Enzi: Innovation Fosters Success in Fight Against Rare and Neglected Pediatric Diseases

 Washington, D.C. – At today’s U.S. Senate Health, Education, Labor and Pensions (HELP) Committee hearing, “Treating Rare and Neglected Pediatric Diseases:  Promoting the Development of New Treatments and Cures,” Ranking Member, Senator Mike Enzi (R-Wyo.) emphasized the need to increase research and development of rare and neglected diseases, and called on Congress to ensure federal policies foster innovative treatments for rare pediatric diseases.

“Congress created the framework for successful partnerships among federal agencies and nongovernmental organizations to combat rare pediatric diseases.  Moving forward, we need to focus on identifying barriers that discourage development of new treatments and cures, and encourage incentives for investments in finding them,” Enzi said.

Rare diseases afflict nearly 30 million Americans. Most of those conditions have no treatment or cure, and companies are unlikely to undertake the expensive, lengthy and difficult research to develop a drug if they cannot recover their investment. 

“The Food and Drug Administration and the National Institutes of Health have taken on a wide range of initiatives that have sped up development of new drugs and resulted in the approval of over 300 products to treat rare and neglected diseases,” Enzi said.  “The Orphan Drug Act, the Food and Drug Administration Amendments Act, and the National Institutes of Health Reform Act all represent significant steps toward success in defeating rare diseases.”

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