Considering Effectiveness in Efficacy trials

Melissa Gilliam MD, MPHAssociate ProfessorSection Chief, Family Planning and Contraceptive ResearchThe University of Chicago

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

Background

U.S. Pregnancies: Unintended vs. Intended

Henshaw SK. Fam Plann Perspect. 1998;30:24-29.

Unintended

Intended

Unintended births

Elective abortions

49%:

22.5%

26.5%

51%

The small proportion of women who do not

use contraceptives . . .Not

using7%

Using93%

Using53%

Not using47%

Women at risk of unintended pregnancy,

1995 (42 million)

Women experiencing unintended pregnancies,

1994 (3 million)

. . . account for roughly half of all

unintended pregnancies

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

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.

Efficacy and Effectiveness

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 testingEffectiveness 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?

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

Introducing Effectiveness into Clinical Trials

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

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

Practical methods of combining efficacy and effectiveness

1. Diversity among research participants

• Recruitment• Retention

2. Improved measures of acceptability

3. Adding technology

Diverse Population: Recruitment strategies

• 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 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

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

Minnis and colleagues, 2003“The intrauterine device is widely liked by users”

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

Adding Tools: Technology• Measuring compliance

– Monitored pill packs– Personal data assistant– Electronic medicine

dispensers

• Improving compliance– Two way pagers– Timers– Instant messaging– Electronic mail

Efficiency trials: Is it worth it?

“Go” or “No Go”

New directions in contraception-Institute of medicine

Phase I

Acceptability

II III IV

Phase I II III IV

“Is it a go?”W

omen

Partners

Provid

ers

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

Does it work? Can it work? Is it worth it?

Contraception Volume 61, Issue 1 , January 2000, Pages 9-25

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

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.

Questions?

Thank you