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

Details

• Alternative Title:
  Health Aff (Millwood)
• Personal Author:
  Feng, Lisa B. ; Grosse, Scott D. ; Green, Ridgely Fisk ; Fink, Aliza K. ; Sawicki, Gregory S.
• Description:
  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.
• Subjects:
  Adolescent Adult Age Factors Aminophenols Child Cohort Studies Cost Savings Cystic Fibrosis Databases, Factual Female Hospitalization Humans Male Middle Aged Precision Medicine Prescriptions Quinolones Retrospective Studies Risk Assessment Sex Factors Treatment Outcome United States United States Food And Drug Administration Young Adult

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• Source:
  Health Aff (Millwood). 37(5):773-779
• Pubmed ID:
  29733727
• Pubmed Central ID:
  PMC6038921
• Document Type:
  Journal Article
• Funding:
  CC999999/Intramural CDC HHSUnited States/
• Volume:
  37
• Issue:
  5
• Collection(s):
  CDC Public Access
• Main Document Checksum:
  urn:sha256:a86fe140c548b3b90700ce4e433a175e947aa3ee53e593acdab4b3deb989dd24
• Download URL:
  https://stacks.cdc.gov/view/cdc/56873/cdc_56873_DS1.pdf
• File Type:
  [PDF - 345.16 KB ]