Above: Gottheimer announces new bipartisan legislation — Cameron’s Law — to boost investment in finding cures for diseases with no FDA-approved treatment, known as ‘orphan diseases.’
On Friday, March 6, 2020, U.S. Congressman Josh Gottheimer announced new bipartisan legislation — Cameron’s Law — which will fully restore the Orphan Disease Research Tax Credit, following the 2017 Tax Hike Bill cutting the Tax Credit in half and making treatment for rare diseases even rarer. 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 and grow the talent pipeline for life sciences jobs in New Jersey through training partnerships and apprenticeships.
This bipartisan legislation, cosponsored by U.S. Congressman Fred Upton (MI-6), is named after five-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.
“Here in New Jersey, we have some of the best hospitals and medical facilities in the country, the best doctors, medical school graduates, nurses, and life sciences leaders — and we need to keep growing that talent pipeline so that we can come together to cure and treat rare diseases, some known as orphan diseases — those that often don’t get the attention and investment they need and deserve,” said Congressman Josh Gottheimer (NJ-5). “With Cameron’s Law, we will invest and restore the Orphan Drug Research Tax 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.”
In 1983, the Orphan Drug Act passed with broad bipartisan support and was signed into law by then-President Ronald Reagan. Prior to the 1983 bill, only 38 orphan drugs for rare diseases had been approved for use in the U.S. By 2014, with the help of the Orphan Drug Research Tax Credit and research incentives laid out in the Orphan Drug Act, 373 drugs for rare diseases had been approved.
Through the Orphan Drug Research Tax Credit, those with mesothelioma, multiple sclerosis, cystic fibrosis, and bone marrow disorders have benefitted from discoveries in labs across New Jersey and the U.S.
New Jersey is one of the leading centers in the world for life sciences and innovation, with 12 of the world’s top 20 research-based biopharmaceutical companies.
More than 372,000 New Jersey jobs are supported by the life sciences sector with $96 billion in total economic output annually. New Jersey has been ranked the number one state in the nation for biotech growth potential and the number two state for biotech strength.
Video of the announcement can be found HERE.
Gottheimer’s remarks as prepared for delivery are below.
We’re here to discuss how New Jersey can continue leading in the life sciences sector through expanded research and development, job creation, and steps we must take, including new legislation, to invest in cures for rare diseases.
It’s important that we come together to tackle diseases that are affecting so many families. And while it’s different from what has brought us together this morning, we’re also coming together as our community here in North Jersey is facing the threat of coronavirus.
We now know of two presumed positive cases of the virus in Bergen County, and one of our schools has shut down temporarily. I’m continuing to stay in close contact with hospital officials, health care providers, and our local and state leaders to ensure they have what they need to treat patients and to help make sure an outbreak is contained.
This week, back in Washington, the House and Senate both passed bipartisan emergency funding to immediately address the coronavirus outbreak and help keep North Jersey families safe, and the President signed this into law this morning.
Here in New Jersey, we have some of the best hospitals and medical facilities in the country, the best doctors, medical school graduates, nurses, and life sciences leaders — and just like we are coming together to combat coronavirus and keep our communities safe, we need to keep coming together to cure and treat rare diseases, some known as orphan diseases — those that often don’t get the attention and investment they need and deserve.
An orphan disease is a disease with no FDA-approved treatment. We are making progress on these fronts, but we have a long way to go. In total, the FDA approved 76 orphan indications last year — brand-new treatments for rare diseases and new discoveries from existing medications that can be used for other conditions. Researchers are taking drugs that are already approved for use and seeing if they can find a breakthrough for those with an orphan disease. Just last year, 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.
With 12 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 cancer treatment drug development, and second for heart and stroke drugs and development. In 2017, 50 percent of all new FDA approvals came from companies with a New Jersey footprint.
Just last year, 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.
In 2018, after more than a decade of trying, Amicus Therapeutics, also in New Jersey, won FDA approval for a drug treating Fabry disease, an inherited rare disorder which can lead to fatal organ damage.
Right here in Fair Lawn, Zimmer Biomet has been a leader in cutting-edge breakthrough technology for medical devices that lead people to live longer and healthier lives. Just last year, Zimmer Biomet won FDA approval for its first spinal tether device to treat scoliosis among young patients in particular.
