The Hill - The Race to Fight Childhood Cancer

Op-Ed

By George Butterfield, Markwayne Mullin, and Michael McCaul

Receiving a cancer diagnosis is scary and life changing. Even more devastating are the tragic instances of cancer in our nation's children. Over 15,000 children are diagnosed with some form of cancer each year. Pediatric cancer is the number one disease killer of children. Together, working across the aisle to put children first, we set out with an aim to help children battling cancer celebrate a long life full of birthdays.

This week, the House will take up legislation to help in our fight against childhood cancer. Specifically, by passing our bill, the Research to Accelerate Cures and Equity - or "RACE" -- for Children Act, new treatment options will be available to those 15,000 children diagnosed this year with pediatric cancer.

This critical piece of legislation fits within the broader themes of the Food and Drug Administration's (FDA) Reauthorization package -- aligning statute with current medical practices. RACE updates the 2003 PREA law -- the Pediatric Research Equity Act, which requires studies of adult drugs in children during the drug development process.

While PREA has been successful in hundreds of cases in providing critical information on a drug's use in children, it has never been applied to a pediatric cancer drug, despite the need being greatest for pediatric cancer patients who are currently treated with "off-label" adult cancer drugs. This came about due to how the current PREA law was written, providing broad exemptions for conducting studies on drugs that affected pediatric cancer patients.

For example, if children were not afflicted with the condition the drug was targeting in adults, or the drug had received "orphan" designation, then drug sponsors were given an automatic "waiver" from completing these studies. When PREA became law nearly 15 years ago, these exemptions made sense; children by and large are not afflicted with the same type of cancers as adults (i.e. children typically do not get breast, prostate, lung, or other common types of adult cancers). Now, however, in 2017, oncology drugs are no longer manufactured to target the specific region of the body where the cancer appears. Rather, they are produced by "molecular targeting" -- targeting genes and proteins that are common across different types of adult and pediatric drugs.

To that end, our bill gives the FDA the authority to require a pediatric investigation into an adult drug if it uses molecular targeting, and the same target is "substantively relevant" to the growth of a pediatric cancer. This will allow doctors to know the dosage, safety and efficacy in children and provide accurate labeling for pediatric use. Additionally, this bill requires adult molecular targeting drugs that receive an orphan designation to undergo such a pediatric investigation.

The future of drug development is now, and we must not forget that our most vulnerable of populations do not have special interests advocating on their behalf. Parents and family members of loved ones affected by cancer must be the advocates for our children so our laws keep up with innovative cures. Already, the links between molecular targets in adult cancers and pediatric cancers have saved children's lives. The ALK gene appearing in adult lung cancers has also been found in certain forms of pediatric neuroblastoma, and ALK-inhibiting treatments for adults have been similarly effective in certain neuroblastoma cases. The FDA is moving to a new world where drugs are approved based on their target, rather than a specific part of the body. The cancer drug Keytruda was recently approved by the FDA for use in both adults AND children for its treatment of cancers that contain solid tumors with the target known as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR).

Today, for the children fighting the most challenging forms of cancer, treatment can be limited. Over 2,000 children die of a pediatric cancer every year and there are over 500,000 pediatric cancer survivors, most of whom suffer from chronic illnesses brought on by toxic cancer treatments. The RACE for Children Act could be the game-changer that finally offers children and their families the best standard of care possible.

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