Rare Disease Report

Horizon Pharma Establishes First Clinical Care Team Endowment at CNRDI

FEBRUARY 26, 2018
Mathew Shanley
Horizon Pharma is breaking new ground and collaborating to support clinical care teams that dedicated themselves to rare disease patients.

This morning, Children’s National Health System and Horizon Pharma plc have announced the creation of the Horizon Pharma Clinical Care Endowment, the first of its kind at the Children’s National Rare Disease Institute (CNRDI). With the endowment, rare disease patients will be provided with both earlier intervention and more consistent treatment over the course of their lifetimes.

The endowment is made possible by a $3 million commitment over 6 years from Horizon Pharma USA. The financial contribution is the second multi-million-dollar gift received by the CNRDI in 2018, and comes just one month after Retrophin, Inc. donated the same amount to assist in the creation of the Retrophin Rare Disease Network at Children’s National.

Each expert care team within the Horizon Pharma Clinical Care Endowment will consist of a clinical geneticist and support team members, including: genetic counselors, nutritionists and social workers, all of whom specialize in the care of children with rare diseases.

“Patients and families with rare conditions deserve a medical home – a trusted place with the medical knowledge to provide quick, clear answers and the expert care they need,” says Marshall Summar, MD, director of the CNRDI.  “We are grateful for Horizon and their support of our mission to make the Children’s National Rare Disease Institute that home. This endowment will support a dedicated team that can provide optimal, comprehensive care to more patients and ensure that families have a trusted source for all aspects of their health care.”

In January, Rare Disease Report sat down with Dr Summar. Throughout the interview, he stressed why contributions like the one made by Horizon Pharma are so exciting, but also vital, for members of the rare disease community. He hinted that he anticipated more companies to follow the example set by Retrophin.

“We are starting to talk to some other companies who are very interested in doing something similar,” he said. “This has caused a lot of buzz across the board, particularly for those of us in the medical field. Obviously, Retrophin gets a huge pat on the back for being the first one in, but we hope they’re not the last.”

Horizon’s commitment marks the first donor-funded endowment at the CNRDI, which is focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases.

“The Children’s National Rare Disease Institute is an important step in better defining the journey from diagnosis to treatment for people living with rare disease and their families,” said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. “We consistently hear stories from people who spent many years in search of a diagnosis, followed by more years searching for the right team of specialists – suffering through a painful journey with devastating consequences. Our hope is that the Horizon Pharma Clinical Care Endowment and the Institute will disrupt this all-too-familiar rare disease trajectory by joining exceptional rare disease specialists together in one place.”

According to the NIH, rare diseases are conditions that affect fewer than 200,000 people in the United States. Of the 7,000 rare diseases known to exist, fewer than 500 (approximately 5%) have a known FDA-approved treatment.

This Wednesday is World Rare Disease Day. For updates on events that are taking place to observe and commemorate the event around the world, follow Rare Disease Report on Facebook and Twitter.

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