We’re lucky to have so many of these cutting-edge research operations here in my District and across New Jersey. More than 372,000 New Jersey jobs are supported by the life sciences sector with $96 billion in total economic output annually. There is nowhere to go but up. New Jersey has been ranked the number one state in the nation for biotech growth potential and the number two state for biotech strength.
We may have some of the best facilities in the country here in the Garden State, but they’re facing obstacles. 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 labor costs and our high taxes. Now, I helped lead the charge to eliminate the federal Medical Device Tax this past December, but the cost of doing business in New Jersey has continued to rise.
Compounding these difficulties, during the past decade, New Jersey’s biopharma industry has experienced increasing competition from other states that have also been trying to raise their quotient of biopharma start-ups.
California and Massachusetts have, respectively, created ten and four times the number of companies compared with New Jersey. That’s 885 biopharma companies founded in California between 2012 and 2015, and 430 in Massachusetts — with only 89 in New Jersey. Medical device companies are also being outpaced by increased employment in states like Massachusetts, Indiana, and Minnesota.
Difficulties have also caused a decline in New Jersey’s biopharma employment — mainly in manufacturing. While R&D employment was flat from 2005 to 2015, manufacturing employment declined 3.6% per year. And both New Jersey’s R&D and manufacturing employment trailed most of our peer states.
Growing the talent pipeline in New Jersey is now more important than ever. Co-op programs, training agreements, apprenticeships, and partnerships with local schools — like those from Zimmer Biomet and NJMEP and others — are perfect opportunities to bounce back from this employment decline.
Known as “the medicine chest of the world,” New Jersey has been a major global hub for biopharma for more than a century.
Yet in recent years, New Jersey’s biopharma industry has not kept up with other states, particularly California and Massachusetts. There is no reason why we shouldn’t be getting more of these jobs, especially given our incredible workforce and the training and skills that our colleges and universities teach. Many of the life sciences companies operating here have recruitment and apprenticeship programs with Rutgers, NJIT, Stevens, and many others. We’re also seeing this growth at our local community colleges, including ten fully accredited programs for health professionals at Bergen Community College..
I want us to double down so that we expand in the years ahead – and continue leading the way in medicine, developing the next cures for orphan diseases, and developing cutting edge medical devices that allow people to walk again and live normal, healthy lives.
Each year, 22,000 students graduate with life sciences degrees from New Jersey’s 63 colleges, universities, and technical schools — many right here in my District – from lab techs to engineers. We need to harness this talent and create a chain of opportunity for them to succeed. This is good for not only the life sciences sector but also the manufacturing sector, which is why I’m glad NJ MEP is here with us today. Nearly 73,000 of New Jersey’s manufacturing jobs support the life sciences sector, so this is a critical partnership that we need to grow.
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.
It also presents a significant opportunity to cure rare diseases that affect our residents, including many families and children I’ve had the honor to meet and work with throughout my time in Congress. Last year, I stood with Sean O’Malley at Hackensack hospital, who beat the odds and is now in remission from pediatric cancer and living a normal health life.
Today, I’m very lucky to be joined by Rob Hyman, whose five-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 recently 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.
I heard this first-hand last week during Rare Disease Week, when I sat down with many of my constituents who face debilitating and life-threatening rare diseases. I lost my mom to a rare disease, Sarcoidosis, a year and a half ago.
In 1983, the Orphan Drug Act passed with broad bipartisan support and was signed into law by President Ronald Reagan. Before that, just thirty-eight orphan drugs for rare diseases had been approved for use in the United States. By 2014, partly as a result of the research incentives laid out in the Orphan Drug Act, 373 drugs for rare diseases had been approved. This progress has been critical, but we still have a long way to go.
The recent Tax Hike Bill that also gutted our State and Local Tax deduction, also sent us in another 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.
That is why today I am announcing that, next week, with Congressman Fred Upton from Michigan, 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.
Conversations like the one we’re having here today are incredibly important. Just like I’m fighting in Congress to make sure our New Jersey life sciences leaders have what they need to be able to find cures for rare diseases, we need to be fighting together here to make sure we’re growing the talent pipeline, growing jobs, and boosting R&D here in the Garden State.
I know, in the greatest country in the world, we can come together to solve problems and give everyone a fighting chance. 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.