Abstract
Objectives: Cystic fibrosis (CF) is a rare genetic disease with no cure. Until recently, treatment has targeted symptoms of the disease and not the disease-causing genetic defect. Ivacaftor is included in a new class of breakthrough drugs targeting the genetic defects of CF. We sought to estimate the long-term cost-effectiveness of ivacaftor from a US payer perspective. Methods: We developed an individual-level microsimulation model that followed a cohort of heterogeneous US CF patients over a lifetime. The primary outcome of interest was quality-adjusted life years (QALYs). We also compared unadjusted life years, count of acute pulmonary exacerbations, and count of lung transplants over a lifetime between patients treated with ivacaftor plus best supportive care and patients treated with best supportive care alone. We conducted one-way and probabilistic sensitivity analyses to test the impact of various model inputs and uncertainties. Results: We found a substantial increase in QALYs, life years, and treatment costs over a lifetime for patients treated with ivacaftor plus best supportive care versus best supportive care alone. Discounted results for ivacaftor were 22.92 QALYs and $8 797 840 in total lifetime costs compared to 16.12 QALYs and $2 336 366 lifetime costs for best supportive care alone. The incremental cost-effectiveness ratios (ICERs) were $950 217 per QALY. Results from the probabilistic sensitivity analysis indicated a 0% chance that ivacaftor was cost-effective at a willingness-to-pay (WTP) threshold of $500 000 per QALY. Conclusions: Treatment with ivacaftor plus best supportive care versus best supportive care alone is not cost-effective at or near commonly accepted WTP thresholds.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 1332-1339 |
| Number of pages | 8 |
| Journal | Value in Health |
| Volume | 23 |
| Issue number | 10 |
| DOIs | |
| State | Published - Oct 2020 |
Bibliographical note
Funding Information:Author Contributions: Concept and design: Wherry, Williamson, Chapman, Kuntz Acquisition of data: Wherry, Williamson Analysis and interpretation of data: Wherry, Williamson, Chapman, Kuntz Drafting of the manuscript: Wherry Critical revision of the paper for important intellectual content: Wherry, Chapman, Kuntz Statistical analysis: Wherry, Kuntz Obtaining funding: Kuntz Administrative, technical, or logistic support: Chapman Supervision: Kuntz Conflict of Interest Disclosures: Dr Williamson reported receiving personal fees from United Healthcare outside the submitted work. Dr Chapman reported receiving grants from the California Healthcare Foundation, the Laura and John Arnold Foundation, the NE States Consortium Systems Organization, and Blue Shield of California Foundation during the conduct of the study, and dues for an annual policy summit from Aetna, America's Health Insurance Plans, Anthem, Blue Shield of California, CVS, Express Scripts, Harvard Pilgrim Health Care, Allergan, United Healthcare, Kaiser Permanente, Premera, AstraZeneca, Genentech/Roche, GlaxoSmithKline, Johnson & Johnson (Janssen Research and Development, LLC), Merck, the National Pharmaceutical Council, Alynlam, Sanofi, Cambia Health Services, Health Care Service Corporation, ?park Therapeutics, Prime Therapeutics, Mallinckrodt, Regeneron, Biogen, Novartis, Editas, Health Partners, LEO Pharma, and Boehringer-Ingelheim outside the submitted work. No other disclosures were reported. Funding/Support: Model development was supported by the Institute for Clinical and Economic Review.
Publisher Copyright:
© 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research
Keywords
- cost-effectiveness, cystic fibrosis, ivacaftor, microsimulation model, percent predicted forced expiratory volume in 1 second (ppFEV)
