Sen. Brown Introduces Legislation to Spur Private-Sector Innovation Aimed at Treating Rare and Neglected Pediatric Diseases

Bipartisan Bill Creates Incentives for Companies to Develop Treatments for Rare, Devastating Illnesses

WASHINGTON, D.C. - U.S. Sen. Sherrod Brown (D-OH) this week introduced bipartisan legislation, the Creating Hope Act of 2010, to spur private-sector innovation aimed at treating rare and neglected pediatric diseases. The legislation builds on existing law to increase incentives for the development of treatments for disabling and deadly diseases, with a focus on rare conditions that may otherwise fail to attract sufficient research and development funding.

"We are falling woefully and inadequately short in our efforts to cure and treat rare and neglected pediatric diseases and conditions," said Brown. "The Creating Hope Act is aimed at doing exactly that-creating hope-for the millions of American children suffering from rare and pediatric diseases by increasing the incentives for pharmaceutical companies to expand research and development into these devastating illnesses."

Last month, Brown called for-and served as co-chairman, alongside Chairman Tom Harkin (D-IA) -a hearing of the Senate Health, Education, Labor, and Pensions (HELP) Committee entitled "Treating Rare and Neglected Pediatric Diseases: Promoting Development of New Treatments and Cures."

The need for new therapies to prevent and treat rare and neglected diseases is enormous. The National Institutes of Health (NIH) estimates that there are more than 6,000 rare diseases as defined by the Orphan Drug Act. Others, such as tuberculosis, malaria, and dengue fever are neglected because they affect impoverished populations in developing countries. Yet, of these diseases, fewer than 300 are of interest to the pharmaceutical industry. Because rare and neglected diseases cannot guarantee the same return on investment as the more common diseases that affect larger and often wealthier populations, there can be little incentive for these companies to invest in the research and development required to create new therapies.

The Creating Hope Act, introduced with Senators Sam Brownback (R-KS) and Al Franken (D-MN), amends provisions of the Food and Drug Administration (FDA) Amendments Act, which was signed into law in 2007. These provisions established an incentive for pharmaceutical companies to develop innovative therapeutics for neglected tropical diseases. Under this law, companies that develop new drugs and biologics for neglected tropical diseases are eligible for a "priority review voucher" entitling them to expedited review of another drug produced by that manufacturer.   Because this voucher can be used to expedite the marketing of a "blockbuster" or "me-too" drug, it provides a strong financial incentive for the development of treatments for otherwise neglected diseases. 

The Creating Hope Act improves upon this important incentive not only by increasing the commercial value of the priority review voucher by making it transferable, but by expanding priority review voucher eligibility to include rare pediatric diseases.

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