The Comparative-Effectiveness Controversy - Pharmaceutical Technology

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The Comparative-Effectiveness Controversy
Government funding is slated to boost comparative studies of prescription drugs.

Pharmaceutical Technology
Volume 33, Issue 5, pp. 30-36

To ensure broad input on this important issue, IOM also established an online system for people to submit specific proposals for CE studies. The panel is ranking these submissions according to disease burden, disease severity, variation in care, cost, public interest, and current gaps in information. The questionnaire requests a brief summary of the research question, study design, study population, and which alternatives should be compared.

A new Federal Coordinating Council for CE Research will monitor how well HHS meets the IOM priorities in awarding CE research grants and will coordinate government-funded CE research initiatives. The 15-member council includes top officials from HHS, NIH, AHRQ, the US Food and Drug Administration, and other federal agencies. In the spirit of transparency, HHS will publish information about grants and contracts awarded under this program, will disseminate the research findings that result, and will report annually to Congress on the program.

One effect of government investment in CE research would be to develop standards and to improve methods for conducting comparative studies and to develop innovative trial designs for real-world assessment of medical treatments. At the IOM public meeting, Bryan Luce, senior vice-president of United BioSource, urged "true transformational thinking" in designing CE research studies. Without it, he said, we will "waste vast amounts of money answering the wrong questions, or the right questions too late."

Existing models

Although ARRA funding will spur activity in this area, a good deal of CE research has been going on for some time. The Medicare Modernization Act of 2003 provided limited funding to AHRQ for research to determine the clinical effectiveness and appropriateness of various health services, including prescription drugs. AHRQ now has a $50-million Effective Health Care program, which synthesizes existing clinical information.

Comparative-cost issues are examined more explicitly by private-sector comparative-research programs supported by health-insurance companies and payers. The Blue Cross and Blue Shield Association's Technology Evaluation Center has been reviewing clinical evidence since 1985 to assess the effectiveness of certain medical procedures, drugs, and medical devices. The Drug Effectiveness Review Project (DERP) at the Oregon Health and Science University provides state Medicaid agencies and large insurers with comparative information on the efficacy and safety of new, high-cost medicines; on widely used drugs; and on therapies that are frequently used for off-label indications. Consumer Reports's Best Buy Drugs program uses the DERP analyses to develop information comparing the effectiveness and costs of widely used medicines.

Several foreign CE research programs have established models for CE advocates in the US. The National Institute for Health and Clinical Excellence (NICE) in the United Kingdom provides Britain's National Health Service with recommendations about the coverage of new drugs and diagnostics and about clinical best practices. NICE sets a clear cost-effectiveness threshold that has led to controversial no-coverage decisions on several new, but costly, therapies.

Australia's Pharmaceuticals Benefits Advisory Committee reviews clinical evidence and makes coverage recommendations to the nation's health minister. Germany's Institute for Quality and Efficiency evaluates drugs and health services to help a Federal Joint Committee determine reimbursement and benefits. These programs generally assess commonly used drugs and established medical procedures and rely on literature searchers and epidemiological surveys to support recommendations.

Industry involvement

Pharmaceutical manufacturers consequently have been sponsoring more comparative studies on their own 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. FDA is demanding more postapproval safety studies comparing new drugs to existing therapies. Premarket safety and efficacy studies in Europe and other regions routinely test new products with comparators instead of placebos. And although FDA generally requests placebo-controlled studies, comparative clinical information can be critical for gaining agency approval and market access, particularly in crowded therapeutic classes or for new products that raise safety issues.

GlaxoSmithKline (London) is testing its long-acting type-2 diabetes treatment Syncria (albiglutide) against several active comparators such as metformin, insulin, sulphonylurea, and other drugs. The multiarm, 4000-patient study aims to show clear benefits over existing treatments for a therapy that may only require weekly or less frequent dosing.


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