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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