By Dr. Alan Goldhammer, Deputy Director for Regulatory Affairs, Pharmaceutical Research Manufacturers of America
First, it’s important to know that “rare diseases
Currently, there are 303 rare disease medicines in development -- 139 of which are aimed at treating rare forms of cancer, such as solid tumors of the liver and thyroid, cancer of the blood and melanoma. There are 16 drugs in development for autoimmune disorders, 28 for infectious diseases, 35 for neurological disorders and many more.
It's because of medical innovations like these that I'm here today as a cancer survivor. I owe my life to my faith, the love and support of my family, and to the miracle medicines made by the men and women of America's research-based pharmaceutical companies. Advancements in care take decades and billions of dollars to research and develop. Policymakers at all levels of government should keep patients in mind when developing health policy and always ask themselves, “Will this help or hinder the search for future cures?