PhRMA report reveals nearly 600 drugs in US paediatric pipeline

According to a new report from the Pharmaceutical Research and Manufacturers of America (PhRMA), there are nearly 600 paediatric medicines currently in clinical development in the US. 

The research found that in the country, there are more than 2,100 industry-sponsored paediatric clinical trials underway, testing 580 investigational medicines and involving more than 1.2 million patients across a variety of therapeutic areas, including diseases where there is significant unmet medical need.

Medicines in development include:

• A gene-edited cell therapy that could potentially be a treatment for sickle cell disease
• A monoclonal antibody (mAb) approved to treat asthma in adults and children aged 12 years and older being tested in children aged six to 11
• The first DDP-4 inhibitor approved for adults with type 2 diabetes in the United States being tested in children aged 10 to 17. 

Other findings in the report show that researchers are testing medicines approved for use in adults to determine safe and effective dosage levels for children. Current investigations include:  

• 125 treatments for genetic diseases including medicines for cystic fibrosis
• 86 treatments for cancer 
• 75 medicines for infectious diseases such as HIV/AIDS, ear infections, pneumonia and hepatitis
• 55 medicines for skin disorders, including atopic dermatitis.

The research suggests that the Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA) work together to foster paediatric drug development in the US. These acts were permanently reauthorised in 2012 and create an approach that yields safety and efficacy information on the use of medicines in children and enables biopharmaceutical companies to continue to make significant investments in paediatric drug research, says the report. 

However, the report concludes that while America’s biopharmaceutical researchers have made progress to overcome the scientific challenges associated with paediatric drug development, more work remains to develop innovative treatment options for pediatric patients.