The global market for pediatric medicines registers a compound annual growth rate of 3.8% and is expected to reach more than $100 billion by 2019, according to a 2014 BCC Research report.
That’s due to the industry designing and implementing more trials in the pediatric population, as well as an increased focus on orphan drug diseases affecting children, according to the report.
Also aiding in the development of pediatric medications are two laws: the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA), which were permanently reauthorized in 2012.
PREA requires sponsors of certain new drug applications to study their products in children. BPCA provides incentives for sponsors to voluntarily conduct studies in pediatrics by granting them an additional six months of market exclusivity. An update of PREA, the Research to Accelerate Cures and Equity (RACE) for Children Act, recently passed the U.S. Senate. This law contains explicit guidance for cancer drug development that could expand the number of pediatric cancer drug treatments.
New pediatric drugs
Several new medications have recently been approved for use in the pediatric population. Mort Goldman, PharmD, FCCP, BCPS, senior consultant, Pharmacy Consulting International, lists some of these new drugs and their anticipated costs:
- Brineura (cerliponase alfa). An enzyme replacement therapy indicated to slow loss of ambulation in pediatric patients with Batten disease. The annual cost per patient to treat this disease with Brineura could be 1.5 million.
- Bavencio (avelumab). Immunotherapy for the treatment of patients aged 12 years and older with metastatic Merkel cell carcinoma. Annual cost per patient may total up to $90,000.
- Emflaza (deflazacort). An oral corticosteroid indicated to treat patients aged 5 years and older with Duchenne muscular dystrophy. Annual cost per patient of up to $100,000.
- Defitelio (defibrotide sodium). An anticoagulant for the treatment of adults and children who develop hepatic veno-occlusive disease with additional abnormalities following hematopoietic stem cell transplantation. One 21-day treatment course may cost up to $85,000.