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New Bill Would Add More Rare Disease Experts and Patients to FDA Drug Review Process
What happened: Two women are working to change how the FDA reviews drugs for rare diseases. Lindsey Sutton — a Californian who lives familial chylomicronemia syndrome, or FCS — and Melissa Goetz, whose daughter was diagnosed with FCS, helped write the HEART Act after learning the Food and Drug Administration (FDA) does not require rare disease specialists or patients to sit in or serve on clinical trial committees to develop new treatments.
