Considering Effectiveness in Efficacy trials

1. Considering Effectiveness in Efficacy trials Melissa Gilliam MD, MPH Associate Professor Section Chief, Family Planning and Contraceptive Research The University of Chicago

2. Overview • Background • Efficacy and Effectiveness • What we can learn from other disciplines • Practical approaches to adding effectiveness • Diversity • Better tools • Technology • Putting it all together • Efficacy, Effectiveness and Efficiency

3. Background

4. U.S. Pregnancies: Unintended vs. Intended Intended 51% 49%: Unintended 22.5% Unintended births Elective abortions 26.5% Henshaw SK. Fam Plann Perspect. 1998;30:24-29.

5. The small proportion of women who do not use contraceptives . . . . . . account for roughly half of all unintended pregnancies Women at risk of unintended pregnancy, 1995 (42 million) Women experiencing unintended pregnancies, 1994 (3 million)

6. Disparities in unintended pregnancy Rates are highest among: • Women aged 15–24 • Unmarried women • Black and Latino women • Women below 200% of the federal poverty level

7. Adherence and method selection • Leading methods: The oral contraceptive pill (OCs) and sterilization • White women more likely to use OCs • African American and Latino women are more likely to use sterilization • Poor and low-income women are more than twice as likely as higher income women to use the three month injectable.

8. Efficacy and Effectiveness

9. Archibald Cochrane Efficacy study “Can it work?” • Under ideal circumstances does the drug in question do more good than harm? • A very essential first step in drug testing Effectiveness study “Does it work?” • Beyond the ideal circumstances of an efficacy trial • Will the contraceptive work in the not so ideal circumstances of clinical practice?

10. What affects effectiveness? • Patient adherence • Personal characteristics • Partner characteristics • Social and cultural context for method use • Aspects of the contraceptive method • Health care delivery system • Provider adherence • Coverage of the method

11. Introducing Effectiveness into Clinical Trials

12. What can we learn from other disciplines? • Shifting paradigms: Social science • Methods: Cultural sensitivity/cross cultural research • Theory: Ecological frameworks • Adding tools: Business • Marketing analyses • Complex decision analyses

13. Shifting paradigms: Bronfenbrenner’s Ecological Theory of Human Development • Human development placed in context of social entities: “like a set of Russian dolls” • Family • Neighborhood • Community • Society • Behavior is a function of the person and the environment • Bioecological model

14. Practical methods of combining efficacy and effectiveness • Diversity among research participants • Recruitment • Retention • Improved measures of acceptability • Adding technology

15. Bilingual research team Spanish language study materials Working through community-based agencies Befriending staff of community agency Staff participation in research Agency staff serve as primary recruiters Snow-ball recruitment Engage community leaders in project Engage mothers, partners and family members in project Provide food, transportation, childcare Provide opportunities for feedback Engage participants as “experts” Diverse Population: Recruitment strategies

16. Diverse populations: Retention strategies • Convenient hours of operation • Convenient locations • Alliances with local health care facilities • Working through social workers and providers who are already trusted in community • Offer meaningful incentives • Upfront qualitative research to determine the population’s needs • Dissemination of results back to the community

17. Better measures of acceptability • Current methods include surrogates: • hypothetical acceptability through surveys • Uptake • Continuation • Acceptability studies and measures of acceptability do not predict actual use* • Acceptability within a narrow population may not predict widespread use • “The intrauterine device is widely liked by users” Minnis and colleagues, 2003

18. Adding tools: Measures • Contraceptive characteristics • Assessing method characteristics • ranking methods • Shared decision analysis • Deciding on alternative medical choices in context of personal situation and preferences • Vignettes to evaluate additional variables • Characteristics: Libido, Bleeding, Amenorrhea • Use behaviors

19. Adding Tools: Technology • Measuring compliance • Monitored pill packs • Personal data assistant • Electronic medicine dispensers • Improving compliance • Two way pagers • Timers • Instant messaging • Electronic mail

20. Efficiency trials: Is it worth it?

21. “Go” or “No Go” Phase I II III IV Acceptability Women Partners Providers “Is it a go?” Phase I II III IV New directions in contraception-Institute of medicine

22. Is it worth it? • Affect contraceptive access and knowledge • Commitment to the medically underserved • Access to new methods through clinical trials • Development of culturally acceptable contraceptive methods

23. Does it work? Can it work? Is it worth it? Contraception Volume 61, Issue 1 , January 2000, Pages 9-25

24. Putting it all together: The drum roll • Add theoretical frameworks • Add theory early • Qualitative research • Better measures • “go” or “no go” • Diverse study population • Retention and recruitment • Actual contraceptive use behaviors • Technology • Technological divide • Efficiency

25. References 1. Boonstra H, Duran V, Northington Gamble V, Blumenthal P, Dominguez L, Pies C. 2000. The “boom and bust phenomenon”: the hopes, dreams, and broken promises of the contraceptive revolution. Contraception 61:9-25. 2. Institute of Medicine. 2005. Improving contraceptive use and acceptability. In: Nass SJ, Strauss JF III, editors. New frontiers in contraceptive research: A blueprint for action. Washington DC: The National Academies Press. 3. Minnis AM, Shiboski SC, Padian NS. 2003. Barrier contraceptive method acceptability and choice are not reliable indicators of use. Sex Transm Dis 30(7):556-561. 4. O’Connor AM, Legare F, Stacey D. 2003. Risk communication in practice: the contribution of decision aids. BMJ 327(7417):736-740. 5. Severy LJ. 1999. Acceptability as a critical component of clinical trials. Adv Pop 3:103-122.

26. Questions? Thank you