Currently, treatments exist for only 200 of the approximately 7,000 rare diseases, defined as illnesses that affect fewer than 200,000 people. Combined, however, rare diseases affect more than 20 million Americans, according to the report.
The International Rare Diseases Research Consortium is advocating for at least 200 more treatments by 2020. In the fall the National Institutes of Health will open a center to speed genetic discoveries into usable therapies. In addition, the Senate recently introduced the Creating Hope Act, which would provide drug makers a voucher for fast FDA evaluation of their next blockbuster drug if they develop a therapy for a rare or neglected disease that disproportionately affects children.
The Food and Drug Administration is encouraging manufacturers to “repurpose” old drugs for rare diseases. Conversely, NIH Director Francis Collins, MD, PhD, says rare disease drug development may aid development of drugs for common diseases.
Read the Washington Examiner report on the push for more drugs for rare diseases.
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