TCNJ

24 The College of New Jersey Magazine The nation's great hope for treating rare disease GAYATRI RAO in the seven-year medical program administered by TCNJ and Rutgers New Jersey Medical School. "It was a marvelous experience," says Rao, who credits the program with sparking her desire to use government policy and regulation to improve the quality of people's lives on the broadest scale possible. Because most rare diseases are genetic, they tend to appear in childhood, often with debilitating or deadly consequences. Rao later picked up a JD and a master's degree in bioethics at the University of Pennsylvania. She worked for a time on healthcare and food and drug issues at an international law firm before moving to the FDA, which oered her the perfect marriage of law, policy, and public health. The stakes are high: Because most rare diseases are genetic, they tend to appear in childhood, often with debilitating or deadly consequences. Patricia Weltin, founder and CEO of the Rare Disease United Foundation and the mother of two daughters with a life-threatening rare disease known as Ehlers-Danlos syndrome, says that being the parent of a child with such an ailment can be harrowing. Yet she has nothing but praise for Rao's open and responsive attitude toward patient advocates like herself. "I have a direct line to her," says Weltin, adding that Rao invited her to bring an exhibition of paint- ings of children with rare diseases to the FDA earlier this year. Titled "Beyond the Diagnosis," the project is intended to raise public awareness of rare diseases. And Claudia Morris, a professor of pediatrics and emergency medicine at Emory University, says the support she's gotten from the oce has been critical to her research. Morris received $1.6 million from the OOPD in 2015 to run a five-year clinical trial of a promising new treatment for sickle cell anemia — a rare blood disease that strikes in infancy, causing organ damage, premature death, and excruciating pain. "I'm very grateful for what they do," Morris says. Rao, meanwhile, is grateful for the opportunity to improve the lives of millions of people. But she also knows that much work remains to be done. Says Rao, "We get many more amazing studies than we can fund." DUCHENNE MUSCULAR DYSTROPHY. Amyotrophic lateral sclerosis. Muscular spinal atrophy. All are considered rare diseases. And as such, you might reasonably assume they aect small numbers of people. But as head of the federal organization charged with promoting the development of new drugs and medical devices for treating such diseas- es, Gayatri Rao '99 knows better. Rare diseases, she explains, are so labeled if they aict fewer than 200,000 people in the United States. But because there are so many rare diseases — approximately 7,000 — combined they aect 30 million Americans. Yet there are typically few, if any, treatments for them. As director of the Oce of Orphan Products Development at the Food and Drug Administration, Rao is helping to change that. Orphan products are ones that pharmaceutical companies and device manufacturers won't develop because the market for them is too small. Congress enacted the Orphan Drug Act in 1983 to encourage the development of such drugs through financial incentives like tax breaks, fee waivers, and market exclusivity — incentives that Rao administers. From a sprawling government campus in a wooded suburb just north of Washington, D.C., she also distributes grant funding to help drive research and encourage de- velopment of new drugs and devices, pumping $15 million in grants each year into clinical trials alone. Thanks to those eorts — and, says Rao, to advances in medical science and support from patient advocacy groups — applications to the OOPD by companies seeking the incentives created by the Orphan Drug Act have jumped by double digits in recent years, and there are now more than 600 FDA-approved drugs for rare diseases on the market. Rao first became interested in the intersection between medicine and public policy while enrolled

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