Precision Medicine In Action: The Impact Of Ivacaftor On Cystic Fibrosis-Related Hospitalizations.
Lisa B Feng, Scott D Grosse, Ridgely Fisk Green, Aliza K Fink, Gregory S Sawicki
Author Information
Lisa B Feng: Lisa B. Feng ( lfeng@cff.org ) is senior director for policy and advocacy at the Cystic Fibrosis Foundation, in Bethesda, Maryland.
Scott D Grosse: Scott D. Grosse is a research economist at the National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention (CDC), in Atlanta, Georgia.
Ridgely Fisk Green: Ridgely Fisk Green is a Carter Consulting, Inc. contractor in the Office of Public Health Genomics, CDC.
Aliza K Fink: Aliza K. Fink is director of epidemiology at the Cystic Fibrosis Foundation.
Gregory S Sawicki: Gregory S. Sawicki is an assistant professor of pulmonology at Boston Children's Hospital, in Massachusetts.
Cystic fibrosis is a life-threatening genetic disease that causes severe damage to the lungs. Ivacaftor, the first drug that targeted the underlying defect of the disease caused by specific mutations, is a sterling example of the potential of precision medicine. Clinical trial and registry studies showed that ivacaftor improved outcomes and reduced hospitalizations. Our study used US administrative claims data to assess the real-world effectiveness of ivacaftor. Comparing twelve-month rates before and after starting the use of ivacaftor among people who initiated therapy during 2012-2015, we found that overall and cystic fibrosis-related inpatient admissions fell by 55 percent and 81 percent, respectively. There was a comparable reduction in inpatient spending. Ivacaftor appears to be effective for multiple mutations that cause the disease, as suggested by the fact that during the study period, ivacaftor's use was extended to nine additional mutations in 2014. Examination of evidence from clinical trial, clinical care, and administrative data sources is important for understanding the real-world effectiveness of precision medicines such as ivacaftor.
