Economic evaluation of health programmes

1. Economic evaluation of health programmes Department of Epidemiology, Biostatistics and Occupational Health Class no. 18: Economic Evaluation using Patient-Level Data I Nov 10, 2008

2. Plan of class • Discuss assignment no. 3 • Collecting patient-level data alongside RCTs – some issues • Analysis of uncertainty

3. Question 1 • Excellent vignettes • Great sample. • Wonderful to be able to use Dr Bayoumi’s program – thank you Stephanie! • Went well beyond what was required. • Very good analysis of systematic patterns.

4. Question 2 • Almost everyone did correctly. • Careful with rounding errors • Could use discounting

5. Question 3

6. Welfarism vs extra-welfarism: How do we measure social welfare? Impacts on individuals – hence society: Health care resources Impacts on other resources available

7. From Arrow to Sen Everyone matters equally so distributional consequences matter No basis for evaluating distributional consequences Not possible to meld peoples preferences into a reasonable social welfare function

8. Welfarist vs extra-welfarist perspectives on economic evaluation

9. Welfarist vs extra-welfarist perspectives on economic evaluation Sources: Brouwer and Koopmanschap 2000; Birch and Donaldson 2003; Brouwer et al. 2008

10. Arguments against welfarism • Peoples’ own present utilities (subjective evaluations of well-being) may not reflect their ultimate self-interest (e.g., due to not taking long-term consequences into account) • Utilities may be small but nonetheless we may have a moral obligation to provide care • Welfarism likely to ignore externalities that people may not internalize

11. Arguments against extra-welfarism • No objective basis for measuring social welfare • Even communitarian approaches may be inadequate • Impossible to construct a non-dictatorial social welfare function (Kenneth Arrow) • Somewhat ad hoc character – not really scientific

12. Is extra-welfarism necessarily paternalistic? • Paternalism: Influencing someone into doing something they may not want to do, out of a (correct or incorrect) belief that this is what is best for them. • Extra-welfarism can reflect a paternalistic viewpoint • Sometimes questionable, e.g., abortions viewed as bad • But this can be clearly legitimate - take into account externalities (e.g., vaccines) • But, can also be grounded in moral obligation • Care for people with severe disabilities or Alzheimers who may not benefit greatly from such care

13. Collecting economic data alongside clinical trials

14. Issues with adding economic component to an RCT • Useful complement to efficacy data – very common in UK; and, relatively cheap • However: • Is comparison condition relevant? • Non-standard, more intensive measurement of outcomes that could affect results • Use of intermediate health outcomes • Inadequate follow-up period or sample size • Need to abstract from protocol-driven costs • Artificial rules mandated by protocol (selection of subjects, keeping them in study, adherence to Tx)

15. What to do? • ‘Pragmatic’ trials: • Patients representative of typical caseload • Routine follow-up • Meaningful and wide range of outcomes • Longer follow-up if relevant • Usually larger sample size • Unblinded patients and physicians • Not often done • Modeling! • With adjusted trial data

16. Data collection methods • Add sheets to case report forms in hospital for hospital-based study • More or less detailed depending on what is being evaluated • Administrative data • Questionnaires and diaries • Accuracy of patient recall is an issue • Ideally monitor every 30 days

17. Statistical analysis of economic data • Nature of economic data • Skewed distribution • Missing data • Hopefully missing at random • Censored data • Difficulties with ICERs