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PURPOSE: To discuss the challenges and potential improvement strategies of cost-effectiveness analyses performed for therapeutics targeting inherited retinal diseases. DESIGN: Perspective METHODS: Literature review with discussion of current limitations and improvement recommendations. RESULTS: Cost-effectiveness analysis (CEA) performed for inherited retinal diseases (IRD) therapeutics has multiple limitations. First, the available methods used to measure health-related quality of life and health utilities can be inaccurate when used in IRDs. Second, the financial burden to patients and society by vision impairment associated with IRDs has been inadequately studied and includes a variety of expenditures ranging from direct costs of IRD specialty healthcare to indirect expenses associated with daily living activities. Third, our collective understanding is limited in the areas of IRD natural history and health benefits gained from new IRD treatments (e.g. gene therapies). Additionally, the therapeutic effect from a patient perspective and its duration of action is not fully understood. Due to the scarcity of data, CEA for newly approved therapies has relied on assumptions and creations of predictive models for both costs and health benefits for these new therapeutics in order to calculate the incremental cost-effectiveness ratio (ICER). CONCLUSIONS: CEA studies performed for IRD therapeutics have been limited by the currently established health utilities in ophthalmology and the lack of disease-specific information. The assumptions and extrapolations in these studies create substantial uncertainty in ICER results. An improved framework is required for CEA of IRD therapeutics in order to determine the cost-effectiveness of each therapy brought from clinical trials to clinical practice. TABLE OF CONTENTS STATEMENT: Cost-effectiveness analysis (CEA) studies performed for inherited retinal disease (IRD) therapeutics have been limited by the currently established health utilities in ophthalmology which lack disease specific information. The assumptions and extrapolations made in these studies are susceptible to large amounts of uncertainty in incremental cost-effectiveness ratio calculations. An improved framework for CEA of IRD therapeutics would better inform policy makers and other stakeholders, as these therapies progress from preclinical testing to clinical trials, and finally to clinical practice

The author examines efforts by managers of a Japanese-owned auto parts plant to avoid hiring union sympathizers in 1993–94. Data from confidential questionnaires in which job applicants stated whether they would vote for union representation in their current or most recent job were matched with outcome data provided by management. Pro-union applicants were much more likely than other applicants to withdraw their applications or quit shortly after being hired. The author finds only weak evidence, however, that management overtly favored antiunion job applicants in making job offers.

Objective This study measured satisfaction of US and Canadian child psychiatrists with quality of care in their field and behaviors of their payers. Method US and Canadian child psychiatrists were surveyed concerning time spent on billing paperwork, waiting times for psychiatrist reimbursement and patient treatment, and their satisfaction with psychiatric services and behaviors of payers. Initial and two nonresponder follow-up mailings of the survey were sent in 1992 to all 83 Canadian members of the American Academy of Child and Adolescent Psychiatry and a random sample of 212 US members. Results Canadians were significantly more satisfied than Americans with the quality and affordability of services for both inpatients and outpatients. Canadians were less likely to think that third-party payers were annoying or affected their clinical decision-making. Canadians spent less time on billing-related paperwork and received payment more promptly. Canadians, however, reported significantly longer waits for both inpatients and mildly ill outpatients, but not for severely ill outpatients. Conclusions Canadian child psychiatrists were significantly more satisfied with the quality of psychiatric services and the behaviors of their payers, but Canadian patients experienced longer waits.

Objective: To conduct a cost-effectiveness analysis of telestroke—a 2-way, audiovisual technology that links stroke specialists to remote emergency department physicians and their stroke patients—compared to usual care (i.e., remote emergency departments without telestroke consultation or stroke experts). Methods: A decision-analytic model was developed for both 90-day and lifetime horizons. Model inputs were taken from published literature where available and supplemented with western states’ telestroke experiences. Costs were gathered using a societal perspective and converted to 2008 US dollars. Quality-adjusted life-years (QALYs) gained were combined with costs to generate incremental cost-effectiveness ratios (ICERs). In the lifetime horizon model, both costs and QALYs were discounted at 3% annually. Both one-way sensitivity analyses and Monte Carlo simulations were performed. Results: In the base case analysis, compared to usual care, telestroke results in an ICER of $108,363/QALY in the 90-day horizon and $2,449/QALY in the lifetime horizon. For the 90-day and lifetime horizons, 37.5% and 99.7% of 10,000 Monte Carlo simulations yielded ICERs $50,000/QALY, a ratio commonly considered acceptable in the United States. Conclusion: When a lifetime perspective is taken, telestroke appears cost-effective compared to usual care, since telestroke costs are upfront but benefits of improved stroke care are lifelong. If barriers to use such as low reimbursement rates and high equipment costs are reduced, telestroke has the potential to diminish the striking geographic disparities of acute stroke care in the United States. Neurology ® 2011;77:1590–1598

This study measures the impact of labor and corporate political action committee (PAC) contributions on the voting of members of the House of Representatives on labor issues during 1979–80. It also analyzes the allocation of labor PAC contributions among House candidates. PAC contributions were found to have a significant direct effect on roll-call voting, even controlling for the Representative's political party and characteristics of the constituency. Since PAC money also affects roll-call voting indirectly (by influencing which party wins elections), the overall impact of PAC money on Congressional voting is probably substantial. The author also finds that labor PACs have focused more on influencing the outcome of elections than on currying favor with powerful members of the House who are likely to be re-elected anyway.

This study analyzes state-level data for 1959–78 to determine whether the rapid growth of teacher unionism during those years was primarily a result or a cause of the public sector bargaining laws adopted during the same period. The author finds, contrary to the view of some scholars, that the enactment of laws requiring public sector employers to bargain with majority representatives of their employees was the single most important cause of the growth in the proportion of teachers covered by union contracts. Although the growth in teacher unionism in turn encouraged the adoption of some new or stronger bargaining laws, this effect was relatively weak. More important predictors of new bargaining laws included the extent of political patronage in a state and the bargaining laws adopted by neighboring states.