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January 2019

Development of Therapeutics for Children—A Tricky Balancing Act

Author Affiliations
  • 1Duke University Medical Center, Durham, North Carolina
JAMA Pediatr. 2019;173(1):18-19. doi:10.1001/jamapediatrics.2018.4026

Consider this possibility: you develop abdominal pain and a headache. The pain is severe enough that it prompts you to visit your primary care clinician. She diagnoses you with severe hypertension and knows there are drugs that can help you. She recognizes that she has to treat you; however, there is no information on the right dose of these drugs for this disease or their safety or efficacy in people like you. Suddenly, she remembers an article she read in medical school about doses used to treat hypertension in nonhuman primates. She shares all of this information with you. You think, “This sounds crazy,” but since you feel profoundly ill, you decide to accept her recommendation. She starts with the nonhuman primate dose, applies a so-called human correction factor, and gives you the prescription. (She also crosses her fingers behind her back—you do not notice.) You pray.

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