Systematic Literature Review of Clinical and Economic Outcomes of Micro-Invasive Glaucoma Surgery (MIGS) in Primary Open-Angle Glaucoma.

INTRODUCTION: Primary open-angle glaucoma is estimated to affect 3% of the population aged 40-80 years. Trabeculectomy is considered the gold standard in surgical management of glaucoma; however, it is a technically complex procedure that may result in a range of adverse outcomes. Device-augmented, minimally invasive procedures (micro-invasive glaucoma surgeries, MIGS) have been developed aiming for safer and less invasive intraocular pressure (IOP) reduction compared with traditional surgery. METHODS: This paper presents results from a systematic literature review conducted in accordance with National Institute for Health and Care Excellence requirements for the Medical Technology Evaluation Programme via multiple databases from 2005 to 2016. For clinical outcomes, randomized clinical trials (RCTs) comparing MIGS with trabeculectomy or other therapies, observational studies, and other non-RCTs were included. Clinical outcomes reviewed were the change from baseline in mean IOP levels and change in topical glaucoma medication. Safety was assessed by reported harm and adverse events. For economic evidence, trials on cost-effectiveness, cost-utility, cost-benefit, cost-consequences, cost-minimization, cost of illness, and specific procedure costs were included. Risk of bias was assessed for clinical studies using the Cochrane Risk of Bias tool. RESULTS: A total of nine RCTs (seven iStents®, one Hydrus®, and one CyPass®), seven non-RCTs (three iStent®, three CyPass®, and one Hydrus®), and 23 economic studies were analyzed. While various forms of trabeculectomy can achieve postoperative IOP of between 11.0 and 13.0 mmHg, MIGS devices described in this review were typically associated with higher postoperative IOP levels. In addition, MIGS devices may result in increased hypotony rates or bleb needling in subconjunctival placed devices, requiring additional medical resources to manage. There is limited available evidence on the cost-effectiveness of MIGS and therefore it remains unclear whether the cost of using MIGS is outweighed by cost savings through decreased medication and need for further interventions. CONCLUSION: Larger randomized trials and real-world observational studies are needed for MIGS devices to better assess clinical and economic effectiveness. Given the shortage of published data and increasing use of such procedures, living systematic reviews may help to provide ongoing and timely evidence-based direction for clinicians and decision makers. This review highlights the current unmet need for treatments that are easy to implement and reduce long-term IOP levels without increasing postoperative aftercare and cost. FUNDING: Santen GmbH, Germany.

Systematic literature review of treatments for management of complications of ischemic central retinal vein occlusion.

BACKGROUND: To understand the clinical and economic outcomes of treatments for managing complications of ischemic central retinal vein occlusion (iCRVO). METHODS: We 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. RESULTS: A 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. CONCLUSIONS: There 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.

Gene therapies: the challenge of super-high-cost treatments and how to pay for them.

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.

Bringing regenerative medicines to the clinic: the future for regulation and reimbursement.

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.

Hidden efficiencies: making completion of the pediatric vaccine schedule more efficient for physicians.

The objective of this work is to demonstrate the potential time and labor savings that may result from increased use of combination vaccinations. The study (GSK study identifier: HO-12-4735) was a model developed to evaluate the efficiency of the pediatric vaccine schedule, using time and motion studies. The model considered vaccination time and the associated labor costs, but vaccination acquisition costs were not considered. We also did not consider any efficacy or safety differences between formulations. The model inputs were supported by a targeted literature review. The reference year for the model was 2012. The most efficient vaccination program using currently available vaccines was predicted to reduce costs through a combination of fewer injections (62%) and less time per vaccination (38%). The most versus the least efficient vaccine program was predicted to result in a 47% reduction in vaccination time and a 42% reduction in labor and supply costs. The estimated administration cost saving with the most versus the least efficient program was estimated to be nearly US $45 million. If hypothetical 6- or 7-valent vaccines are developed using the already most efficient schedule by adding additional antigens (pneumococcal conjugate vaccine and Haemophilus influenzae type b) to the most efficient 5-valent vaccine, the savings are predicted to be even greater. Combination vaccinations reduce the time burden of the childhood immunization schedule and could create the potential to improve vaccination uptake and compliance as a result of fewer required injections.

Cataracts in airline pilots: prevalence and aeromedical considerations in Japan.

INTRODUCTION: Despite modern aviation being increasingly less dependent on human factors, the pilot is still part of the aircraft systems and vision is critical for flight safety. The incidence of cataract increases with age, but as most epidemiological studies focus on senior age groups, there is no data relevant to working age groups. PURPOSE: The aim of our study was to elucidate the prevalence of lens opacity in Japanese airline pilots. METHODS: A retrospective cohort study was conducted at the Japan Aeromedical Research Center. Medical records for all ophthalmological assessments performed in the 12-mo period prior to March 2008 were reviewed. Data collected included whether there was documented lens opacity or a history of previous cataract surgery. RESULTS: Over 12 mo, 3780 pilots underwent slit-lamp examination with an ophthalmologist as part of their scheduled medical and 105 pilots were identified with cataract. Out of these, 59/105 were bilateral, 29/105 were congenital types (of which 19 were bilateral), and 12/105 pilots gave a history of previous cataract surgery. In all 105 pilots, the visual acuity was sufficient to continue the privileges of their licenses. CONCLUSIONS: This study offers insight into the clinical iceberg of early cataracts in persons of working age. Mild and early lens opacities can cause significant glare and haze, and changes in color vision, which might compromise pilot performance even in the absence of decreased visual acuity. Cataracts in otherwise fit pilots have important aeromedical significance which requires further consideration.