Congressman Proposes Legislation to Combat Rare Diseases Impacting Women

Congressman Promotes Legislation to Combat Rare Diseases Impacting Women

Rare Daily Staff

U.S. Congressman Josh Gottheimer, D -New Jersey, unveiled legislation to help fund new efforts to increase the number of women in clinical research trials for rare diseases and blood disorders.

The Securing Equal Access to Research, Care, and Health, or SEARCH Act, would require the National Institutes of Health to produce an action plan within 180 days to highlight the rare disease and health complications that uniquely impact women.

It would fund recruitment campaigns for women in NIH clinical research trials for rare diseases and require the U.S. Centers for Disease Control and Prevention and the U.S. Department of Health and Human Services to increase the number of women in federal bleeding disorder programs that provide funding for research, surveillance, prevention, and services through public awareness campaigns.

The legislation would also create a task force between the HHS, NIH, the U.S. Food and Drug Administration and the Centers for Medicare and Medicaid Services, and the private sector to produce a report on the rare diseases that disproportionately impact women, helping ensure more funding goes toward diseases and conditions that impact women.

“There is a clear gap in research, awareness, diagnosis, management, treatment, and cures of diseases and disorders affecting women. We must address the inequality between men and women in research, clinical trials, and awareness,” said Gottheimer, who is a member of the Rare Disease Caucus. “It must be taken seriously and fixed and there’s no better time than the present.”

Up to 85 percent of clinical trials fail to recruit or retain a sufficient sample size, leading to failures to meet targets in four out of every five trials. For rare diseases more commonly impacting women, like Rett syndrome, it can be even harder to start and maintain proper trials.

Gottheimer said because women tend to have rare diseases diagnosed later than men, bias in cures or treatments for women start early and leave lasting impacts. Reports indicate that women are directed to a hospital and specialists later than men following the onset of symptoms, which delays diagnosis and care. This can often lead to a rapid progression of the disease.

“I have witnessed the disparities women face and have faced for decades,” said Hemophilia Association of New Jersey Executive Director Stephanie Lapidow. “Women are overlooked, misdiagnosed, or not diagnosed at all and it has become clear that legislation of this kind is not only necessary but critical for women, not only with bleeding disorders but also for women in the rare disease community at large.”