1.) Health Reform Targets Cost and Coverage
2.) Transparency Shapes Pharmaceutical R&D
3.) Designing Clinical Research for Better Patient Care
In 1989 the Health Care Financing Administration, now the Centers for Medicare and Medicaid Services, published proposed coverage
criteria. This was the first time the governing body of Medicare had attempted to make its decision-making process explicit,
and included within these proposed criteria was a stipulation of cost-effectiveness.2The proposed cost effectiveness criteria created controversy among medical device manufacturers, professional societies, providers,
and insurers, but remained in circulation until quietly withdrawn 10 years later.3 Since then, cost-effectiveness methodologies have continued to develop and cost-effectiveness data remain in demand, though
their use in coverage decisions is not explicit.
Although the proposed Medicare cost-effectiveness criteria were withdrawn, changing U.S. demographics are making economic
analyses more relevant. In '07 the first baby boomer reached age 65, and Medicare is bracing for an onslaught of services
to be rendered in the coming decades.
The RAND elderly model4 prompted one reviewer to state that "cost-effectiveness has no necessary relation to affordability at a societal level."5 Indeed, each individual insurer may have their own defined threshold by which a technology would be accepted, based on the
size and characteristics of a particular patient population. What might be deemed affordable in 2008 may not be in 2018, and
Medicare's increased financial burden will most likely make economic analyses a more prominent component within the decision-making
process.
Some background
Payer groups, academic institutions, and technology manufacturers use economic analyses to make informed decisions about which
treatments generate cost-effective outcomes for individual patient types. While such arguments are not necessary to receive
product approval, they must be considered in the development of reimbursement and marketing strategies.
Incorporating costs and outcome considerations into a treatment decision for a patient seems intuitive. Economics provide
a substantial toolbox for developing these arguments. Garber6 states that "it is possible to define a 'cut off' cost-effectiveness ratio, such that interventions whose cost-effectiveness
ratios exceed the cut-off are not acceptable, while those with lower cost-effectiveness ratios are."
While such a decision threshold is intuitively appealing, it is difficult to implement. Risk profiles differ and are often
not comparable among patients. New therapies have different effects on different patient subgroups, and the majority of clinical
trials are not appropriately sized to estimate treatment effects in all subgroups of interest.
Furthermore, the health care environment is not static. Multiple new therapies and diagnostics enter the market constantly
and change patient care pathways. Gelijns and Rosenberg7 have observed (and cited examples) that incremental improvements in medical technology tend to expand the patient population
eligible for a particular therapy.
Laparoscopic procedures allow surgery for patients who may have not been candidates for—or who may have declined—more invasive
procedures. Improved diagnostic imaging such as computed tomography (CT) or magnetic resonance imaging (MRI) allows diagnostic
services to take place on patients who may have required surgery to make an accurate diagnosis. Because of its noninvasive
nature, the use of such technology has "dramatically broadened the technology's indications of use, thereby probably offsetting
any savings resulting from the reduction in exploratory surgery or other interventions."7
Incremental increases in the number of procedures have significant implications for intervention comparisons. The rational
and straightforward decision-making described earlier by Garber becomes more complicated because changes in practice patterns
are dynamic and cost-effectiveness ratios could vary among new subsets of patient populations eligible for such procedures.
The art of clinical trial design is already complex given that multiple audiences (FDA, CMS, commercial insurers, medical
community) must be convinced of a given therapy's benefit, and the respective criteria for each audience are rarely the same.
