This blog was originally published in February and updated on July 10, 2017.
TODAY is the day to reach out to your Members of Congress about the RACE for Children Act. This legislation is included in a larger FDA User Fee bill that is expected to be voted on Wednesday.
Here's why the RACE Act is so important:
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 based on the Research to Accelerate Cures and Equity for Children (RACE) Act was introduced this year by Senators Bennet (D-CO) and Rubio (R-FL) and by Reps. McCaul (R-TX) and Butterfield (D-NC) that would eliminate those exemptions and improve opportunities for more studies in childhood cancer. Today, we ask you to please urge Members of Congress to pass the RACE Act as part of FDA user fee legislation currently under consideration.
This legislation would:
- Require that companies developing cancer drugs do PREA studies of their drugs in children when the molecular target of their drug is relevant to a children's cancer; and
- End the exemption of PREA obligations for cancer drugs with orphan designations if the molecular target of their drug is relevant to a children's cancer.