WASHINGTON, D.C. — To recognize Rare Disease Week, U.S. Congressman Josh Gottheimer (NJ-5) spoke at The Hill’s COVID-19 & Rare Disease Patients event to discuss what can be done to help us invest more in research and development for cures for orphan or rare diseases, which currently have no FDA-approved treatments.

Gottheimer highlighted his bipartisan legislation — Cameron’s Law — 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. 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.

“As we recognize Rare Disease Week, I’m continuing the fight to pass Cameron’s Law — bipartisan legislation I’ve authored to fully restore the Orphan Drug Tax Credit and to develop new tools to fight against rare diseases that affect far too many in our great state and country,” said Congressman Josh Gottheimer (NJ-5). “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. Cameron’s Law will allow us to give our best and brightest a fighting shot to find effective treatments for rare diseases and, eventually, find a cure to the most ruthless diseases that plague our loved ones.”

This bipartisan legislation is named after 7-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. Cameron’s Law is being cosponsored by Congressman Fred Upton (MI-6), a Republican and member of the Problem Solvers Caucus.

Gottheimer was joined at The Hill’s virtual event by U.S. Congressman Brad Wenstrup (OH-2) and representatives from EveryLife Foundation for Rare Diseases, Prader-Willi Syndrome Association, USA, and Boston Children’s Hospital. 

The panel explored what is at stake for the nearly 30 million Americans with rare diseases and the critical scientific research landscape they rely on.

Watch the Rare Disease Week event here.

Earlier this Congress, Gottheimer joined with Representatives Chrissy Houlahan (PA-6), Stephanie Murphy (FL-7), and Eric Swalwell (CA-15) to fight to restore the Orphan Drug Tax Credit (ODTC) within negotiations of the reconciliation package. 

The Members wrote in a letter to Speaker Nancy Pelosi and Chairman of the House Ways and Means Committee Richard Neal, saying, “We urge you to preserve the [Orphan Drug Tax Credit] in its current form as the Build Back Better reconciliation package is negotiated and finalized in the coming days. By maintaining the ODTC, we can continue to incentivize life-saving research and find treatments and cures for millions suffering from some of society’s most vexing diseases.” The full letter can be found here.

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 United States. By 2014, through 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 14 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.

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