Potential lifetime quality of life benefits of choroideremia gene therapy: projections from a clinically informed decision model

Upon completion of this activity, participants will be able to: 1. Describe the estimated lifetime benefit of gene therapy for the retinal dystrophy choroideremia, according to a Markov model of disease progression informed by clinical data of AAV.REP1 and voretigene neparvovec. 2. Illustrate the effects of discount rate and sensitivity analysis on estimated lifetime benefit of gene therapy for choroideremia, according to a Markov model of disease progression informed by clinical data of AAV.REP1 and voretigene neparvovec. 3. Identify clinical implications of the estimated lifetime benefit of gene therapy for choroideremia, according to a Markov model of disease progression informed by clinical data of AAV.REP1 and voretigene neparvovec. Describe the estimated lifetime benefit of gene therapy for the retinal dystrophy choroideremia, according to a Markov model of disease progression informed by clinical data of AAV.REP1 and voretigene neparvovec. Illustrate the effects of discount rate and sensitivity analysis on estimated lifetime benefit of gene therapy for choroideremia, according to a Markov model of disease progression informed by clinical data of AAV.REP1 and voretigene neparvovec. Identify clinical implications of the estimated lifetime benefit of gene therapy for choroideremia, according to a Markov model of disease progression informed by clinical data of AAV.REP1 and voretigene neparvovec. In support of improving patient care, this activity has been planned and implemented by Medscape, LLC and Springer Nature. Medscape, LLC is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Medscape, LLC designates this Journal-based CME activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. 1.0 Release date: 17 July 2019 Expiration date: 17 July 2020 Post-test link: https://medscape.org/eye/posttest913835 Sobha Sivaprasad has disclosed the following relevant financial relationships: Served as an advisor or consultant for Allergan, Bayer, Boehringer Ingelheim, Roche, Heidelberg, Optos. Served as a speaker or a member of a speakers bureau for Bayer, Allergan, Novartis, Optos. Received grants for clinical research from: Bayer, Boehringer Ingelheim, Allergan, Novartis, Optos. REM has disclosed the following relevant financial relationships: Served as an advisor or consultant for Nightstar Therapeutics plc; Spark Therapeutics, Inc. Served as a speaker or a member of a speakers bureau for Nightstar Therapeutics plc. Received grants for clinical research from Nightstar Therapeutics plc. CLHH, JKJ, JAS, RPV, and DAB have disclosed no relevant financial relationships. Laurie Barclay, MD: freelance writer and reviewer. Laurie Barclay, MD, has disclosed no relevant financial relationships.