RIDGEWOOD, NJ— Today, March 2, 2023, during Rare Disease Week, U.S. Congressman Josh Gottheimer (NJ-5) 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 Congressman Josh 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 today, 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.
Gottheimer was joined at Valley Hospital today by Rob Hyman, founder of the CAM Foundation and the father of Cameron Hyman, Valley Health System President of Clinical Integration & Physician Enterprise Dr. Robert Brenner, Valley Health System Director of the Center for Comprehensive Heart Failure Care Dr. Kariann Abbate, and Valley Hospital medical professionals.
Below: Gottheimer at Valley Hospital in Ridgewood.
Below: Gottheimer with Rob Hyman, whose 8-year-old daughter, Cameron Hyman, has a rare disease called Sanfilippo Syndrome.
Gottheimer’s remarks as prepared for delivery:
Good afternoon. It’s great to be here in Ridgewood at the Valley Hospital. 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 everyday for our families. I cannot thank all of you enough for everything you do for our community and to help improve the health and lives of millions of Americans.
It’s Rare Disease Week, and I’m here today to announce two new bipartisan bills that I’m introducing to help our incredible New Jersey life sciences sector boost research and development to find treatments and cures for rare diseases — sometimes known as orphan diseases.
A rare disease is one that impacts fewer than 200,000 people in the U.S. Rare diseases often have no FDA-approved treatments, and, today, we’ve heard about some of our New Jersey families impacted by a rare disease. We are making progress on these fronts, but we have a long way to go. More than 90% of rare diseases still have no FDA-approved treatment.
Rare disease research is critical because not only can it lead to a cure or treatment for those inflicted, but the research can also be a gateway to breakthroughs for other treatments and cures too.
We will also be announcing, today, a new federal investment clawed back from Washington to Valley Hospital for next-generation patient monitoring technology — to help make sure our families are safe and always receiving the best care possible.
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 — and just like we came together during the pandemic to keep our communities safe, we need to keep coming together to cure and treat rare diseases — those that often don’t get the attention and investment they need and deserve.
We need to make sure that New Jersey can continue leading in the life sciences sector through expanded research and development, job creation, and take steps — including with new legislation — to invest in cures for rare diseases.
With 14 of the world’s top 20 research-based biopharmaceutical companies here in our state, New Jersey is one of the leading centers in the world for life sciences and innovation. 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 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 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 2 to 17.
Known as “the medicine chest of the world,” New Jersey has been a major global hub for biopharma for more than a century.
I want us to double down, so that we expand in the years ahead – and continue leading the way in medicine and bio jobs, helping to develop the next cures for orphan diseases, and developing cutting edge medical devices that allow people to walk again and live normal, healthy lives.
After all, we are on the cutting edge, with more than half of the twenty best global pharmaceutical companies operating here. This represents a significant opportunity to attract more companies, grow revenues, attract more jobs here, and continue leading in improving the health of people all around our nation.
And when we find those cures, it’ll help the amazing medical professionals — like here at Valley — care for patients.
But there are challenges. First, it’s expensive to do business in our state and we need to make it more affordable. Since the 1990s, the cost of doing business in New Jersey has increased more than most of our peers. It is more than 10% higher than the U.S. average, primarily because of the state’s high taxes and red tape — and the cost of doing business in New Jersey has continued to rise. Now, I helped lead the charge to eliminate the federal Medical Device Tax, and we are working to restore SALT, the State and Local Tax Deduction, to help cut property taxes, cut outdated regulations, and work with the state legislature to provide other tax incentives for businesses who relocate here.
Second, in 1983, to encourage research in rare diseases, Congress passed and President Reagen signed into law the bipartisan Orphan Drug Act. To help offset the incredibly costly, and far too often unsuccessful research into rare diseases, it included a R&D tax credit for life sciences companies. Only 12 percent of all drug research projects lead to a go-to-market drug. Before the tax credit, only thirty-eight orphan drugs for rare diseases had been approved for use in the United States. As of 2022, the number of orphan drugs approved has reached more than 650. This progress has been critical, but we still have a long way to go.
The 2017 Tax Hike Bill also gutted our State and Local Tax deduction and sent us in the wrong direction — cutting the Orphan Drug Research Tax Credit in half — making treatment for rare diseases even rarer. Yes, I was shocked, too. Thanks to the Orphan Tax Credit, and our desire to win, individuals with mesothelioma, multiple sclerosis, cystic fibrosis, and bone marrow disorders have benefitted from discoveries in labs across New Jersey and our great country.
