1. Schneider Institute for Health Policy Heller Graduate School Brandeis University 1 by Donald S. Shepard, Ph.D. Schneider Institute for Health Policy Heller School, MS 035 Brandeis University Waltham, MA 02454-9110 USA Tel: 781-736-3975 Fax: 781-736-3965 Web: http://www.sihp.brandeis.edu/shepard E-mail: Shepard@Brandeis.edu Monday, Oct. 1: Policy Uses of Cost-Effectiveness Analysis – HIV prevention and control

2. Schneider Institute for Health Policy Heller Graduate School Brandeis University 2 Practical information Teaching assistant: Jsuaya@Brandeis.edu Administrative assistant: Linda Purrini, Next to library in Heller 781-736-3930 Purrini@Brandeis.edu Cost of packet: $6.00

3. Schneider Institute for Health Policy Heller Graduate School Brandeis University 3 Cost-Effectiveness of Sildenafil, 1 Abstract Background: Coverage of sildenafil by health insurance plans is a contentious issue. Objective: To evaluate the cost-effectiveness of sildenafil treatment for erectile dysfunction. Design: A Markov decision model to estimate the incremental cost-effectiveness of sildenafil compared with no drug therapy.

4. Schneider Institute for Health Policy Heller Graduate School Brandeis University 4 Data Sources: Values for the efficacy and safety of sildenafil and quality-of-life utilities were obtained from the published medical literature. Base-case values were chosen to bias against sildenafil use. Target Population: Men 60 years of age with erectile dysfunction. Cost-Effectiveness of Sildenafil, 2

5. Schneider Institute for Health Policy Heller Graduate School Brandeis University 5 Time Horizon: Lifetime. Perspective: Societal and third-party payer. Intervention: Sildenafil or no treatment in identical hypothetical cohorts. Outcome Measures: Cost per quality-adjusted life- year (QALY) gained. Cost-Effectiveness of Sildenafil, 3

6. Schneider Institute for Health Policy Heller Graduate School Brandeis University 6 Results of Base-Case Analysis: The cost per QALY gained for sildenafil treatment compared with no therapy was $11 290 from the societal perspective and $11 230 from the third-party payer perspective. Cost-Effectiveness of Sildenafil, 4

7. Schneider Institute for Health Policy Heller Graduate School Brandeis University 7 Results of Sensitivity Analysis: From the societal perspective, the cost per QALY gained associated with sildenafil was less than $50 000 if treatment-related morbidity was less than 0.8% per year, mortality was less than 0.55% per year, treatment was successful in more than 40.2% of patients, or sildenafil cost less than $244 per month. The results were sensitive to variation of erectile dysfunction utilities, but cost per QALY gained was less than $50 000 if successful treatment increased utility values by 0.05 or more on a scale of 0 (death) to 1 (perfect health). Cost-Effectiveness of Sildenafil, 5

8. Schneider Institute for Health Policy Heller Graduate School Brandeis University 8 Conclusions: In an analysis biased against use of sildenafil, the cost-effectiveness of sildenafil treatment compared favorably with that of accepted therapies for other medical conditions. Cost-Effectiveness of Sildenafil, 6

9. Schneider Institute for Health Policy Heller Graduate School Brandeis University 9 Markov model for erectile dysfunction

10. Schneider Institute for Health Policy Heller Graduate School Brandeis University 10

11. Schneider Institute for Health Policy Heller Graduate School Brandeis University 11

12. Schneider Institute for Health Policy Heller Graduate School Brandeis University 12 Sildenafil, sensitivity analysis

13. Schneider Institute for Health Policy Heller Graduate School Brandeis University 13 Objectives This lecture addresses how cost-effectiveness analysis could contribute to a current policy question facing developing countries: the screening and treatment of pregnant women for HIV to reduce mother-to-child transmission, and other issues around controlling HIV.

14. Schneider Institute for Health Policy Heller Graduate School Brandeis University 14 Reducing HIV risk factors: Needs and challenges* *  Inciardi JA, Surratt HL, Telles PR. Sex, Drugs and HIV/AIDS in Brazil. Boulder Co: Westview, 2000. Chapter 4, HIV-AIDS prevention-intervention in Rio de Janeiro, 61-74. ItemBefore (N = 1544) After (N = 782) Statistical significance Some cocaine use in prior 30 days 100%90% Days with cocaine use in prior 30 days 17.3 days12.9 daysP <.001 Times used condoms during prior 30 days 1.73.7P<.003

15. Schneider Institute for Health Policy Heller Graduate School Brandeis University 15 Facts around anti-viral treatment of HIV* * Freedberg KA et al. “Cost-effectiveness of combination antiretroviral therapy for HIV disease.” N Engl J Med 2001; 334(11):824-31.

16. Schneider Institute for Health Policy Heller Graduate School Brandeis University 16 The Cost Effectiveness of Combination Antiretroviral Therapy for HIV Disease. Freedberg, Kenneth A. at al.

