# Presentation: 552932

## Considering Effectiveness in Efficacy trials

**Melissa Gilliam MD, MPH**
- Associate Professor
- Section Chief, Family Planning and Contraceptive Research
- The 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

- 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%**

**Notes:**
Unintended pregnancy continues to be a major public health issue in the United States
Each year, half of all pregnancies in the United States are unintended
Half of unintended pregnancies - or one in four pregnancies overall - end in abortion
Abortion rate in US is highest of all Western developed countries and Japan

## The small proportion of women who do not use contraceptives . . .

**Women at risk of **
**unintended pregnancy,**
**1995 (42 million)**

**Women experiencing unintended pregnancies,**
**1994 (3 million)**

**. . . account for roughly half of all unintended pregnancies**

**Notes:**
Source: AGI, Fulfilling the Promise: Public Policy and U.S. Family Planning Clinics, New York: AGI, 2000.

## 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

- 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 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?

## 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

- Diversity among research participants
- Recruitment
- Retention
- Improved measures of acceptability
- 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?”

- Women
- Partners
- Providers

## 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