Luxturna: FDA documents reveal the value of a costly gene therapy

Updating Cost-Effectiveness — The Curious Resilience of the $50,000-per-QALY Threshold

Peter J. Neumann, Joshua T. Cohen, Milton C. Weinstein · New England Journal of Medicine · 2014 · 2.3K citations

Pharmacy, Health Policy, Pharmacoeconomics +17

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Stephen R. Russell, Jean Bennett, Jennifer Wellman et al. · The Lancet · 2017 · 1.9K citations

Medicine, Ophthalmology, Vision Science +14

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

James Bainbridge, Manjit Mehat, Venki Sundaram et al. · New England Journal of Medicine · 2015 · 737 citations · Full text

Medicine, Genetics, Ophthalmology +17

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Artur V. Cideciyan, Samuel G. Jacobson, William A. Beltran et al. · Proceedings of the National Academy of Sciences · 2013 · 440 citations · Full text

Medicine, Ophthalmology, Vision Science +17

Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012

Nicholas S. Downing, Jenerius A. Aminawung, Nilay D. Shah et al. · JAMA · 2014 · 409 citations · Full text

Medicine, Pharmacology, Clinical Pharmacology +16