Gene therapies have the potential to cure rare conditions that often have no current efficacious treatments with a one-time treatment episode, relieving substantial unmet need and having profound positive impact on patients' lives. However, with the first gene therapy now licensed and priced at around US$1 million per patient, cost and uncertain funding mechanisms present a potential barrier to patient access. In this article, we discuss the unique challenges presented by gene therapies, particularly concerning the uncertainty inherent in their clinical evidence package at launch and their affordability within strained healthcare budgets. We present several payment models that would allow for sustainable reimbursement of these innovative technologies and make recommendations pertinent both to those developing gene therapies and to those paying for them.

Significant investments in regenerative medicine necessitate discussion to align evidentiary requirements and decision-making considerations from regulatory, health system payer and developer perspectives. Only with coordinated efforts will the potential of regenerative medicine be realized. We report on discussions from two workshops sponsored by NICE, University of Alberta, Cell Therapy Catapult and Centre for Commercialization of Regenerative Medicine. We discuss methods to support the assessment of value for regenerative medicine products and services and the synergies that exist between market authorization and reimbursement regulations and practices. We discuss the convergence in novel adaptive licensing practices that may promote the development and adoption of novel therapeutics that meet the needs of healthcare payers.

BackgroundTo understand the clinical and economic outcomes of treatments for managing complications of ischemic central retinal vein occlusion (iCRVO).MethodsWe conducted a systematic literature review by searching multiple databases and ophthalmology conferences from 2004 to 2015. Studies published in English language and populations of age ≥45 years were included. For clinical endpoints, we defined eligibility criteria as randomized controlled trials, prospective before-and-after study designs, and non-randomized studies reporting on treatments in patients with iCRVO. For economic endpoints, all types of study design except cost-of-illness studies were included. We evaluated the definitions of ischemia, clinical and economic endpoints, and rate of development of complications. Risk of bias was assessed for clinical studies using the Cochrane risk-of-bias tool.ResultsA total of 20 studies (1338 patients) were included. Treatments included anti-vascular endothelial growth factors (anti-VEGFs), steroids, and procedures primarily targeting macular edema and neovascularization. Ischemia was not defined consistently in the included studies. The level of evidence was mostly low. Most treatments did not improve visual acuity significantly. Development of treatment complications ranged from 11 to 57 %. Incremental cost-effectiveness ratios reported for anti-VEGFs and steroids were below the accepted threshold of GB£30,000, but considering such treatments only ameliorate disease symptoms they seem relatively expensive.ConclusionsThere is a lack of evidence for any intervention being effective in iCRVO, especially in the prevention of neovascularisation. iCRVO poses a significant clinical and economic burden. There is a need to standardize the definition of ischemia, and for innovative treatments which can significantly improve visual outcomes and prevent neovascular complications.

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of sixteen countries from the region according to different criteria: characteristics, quality (using Drummond's checklist), use of local data, addressed inputs limitation and results transferability. Results: Economic evaluations are used in CEE countries for informing decision making, while critically considering methodology, quality and study's reliability. Experts acknowledged limited generalizability of study results both between and within geographic regions. Meanwhile, despite these constraints, facing limited health technology assessment (HTA) capacity experts were still using foreign evidence. At the same time, the usefulness of studies published in CEE and former Soviet countries to inform their decision making is limited because of insufficient transparency in reporting, unaddressed uncertainty, limited insight on inputs and transferability of results. Although local costs, baseline risk and resource use data are required, experts accept evidence originating from health care settings outside CEE and former Soviet countries regarding relative effect and utilities values. ConClusions: HTA priority setting and transferability assessment of economic evidence are important issue in health care decision making in CEE and former Soviet countries, since HTA research capacity is limited and local evidence is scarce. For this purpose, quality, transparency, and transferability should be addressed explicitly in published economic evaluations originating from CEE and former Soviet countries.