Cost-effectiveness analysis of lumacaftor and ivacaftor combination for the treatment of patients with cystic fibrosis in the United States

[1] MacKenzie T(2014)Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation patient registry Ann Intern Med 161 233-241

[2] Gifford A(2015)Lumacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR N Engl J Med 373 220-231

[3] Sabadosa K(2011)Pricing and reimbursement of orphan drugs: the need for more transparency Orphanet J Rare Dis 6 42-23

[4] Quinton H(2010)Orphan drug pricing and payer management in the United States: are we approaching the tipping point? Am Health Drug Benefits 3 15-1191

[5] Knapp E(2012)What is wrong with orphan drug policies? Value Health 15 1185-17

[6] Goss C(2015)Orkambi's slick unveiling puts insurers in a bind Manag Care 24 16-1335

[7] Wainwright CE(2017)Forecasting the long-term clinical and economic outcomes of Lumacaftor/Ivacaftor in cystic fibrosis patients with homozygous phe508del mutation Value Health 20 1329-1103

[8] Elborn JS(2008)Incentives for orphan drug research and development in the United States Orphanet J Rare Dis. 3 33-139

[9] Ramsey BW(2016)Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine JAMA 316 1093-66

[10] Marigowda G(1999)The cost of medical care for patients with cystic fibrosis in a health maintenance organization Pediatrics 103 e72-400

[1] MacKenzie T(2014)Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation patient registry Ann Intern Med 161 233-241

[2] Gifford A(2015)Lumacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR N Engl J Med 373 220-231

[3] Sabadosa K(2011)Pricing and reimbursement of orphan drugs: the need for more transparency Orphanet J Rare Dis 6 42-23

[4] Quinton H(2010)Orphan drug pricing and payer management in the United States: are we approaching the tipping point? Am Health Drug Benefits 3 15-1191

[5] Knapp E(2012)What is wrong with orphan drug policies? Value Health 15 1185-17

[6] Goss C(2015)Orkambi's slick unveiling puts insurers in a bind Manag Care 24 16-1335

[7] Wainwright CE(2017)Forecasting the long-term clinical and economic outcomes of Lumacaftor/Ivacaftor in cystic fibrosis patients with homozygous phe508del mutation Value Health 20 1329-1103

[8] Elborn JS(2008)Incentives for orphan drug research and development in the United States Orphanet J Rare Dis. 3 33-139

[9] Ramsey BW(2016)Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine JAMA 316 1093-66

[10] Marigowda G(1999)The cost of medical care for patients with cystic fibrosis in a health maintenance organization Pediatrics 103 e72-400