A congressional advisory commission has concluded that promising rare disease therapies are failing to reach American patients because of gaps in funding, data, regulation and manufacturing, and that the country's long-held lead in the field is eroding.
The National Security Commission on Emerging Biotechnology, a bipartisan legislative advisory body chaired by Sen. Todd Young of Indiana, released a white paper identifying four main hurdles and recommending an all-of-government approach to rare disease. Vice Chair Michelle Rozo said in a statement that emerging biotechnology now makes it possible to cure rare diseases rather than manage symptoms for life, and that what the country lacks is a national strategy to channel that innovation.
