Clinical trials provide a critical base of evidence for evaluating whether a medical product is effective before the product is approved for marketing. Women’s participation in clinical trials has improved in many areas, but we need to continue this momentum. The FDA Office of Women’s Health (OWH) was established by congressional mandate to advocate for the participation of women in clinical trials and the analyses of data by sex. OWH supports studies to track the participation of women in clinical trials and collaborates with FDA's centers and external partners to conduct trainings, workshops, outreach, and research to improve clinical trials design, recruitment, and analyses and to support FDA regulatory policy and decision-making.
Why is this work so important?
Adequate participation of women in clinical trials allows representation of the population most likely to use a disease therapy.
Analyzing data by sex allows researchers to determine if there are any sex differences in the response to medical treatment.
Analyses for sex differences can provide insight into the scientific basis for individual therapy differences and provide future directions for research.
FDA Guidance, Regulations and Reports
FDA has a variety of statutory, regulatory, and policy-related tools that provide a framework for guiding medical product sponsors in the inclusion and analysis of women and demographic subgroups in clinical trials.
White Paper: Successful Strategies for Engaging Women and Minorities in Clinical Trials (PDF 1.2MB)
NIH Outreach Toolkit: How to Engage, Recruit, and Retain Women in Clinical Research
In-Person Training: FDA's Clinical Investigator Training Course
The Science of Sex and Gender in Human Health: Three free CE Courses developed with the NIH Office of Research on Women's. Health. This online series of courses was designed to provide a foundation for sex and gender accountability in medical research and treatment.
Diverse Women in Clinical Trials Webinar Series (in conjunction with The Association of Clinical Research Professionals): Three-part series providing clinical research professionals with proven strategies for recruiting and retaining diverse women in research and best practices for preparing for FDA inspections. All employees of the Department of Health and Human Services may access these materials at no cost.
- “Diverse Women in Clinical Trials Part I: Planning for Diversity” available through October 31, 2017.
- “Diverse Women in Clinical Trials Part II: Recruitment and Retention” available through November 8, 2017.
- “FDA BIMO Compliance and Enforcement of Drugs and Devices” available through November 15, 2017.
Development of the US Women’s Health Coordinated Registries Network – Nilsa Loyo-Berrios, PhD, CDRH (2016)
Individual patient-data meta-analysis and postmarket analysis as a method for improving data quality in demographic subgroups. (Support for implementation of FDASIA Section 907 Action Plan) – Daniel Canos, PhD, CDRH (2015)
Zusterzeel R., O’Callaghan KM., Canos DA., Sanders WE., Marinac-Dabic D., Strauss DG. Improving the Safety and Effectiveness of Medical Device Therapy in Women. J Womens Health (Larchmt). 2016 May;25(5):428-30. doi: 10.1089/jwh.2015.5605.
Inclusion of Minority Racial/Ethnic Subjects in OB-GYN Device Applications and Device Labeling, 2005-2015 – Christopher Ronk, SD, CDRH (15)
Collection, Analysis, and Availability of Demographic Subgroup Data for FDA-Approved Medical Products – Annie Pariser, MD, CDER (14)
Evaluation of gender-related clinical pharmacology information in the labelings on adverse events and outcomes – Lei Zhang, PhD, CDER (2010)
Women in HIV trials: a comprehensive review and meta-analysis for safety – Greg Soon, PhD, CDER (2010)
Identification of Sex Differences in Adverse Outcomes for New Molecular Entities (NMEs) Approved from 2000-2002 – Yongsheng Yang, Ph.D., CDER (2007)
Yang Y, Carlin AS, Faustino PJ, Paga´n Motta MI, Hamad ML, He R, Watanuki Y, Pinnow EE, Khan MA, Participation of Women in Clinical Trials for New Drugs Approved by the Food and Drug Administration in 2000–2002. J of Women’s Health March 2009; 18(3):303-310.
Participation of Women in Clinical Trials and Gender Analysis of Data in Original NDAs Approved 2000-2002 – Yongsheng Yang, Ph.D., CDER (2006)
Gender Differences and Impact of Pharmacogenomics in Rheumatoid Arthritis – Shashi Amur Ph.D., CDER (2006)
Arasappan D, Tong W, Mummaneni P, Fang H, Amur S, Meta-analysis of microarray data using a pathway-based approach identifies a 37-gene expression signature for systemic lupus erythematosus in human peripheral blood mononuclear cells. BMC Medicine May 2011; 9:65.
Amur S, Parekh A, Mummaneni P. Sex differences and genomics in autoimmune diseases. Journal of Autoimmunity May 2012; 38(2-3):J254-J265.
Impact of Gender Analysis and Pharmacogenomics on Clinical Efficacy, Safety, and Pharmacokinetics of Drugs Used for the Treatment of Alzheimer’s Disease – Angela Men, Ph.D., CDER (2006)
Women in HIV Trials: A Comprehensive Review and Meta-Analysis – Greg Soon, Ph.D., CDER (2006)
Soon GG, Min M, Struble KA, Chan-Tack KM, Hammerstrom T, Qi K, Zhou S, Bhore R, Murray JS, Birnkrant DB. Meta-Analysis of Gender Differences in Efficacy Outcomes for HIV-Positive Subjects in Randomized Controlled Clinical Trials of Antiretroviral Therapy (2000–2008). AIDS Patient Care and STDs August 2012; 26(8):444-453.
Statistical Analysis of Gender Specific Data from New Drug Application (NDA) Submissions – Ohidul Siddiqui Ph.D., CDER (2006)
Evaluation of Availability and Quality of Information Available on Females Included in Mechanical Device Implant Trials – Kathryn O’Callaghan B.S., CDRH (2006)
Women’s participation in clinical drug trials for unstable angina and myocardial infarction. Ann Farrell, MD, CDER (2000)
Development and expansion of a pilot tracking system for monitoring the barriers to the enrollment of women in clinical trials. Toigo, Teresa, RPH, MBA, OC/OEA (1995)
Conference: Dialogues on Diversifying Clinical Trials: Successful Strategies for Engaging Women and Minorities, September 22-23, 2011, Washington, DC (Held in Collaboration with the Society for Women’s Health Research)
Workshop: Exploration of Public Policy Development Regarding the Study and Analysis of Sex Differences in the Clinical Evaluation of Cardiovascular Medical Products, June 2, 2008, Kathryn M. O’Callaghan, B.S., CDRH
Workshop: Gender Differences in Cardiovascular Device Trials, December 9, 2008, Kathryn M. O’Callaghan, B.S., CDRH
Workshop: Gender Studies in Product Development: Scientific Issues and Approaches. November 1995
The FDA Office of Women’s Health established a new CE lecture series for FDA reviewers to provide the latest evidence about sex differences in disease presentation, treatment, and outcomes. Launched in March 2016, the series features scientific developments and regulatory best practices in sex and gender-specific women’s health.
- Successful Strategies for Engaging Women and Minorities in Clinical Trials (PDF - 1.2MB)
- Outreach Toolkit: How to Engage, Recruit, and Retain Women in Clinical Research (NIH Office of Research on Women's Health)
- Inclusion of Women in Research (NIH Office of Research on Women's Health)