The Race Against the Clock: A New Bill Providing Hope for Children Fighting the Ultimate Battle

Nicholas M. Fiorello

Roughly forty-three children are diagnosed with cancer daily.[i]  In total, approximately 1190 children are expected to lose their lives this year in the United States alone, as a result of battling pediatric cancer.[ii]  Pediatric cancer is the leading cause of death in children and adolescents (ages 1–14) in the U.S.[iii]  More than 40,000 children suffer through cancer treatment every year and, to add insult to injury, roughly 15,700 more children will be diagnosed with pediatric cancer this year alone.[iv]  A child of any age, ethnicity, gender, or socio-economic group can fall victim to a cancer diagnosis.[v]

Although advances in the medical field have altered the landscape of adult medicines, children are usually ignored.  Cancer causes the most disease-related deaths among children in the country, but yet, children are usually not the direct recipients of the newest and most promising drugs.  Children are usually placed on the back-burner in the world of drug development, especially in pediatric oncology.  This unfortunately relates to statistics and profit, with an emphasis on profit. 

Pediatric cancers are very rare, as less than 1% of all newly diagnosed cancer each year in the United States consist of one of the multitude of forms.[vi]  With only 1% of cancer patients in one specific class, why would a pharmaceutical company use their precious resources (time, energy, money) on a small percentage, offering little in return?  The pediatric cancer “market” does not offer a high enough return for these companies, where other markets offer astronomical returns.[vii]  Developing and testing drugs is an expensive experience that lasts many years, if not longer.

Children, not only in pediatric oncology, but in general, have always been cast-aside by pharmaceutical companies due to higher earning potential in the adult market.  This leads to a lack of progress in the pediatric drug development field.  Children are treated with medicines, either only approved for, or, only tested on adults.[viii]  A lack of drugs specifically tailored for children places a lot of pressure on a treating doctor as they are forced to estimate appropriate dosages based on the relative weight of the child.[ix]  This practice is called “off-label” prescribing and is a custom doctors regularly perform. But this can be especially dangerous due to the differences between how adults and children metabolize.[x]

           Beginning in the 90s and into the early 2000s, the government attempted to increase the number of drugs available to treat children through legislation.  The government decided to use a “carrot and stick” approach to attack this issue.  The Best Pharmaceuticals for Children Act (“BPCA”), or the “carrot,” provides incentives to pharmaceutical companies for conducting tests on children regarding an adult drug.[xi]  In exchange for conducting pediatric tests, the Food and Drug Administration (“FDA”) would grant an additional six months of market exclusivity to that company for that drug, meaning the patent for that drug would not expire for an additional six months.[xii]  The Pediatric Research Equity Act (“PREA”), or the “stick,” granted the FDA the authority to mandate pediatric testing.[xiii]  Both laws have had moderate success in updating labeling for pediatric use, but very little progress has been made in pediatric oncology due to many loopholes, exceptions and waivers riddled in the laws.[xiv]

In February 2017, legislation was introduced to patch these holes, creating hope for children fighting cancer.  The Research to Accelerate Cures and Equity for Children Act (“RACE”) was proposed to close these loopholes.[xv]  Upon introduction, this bill was directed to update law to correlate with modern medicine practices and would no longer allow broad exemptions by pharmaceutical companies.[xvi]  Last summer, this Bill passed in the House and the Senate.  On August 3, 2017, the RACE for Children Act was signed into law by the President.  RACE was included in Title V of the FDA Reauthorization act, amending the prior law, PREA.  Coming from someone who has been touched by the horrendous disease, I do not see a reason why children, our future, should continue to be cast aside in favor of mere monetary gain.  I am on board to RACE for a cure, are you?

[i].                Geoff Duncan, Childhood Cancer StatisticsCuresearch (August 5, 2016),
[ii].               Nat’l Cancer Inst., Childhood Cancers, (April 5, 2017),
[iii].              Nat’l Cancer Inst., Childhood Cancers, (April 5, 2017),
[iv].               Duncan, supra note 1.  12% of the children diagnosed with cancer this year will not survive.  Id.
[v].                Id.
[vi].               Peter C. Adamson et al., Drug Discovery in Paediatric Oncology: Roadblocks to Progress, 11 Nature Revs. Clinical Oncology 732, 734 (2014).
[vii].              Id.
[viii].             Lisa Jerles, The Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act—Helping or Hurting America’s Children?, 6 Cardozo Pub. L. Pol’y & Ethics J. 515, 517 (2008).
[ix].               Id.
[x].                Id. at 527.
[xi].               Cures for All: US Lawmakers Should Give Drug Firms the Confidence to Test Cancer Therapies in Childrensupra note 18, at 466.
[xii].              Editorial, Cures for All:  US Lawmakers Should Give Drug Firms the Confidence to Test Cancer Therapies in Children, 535 Nature Int’l Wkly J. Sci. 465 (2016).
[xiii].             Id. at 466.
[xiv].             Id.
[xv].              Id.
[xvi].             Id.

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