The effectiveness and value of novel treatments for cystic fibrosis

Tice, Jeffrey A.; Kuntz, Karen M.; Wherry, Kael; Seidner, M.; Rind, David M.; Pearson, Steven D.

doi:10.18553/jmcp.2021.27.2.276

Cited by 6 publications

(4 citation statements)

References 9 publications

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“…Wholesale acquisition costs of CFTR modulators can range from $272 623 to $311 741 per year and raise concerns about affordability and access . However, the final reimbursement cost is based on agreements with health authorities or private payers and is variable.…”

Section: Discussion and Observationsmentioning

confidence: 99%

“…Wholesale acquisition costs of CFTR modulators can range from $272 623 to $311 741 per year and raise concerns about affordability and access. 106 However, the final reimbursement cost is based on agreements with health authorities or private payers and is variable. In the US, CFTR modulator therapies were prescribed to 91% (range, 75.9%-100%) of people with eligible CFTR variants in 2021, funded by both private and public payers.…”

Section: Practical Considerations and Application Of Evidencementioning

confidence: 99%

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Cystic Fibrosis

Ong

Ramsey

2023

JAMA

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ImportanceCystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in the US and approximately 89 000 worldwide. Absent or decreased function of the CFTR protein is associated with multiorgan dysfunction and shortened life expectancy.ObservationsCFTR is an anion channel in the apical membrane of epithelial cells. Loss of function leads to obstructed exocrine glands. Of people with cystic fibrosis in the US, approximately 85.5% have the gene variant F508del. Manifestations of cystic fibrosis in patients with the F508del gene variant begin in infancy with steatorrhea, poor weight gain, and respiratory symptoms (coughing, wheezing). As people with cystic fibrosis age, chronic respiratory bacterial infections cause loss of lung function and bronchiectasis. With the availability of universal newborn screening in multiple countries including the US, many people with cystic fibrosis are asymptomatic at diagnosis. With multidisciplinary care teams that included dietitians, respiratory therapists, and social workers, treatment of cystic fibrosis can slow disease progression. Median survival has improved from 36.3 years (95% CI, 35.1-37.9) in 2006 to 53.1 years (95% CI, 51.6-54.7) in 2021. Pulmonary therapies for patients with cystic fibrosis consist of mucolytics (eg, dornase alfa), anti-inflammatories (eg, azithromycin), and antibiotics (such as tobramycin delivered by a nebulizer). Four small molecular therapies, termed CFTR modulators, that facilitate CFTR production and/or function have received regulatory approval. Examples are ivacaftor and elexacaftor-tezacaftor-ivacaftor. For example, in patients with 1 F508del variant, the combination of ivacaftor, tezacaftor, and elexacaftor improved lung function from −0.2% in the placebo group to 13.6% (difference, 13.8%; 95% CI, 12.1%-15.4%) and decreased the annualized estimated rate of pulmonary exacerbations from 0.98 to 0.37 (rate ratio, 0.37; 95% CI, 0.25-0.55). Improved respiratory function and symptoms have lasted up to 144 weeks in postapproval observational studies. An additional 177 variants are eligible for treatment with the elexacaftor-tezacaftor-ivacaftor combination.ConclusionCystic fibrosis affects approximately 89 000 people worldwide and is associated with a spectrum of disease related to exocrine dysfunction, including chronic respiratory bacterial infections and reduced life expectancy. First-line pulmonary therapies consist of mucolytics, anti-inflammatories, and antibiotics, and approximately 90% of people with cystic fibrosis who are 2 years or older may benefit from a combination of ivacaftor, tezacaftor, and elexacaftor.

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Section: Discussion and Observationsmentioning

confidence: 99%

Section: Practical Considerations and Application Of Evidencementioning

confidence: 99%

Cystic Fibrosis

Ong

Ramsey

2023

JAMA

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“…The cost‐effectiveness of ETI has been extensively evaluated by the Institute for Clinical and Economic Review (ICER) 8 . Results of their analyses showed that CFTR modulator therapies plus best supportive care substantially improves patient health outcomes compared with best supportive care.…”

Section: Pharmacoeconomics Of Pk Enhanced Etimentioning

confidence: 99%

Pharmacokinetic Enhancement of Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis: A Cost Reduction Strategy to Address Global Disparities in Access

Hong,

Zampoli,

Beringer

2024

Clin Pharma and Therapeutics

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“…5 This cost is unjustifiably high. CFTR modulators are overpriced compared with quality life years gained 6 and the estimated cost of producing is 90% less than the list price. 7 The fact that Vertex is drastically decreasing its copay assistance program while earning these jaw-dropping profits truly exposes its corporate greed.…”

mentioning

confidence: 99%