NIH to Publish Updated List of Priority Therapeutic Areas for Pediatric Research
The National Institutes of Health (NIH) is preparing to release a new report detailing the therapeutic needs of pediatric patients under the Best Pharmaceuticals for Children Act (BPCA), a piece of legislation aimed at promoting the safe prescribing of medicines for children.
The 2002 law, similar to the Pediatric Research Equity Act (PREA), allows FDA to work with sponsors of certain therapeutic products to conduct postmarketing studies involving children in return for additional months of marketing exclusivity. Many sponsors decline or are unable to conduct pediatric studies, either due to lack of interest, ethical or logistical concerns, insufficient enrollment or market-based concerns.
Because relatively few pediatric clinical trials take place, most drugs used in children are prescribed off-label and without the backing of the same rigorous studies of its on-label indications, creating the potential for unknown adverse events to occur in children. "All of these factors contribute to the lack of adequately collected pharmacokinetic, pharmacodynamic, safety, and efficacy data in children and can increase a child's risk for unknown and/or adverse effects," explained NIH in a Federal Register posting on 25 October.
In light of this gap between the needs of pediatric patients to have accurate data to inform prescribing habits and the behavior of the market, the BPCA also directs NIH's National Institute of Child Health and Human Development to compile a list known as the Priority List of Needs of Pediatric Therapeutics (PLNPT) at least once every three years. The most recent update to the list occurred in 2011.
The PLNPT authorizes additional incentives for sponsors of on-patent drugs that meet various criteria to sponsor new clinical trials and submit the resulting data to FDA for labeling changes meant to inform pediatric prescribing.
NIH said the criteria to be a part of the list is based upon six general factors: the availability of information regarding safe and effective pediatric prescribing, the potential health benefits for children, the need for reformulation of products, therapeutic gaps in pediatric pharmaceuticals, pediatric-specific diseases without adequate treatment, and the suitability of infrastructure to conduct pediatric testing.
The PLNPT is scheduled to be posted online on 26 October 2012 on NIH's BPCA website, said NIH in response to an inquiry by Regulatory Focus.