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Science Translational Medicine: 'Creating Hope Act' incentivizes pediatric drug R&D
Date:1/19/2011

Washington, DC Recent legislative and regulatory actions make great strides toward establishing much needed incentives for pharmaceutical companies and others to develop and test more medications for pediatric rare diseases, including pediatric cancers, according to commentary by experts from Children's National Medical Center. The commentary appears in the January 19 issue of Science Translational Medicine.

"Pediatricians who treat children with serious and life-threatening diseases often find themselves face to face with the inadequacies of pediatric drug development," write Edward Connor, MD, director of the Office of Innovation Development and Investigational Therapeutics at Children's National Medical Center, and Pablo Cure, MD, MPH, Laboratory for Entrepreneurial Achievement in Pediatrics (LEAP) Scholar in the Office of Innovation Development and Investigational Therapeutics.

In a previous commentary for this journal, Dr. Connor noted that despite the fact that children make up nearly 40 percent of the world's population, little research is done to test the effects of "adult therapies" on children, who have unique needs requiring tailored care more sophisticated than merely smaller doses of adult therapeutics. The demand for pediatric drugs, especially those for rare diseases like childhood cancer, is relatively low compared to the demand for adult drugs, as fewer children develop rare and serious illnesses. To maximize efficacy and return on investment, pharmaceutical developers sometimes focus their efforts on therapeutics that address illnesses common to larger populations.

Pediatric specialists frequently have to prescribe therapeutics for children "off-label" because many common drugs and devices for treating diseases are only tested in adults but are necessary for the treatment of children. Resear
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Contact: Jennifer Stinebiser
jstinebi@cnmc.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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