But with this credit cut in half back in 2017, we’re not where we should be, and we are putting at risk future progress in our fight against rare diseases. I’ve heard this first-hand when I’ve sat down with many of my constituents who face debilitating and life-threatening rare diseases. Personally, I lost my mom to a rare disease, Sarcoidosis, four and a half years ago. May her memory be a blessing.
Today, I’m grateful to be joined by Rob Hyman, whose 8-year-old daughter, Cameron Hyman, has a rare disease called Sanfilippo Syndrome, which affects the central nervous system — specifically the brain and spinal cord. Right now, there are no FDA- approved treatments for Sanfilippo Syndrome. It’s one of the reasons I’m here today – because we need to change that.
Cameron’s family launched the CAM Foundation, to raise funds to help invest in trials and to find a cure for this degenerative disease. Cameron is far from alone in the fight for cures for rare diseases. There are nearly 30 million people in our country who have been diagnosed with a rare disease, meaning none of us are very far from these diseases — or very far from seeing all the work that we still need to do to make sure more cures are found.
Today, along with Republican and fellow Problem Solver, Congressman Don Bacon from Nebraska, I will be formally introducing new bipartisan legislation — Cameron’s Law — which will fully restore the previous slashing of the Orphan Disease Research Tax Credit. We will invest and restore the credit to its original levels and give our best and brightest — many of them right here in Jersey — a fighting shot to find effective treatments for rare diseases and, eventually, find a cure to the most ruthless diseases that plague our families, loved ones, and neighbors. These drugs are often then used to cure other diseases, too.
This tax credit will give families like Cameron’s 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.
While important research and development for COVID-19 vaccines and treatments soared, other rare disease clinical research unfortunately dwindled. The pandemic also added increased costs, delays, and uncertainty to clinical trials for rare diseases. This halted the collection of valuable data from the experiences of thousands of trial participants and drastically delayed the path of many critical treatments getting to market and to patients who need them.
Much of this clinical research is still stalled, while millions of patients are waiting for breakthrough treatments and cures for the diseases that threaten their lives. This is a particular issue for rare diseases, which currently have no FDA-approved treatments, and trials are more difficult to conduct.
In May 2020, the National Institutes of Health (NIH) then-Director estimated that $10 billion of research would be lost as a result of the pandemic. Given the tumultuous two years that have followed, that estimated loss of investment could grow.
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.
That’s why, with Congressman Bacon, I’m also introducing the bipartisan Rare Disease Clinical Trial Pandemic Disruption Act. This bipartisan bill will help get lifesaving clinical research back on track, after work toward COVID-19 vaccines and other pandemic-driven interruptions disrupted research and development for rare disease cures.
The bill will incentivize clinical research that was halted by the pandemic. It will allow 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.
It will help in our efforts to find cures for rare diseases that affect far too many by incentivizing companies to resume clinical research, and help get life saving research and trials back on track as they were before the industry was hit with pandemic-driven interruptions.
Finally, I’m also incredibly proud to announce today that, by working closely with Valley Hospital, we successfully clawed back more than $600,000 to help enhance their AUGi Project — a next-generation AI technology that will be used to reduce patient falls and keep people safe.
This monitoring technology can help identify patient movements that may suggest their exiting the bed or getting up from a chair and will alert hospital staff or caregivers to their device.
What’s great is that this technology allows for monitoring, but without streaming video to protect a patient’s privacy. Essentially, it creates a digital outline which looks like a stick figure over the person’s blurred image. It can even detect if a patient is getting out of bed before a traditional bed alarm can.
This incredible technology is an extra pair of eyes for our health care workers and it’s an extra layer of protection for our families. The hospital’s pilot in the neurology unit has seen a 28 percent reduction of falls with injury — an amazing feat.
This investment will be used to ensure that this state-of-the-art technology is included in every medical, surgical, critical care, and emergency department patient room at Valley Hospital currently under construction.
I know this new technology is bringing peace of mind to so many North Jersey families who have loved ones here at Valley.
Today, even with the challenges we are facing, I hope you are filled with hope. I know that I am. In the greatest country in the world, if we can come together to solve problems and give everyone a fighting chance, I know that our best days will always be ahead of us. Once again, thank you for joining me here today.
Thank you for everything you all do, God bless you, and may God continue to bless and watch over brave families like the Hymans and our great country.
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