, institutional providers, insurers, purchasers,
state government, consumers and experts in health care quality, financing and administration.
Their recommendations for addressing the effects of the changes in health care financing and
delivery will help public and private policy makers define appropriate consumer protection and
quality standards.
CONCLUSION
The coverage process of the Medicare program has evolved over the years to meet the rapidly
changing demands placed on it by the ever-increasing advances in medical science and
technology. Like all health care insurers, it is our ongoing responsibility to define, clarify,
review, and modernize the benefits under the Medicare program.
HCFA has been a leader in the trend in our health care industry toward evidence-based decisions
and policies that provide needed scientific data to support medical effectiveness and outcomes.
In today's environment, Medicare will continue to make coverage decisions and modify them
whenever necessary to reflect new findings and data. It is vital that providers, suppliers,
manufacturers and beneficiaries understand and participate in this process.
For more than 30 years, the Medicare coverage process has assured access to medical advances
for beneficiaries while protecting them from unproven services, an accomplishment that
continues to benefit the entire health care system. We plan to build on this success and to ensure
that beneficiaries have access to those state-of-the-art technologies that preserve and support
maximum well-being and quality of life.
MEDICARE COVERAGE OF LUNG VOLUME REDUCTION SURGERY (LVRS)
PRELIMINARY REPORT TO CONGRESS
APRIL 17,1997
In its FYI 997 Appropriations Conference Report, Congress required HCFA, with the advice and
technical assistance of AHCPR, to report to Congress on the treatment of end-stage emphysema
and chronic obstructive pulmonary disease and on unilateral and bilateral lung volume reduction
surgery (LVRS) and supplemental surgical methods. HCFA was asked to review all available
studies and, based on this review, make a recommendation as to the appropriateness and.
conditions of Medicare coverage for such procedures. This preliminary report responds to that
requirement. Additional information on the protocol will be forwarded at a later date.
Summary
Lung Volume Reduction Surgery encompasses procedures intended to improve lung function for
emphysema patients and relieve debilitating symptoms of the disease. LVRS diffused rapidly
from 1993-1996, despite a paucity of medical evidence concerning its safety and efficacy.
Moreover, there are considerable debates within the medical community not only about the
appropriateness of surgical intervention, but also concerning the relative merits of a variety of
surgical approaches, appropriate selection of patients, and the contribution of comprehensive
pulmonary rehabilitation..
In December 1995, HCFA issued a noncoverage policy for a LVRS procedures based on the lack
of medical evidence and potential for extensive morbidity and mortality among Medicare
beneficiaries. HCFA also requested that the Center for Health Care Technology (CHCT) of the
Agency for Health Care Policy and Research (AHCPR) undertake a complete review of available
evidence, published or unpublished. Over the same period of time, the National Heart, Lung and
Blood Institute (NHLBI) also became interested in the medical issues concerning LVRS. A
September 1995 workshop sponsored by NHLBI called for controlled studies. In April, 1996,
the CHCT report was released. Based on an analysis of approximately 2800 patients from 27
institutions, the report concluded that the available data did not permit a scientific conclusion
regarding the risks and benefits of LVRS. The report recommended that since some patients
appeared to benefit in the short run, Medicare coverage might be provided within a controlled
clinical study. Later that month, HCFA and NHLBI signed an agreement to participate in such a
study. As part of this agreement, NHLBI would design and fund the study and HCFA would
provide reimbursement for the medical care services provided to Medicare's beneficiaries in the
study.
The clinical study, for which protocol has not been finalized, may include as many as 9000
patients. The study represents a precedent setting cooperative effort between the nation's largest
payer and its premier science agency for research on pulmonary diseases and may serve as a
model for future assessments of important new technologies. Medicare beneficiaries will be
provided controlled access to a promising but in many ways unproven procedure, while
scientifically valid data to guide future use and reimbursement decisions are generated.
As is true for many new technologies in the U.S. health care system, many parties are interested
its rapid diffusion. Emphysema affects millions of individuals with substantial disability,
morbidity and mortality. LVRS offers hope of improved quality of life for some of these
patients. Because of the potentially large number of patients that might be eligible for this
surgery, there is also considerable financial interest in LVRS on the part of providers and
manufacturers. Therefore, it is understandable that there would be questions about the cautious
approach to LVRS represented by the clinical study. This report will provide comprehensive
answers to such questions. It provides detailed explanations of the study, an assessment of recent
data concerning LVRS and details of how modifications to Medicare coverage policy will be
made.
What Do Recent Published Data Suggest About the Appropriate Course of Action for Medicare?
We reviewed 21 recently published articles regarding LVRS for this report. A set of evidence-
based criteria for systematic review was applied, including examination of study design,
outcomes measured and patient follow-up. In addition, we examined Medicare databases in
order to provide supplemental information on patients' morbidity and mortality following
surgery.
The conclusion from this review is essentially the same as that of the AHCPR/CHCT assessment
and an assessment for the American Lung Association. While some patients appear to benefit for
a short period of time in terms of selected measures of lung function and exercise capacity, the
most critical questions concerning the risks and benefits of LVRS for particular patients cannot
be adequately answered by existing data. Indeed, the data review heightened the importance of
producing scientifically valid evidence concerning the durability of any benefits and mortality
during a reasonable post-surgical period. Thus, the most appropriate course for Medicare on
behalf of its beneficiaries remains the cautious and scientific path reflected in the NHLBI/HCFA
clinical study. That is, the data suggest enough potential for benefit that some Medicare
coverage should be provided, but only under the conditions present in a clinical study that will
generate the medical evidence needed to answer critical questions about the value of this surgery.
