My daughter Megan and I were honored to attend as special guests of President Trump for his address two weeks ago to a Joint Session of Congress. We are grateful beyond words for his commitment to accelerating and increasing the number of new medicines to treat the millions of people around the world suffering from rare genetic diseases. It was also a public affirmation of President Trump’s promise that he would appoint an FDA commissioner who would reform the “burdensome approval process at the Food and Drug Administration [that] keeps too many advances, like the one that saved Megan’s life, from reaching those in need.” The medicine that saved Megan’s life was approved in 2006 in an expedited pathway. It was a good example as the President noted of the system working in the best interests of patients. Too many new medicines today for too many diseases though, especially rare diseases, are not reaching patients as fast as necessary.

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