ArticleHealth affairs (Project Hope)2018
Precision Medicine In Action: The Impact Of Ivacaftor On Cystic Fibrosis-Related Hospitalizations.
Article in Health affairs (Project Hope), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 19 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed.
- Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial.The Lancet. Respiratory medicine · 2025Trial
- Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis: Impact on Hospitalizations and Health Care Resource Utilization in a Universal Health Care Setting.Pulmonary therapy · 2025Article
- Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.Thorax · 2024Article
- The Rapid Reduction of Infection-Related Visits and Antibiotic Use Among People With Cystic Fibrosis After Starting Elexacaftor-Tezacaftor-Ivacaftor.Clinical infectious diseases : an official publication of the Infectious Diseases Society of America · 2022Article
- The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems.Orphanet journal of rare diseases · 2021Article
- A Review of Trikafta: Triple Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy.Cureus · 2021Review
- Long-Term Impact of Ivacaftor on Healthcare Resource Utilization Among People with Cystic Fibrosis in the United States.Pulmonary therapy · 2021Article
- Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review.Journal of clinical medicine · 2021Review
- Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021Observational
- Genomic, transcriptomic, and protein landscape profile of CFTR and cystic fibrosis.Human genetics · 2021Article
- With newer tools for gene editing, is it time to revisit genetic therapy for cystic fibrosis?Genes & diseases · 2021Article
- Prognosis-Based Early Intervention Strategies to Resolve Exacerbation and Progressive Lung Function Decline in Cystic Fibrosis.Journal of personalized medicine · 2021Article
- How Clinically Efficient Is Lumacaftor/Ivacaftor for Cystic Fibrosis Patients? An Updated Literature Review.Cureus · 2020Review
- Two Unanticipated Pregnancies While on Cystic Fibrosis Gene-Specific Drug Therapy.Journal of patient experience · 2020Article
- Practitioner Due Diligence: Real-World Lumacaftor/Ivacaftor Use.The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG · 2020Article
- In-vitro evaluation of a ciprofloxacin and azithromycin sinus stent for Pseudomonas aeruginosa biofilms.International forum of allergy & rhinology · 2020Article
- Genetic Support for Longevity-Enhancing Drug Targets: Issues, Preliminary Data, and Future Directions.The journals of gerontology. Series A, Biological sciences and medical sciences · 2019Review
- Clinical impact and cost-effectiveness of a 176-condition expanded carrier screen.Genetics in medicine : official journal of the American College of Medical Genetics · 2019Article
- Use of ivacaftor in late diagnosed cystic fibrosis monozygotic twins heterozygous for F508del and R117H-7T - a case report.BMC pulmonary medicine · 2019Article
Corrections and comments
- Erratum issuedErrata.2019
Authors and funding
5 authors.
Funding
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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.