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 Statistics, Curesearch (August 5, 2016),
http://curesearch.org/Childhood-Cancer-Statistics.
[ii]. Nat’l
Cancer Inst., Childhood
Cancers, Cancer.gov (April 5, 2017),
https://www.cancer.gov/types/childhood-cancers.
[iii]. Nat’l
Cancer Inst., Childhood
Cancers, Cancer.gov (April 5, 2017),
https://www.cancer.gov/types/childhood-cancers.
[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 Children, supra 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.
