Cystic Fibrosis: Breakthrough Drugs at Break-the-Bank Prices

Dodge JA , Lewis PA , Stanton M Cystic fibrosis mortality and survival in the UK: 1947-2003. Eur Respir J. 2007; 29: 522-6.

Quinton PM , Bijman J . Higher bioelectric potentials due to decreased chloride absorption in the sweat glands of patients with cystic fibrosis. N Engl J Med 1983; 308(20): 1185-9.

Riordan JR , Rommens JM , Kerem B Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989; 245(4922): 1066-73.

Ramsey BW , Davies J , McElvaney NG A CFTR otentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011; 365(18): 1663-72.

Wainwright CE , Elborn JS , Ramsey BW Lumacaftor-Ivacaftor in patients with cystic fibrosis homozygous for phe508del CFTR. N Engl J Med. 2015; 373(3): 220-31.

Cholon DM , Quinney NL , Fulcher ML Potentiator ivacaftor abrogates pharmacological correction of DeltaF508 CFTR in cystic fibrosis. Sci Transl Med. 2014; 6(246): 246ra296.

O'Sullivan BP , Orenstein DM , Milla CE . Pricing for orphan drugs: will the market bear what society cannot? JAMA. 2013 Oct 2;310(13):1343-4.

Walker J . Arkansas reaches settlement in cystic fibrosis drug suit: state will revise eligibility criteria for renewing prescriptions for Vertex Pharmaceuticals’ Kalydeco. Wall Street Journal. February 5, 2015. http://www.wsj.com/articles/arkansas-reaches-settlement-in-cystic-fibrosis-drug-suit-1423162197. Accessed September 23, 2015.

Weisman R . Cystic fibrosis drug could bring millions to executives: Critics decry plan to compensate Vertex executives if costly new drug is profitable. Boston Globe. May 1, 2015. https://www.boston-globe.com/business/2015/04/30/bonuses-granted-senior-vertex-executives-are-increasingly-rare-compensation-specialists-say/QWQnwztbou0TYAER9ykhnJ/story.html. Accessed September 23, 2015.

Cohen D , Raftery J . Paying twice: questions over high cost of cystic fibrosis drug developed with charitable funding. BMJ. 2014; 348: g1445.

Pollack A . Deal by Cystic Fibrosis Foundation raises cash and some concern. New York Times. November 19, 2014. http://www.nytimes.com/2014/11/19/business/for-cystic-fibrosis-foundation-venture-yields-windfall-in-hope-and-cash.html. Accessed September 23, 2015.

Tefferi A , Kantarjian H , Rajkumar SV In support of a patient-driven initiative and petition to lower the high price of cancer drugs. Mayo Clin Proc. Aug 2015; 90(8): 996-1000.

Pollack A . Sanofi halves price of cancer drug Zaltrap after Sloan-Kettering rejection. New York Times. November 8, 2012. http://www.nytimes.com/2012/11/09/business/sanofi-halves-price-of-drug-after-sloan-kettering-balks-at-paying-it.html?_r=0. Accessed September 23, 2015.