US biopharmaceutical companies are currently researching 282 medicines to help meet the unique health care needs of children and adolescents, according to new industry data.
The 282 potential new products, which are all either now in clinical trials or under review by the Food and Drug Administration (FDA), include:
– 54 potential new treatments for cancer, which is still the leading cause of death by disease among US children;
– 49 for infectious diseases, which result in more than 164 million missed schooldays annually in US public schools because of the spread of infectious diseases;
– 48 for genetic disorders, including medicines for cystic fibrosis, which affects 30,000 American children and adults; and
– 25 for neurological disorders, including medicines for epilepsy, which affects more than 300,000 US children aged under 14.
Potential new drugs for the treatment of children with arthritis, diabetes, eye problems, gastrointestinal disorders, psychiatric problems, respiratory disorders and skin conditions are also currently in R&D, according to the report, which is published by the Pharmaceutical Research and Manufacturers of America (PhRMA).
Moreover, in addition to creating new medicines specifically for children, biopharmaceutical research companies are testing many existing treatments to determine safe and effective dosage levels for children, it adds.
In 2007, the US Congress reauthorised both the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA), which the industry group notes have been highly successful at generating new knowledge about medicines for use in children. They have led to hundreds of paediatric studies covering more than 16 broad categories of diseases that affect children, and the FDA has reported that the research conducted as a result of these two Acts has led to 424 paediatric labelling changes since 1998.
Both PREA and BPCA are due to expire on October 1 this year, unless they are reauthorised or made permanent by Congress, which PhRMA is calling for.
“Reauthorisation of these pivotal programmes is essential to continued progress in children’s health,” said PhRMA chief executive John J Castellani. “Permanent reauthorisation would provide much-needed predictability and certainty, to the benefit of regulators, innovative companies and, ultimately, children.”
– PREA requires drug sponsors to conduct paediatric studies for certain products unless the FDA grants a waiver or deferral, while BPCA is voluntary for sponsors.
In a report issued last summer, the US Government Accountability Office (GAO) said that at least 130 products – 80 under PREA and 50 under BPCA – had been studied for use in children since the Acts were last reauthorised in 2007. However, it added that the FDA could not be certain how many additional products might have been studied because it does not track applications submitted under PREA.
Under BPCA, the FDA had granted six months’ paediatric exclusivity to the sponsors of 44 of the 50 drugs in exchange for conducting paediatric studies, the GAO reported, but it also pointed out that, because BPCA is voluntary, sponsors may decline FDA requests for such studies.
