An Economic Evaluation of Voretigene Neparvovec for the Treatment of Biallelic RPE65-Mediated Inherited Retinal Dystrophies in the UK

Author:

Viriato Daniel,Bennett Natalie,Sidhu Raisa,Hancock Elizabeth,Lomax Hannah,Trueman David,MacLaren Robert E.

Funder

Novartis Pharma AG

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),General Medicine

Reference42 articles.

1. RetNet: summaries of genes and loci causing retinal diseases. https://sph.uth.edu/retnet/sum-dis.htm. Accessed 16 Jan 2020.

2. Russell S, Bennett J, Wellman JA, Chung DC, Yu Z-F, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390:849–60. https://doi.org/10.1016/S0140-6736(17)31868-8.

3. National Institute for Health and Care Excellence (2019) Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations. https://www.nice.org.uk/guidance/hst11. Accessed 16 Jan 2020.

4. National Library of Medicine (US) Retinitis pigmentosa. https://ghr.nlm.nih.gov/condition/retinitis-pigmentosa. Accessed 16 Jan 2020.

5. RNIB (2017) Retinitis pigmentosa and other inherited retinal dystrophies. https://www.rnib.org.uk/sites/default/files/Understanding_Retinitis_Pigmentosa.pdf. Accessed 16 Jan 2020.

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