Nearly 300 Medicines in Development to Meet Unique Needs of Children

Nearly 300 Medicines in Development to Meet Unique Needs of Children

Washington, D.C. (January 12, 2012)--America’s biopharmaceutical companies are researching 282 medicines to help meet the unique health care needs of children and adolescents, according to a new report released today by the Pharmaceutical Research and Manufacturers of America (PhRMA).  The medicines are currently in clinical trials or under review by the Food and Drug Administration (FDA).

Considerable progress in the fight against diseases that impact children has been achieved in recent decades, with medicines playing a key role. Infant mortality has sunk to record lows. New vaccines protect children against many childhood diseases. Antibiotics prevent deaths from pneumonia and other infectious diseases. Thanks in part to major treatment advances, 82 percent of children diagnosed with cancer will survive five years or longer, compared to 58 percent three decades ago, according to the American Cancer Society.

Biopharmaceutical research companies are working to continue this progress and to meet new health challenges specific to children. The nearly 300 medicines in development listed in the new report reflect these ongoing efforts. These include:

• 54 for cancer which, despite significant progress, is still the leading cause of death by disease among American children.

• 49 for infectious diseases, resulting in more than 164 million missed school days annually in American public schools due to the spread of infectious diseases.

• 48 for genetic disorders, including medicines for cystic fibrosis, which affects 30,000 American children and adults.

• 25 for neurologic disorders, including medicines for epilepsy, which affects more than 300,000 school children under age 14 in the United States.

“These medicines offer hope that the significant improvements achieved in children’s health over the past few decades will continue and even accelerate,” said PhRMA President and CEO John J. Castellani. “New medicines and new knowledge about pediatric use of existing medicines will help give our children and grandchildren the opportunity they deserve to live long, healthy lives. Nothing is more important or gratifying.”

In addition to creating new medicines specifically for children, biopharmaceutical research companies are testing many existing medicines to determine safe and effective dosage levels for children. In 2007, Congress reauthorized both the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA), highly successful programs that generate new knowledge about medicines for use in children. PREA and BPCA are set to expire on October 1, 2012 unless reauthorized or made permanent by Congress. 

BPCA and PREA have led to hundreds of pediatric studies covering more than 16 broad categories of diseases that affect children.  The research conducted as a result of BPCA and PREA has led to nearly 425 pediatric labeling changes since 1998, according to the FDA.

“Reauthorization of these pivotal programs is essential to continued progress in children’s health,” said Castellani. “Permanent reauthorization would provide much-needed predictability and certainty, to the benefit of regulators, innovative companies and, ultimately, children.”


The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading pharmaceutical research and biotechnology companies, which are devoted to inventing medicines that allow patients to live longer, healthier, and more productive lives. PhRMA companies are leading the way in the search for new cures. PhRMA members alone invested an estimated $49.4 billion in 2010 in discovering and developing new medicines. Industry-wide research and investment reached a record $67.4 billion in 2010.

Contact: Preet Bilinski                                                     

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