Precision Medicine In Action: The Impact Of Ivacaftor On Cystic Fibrosis-Related Hospitalizations

Abstract

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.

Keywords: cystic fibrosis; precision medicine; rare disease; real world evidence.

Exhibit 3. Mean inpatient spending during the twelve months before and twelve months after filling the first ivacaftor prescription, by age group, 2012–15

SOURCE Authors’ analysis of data from the Truven Health Analytics MarketScan Commercial Research database. NOTES Patients’ diagnosis codes, prescription fills, insurance coverage, and age are explained in the notes to exhibit 1. Inpatient admissions were for all causes. Total payments (allowable charges) on inpatient services during the twelve months before and the twelve months after filling the first ivacaftor prescription were calculated and adjusted to 2016 medical prices using the Personal Consumption Expenditures health price index of the Bureau of Economic Analysis, an unbiased measure of US medical inflation.