The small population of children with cancer provides little market incentive for the biopharmaceutical industry to develop new pediatric oncology drugs. Children with cancer are currently treated with drugs that were developed several decades ago for adults, and research documents that they can be very damaging to their physical and intellectual development. Childhood cancer remains the leading cause of disease-related death in our children, and new and better therapies are sorely needed for children battling cancer.
Responding to this problem, Congress passed the Best Pharmaceuticals for Children Act (BPCA) in 2002 and the Pediatric Research Equity Act (PREA) in 2003 which provide critically important information on the safe and effective use of medications in the pediatric population, advancing the health of children. While BPCA and PREA have yielded important new safety and labeling information for other children’s diseases, the laws have had a very modest impact on childhood cancer.
The two laws act in tandem as both carrot and stick to encourage new drug development for childhood diseases. BPCA provides an incentive of six months of exclusivity – or patent extension – for drugs that are approved for use in children. PREA, the “stick” portion of the approach, requires pharmaceutical companies who are developing a drug for adult indications also test the drug in children. Two exceptions to this requirement constrain their impact for children with cancer. First, pediatric studies of a drug can only be required in the same disease (“indication”) for which it is being studied in adults. Since children do not develop lung, breast or prostate cancer, for example, drugs under development for adult cancers do not have to be tested in kids. A second exemption from the PREA requirement applies to any drug being developed for a “rare” disease. Rare diseases are defined in US law as those that are diagnosed in fewer than 200,000 people per year in the US. Unfortunately, most cancers meet the definition of a rare disease. These exceptions have resulted in PREA having virtually no effect in stimulating more treatments for childhood cancer.
That may be about to change, however. Legislation titled the Research to Accelerate Cures and Equity for Children (RACE) Act has just been introduced by Senators Bennet (D-CO) and Rubio (R-FL) and by Reps. McCaul (R-TX) and Speier (C-CA) that would eliminate those exemptions and improve opportunities for more studies in childhood cancer. Furthermore, the legislation would determine whether childhood cancer studies are warranted based on the “method of action” or affected biomarkers, rather than the site of the cancer; which is now the basis of most cancer drug development.
With a legislative opportunity coming up in the next year through a larger ‘must pass’ bill, the Children’s Cause is encouraging childhood cancer advocates to urge their senators and representatives to cosponsor this legislation. Click the button below to use our form and send a message to your Members of Congress asking for their support!