These conclusions result from several inherent weaknesses in the published data concerning
LVRS. First, the published articles present case series data rather than evidence from clinical
studies. While the authors involved should be commended for reporting outcomes data, case
series are generally considered as a weak and potentially misleading form of evidence concerning
the effect of therapeutic interventions. Moreover, while an important test for the value of LVRS97. Moreover, 40% are hospitalized post-surgery, averaging 2.1 admissions and 30 hospital
days in the 12 months following the surgery. Sixteen percent of the patients had either a long
term care hospital admission (averaging 30 days) or a rehabilitation hospital admission
(averaging 24 days). The fact that LVRS patients are a high risk for both morbidities requiring
hospital stays and mortality during the post-surgical period requires careful examination. In
particular, the controlled study will allow us to distinguish between LVRS's impact on these
measures, and the impact of the disease itself.
The NHLBI/HCFA Clinical Study
The study is a multi-center clinical trial designed to determine the efficacy of two surgical
approaches to bilateral lung volume reduction, median sternotomy and video assisted
thoracoscopy. Efficacy will be determined with regard to a variety of patient outcomes including
lung function measures, exercise tolerance, medical care service use, a variety of quality of life
measures and mortality. Eighteen study centers, including 21 hospitals has been chosen for the
study. The study is scheduled to be completed in 5 years.
Patients will be randomized to either of two surgical groups with maximal medical therapy, or a
group that will receive only maximal medical therapy. Maximal medical therapy includes state
of the art medical care and intensive pulmonary rehabilitation. The randomized design is chosen
for two reasons. First, current data do not allow for 97. Moreover, 40% are hospitalized post-surgery, averaging 2.1 admissions and 30 hospital
days in the 12 months following the surgery. Sixteen percent of the patients had either a long
term care hospital admission (averaging 30 days) or a rehabilitation hospital admission
(averaging 24 days). The fact that LVRS patients are a high risk for both morbidities requiring
hospital stays and mortality during the post-surgical period requires careful examination. In
particular, the controlled study will allow us to distinguish between LVRS's impact on these
measures, and the impact of the disease itself.
The NHLBI/HCFA Clinical Study
The study is a multi-center clinical trial designed to determine the efficacy of two surgical
approaches to bilateral lung volume reduction, median sternotomy and video assisted
thoracoscopy. Efficacy will be determined with regard to a variety of patient outcomes including
lung function measures, exercise tolerance, medical care service use, a variety of quality of life
measures and mortality. Eighteen study centers, including 21 hospitals has been chosen for the
study. The study is scheduled to be completed in 5 years.
Patients will be randomized to either of two surgical groups with maximal medical therapy, or a
group that will receive only maximal medical therapy. Maximal medical therapy includes state
of the art medical care and intensive pulmonary rehabilitation. The randomized design is chosen
for two reasons. First, current data do not allow for a conclusion regarding the relative risks and
benefits of LVRS or the contribution to outcomes of maximal medical therapy. Therefore, there
should be no pre-disposal as to the preferred course of treatment. Second, because there is no
existing data on the natural history for this group of patients, many of the most critical questions
about the risks and benefits of LVRS can only be answered within a randomized design.
The final study protocol is being determined by the Executive Steering Committee comprising
the principal investigators and investigators from the study's coordination center is expected to be
completed by the end of May. Patient enrollment will likely begin this summer. The study also
includes a Data Safety monitoring Board (DSMB) which regularly reviews the study data. If at
any time the DSMB finds that data conclusive -- that is, one of the study treatments is providing
clear benefit or harm -- they will recommend either the study be terminated or the protocol be
modified.
Future Medicare Coverage Decision and the Clinical Study
Soon Medicare reimbursement for LVRS is provided only within the context of the clinical
study. However, HCFA is committed to modifying this policy rapidly in response to new and
scientifically conclusive data regarding LVRS. The explicit contingencies for modification of
Medicare coverage are part of the agreement between HCFA and NHLBI.
First, current coverage is conditioned on the study protocol and changes automatically in
response to study design and analysis of evidence. The initial protocol required approximately
1300 patients each in surgery and maximal medical therapy. These patients are expected to be
enrolled within 18 to 24 months of the implementation of the study. Unless the data demonstrate
harmful effects, Medicare coverage will be continued in a manner consistent with the evidence.
More importantly, Medicare will respond immediately to any finding of conclusive evidence
by the DSMB and NHLBI. For example, if the DSMB were to recommend and NHLBI agree
that some group of patient were achieving a clear benefit from LVRS, coverage would be
expanded. Depending on the evidence, such expansions might take place in the study centers, or
new criteria established for its provision in hospitals outside of the study. Likewise, a finding of
clear ineffectiveness or harm for some patients would trigger a noncoverage policy targeted to
that group.
Conclusion
We believe that the scientific, yet flexible approach to coverage of LVRS outlined in this report
remains the appropriate policy course for Medicare. HCFA strives to assure access to safe and
effective services for Medicare beneficiaries, while not providing reimbursement for ineffective
and harmful services. Despite a number of published articles regarding LVRS, a scientific
conclusion concerning the relative risks and benefits of these procedures cannot be made from
existing data. By providing coverage within the clinical study, Medicare is making a precedent
setting effort to provide controlled assess to a promising, yet unproven procedure while assuring
that the needed medical evidence is created.