As physicians and policymakers gear up for healthcare reform and the elusive goal of improved outcomes at lower costs, providers and payors are increasingly looking to comparative effectiveness research (CER) to guide clinical practice and reimbursements, evidenced by a solid investment on the part of the government, argued the authors of a perspective article in the New England Journal of Medicine.  

The American Recovery and Reinvestment Act of 2009 allocated $1.1 billion to CER, followed by the Patient Protection and Affordable Care Act’s creation of the Patient-Centered Outcomes Research Institute, which has a projected annual budget of $500 million.

“CER treats effectiveness as a balance of benefits and harms; when the risks associated with a procedure outweigh its clinical benefits, it is appropriate and ethical to limit its use. Both the clinical need and the desire to avoid wasteful expenditures were part of the rationale for subjecting these procedures to comparative studies,” said Adam G. Elshaug, MPH, PhD, and Alan M. Garber, MD, PhD, from the International Program in Health Policy and Practice at the Commonwealth Fund in New York City, and the Center for Health Policy and Center for Primary Care and Outcomes Research at Stanford University in Stanford, Calif., respectively.

Elshaug and Garber highlighted two ambiguous osteoporotic vertebral fracture treatments, percutaneous vertebroplasty and kyphoplasty. Poorly designed early studies indicated that the procedures, both of which involve the injection of cement into the vertebral body (kyphoplasty also implants a balloon), yielded superior outcomes to conventional symptomatic treatment.

Subsequent CER has shown, however, that kyphoplasty is largely ineffective compared with placebo, while vertebroplasty research “at best cast[s] doubt on the magnitude of any benefits,” the authors contended. In these cases, CER has led Canadian and American payors to begin to reverse decisions and refuse reimbursement for the procedures.

A 50 percent reduction in both procedures would save Americans $450 million annually, while an 80 percent reduction would cut Medicare costs by $325 million each year, the authors estimated. Actual figures are likely to be substantially higher because, as the authors pointed out, these data are based on costs, not charges and payments.

Comparative effectiveness studies are imperfect. Persistent difficulties that policymakers and physicians need to be aware of include distinguishing the effects of treatment from the natural course of the condition and specific evidence identifying individuals or groups that would benefit from treatment even with blunted aggregate effectiveness.

“If nothing else, well-designed studies demonstrating ineffectiveness can help redirect research toward the development of alternatives,” Elshaug and Garber maintained. Whereas when comparative effectiveness trials hold their ground, research “may also improve the healthcare system by freeing up resources to be used for safer and more effective forms of care.”

Another significant challenge to implementing effective treatment is the status quo. Although treatments are commonly adopted before sufficiently replicated studies confirm their effectiveness, once procedures become the standard, effective or not, a high burden of proof must be met to convince providers to change course and drop treatment.

“Of course, savings will be derived from CER only if practice changes,” the authors noted. “The benefits for patients are large, as are the potential savings. Support for CER, reinforced by appropriate payment changes, is likely to represent a very good investment for the federal government and U.S. taxpayers.”