Considering costs

The big question is whether investment in CER will steer providers and patients to more effective care and reduce inappropriate expenditures. Instead of drug-to-drug cost assessments, pharma companies want CER to compare medicines to surgery or other treatments, and assess the value of disease management, care coordination, and benefit design. Focusing on drugs and devices "misses the point," according to Gail Wilensky of Project HOPE. "The real explosion in costs is in medical procedures."

However, pharmaceuticals are an easy CER target because so much drug data is available from clinical trials, outcomes studies, and adverse-event reports. The clamor for information on pharmaceutical value and quality ensures that more studies will be conducted in this area. AHRQ's research program began in 2005 with an emphasis on pharmaceuticals to support the then-new Medicare drug benefit, along with instructions from Congress not to use CE information in making coverage decisions. But at an AHRQ advisory meeting, Anthony Wisniewski, head of health policy for the US Chamber of Commerce, said that it is unrealistic to ignore cost-effectiveness issues in CER. Analysis of costs should not be the first priority, he noted, but "it must remain a critical component" of CER activity.

The Future of CER

Analysis of drug effectiveness and cost-effectiveness is nothing new for insurers and payers. Blue Cross and Blue Shield Association's Technology Evaluation Center has reviewed clinical evidence since 1985 to determine the effectiveness of drugs and medical technologies. The Drug Effectiveness Review Project (DERP) at the Oregon Health and Science University provides comparative efficacy and safety information on high-cost, heavily used medicines, as well as drugs frequently used off-label. Consumer Reports' Best Buy Drugs program uses the DERP analyses to provide advice on medical appropriateness and affordability of widely used medicines.

Foreign CER programs have spurred interest in US initiatives. Most prominent is the National Institute for Health and Clinical Excellence (NICE) in the United Kingdom, which provides Britain's National Health Service with recommendations on the coverage of new drugs and diagnostics. NICE sets a clear cost-effectiveness threshold that has led to controversial no-coverage decisions on a number of new, but costly, therapies.

Pharma companies have also been sponsoring more of their own comparative studies to meet regulatory and reimbursement requirements. Payers and formulary committees increasingly want to see data indicating superior efficacy or safety of new drugs compared to available treatments. There are more postapproval comparator studies to meet FDA requirements, and comparative clinical information is increasingly important for gaining market access, particularly in crowded therapeutic classes or for new products that raise serious safety issues. There, of course, sponsors write the protocols, select the comparators, and control the results.

The future promises more oversight of industry studies, along with more CER. Pharma is lobbying hard for such research to focus on clinical value and outcomes, as opposed to cost-effectiveness. Drug and device makers have enlisted support from patient and medical groups to form the Partnership to Improve Patient Care, which has signed up former Congressman Tony Coelho to carry the banner for ensuring that CE studies are well-designed, promote continued medical innovation, and protect patient access to "advanced treatment options." The National Pharmaceutical Council has narrowed its agenda to focus on CER issues and plans a mid-June symposium on the CER landscape in the US and abroad.

Policymakers insist that CER will not lead to coverage denials, but will steer doctors and providers to treatments offering greater benefits for patients and will support flexibility to address special needs. According to Wilensky, the important questions are whether CE data has credibility, whether research practices are open and transparent, and whether studies are objective and not politically motivated.

Better Methods

One positive result of a broader CER program is to develop standards and improve trial designs for real-world assessment of medical treatments. AHRQ plans more investment in methods for designing CER studies, particularly those involving under-represented populations, and is holding a symposium June 1-2 on the subject. NIH is offering grants to improve CER research practices, assess quality of life measures and establish patient registries. At the IOM public meeting, Bryan Luce, senior vice president of UBC, urged "true transformational thinking" in designing CE research studies; otherwise, he said, we will "waste vast amounts of money answering the wrong questions, or the right questions too late."

Now NIH is proposing a number of studies addressing pharmaceutical efficacy and costs for ARRA-funded challenge grants. Research officials would like to assess costs along with risks and benefits of commonly used cancer treatments, and compare those costs and other factors for treating autoimmune rheumatic and skin diseases. The NIH "wish list" includes comparisons of efficacy, outcomes and costs of treatments for kidney stones, for fibromyalgia and for patients newly diagnosed with type 2 diabetes.

Interest is high in the IOM committee's deliberations, as dozens lined up at a March public meeting to propose CER study methods and topics to explore. Similarly, there was standing-room-only at a meeting of AHRQ's National Advisory Council, which heard presentations on how the agency should invest its ARRA funds.

Jill Wechsler is Pharmaceutical Executive's Washington correspondent. She can be reached at jwechsler@advanstar.com