Under the FDA Safety and Innovation Act, manufacturers who get a drug for a rare pediatric disease approved and on the market earn a voucher requiring the FDA to review a second drug within 6 months of submission of an application for its approval.
The U.S. Food and Drug Administration (FDA) reauthorization bill signed into law by President Obama in July adds provisions intended to bolster drug development for rare childhood diseases such as cancers.
Known as the FDA Safety and Innovation Act, the bill establishes a more stable business environment for drug developers by permanently authorizing the Best Pharmaceutics for Children Act and the Pediatric Research Equity Act.
Unfortunately, those 2 acts have given little economic incentive to develop drugs that are specific for rare pediatric diseases, contributing to a long-standing gap for childhood therapeutics, including those for cancer. “The gap is growing as our knowledge of molecular mechanisms increases, and we are learning that diseases require specific pediatric therapies, which one can't simply move directly from the adult to the pediatric realm,” comments Peter Adamson, MD, chair of the Children's Oncology Group and chief of clinical pharmacology and therapeutics at the Children's Hospital of Philadelphia.
The FDA bill tackles this lack of incentives head-on in an unusual way: Manufacturers who get a drug for a rare pediatric disease approved and on the market earn a voucher requiring the FDA to review a second drug within 6 months of submission of an application for its approval. Proponents say the vouchers will be significant assets for companies and will act as major incentives to create pediatric drugs.
However, the act does not incorporate proposals to extend the existing legal requirements by mandating consideration of appropriate pediatric testing of molecularly targeted oncology drugs approved for adult use that may be effective against other types of cancer in children.
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