i. b. , z. m. , p. c. , l. h. , and a. h. are employees of pfizer inc . and hold significant stocks and shares of pfizer inc .
 approximately 50% of rare diseases are evident in children . fatal disease prognosis and lack of treatments causes 30% of affected children to not live past their fifth birthday . 
 this clear sense of urgency demands innovation and acceleration in drug development . 
 a case study is discussed highlighting the need for datarich phase i study design , extensive use of modeling and simulation , use of diverse data sources , and input from collaborators to respond to this urgent call . 
CONFLICT OF INTEREST