During Rare Disease Week, Gottheimer introduces two bipartisan bills to boost R&D for rare disease cures

NEW JERSEY — During Rare Disease Week, U.S. Congressman Josh Gottheimer (NJ-5) Thursday joined with rare disease advocates, healthcare leaders, and medical professionals at Valley Hospital to announce two bipartisan bills to boost research and development to find cures and treatments for rare diseases.

Gottheimer is introducing these two bills with fellow Problem Solvers Caucus member Congressman Don Bacon (NE-2). Gottheimer also announced a new $600,000 federal investment successfully clawed back to Valley Hospital for next-generation AI patient monitoring technology, to improve safety and reduce patient falls.

A rare disease is a disease that impacts fewer than 200,000 people in the United States. Rare diseases often have no FDA-approved treatments.

“We must do more to provide hope and support to the families who struggle every day managing a rare disease, and also provide opportunities for our cutting-edge research institutions to finally get us to the desperately needed cures,” said Gottheimer (NJ-5), a member of the bipartisan Rare Disease Congressional Caucus. “The Rare Disease Clinical Trial Pandemic Disruption Act will encourage life sciences leaders to resume their delayed trials, ensuring research and development of critical treatments for rare diseases continues. And the tax credit in Cameron’s Law will give families the resources and hope for curing these rare diseases. It will give everyone here more research opportunities so that we can all come together and continue to ensure New Jersey is a leader in this area.”

“Cameron’s Law will create incentives, through tax credits, for pharmaceutical companies to create drugs to treat these diseases and get them to patients as quickly and safely as possible. Rare diseases have hit close to home, as two of my staffers have been affected by rare diseases, but thankfully drugs and treatments were available to them. It is my hope that like them, all those suffering from rare diseases will have increased access to lifesaving drugs,” said Congressman Don Bacon (NE-2), the lead Republican cosponsor of the bipartisan Cameron’s Law and the Rare Disease Clinical Trial Pandemic Disruption Act. “I’m glad to be a co-lead on the Rare Disease Clinical Trial Pandemic Disruption Act. Many patients are desperately waiting for drugs, and this bill will incentivize pharmaceutical companies to continue development and trials that have been delayed by the pandemic so that they can get these drugs to the people that need them.”

“We live in Bergen County where we have a congressman that represents us, that’s so dedicated to the world of rare disease. He’s on the frontlines. How could we sit there on the sidelines when we have a daughter at home with such a debilitating rare disease? Inevitably, we started the CAM Foundation. It’s been something that we are proud of, something that gives us some purpose,” said Rob Hyman, whose 8-year-old daughter, Cameron Hyman, has a rare disease called Sanfilippo Syndrome. “If we felt like it was falling on deaf ears, if I didn’t know Congressman Gottheimer and his dedication to this world, I don’t know if we’d have the energy for it that we do. When you hear people at the largest levels of government advocating for this, it’s hard to turn your back on your cause. It gives us inspiration. We’re very grateful in our family, and in our household, to the Congressman for not only his support and friendship, but his leadership in the world of rare disease. Of course, I’m very much honored that Cameron’s name is part of something so important like Cameron’s Law. Everything you’ve done for everybody, and all these families is inspiring and it keeps us strong.”

Gottheimer announced the introduction of two bipartisan bills to boost R&D for cures & treatments for rare diseases:

• Cameron’s Law — bipartisan legislation to fully restore the Orphan Drug Tax Credit, following the 2017 Tax Hike Bill cutting the tax credit in half and making treatment for rare diseases even rarer.
  • Cameron’s Law is named after 8-year-old Cameron Hyman from Wyckoff, NJ, who is diagnosed with the rare disease Sanfilippo Syndrome, which affects the central nervous system. Right now, there are no FDA-approved treatments for Sanfilippo Syndrome.
  • With Cameron’s Law, New Jersey’s life sciences leaders will be able to invest more in research and development for cures for orphan diseases — the majority of which have no FDA-approved treatments — and grow the talent pipeline for life sciences jobs in New Jersey through training partnerships and apprenticeships.
• Rare Disease Clinical Trial Pandemic Disruption Act — bipartisan legislation to incentivize clinical research that was halted by the COVID-19 pandemic, by allowing any company that resumes a trial for new or existing orphan drugs that show promise and were halted during the pandemic to be granted a claw-back of an additional six months of exclusivity on the specific product under consideration when it comes to market.
  • This incentive will encourage life sciences leaders to resume their delayed trials, ensuring research and development of critical treatments for rare diseases continues.
  • Find legislative text of the Rare Disease Clinical Trial Pandemic Disruption Act here.

Gottheimer announced a new $600,000 federal investment successfully clawed back for next-generation AI technology for Valley Hospital:

• By working with Valley Hospital, Gottheimer secured a new $600,000 federal investment to enhance Valley’s AUGi Project — a next-generation AI technology that will be used to reduce patient falls and boost safety.
• The monitoring technology can help identify patient movements that may suggest exiting a bed or getting up from a chair and will alert hospital staff or caregivers to the device.
• The technology allows for patient monitoring, but without streaming video to protect a patient’s privacy. It can detect if a patient is getting out of bed faster than a traditional bed alarm.
• Valley Hospital’s pilot program with the technology in its neurology unit saw a 28% reduction of falls resulting in injury.
• This new federal investment will be used to ensure that the state-of-the-art technology is included in every medical, surgical, critical care, and emergency department patient room at Valley Hospital currently under construction.