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  1. Science and Research Special Topics

Women's Health Research Roadmap - Priority Area 1: Advance Safety and Efficacy

Next Section: Priority Area 2

Advance the safety and efficacy and reduce the toxicity of FDA-regulated products used by women.

Proposed research in this priority area should take a comprehensive look at the diseases and conditions primarily affecting women and the areas where additional research is needed to advance our understanding of disease and treatment in women in particular. It should focus on health areas of unmet medical need and scientific gaps related to FDA-regulated products, such as those outlined in the IOM report33 on knowledge gaps in women’s health. Research should explore how sex differences may affect medical product safety and effectiveness or play a role in the use of other regulated products (e.g., cosmetics). Research should also explore the possible effects on diverse and special subpopulations of women across the lifespan (e.g., reproductive age women, pregnant women, post-menopausal women, elderly women).

FDA will use the results of this research to advance the understanding of disease presentation and manifestation in women, the mechanisms of action of regulated products, and women’s response to therapies to treat those diseases. This research should expand FDA’s capacity to effectively evaluate FDA-regulated products used by women throughout all life and disease stages.

Potential health impacts of this research include gaining a better understanding of diseases in specific subpopulations of women (e.g., elderly women,34 pregnant women), which should enhance the ability of patients and their health care professionals to make appropriate treatment decisions. Advances in the science of women’s health, especially in areas of unmet medical need or in diseases or conditions with a large public health burden, will advance women’s health in general.

Objectives

1.1 Advance our understanding about diseases and conditions that primarily affect women, such as breast health, reproductive issues, obstetric and gynecological issues, and biocompatibility issues related to implants used in women

1.2 Expand evaluation of regulated products for disease areas where additional research is needed to advance our understanding of disease prevention, presentation, manifestation, and treatment in women that may be different from those in men; promote innovation in product development; or address disparities affecting women, to include, but not limited to major causes of mortality and morbidity such as:

  • Autoimmune disease
  • Cancer
  • Cardiovascular disease
  • Diabetes
  • HIV/AIDS
  • Lung Disease
  • Neurological conditions
  • Osteopenia/Osteoporosis
  • Psychiatric disorders 

1.3 Improve our understanding of the apparent role of sex-correlated co-morbidities, concomitant medications, other subgroup differences (e.g., age, race, obesity, body size, physiology) and metabolism of drugs and biologics on the toxicity or the safety and efficacy of FDA-regulated products

1.4 Evaluate the role of sex hormones on the efficacy and safety of medical products

1.5 Develop tools and methods in support of development of diagnostics and therapeutics targeting specific subpopulations of women, including, but not limited to:

  • Develop tools and methods to evaluate innovative, new devices and diagnostics specifically designed for use in women (e.g., gynecological devices)
  • Develop tools and methods to enhance the evaluation of devices used in both men and women to take into consideration sex differences like organ size/anatomy/physiology/human factors differences that may affect device performance
  • Foster development of tools and methods to evaluate sex-matched devices and companion diagnostics

1.6 Enhance our understanding of how FDA-regulated products work in sub-populations of women, including, but not limited to the following examples:

  • Elderly Women – Identify sex-specific biomarkers for disease progression and treatment outcome in elderly women, including elderly women with comorbid conditions and those who take multiple therapeutic agents
  • Pregnant Women – Enhance our understanding of the toxicity or the safety and effectiveness of FDA-regulated products used during pregnancy
  • Lactating Women – Evaluate the safety of drugs, biologics, and potential toxicants, including from tobacco or tobacco smoke, and materials used in manufacturing medical devices, that may be transferred into breast milk and to nursing infants
  • Pre- and post-menopausal Women – Investigate how the effects of hormonal changes and reproductive transition in pre- and post-menopausal women may affect the toxicity or the safety and efficacy of FDA-regulated products

Next Section: Priority Area 2 - Improve Clinical Study Design and Analyses


Footnotes:

33 See Institute of Medicine. 2010. Women’s Health Research: Progress, Pitfalls, and Promise. Washington D.C.: The National Academies Press.

34 Generally, considered to be persons who are 65 years old and older.

35 Sex-matched devices are devices that are typically used in both sexes, but for which there are specific versions with specific claims for women. An example is a set of orthopedic implants that have specific sizes and shapes intended for women.

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