Clinical, Featured, Professional Advice / Opinions:

The Challenges of Pediatric Clinical Drug Trials and Drug Product Labeling

By: Shannon Tellier, Associate Student Editor

The disease burden in children outweighs the number of pediatric clinical drug trials currently being conducted.  The lack of data in pediatrics leads to drugs being used off-label and without sufficient knowledge of doses, tolerability, and efficacy.  In 1975, only 22% of products in the electronic Physicians’ Desk Reference had pediatric labeling, which increased to 46% in 2009.1  Even though there has been an increase in the last three decades, the large number of medications without pediatric labeling could lead to detrimental effects in children.  This deficiency in pediatric clinical drug trials is due to many factors: lack of financing, as well as legal and ethical problems.

Since most of the currently available medications do not include pediatric labeling, healthcare professionals face difficult decisions.  The two options include (1) not treating children with potentially beneficial medications or (2) extrapolating doses of medications from adult trials.  The former raises an ethical question and the latter is dangerous because the pharmacokinetic, pharmacodynamic, and toxic properties of drugs in children are quite different from adults.  These extensive differences could lead to dosing errors and adverse events in the pediatric population.

Even though the information gained from pediatric clinical drug trials is extremely valuable, conducting these trials is difficult.  Finding parents willing to enroll their child in a trial is one of the biggest hurdles.  Many parents are hesitant because of the fear of hurting their children by subjecting them to treatment that may not provide immediate benefit.  The idea of their child receiving a placebo also turns parents off.  It becomes easier to recruit children in trials studying potentially lethal illnesses, particularly where current therapies are unsatisfactory.

Within the past couple of decades, there have been numerous legislative acts aimed to increase pediatric drug development.  The FDA Modernization Act (FDAMA) of 1997 offered pharmaceutical companies an extra six months of market exclusivity to conduct pediatric studies and create pediatric labeling.  The Best Pharmaceuticals for Children Act, passed in 2002 and 2007, provided methods for studying drugs in pediatrics.2 It called for (1) identifying and prioritizing drugs that need to be studied in children and (2) conducting studies on priority drugs if manufacturers do not complete them.

Even though these Acts are currently available to promote pediatric drug trials, since the majority of medications still do not carry pediatric labeling, we need to provide additional incentives to encourage more research in the pediatric population.  The extremely valuable information gained through pediatric drug trials will not only reduce the risk of adverse effects in children but will also allow children to safely use medications that have been previously unused in the pediatric population.  Moving pediatric trials forward will take the combined efforts of many healthcare professionals (e.g. pharmacists, pediatricians, researchers, manufacturers and pharmacologists).  Additional pediatric randomized controlled trials will provide the necessary information needed to make evidence-based guidelines instead of using expert opinion, case reports, or trial and error to dose medications in children.


  1. Sachs AN, Avant D, Lee CS, et al.  Pediatric Information in Drug Product Labeling.  JAMA.  2012;18: 1914-1915.
  2. Background of the Best Pharmaceuticals for Children Act.  Accessed May 24, 2012.
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