17. Schneider Institute for Health Policy Heller Graduate School Brandeis University 17 Abstract Background: Combination antiretroviral therapy with a combination of three or more drugs has become the standard of care for patients with human immunodeficiency virus (HIV) infection in the United States. We estimated the clinical benefits and cost effectiveness of three-drug antiretroviral regimens. Combination therapy, 1

18. Schneider Institute for Health Policy Heller Graduate School Brandeis University 18 MethodsMethods: We developed a mathematical simulation model of HIV disease, using the CD4 cell count and HIV RNA level as predictors of the progression of disease. Outcome measures included life expectancy, life expectancy adjusted for the quality of life, lifetime direct medical costs, and cost effectiveness in dollars per quality-adjusted year of life gained. Clinical data were derived from major clinical trials, including the AIDS Clinical Trials Group 320 Study. Data on costs were based on the national AIDS Cost and Services Utilization Survey, with drug costs obtained from the Red Book. Combination therapy, 2

19. Schneider Institute for Health Policy Heller Graduate School Brandeis University 19 ResultsResults: For patients similar to those in the AIDS Clinical Trials Group 320 Study (mean CD4 cell count, 87 per cubic millimeter), life expectancy adjusted for the quality of life increased from 1.53 to 2.91 years, and per-person lifetime costs increased from $45,460 to $77,300 with three-drug therapy as compared with no therapy. The incremental cost per quality-adjusted year of life gained, as compared with no therapy, was $23,000. Combination therapy, 3

20. Schneider Institute for Health Policy Heller Graduate School Brandeis University 20 Results (continued): On the basis of additional data from other major studies, the cost-effectiveness ratio for three-drug therapy ranged from $13,000 to $23,000 per quality-adjusted year of life gained. The initial CD4 cell count and drug costs were the most important determinants of costs, clinical benefits, and cost effectiveness. Conclusions: Treatment of HIV infection with a combination of three antiretroviral drugs is a cost-effective use of resources. Combination therapy, 4

21. Schneider Institute for Health Policy Heller Graduate School Brandeis University 21 Combination therapy, Data sources

22. Schneider Institute for Health Policy Heller Graduate School Brandeis University 22

23. Schneider Institute for Health Policy Heller Graduate School Brandeis University 23 Affordable approaches to controlling perinatal HIV* * Lallemant M, Jourdain G, Le Coeur S, et al. A trial of shortened zidovudine regimens to prevent mother-to-child transmission of Human Immunodeficiency Virus type 1. N Engl Med 2000; 343:982-91. Maternal treatment: Long (from week 28) vs short (from week 35) Infant treatment: Long (6 weeks) vs short (3 days) Breast feeding: Yes or no (formula feeding)

24. Schneider Institute for Health Policy Heller Graduate School Brandeis University 24 Cost-effectiveness of alternative peri-natal AZT regimens

25. Schneider Institute for Health Policy Heller Graduate School Brandeis University 25 Final Assignment of CE module, 1 Purpose: See how cost-effectiveness analysis can be applied to a subject of interest to you. Background: Select and cite an article or report of interest to you that describes the effectiveness or cost-effectiveness of a clinical intervention or program in a human services sector. For suggestions or help in identifying articles, see the instructor or teaching assistant. For example, the instructor’s report, “Economic analysis of anti-viral therapy in Botswana” provides data on the cost-effectiveness of AZT for pregnant women. A copy is available on the web site under “downloads.” Two types of papers are possible. Alternative A: Perform a preliminary cost-effectiveness analysis. If the study is an effectiveness study only, perform a preliminary cost-effectiveness analysis. Clearly indicate the alternatives you wish to compare and the effectiveness measure, and provide illustrative results. Use existing data where readily available, and explicitly assume values for other the unknown data. Interpret the findings. Discuss what kinds of data would be needed to convert the preliminary study into a more valid cost-effectiveness study, and where would they be obtained? Which items do you think are most critical?

26. Schneider Institute for Health Policy Heller Graduate School Brandeis University 26 Final Assignment of CE module, 2 Alternative B: Discuss a cost-effectiveness analysis. If the study is a cost- effectivenessstudy, discuss the appropriateness of the data, analysis, and interpretation, and put the results in a larger context. For example, compare the intervention under study with other interventions for the same disease or the same population. Submission: The length of the paper should be about 6 double spaced pages, including any tables or figures per student. Two or more students may work together and submit a joint paper that is proportionally more extensive, and longer if desired (6 pages per student). Please submit to Linda Purrini, administrative assistant, Heller G6, tel: 781-736- 3930, Purrini@Brandeis.edu. Also, please include a copy a summary, abstract, or full copy of the article with your submission. The paper may also be submitted electronically to Ms. Purrini. Schedule: Due Thursday, Oct. 25, 2001 (10 days after the last class). Evaluation: The module will be graded according to each student’s registration (audit, pass-fail, or letter grade) based on this final exercise, other exercises, and class participation. In addition, the instructors plan to give written comments on this exercise. The instructors look forward to your papers.

27. Schneider Institute for Health Policy Heller Graduate School Brandeis University 27 Next class session Monday, Oct. 8, 6 pm – 9 pm

28. Schneider Institute for Health Policy Heller Graduate School Brandeis University 28 Final class session Monday, Oct. 15, 6 pm – 9 pm

29. Schneider Institute for Health Policy Heller Graduate School Brandeis University 29 Final class, Oct. 15 Help us identify illustrative, empirical, preferably intervention studies outside of the health sector Collect and read the selected studies (distributed later this week) Be prepared to discuss the cost-effectiveness framework: What are the alternative(s)? What are the costs? What are the outcomes (effectiveness)? How do we interpret cost-effectiveness results?