RELEASE: As Part of New “Project Women’s Health” Initiative, Gottheimer Announces Legislation to Combat Rare Diseases Impacting Women

Multi-front effort to boost clinical trials and find treatments and cures

Women often left out or underrepresented in clinical trials

Above: Gottheimer at Cooperman Barnabas Medical Center.

LIVINGSTON, NJ — Today, March 19, 2024, U.S. Congressman Josh Gottheimer (NJ-5) 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.”  

Video of the announcement can be found here.

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 Congressman Josh Gottheimer  (NJ-5), 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.

Below: Gottheimer with rare disease doctors, nurses, and advocates.

Gottheimer’s remarks as prepared for delivery: 

Good afternoon, and thank you again to Mark and Rick, Dr. Phillips, Stephanie, and Suzanne. It’s great to be back here in Livingston at Cooperman Barnabas Medical Center. I say back because I was born here and grew up down the road in North Caldwell. I also had surgery here after I broke my elbow as a kid. I can’t tell you the exact room. It’s a little different than what I remember — and the hospital looks fantastic. I do remember my mom sneaking me apple pancakes from the Ritz diner.

Before I begin, I want to thank all our nurses, doctors, frontline health care workers, our great North Jersey bio and life sciences leaders, and of course our rare disease researchers and advocates for the incredible work they do each and every day for our families. I cannot thank all of you enough for everything you do for our community to help improve the health and lives of countless Americans.

There are a few reasons it’s appropriate that we’re here this morning. March not only is it Women’s History Month, but it’s Blood Disorder Month, and it was just recently Rare Disease week. 

Just yesterday, recognizing the importance of this month, the President signed an Executive order directing federal agencies to deploy federal funds to research health conditions and diseases that disproportionately affect women. As a member of the Rare Disease Caucus in Congress, I couldn’t agree more.  

That is why today, I’m here to announce new legislation to help fund a new effort 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 across our nation. 

It’s all part of my new, multi-front legislative initiative project women’s health which builds on my years of work on this front.  

When it comes to research on rare and blood diseases, including those that disproportionately affect women, there is a shortage of women in clinical trials. My new legislation — the Securing Equal Access to Research, Care, and Health or SEARCH Act — will take that on. 

As a quick refresher, a rare disease is one that impacts fewer than 200,000 people and far too often has no FDA-approved treatments. We just heard from Suzanne about her niece who was diagnosed with Friedreich Ataxia and sadly passed away at the age of 30, just three years before an FDA medication was approved to help delay the effects of her condition. As Suzanne mentioned, if we had had more investments for recruitment and public awareness campaigns for clinical trials, maybe the medication to delay the effects of her niece’s condition would have been discovered in time. 

Personally, I lost my mom, of blessed memory, to a rare disease, Sarcoidosis, four and a half years ago. 

While we are making progress, and have had some big breakthroughs, it’s far too slow, and we still have a long way to go. In fact, 95 percent of the more than 10,000 rare diseases that impact more than 30 million Americans have no FDA-approved treatment.

Rare disease research is critical. Not only can it lead to a cure or treatment for those afflicted, but the research can also be a gateway to breakthroughs for other treatments and cures. 

Here in New Jersey, we are lucky to have some of the best, hospitals and medical facilities, labs, and R&D in the country, the best doctors, medical school graduates, nurses, and life sciences leaders. 

With fourteen of the world’s top twenty research-based biopharmaceutical companies here in our state, we not only have cutting-edge hospitals, but our pharmaceutical and medical device companies have led the way on some of the biggest medical breakthroughs. New Jersey ranks second in the nation for cancer medicines in development and first for heart and stroke drugs and development. In 2017, 50 percent of all new FDA approvals came from companies with a New Jersey footprint.

What’s great is we already have some success stories from rare disease and bleeding disorder research.

In 2019, there were new approvals for the first triple combination therapy for patients with cystic fibrosis and a new gene therapy to treat pediatric patients under two-years-old with spinal muscular atrophy.

And just last year, the FDA approved gene therapies for hemophilia A and hemophilia B — an important advancement in providing treatment options and reducing the need for ongoing, routine therapy.

And Allergan, right here in New Jersey, announced FDA approval for an existing drug application that can now be used to treat lower-limb spasticity in children ages two to seventeen.

I want us to double down, so that we expand in the years ahead. We can lead the way in helping to develop the next cures for orphan diseases and blood disorders too commonly leaving women without the support and treatments they need.  

And when we find those cures, it’ll help the amazing medical professionals — like here at Cooperman Barnabas Medical Center — care for women too often left without the help they need.

As Suzanne intimated, there are clear challenges and obstacles — especially when it comes to research. 

First, participation in clinical trials is poor. Since 1994, fewer than four percent of adults in the United States participate in clinical trials despite increasingly prolonged recruitment periods. What’s worse, 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 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 and underdiagnosed. The CDC even estimates that up to 1 of every 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. Women with bleeding disorders have historically been excluded from research.

Third, because women tend to have rare diseases diagnosed later than men, bias in cures or treatments 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.

The bottom line: There is a clear gap in research, awareness, diagnosis, management, treatment, and cures of diseases and disorders affecting women. It must be taken seriously and fixed. We must do more to provide hope and support to the families who struggle every day to manage a rare disease or bleeding disorder — or any disease, for that matter. We must address the inequality between men and women in research, clinical trials, and awareness. And there’s no better time than the present. 

That’s why, today, as I mentioned earlier, I’m here with medical professionals, advocates, and family members of those taken from rare diseases to announce the Securing Equal Access to Research, Care, and Health or SEARCH Act. This new legislation I’m introducing in Congress will support new research and clinical studies — and hopefully new treatments and cures — for diseases impacting women, including rare diseases and blood disorders.  

First, the legislation 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. As we laid out today, there are many diseases like blood disorders that disproportionately impact women and are often swept under the research and funding mat. 

Second, the SEARCH Act will 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. 

Third, the new legislation requires 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.

Fourth, the SEARCH Act will create a task force between the HHS, NIH, FDA, CMS, and the private sector, including hospital 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.

Finally, in his State of the Union Address earlier this month, the President called on Congress to provide $12 billion to research health conditions and diseases that disproportionately affect women. In Congress, I plan to work overtime in the coming months to make this investment a reality. We must pass legislation to create a new fund for Women’s Health Research at the NIH that will support a nationwide network of cutting-edge research centers in women’s health care.

Today, we’re here as part of my new, legislative fight on multiple fronts to protect women’s health care – whether that’s about research, clinical trials, treatments, and finding cures, or doing everything we can to protect a woman’s fundamental right to make her own personal, private healthcare decisions.

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.

In the greatest country in the world, if we can come together to solve problems and support women’s health, I know that our best days will always be ahead of us. Once again, thank you for joining me here today.

May God bless you, your families, and may God continue to bless the United States of America.

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