As part of new ‘Project Women’s Health’ initiative, Gottheimer announces legislation to combat rare diseases impacting women

LIVINGSTON, NJ (Essex County) — U.S. Congressman Josh Gottheimer (NJ-5) Tuesday announced new legislation, the Securing Equal Access to Research, Care, and Health, or SEARCH Act, to help fund new efforts to increase the number of women in clinical research trials, leading to treatments and cures for rare diseases and blood disorders which affect millions of women in New Jersey and across our nation.

The legislation is part of Gottheimer’s new, multi-front legislative initiative: “Project Women’s Health.”

Gottheimer also highlighted his support for the President’s State of the Union Address earlier this month, which called on Congress to provide $12 billion to research health conditions and diseases that disproportionately affect women. In Congress, Gottheimer plans to work to help make this investment a reality.

“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. It must be taken seriously and fixed and there’s no better time than the present,” said Gottheimer, Rare Disease Caucus Member. “Even with all of the challenges we are facing, I remain optimistic and hopeful. I hope that you are, too. Built on collaborative work with experts and advocates, I’m confident that the SEARCH Act will ultimately lead to life changing medication, treatment, and cures for women impacted by rare diseases and bleeding disorders.”

“The Congressman is an advocate for ensuring equal access to care for all residents in New Jersey. He is focused on addressing disparities and improving healthcare outcomes across the state. We share a deep commitment with the Congressman to the most vulnerable among us,” said RWJBarnabas Health President and CEO Mark E. Manigan. “While we’re very proud of the awesome clinical services we offer here at Cooper and across the system, we are equally proud of that commitment to the most vulnerable as this state’s largest provider of charity care and as the state’s largest providers of care to beneficiaries to the medicare program by two times.”

“Thank you congressman for giving me the opportunity to speak this morning and for leading the charge on the SEARCH Act. It’s an important piece of legislation that champions the health of women, especially those affected by rare disease. The importance of the SEARCH act goes beyond my professional role and in fact connects deeply and is personal to me. In 1999, my family’s life changed forever when my niece who was ten years old at the time was diagnosed with Friedreich’s ataxia — a rare, debilitating, degenerative, neuromuscular disease. Sadly, in 2020, my niece’s battle ended just months after her 30th birthday… just last year… almost three years to the day after she died, the FDA approved a new medication to slow the progression for Friedreich’s ataxia. A breakthrough that arrived too late for my niece, but marked a significant step forward for others. The SEARCH Act represents a beacon of hope for women suffering from rare diseases,” said RWJBarnabas Health Vice President of Women’s Services Suzanne Spernal.

“I have witnessed the disparities women face and have faced for decades. 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,” said Hemophilia Association of New Jersey Executive Director Stephanie Lapidow.

“Although a disease may be rare, thus impacting a small number of people, it does not change the profound impact a condition has on those afflicted, as well as their families. Not uncommonly, due to the limited understanding of these diseases and the small number of people affected, funding for research is often scarce, making it difficult to find treatments,” said RWJBarnabas Director of Cell Therapy and Bone Marrow Transplantation Northern Regions Dr. Adrienne Phillips. “For the individual or family with a disease, getting to a diagnosis may be a long and frustrating road fraught with prolonged diagnosis and treatment delays.”

Challenges Facing Women’s Health Related to Rare Diseases and Blood Disorders:

• First, participation in clinical trials is poor.
  • Since 1994, fewer than 4% of adults in the United States participate in clinical trials despite increasingly prolonged recruitment periods.
  • Up to 85% 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 and multiple sclerosis, it can be even harder to start and maintain proper trials.
• Second, like rare diseases, women with bleeding disorders have often been underrecognized, underdiagnosed, and excluded from research.
  • The CDC estimates that up to 1 in 100 women and girls in the United States — more than 1.5 million Americans — have a bleeding disorder, many of whom aren’t even aware of their condition.
  • The most common bleeding disorder affecting women is von Willebrand disease, or VWD, which results from a deficiency or defect in the body’s ability to produce a certain protein that helps blood clot.
• Third, 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.

Gottheimer’s New Legislation, the Securing Equal Access to Research, Care, and Health or SEARCH Act will:

• Require the National Institute of Health, or NIH, to produce an action plan within 180 days to highlight the rare disease and health complications that uniquely impact women.
  • There are many diseases that disproportionately impact women and are often swept under the research and funding mat.
• Fund recruitment campaigns for women in NIH clinical research trials for rare diseases by increasing advertising at hospitals, doctors’ offices, health centers, and healthcare clinics. This can have a direct and immediate impact on helping Jersey families.
• Require the CDC and HHS to increase the number of women in federal bleeding disorder programs that provide funding for research, surveillance, prevention, and services through public awareness campaigns.
• Create a task force between the HHS, NIH, FDA, CMS, and the private sector, including hospitals and labs in New Jersey, to produce a report on the rare diseases that disproportionately impact women, helping ensure more funding goes toward diseases and conditions that impact women.

Gottheimer was joined by RWJBarnabas Health President and CEO Mark E. Manigan, Cooperman Barnabas Medical Center President and CEO Richard L. Davis, RWJBarnabas Health Senior Vice President Dr. Balpreet Grewal-Virk, RWJBarnabas Health Vice President of Women’s Services, Director of Cell Therapy and Bone Marrow Transplantation Northern Regions Adrienne Phillips, and Hemophilia Association of New Jersey Executive Director Stephanie Lapidow.