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Research Techniques Made Simple: Comparative Effectiveness Research

Vinod E. Nambudiri, MD MBA 1,2 and Abrar Qureshi , MD, MPH 2 1 Department of Internal Medicine, Brigham and Women’s Hospital, Boston, Massachusetts 2 Department of Dermatology, Brigham and Women’s Hospital, Boston, Massachusetts.

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Research Techniques Made Simple: Comparative Effectiveness Research

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  1. Vinod E. Nambudiri, MD MBA1,2 and AbrarQureshi, MD, MPH2 1Department of Internal Medicine, Brigham and Women’s Hospital, Boston, Massachusetts 2Department of Dermatology, Brigham and Women’s Hospital, Boston, Massachusetts Research Techniques Made Simple:Comparative Effectiveness Research

  2. What is Comparative Effectiveness? • Comparative effectiveness research (CER) aims to inform health care decision making • Involves research that compares therapeutics, devices, diagnostic tests, interventions against each other • Weighs evidence on clinical effectiveness, benefits, and harms of different diagnostic and treatment options1 1. AHRQ. “What Is Comparative Effectiveness Research.” Available at: http://effectivehealthcare.ahrq.gov/index.cfm/what-is-comparative-effectiveness-research1/

  3. Methods of CER • Analysis of already-performed studies, usually through the process of systematic review of published literature • Registry reviews • Meta-analyses • Generating novel observations via randomized controlled trials offering comparisons of diagnostics, therapeutics, or procedures against each other • Head-to-head randomized comparisons • Cluster randomized trials

  4. Challenges of CER • Interpretation of results predicated on methodology; poorly designed studies may have limited clinical impact2 • Large sample sizes necessary for sufficient statistical power • Costly to perform large trials • Selection of appropriate endpoints and timing of endpoints often difficult2 2. Williams HC and Delavalle RP (2012) J Invest Dermatol 132:1008-172.

  5. CER Example: Therapeutics • Topical corticosteroid regimens for bullous pemphigoid: high dose versus low dose3 • Randomized controlled trial • 312 patients, moderate or severe bullous pemphigoid • Regimens: 40g (high) vs 10-30g (low) dose for 4 months • Outcomes: disease control; event-free-survival; relapse; side effects • Study designed with 80% power to detect 33% difference in event-free-survival 3. Joly et al. (2009) J Invest Dermatol 129:1681–7.

  6. CER Example: Therapeutics • Kaplan-Meier curves show survival versus time • Significant difference between regimens • Log-rank test used to compare survival • Fewer side effects but higher rate of relapse with lower dose Event-free and disease-free survival curves of BP patients from the study. Kaplan–Meier estimates of event-free survival, i.e., combined outcome of death or life-threatening adverse event occurrence (a) and disease-free survival (b) of BP patients according to treatment regimen ( patients treated with the mild regimen of topical corticosteroids; patients treated with the standard regimen). Reproduced from Joly et al. (2009) J Invest Dermatol 129:1681–7.

  7. CER Example: Diagnostic Tool • Comparison of Skindex-17 versus Skindex-29 for measurement of skin health related quality of life4 • Review of 2487 surveys administered to patients • Single-site study • Calculation of intraclass correlation coefficients (ICC) to determine effectiveness of simpler tool • ICC = 0.957 for symptom questions, ICC = 0.94 for psychosocial impact questions • Cost savings of using shorter survey tool • Research efficiency of a more targeted instrument 4. Sampogna et al (2012) JID; epub 26 July 2012; doi:10.1038/jid.2012.244.

  8. Future for Dermatology CER • Needs assessments of areas for targeted dermatology CER studies • Clinical effectiveness of technologies such as teledermatology and e-visits for patient care • Head-to-head trials of medical therapeutics for common conditions • Translation of CER findings to practice-changing behavior

  9. Further Reading Agency for Healthcare Research and Quality. “What Is Comparative Effectiveness Research.” Available at: http://effectivehealthcare.ahrq.gov/index.cfm/what-is-comparative-effectiveness-research1/ Joly P, Roujeau JC, Benichou J et al. (2009) A comparison of two regimens of topical corticosteroids in the treatment of patients with bullous pemphigoid: a multicenter randomized study. J Invest Dermatol 129:1681–7. Sampogna F, Spagnoli A, Di Pietro C et al. (2012) Field Performance of the Skindex-17 Quality of Life Questionnaire: A Comparison with the Skindex-29 in a Large Sample of Dermatological Outpatients. J Invest Dermatol . Published online 2012 Jul 26; doi: 10.1038/jid.2012.244. Williams HC and Delavalle RP (2012) The growth of clinical trials and systematic reviews in informing dermatological patient care. J Invest Dermatol 132:1008-17.

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