Don’t Leave Them Waiting: How to Bring New Cancer Drugs to Kids

My mom just got home from the hospital. She received excellent care, including round-the-clock infusions of antibiotics that saved her life. Years ago this infection would have killed her.

When kids with cancer are hospitalized, the care is excellent, too. The room may be painted ocean blue, with sea turtles and dolphins. The hospital staff will bring balloons and invite clowns. But the chemo drugs in their I.V. will be nothing to celebrate. Most kids with cancer are treated with medicines from the time when my mom was young.

In the next few weeks, Congress has an opportunity to bring new treatments to kids by passing the Research to Accelerate Cures and Equity (RACE) for Children Act. The RACE Act is needed because it takes years to approve drugs for kids after they are available for adults. Take Gleevec, a drug for a form of leukemia, that has been saving lives of adults since 2001, changing the 5-year survival from 50 percent to 95 percent. It took five years before it received approval for pediatric patients, and another 7 years to become approved for acute lymphoblastic leukemia, the most common type of pediatric cancer.

Very few drugs are studied in kids because it is not profitable to develop cancer drugs for kids. The pediatric oncology market is small. Right now, 295 studies are being conducted of a kind of cancer immunotherapy called PD-1/PD-L1 — only five of these studies are enrolling children.

Last July, the Food and Drug Administration said that testing drugs in pediatric patients is inadequate, and gave Congress suggestions for improving pediatric research. The RACE for Children Act implements these suggestions. It will not cost the taxpayers a penny.

The RACE Act will close a loophole. Under the current law, the Pediatric Research Equity Act, drug companies are required to study drugs in children when they are developing drugs for adults for the same disease. Since its enactment in 2003, there have been 363 PREA studies, but not a single one of these involved a cancer drug. This is because PREA only applies when diseases are exactly the same in kids and adults. For cancer, this means the cancer affects the same part of the body.

Of course, kids don’t get prostate or breast cancer. Advances in oncology, such as targeted therapies, showed that kids’ and adults’ cancers may be treated with the same drugs when they share the same genetic mutation. For instance, a mutation causing lung cancer is sometimes the same as in neuroblastoma, a childhood cancer. The RACE for Children Act asks companies to study drugs based on the genetic origin of cancer, not its location. When something does not work, shouldn’t it be fixed?

The RACE Act will also remove the “orphan” exemption that gives drug companies a waiver from studying new drugs in kids for drugs to treat orphan diseases (those that affect fewer than 200,000 Americans). Ninety percent of cancer drugs developed in the past five years received this exemption – and kids missed out on 90 percent of new discoveries.

The benefits of the proposed legislation are obvious: More studies equals more potential treatments. However, some drug companies are opposed to the extra burden of conducting pediatric cancer studies, despite the fact that they comply with PREA requirements for other diseases.

If the RACE for Children Act does not pass, here is what will happen: As in the past, there will be about 5 to 10 new cancer drugs approved annually for adults, and it will take 25 years to approve just three new drugs developed specifically for kids. Kids will be treated with old drugs or with medicines created for adults for which there have been no studies of the proper dose or effectiveness in children. The new targeted therapies will not be studied in kids at the same time they are studied in adults, and may never be studied. It is unethical to wait to study drugs that could help kids today.

Last month, hundreds of families and kids who have been touched by cancer came to the Capitol Hill to support the RACE Act. The RACE Act has bipartisan support, and has been endorsed by leading hospitals, research institutions, American Society of Clinical Oncology, Children’s Oncology Group, and over 250 advocacy organizations.

The passionate minds at pharmaceutical companies and academic centers have been searching for the causes of disease and have found many treatments. My mom was saved because of their efforts.

Kids with cancer need the same chance.  


Elena Gerasimov is Director of Programs at Kids v Cancer, a nonprofit pediatric cancer advocacy organization.

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