Payer decision-making with limited comparative and cost effectiveness data: the case of new pharmacological treatments for gout

• Published in: BMJ evidence-based medicine Vol. 17; no. 4; pp. 105 - 108
• Main Authors: Meltzer, Michele, Pizzi, Laura T, Jutkowitz, Eric
• Format: Journal Article
• Language: English
• Published: England BMJ Publishing Group Ltd 01.08.2012; BMJ Publishing Group LTD
• Subjects: Alcohol; Allopurinol - economics; Allopurinol - therapeutic use; Clinical medicine; Clinical trials; Clinical Trials as Topic; Comparative Effectiveness Research; Cost-Benefit Analysis; Costs; Decision Making; Decision Trees; Drug dosages; Febuxostat; Gout - drug therapy; Gout - economics; Gout Suppressants - economics; Gout Suppressants - therapeutic use; Humans; Liver; Pharmaceutical industry; Rheumatism; Studies; Thiazoles - economics; Thiazoles - therapeutic use; Uric acid; White people
• ISSN: 1356-5524; 2515-446X; 1473-6810; 2515-4478
• DOI: 10.1136/ebmed-2011-100065

Context The need for comparative effectiveness (CE) data continues to grow, fuelled by market demand as well as health reform. There may be an assumption that new drugs result in improved efficacy compared with the standard of care, therefore warranting premium prices. Gout treatment has recently become controversial, as expensive new drugs enter the market with limited CE data. Methods The authors reviewed published clinical trials and conducted a cost effectiveness analysis on a new drug (febuxostat) versus the standard (allopurinol) to illustrate the limitations in using these data to inform evidence-based decision-making. Findings Although febuxostat trials included allopurinol as a comparator, methodological limitations make comparative effectiveness evaluations difficult. However, when available trial data were input to a decision analytic model, the authors found that a significant reduction in febuxostat cost would be required in order for it to dominate allopurinol in cost effectiveness analysis. This case exemplifies the challenges of using clinical trial data in comparative and cost effectiveness analyses.