Despite the number of clinical trials available in the US, research shows women are often left out or underrepresented.

That's critical for individuals diagnosed with rare diseases because those trials are frequently their only avenue for care.

Rare medical disorders have few FDA approved treatments.

Congressman Josh Gottheimer today announced new federal legislation to both boost pipelines for clinical trials and help find cures for the rare illnesses that disrupt, fortunately affect women.

Melissa Rose Cooper reports.

As a member of the Rare Disease Caucus in Congress, I couldn't agree more with the agenda here that we have to achieve as a country, making sure that we invest more in women's health care.

It's an area that has too often actually been left behind where we haven't invested enough, where we haven't done enough.

And it's part of why we're here this morning.

Congressman Josh Gottheimer joining doctors, nurses and other health advocates at Cooperman Barnabas Medical Center to announce his latest initiative to support women.

The Search Act, which stands for Securing Equal Access to research Care and Help aims to find treatment for rare diseases that disproportionately impact women.

The congressman says the legislation will tackle various research challenges, including poor participation in clinical trials.

Since 94, fewer than 4% of adults in the United States participated in clinical trials, despite prolonged recruitment efforts.

We try to find people, and people just don't raise their hand.

What's worse, up to 85% of clinical trials fail to recruit or retain a sufficient sample size.

So that leads to failures to meet targets and four out of every five trials.

And for rare diseases which impact less than 200,000 people and don't often have FDA approved treatments.

Doctors say it's even harder to maintain proper trials.

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 for the individual or family with the disease.

Getting to a diagnosis may be a long and frustrating road fraught with prolonged suffering, misdiagnosis and treatment delays.

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.

Suzanne Spernal now knows all too well the negative impact not having access to treatment can have.

She lost her 30 year old niece in 2020 who had been battling a rare disease since she was a child.

Just last year, 24 years after she was initially diagnosed and 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, A breakthrough that arrived too late for my niece, but marked a significant step forward for others.

While the congressman says there has been some progress over the years, he says more work still needs to be done.

95% of more than 10,000 rare diseases that impact more than 30 million Americans have no FDA approved treatment.

Right.

It's a lot of people.

These rare diseases affect small amounts of people, right.

But you add them all up.

It's lots and lots of people and a lot of families who are affected.

Rare disease research is critical.

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

Goals Health advocates hope this new legislation will achieve so more lives can be saved.

For NJ Spotlight News, I'm Melissa Rose Cooper.