New bipartisan legislation was introduced in the US Senate last week which seeks to encourage innovative R&D by drugmakers aimed at treating rare and neglected pediatric diseases.

The Creating Hope Act of 2010 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 R&D funding.

“Seven thousand known rare or orphan diseases afflict nearly 30 million Americans – approximately 50% of whom are children,” said Democrat Senator Sherrod Brown, who is co-sponsoring the bill with Republican Sam Brownback and Democrat Al Franken.

The Senators note that while the US National Institutes of Health (NIH) estimates that there are more than 6,000 rare diseases as defined by the Orphan Drug Act - and others such as tuberculosis, malaria, and dengue fever are neglected because they affect impoverished populations in developing countries - fewer than 300 of these diseases 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 R&D required to create new therapies, they add.

The Creating Hope Act amends provisions of 2007’s Food and Drug Administration (FDA) Amendments Act introduced by Sens Brown and Brownback which established an incentive for pharmaceutical companies to develop innovative therapeutics for neglected tropical diseases.

Under this law, companies which 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 proposed new legislation would improve upon this 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.

“Because of the unique set of circumstances that discourage innovation in this field, thinking outside the box becomes a necessity,” says Sen Brown.