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

Feng, Lisa Bo; Grosse, Scott D.; Green, Ridgely Fisk; Fink, Aliza K.; Sawicki, Gregory S.

doi:10.1377/hlthaff.2017.1554

Cited by 31 publications

(32 citation statements)

References 22 publications

(30 reference statements)

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“…[55][56][57] In addition, there are eight new observational studies (five full text, three abstracts only). 13,51,[58][59][60][61][62][63] See Appendix Tables D1 to D15 and our prior report 11 for detailed analyses of the clinical trials of Kalydeco in patients with mutations that respond to Kalydeco. The prior report summarized four RCTs (STRIVE, ENVISION, KONNECTION, and KONDUCT) that evaluated the safety and efficacy of Kalydeco in patients with at least one G551D, non-G551D gating, or R117H mutation.…”

Section: Clinical Benefits Population 1: Kalydeco In Gating and Residmentioning

confidence: 99%

“…There were eight observational studies on long term use of Kalydeco. 13,51,[58][59][60][61][62][63] The two largest and longest are from the same populations (US and UK Registries), but report complementary outcomes. 13,63 These ongoing post-approval safety studies evaluate clinical outcomes and disease progressions in all those in the UK and US registries following commercial availability.…”

Section: New Observational Data For Kalydecomentioning

confidence: 99%

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Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

Somayaji

Nichols

Bell

2020

Expert Opinion on Investigational Drugs

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The Institute for Clinical and Economic Review (ICER) is an independent non-profit research organization that evaluates medical evidence and convenes public deliberative bodies to help stakeholders interpret and apply evidence to improve patient outcomes and control costs. Through all its work, ICER seeks to help create a future in which collaborative efforts to move evidence into action provide the foundation for a more effective, efficient, and just health care system. More information about ICER is available at http://www.icer-review.org. The funding for this report comes from government grants and non-profit foundations, with the largest single funder being the Laura and John Arnold Foundation. No funding for this work comes from health insurers, pharmacy benefit managers, or life science companies. ICER receives approximately 20% of its overall revenue from these health industry organizations to run a separate Policy Summit program, with funding approximately equally split between insurers/PBMs and life science companies. For a complete list of funders and for more information on ICER's support, please visit http://www.icer-review.org/about/support/.

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Section: Clinical Benefits Population 1: Kalydeco In Gating and Residmentioning

confidence: 99%

Section: New Observational Data For Kalydecomentioning

confidence: 99%

Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

Somayaji

Nichols

Bell

2020

Expert Opinion on Investigational Drugs

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“…Short-term disease cost data were queried from a commercial database (Marketscan; Truven Health Analytics), commonly used in health-economics studies (see, e.g., [26][27][28] ). Twenty-five conditions were selected based on having high incidence and specific International Classification of Diseases (ICD) coding.…”

Section: Cost-of-care Database Surveymentioning

confidence: 99%

Clinical Impact and Cost-Effectiveness of a 176-Condition Expanded Carrier Screen

Beauchamp¹,

Taber²,

Muzzey³

2018

Preprint

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PurposeCarrier screening identifies couples at high risk for conceiving offspring affected with serious heritable conditions. Minimal screening guidelines mandate testing for cystic fibrosis and spinal muscular atrophy, but expanded carrier screening (ECS) assesses reproductive risk for hundreds of conditions simultaneously. Although medical societies consider ECS an acceptable practice, the health economics of ECS remain incompletely characterized. MethodsThe clinical impact and cost-effectiveness of a 176-condition ECS panel were investigated using a decision-tree model comparing minimal screening and ECS in a preconception setting.Carrier rates from >50,000 patients informed disease-incidence estimates, while cost and lifeyears-lost data were aggregated from the literature and a cost-of-care database. Model robustness was evaluated using one-way and probabilistic sensitivity analyses.

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“…Ivacaftor, a breakthrough medication for cystic fibrosis ("CF"), is an effective cure but only for three to four percent of the CF population. 52 The FDA approved Eteplirsen for Duchenne muscular dystrophy in 2016 but only for those with a specific genetic mutation, an estimated thirteen to…”

Section: A Restricted Market Sizementioning

confidence: 99%

Gene Therapy's Field of Dreams: If You Build It, Will We Pay?

Hercher

Prince

2019

SSRN Journal

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Long overpromised and underdelivered, gene therapy has at last achieved clinical validation and, with the advent of improved gene-editing technologies such as CRISPR, seems poised to play a rapidly expanding role in medical care. However, some of the intrinsic qualities of gene therapy pose a unique challenge to our health insurance model. Gene therapy is costly for a number of reasons. It is "personalized medicine," which means that treatments are individualized and not for a broad audience. Additionally, the goal of gene therapy is to provide a one-time cure, so the cost is upfront and not spread over time as it would be with conventional drugs or therapeutics. As our experience to date illustrates, these issues of cost may adversely affect access. In this Article, we argue that a lack of broad access to gene therapy will deepen existing health inequities and may create a society of genetic haves and have-nots, where certain genetic diseases become something that happens only to those who cannot afford treatment. This in turn may increase stigma and decrease resources for affected individuals. For these reasons, the success of gene therapy must be considered as inextricable from issues of cost and coverage.

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

Cited by 31 publications

References 22 publications

Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

Clinical Impact and Cost-Effectiveness of a 176-Condition Expanded Carrier Screen

Gene Therapy's Field of Dreams: If You Build It, Will We Pay?

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