Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease.

Importance: Voretigene neparvovec-rzyl, the first gene therapy approved by the US Food and Drug Administration, was approved for the treatment for RPE65-mediated inherited retinal disease (IRD) in December 2017. This gene therapy is associated with high up-front costs and high efficacy, although of unknown duration, and its cost-effectiveness has not been assessed with RPE65 IRD-specific, longitudinal, patient-observation-level data.

Objective: To assess the incremental cost-effectiveness ratio (ICER) of voretigene neparvovec-rzyl compared with standard care for RPE65-mediated inherited retinal disease.

Estimation of impact of mediated inherited retinal disease on quality of life and the potential benefits of gene therapy.

Background/aims: In rare diseases, health-related quality of life (HRQL) data can be difficult to capture. Given the ultrarare nature of -mediated inherited retinal disease (IRD), it was not feasible to recruit a patient sample and collect HRQL data prospectively. The objectives of this study were to develop health state descriptions of -mediated IRD, and to estimate associated patient utilities.

Waiting for organ transplantation: results of an analysis by an Institute of Medicine Committee.

One of the most visible and contentious issues regarding the fairness of the original system of organ procurement and allocation is the argument that it resulted in great disparities in the total amount of time a patient waited for an organ (i.e. the time from registration at a transplantation center to transplant), depending on where he or she lived.