PhRMA calls for regulatory environment to support rare disease research

US biopharmaceutical research companies are developing 452 new medicines for rare diseases, including genetic disorders, neurological conditions, infectious diseases and autoimmune disorders, according to a new report released by the Pharmaceutical Research and Manufacturers of America (PhRMA).

Scientific advances, and a deeper understanding of the human genome, have given researchers new tools to explore rare diseases, which are often more complex than common diseases. According to the US Food and Drug Administration, in the last five years, one third of all new drug approvals were for rare diseases.

PhRMA president and chief executive John Castellani said: “Biopharmaceutical scientists are better equipped than ever before to bring new treatments – and hope – to patients battling one of the nearly 7,000 rare diseases. In order to capitalize on this scientific promise, we need to ensure that we have a policy and regulatory environment that helps foster this vital work.”