Introduction: Voretigene neparvovec (VN) is a gene therapy and the first approved pharmacological treatment for biallelic RPE65-mediated inherited retinal dystrophies (IRD), a rare condition that starts in early life and causes vision to progressively deteriorate towards complete blindness. In a phase III trial, treatment with VN significantly improved functional vision and visual function, and in October 2019 the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies Enhanced Digital Features To view enhanced digital features for this article go to https://doi.org/10.6084/ m9.figshare.11637108.
Objectives: The objective of this analysis was to examine patient-reported productivity losses for Charcot-Marie-Tooth disease (CMT) in EU and US real-world practice. Methods: Adults with CMT were recruited to a two-year international observational study exploring the real-world impact of the disease. Data were collected via CMT&Me, a 'bring your own device' app specifically developed for this study, through which participants were asked questions about demographic and employment variables. This interim analysis examined the impact of CMT on ability to work and study in participants from Germany, Italy, Spain, the UK and the US. Results: Of participants who responded, just over half (52%) reported that they were currently working for pay. Twenty-one percent of all participants reported not working due to a disabled status. A further 7% were unemployed, of whom 62% reported that CMT was a contributing factor. Of those working for pay, 77% reported that their work life was affected by CMT. The highest rates were in the UK (81%), while the lowest were in Italy and Spain (both 74%). The most frequently reported ways in which work life was affected by CMT were type of job (58%), number of days taken as sick leave (31%), and working part-time (28%). Participants reported missing a mean of 1.08 days of work in the past two weeks due to their condition. Of the 5% of participants who reported that they were studying, 74% reported that their study life was affected by CMT. The most frequently reported ways in which study life was affected were choice of school/college/university, and Other (both 52%). Conclusions: CMT is associated with substantial productivity losses, which are comparable across EU countries and the US. Further research is needed to explore the specific indirect costs associated with these productivity losses, and to better manage CMT's impact on patients' work/ study lives.
tiveness analyses were compared with methods specified by NICE as the reference case in its 2004 and 2008 Methods Guides. The review focused on guidance published before June 2013 for medicines treating metastatic breast cancer, metastatic colorectal cancer, metastatic hormone-refractory prostate cancer and metastatic non-smallcell lung cancer. Nineteen technology appraisals published between March 2002 and August 2012 met the inclusion criteria. Results: Common themes or variations that exist between utility values selected by manufacturers and independent academic groups for each metastatic cancer and between the 4 metastatic cancers were analysed. The research also explored the methodological issues that were considered by the Appraisal Committee relating to the selection of utility values. ConClusions: Therefore, this research provides insight to the methodological considerations regarding incorporation of utility values that have informed health technology assessment decision-making in England for 4 metastatic cancers.
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