Time for a change in how new antibiotics are reimbursed: Development of an insurance framework for funding new antibiotics based on a policy of risk mitigation.

Healthcare systems depend on the availability of new antibiotics. However, there is a lack of treatments for infections caused by multidrug resistant (MDR) pathogens and a weak development pipeline of new therapies. One core challenge to the development of new antibiotics targeting MDR pathogens is that expected revenues are insufficient to drive long-term investment. In the USA and Europe, financial incentives have focussed on supporting R&D, reducing regulatory burden, and extending market exclusivity. Using resistance data to estimate global revenues, we demonstrate that the combined effects of these incentives are unlikely to rekindle investment in antibiotics. We analyse two supplemental approaches: a commercial incentive (a premium price model) and a new business model (an insurance model). A premium price model is familiar and readily implemented but the required price and local budget impact is highly uncertain and sensitive to cross-sectional and longitudinal variation in prevalence of antibiotic resistance. An insurance model delivering risk mitigation for payers, providers and manufacturers would provide an incentive to drive investment in the development of new antibiotics while also facilitating antibiotic conservation. We suggest significant efforts should be made to test the insurance model as one route to stimulate investment in novel antibiotics.

Mapping the COPD Assessment Test onto EQ-5D.

OBJECTIVES: To develop an algorithm for estimating EuroQol five-dimensional questionnaire (EQ-5D)-equivalent utilities from the chronic obstructive pulmonary disease (COPD) assessment test (CAT) and evaluate its use in economic evaluations as part of health technology assessments for COPD. METHODS: Data for the three-level EQ-5D (EQ-5D-3L) and the CAT were obtained from two multinational, phase III clinical trials. Three approaches were explored for estimating EQ-5D-equivalent utilities from the CAT: ordinary least-squares (OLS) regression, multinomial logistic regression, and a combination of the two. Estimated utilities were compared with actual EQ-5D-3L utilities, including treatment effect and the impact of disease severity on health, to evaluate the predictive performance of each algorithm. RESULTS: Root mean squared error and mean absolute error analyses showed that an OLS algorithm performed as well as algorithms developed using more complex modeling structures. The OLS regression included EQ-5D-3L utility weights as dependent variables and CAT items as independent variables. Within-sample validation showed systematic overestimation and underestimation within the range 0.5 ≤ EQ-5D-3L ≤ 0.9 (although all mean absolute errors were ≤0.100), with the smallest difference between estimated and actual values within 0.7 < EQ-5D-3L ≤ 0.9. The algorithm underestimated utility near full health and overestimated utility less than 0.5. As a consequence, the change from baseline was lower and the confidence intervals were narrower than those observed with actual EQ-5D-3L data. CONCLUSIONS: In the absence of EQ-5D data, the OLS regression algorithm may provide an estimate of utility for treatment models, but it is likely to underestimate treatment effects. Therefore, it is recommended that utilities be derived directly from the EQ-5D for the purposes of health technology assessments for COPD treatments in the United Kingdom.

Sources and Characteristics of Utility Weights for Economic Evaluation of Pediatric Vaccines: A Systematic Review.

BACKGROUND: Cost-effectiveness analysis of pediatric vaccines for infectious diseases often requires quality-of-life (utility) weights. OBJECTIVE: To investigate how utility weights have been elicited and used in this context. METHODS: A systematic review was conducted of studies published between January 1990 and July 2013 that elicited or used utility weights in cost-effectiveness analyses of vaccines for pediatric populations. The review focused on vaccines for 17 infectious diseases and is presented following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) methodology. RESULTS: A total of 6410 titles and abstracts and 225 full-text articles were reviewed. Of those selected for inclusion (n = 101), 15 articles described the elicitation of utility weights and 86 described economic modeling studies using utilities. Various methods were used to generate utilities, including time trade-off, contingent valuation, and willingness to pay, as well as a preference-based measure with associated value sets, such as the EuroQol five-dimensional questionnaire or the Health Utilities Index. In modeling studies, the source of utilities used was often unclear, poorly reported, or based on weak underlying evidence. We found no articles that reported on the elicitation or use of utilities in diphtheria, polio, or tetanus. CONCLUSIONS: The scarcity of appropriate utility weights for vaccine-preventable infectious diseases in children and a lack of standardization in their use in economic assessments limit the ability to accurately assess the benefits associated with interventions to prevent infectious diseases. This is an issue that should be of concern to those making decisions regarding the prevention and treatment of infectious childhood illnesses.

Assessing Patient-Reported Outcomes in Pediatric Populations With Vaccine-Preventable Infectious Diseases: A Systematic Review of the Literature (the PROCHID Study).

OBJECTIVE: To investigate the use of patient-reported outcomes (PROs) in pediatric populations with vaccine-preventable infectious diseases in high-income Western countries. METHODS: Systematic review of PRO use in populations younger than 18 years with any of 17 infectious diseases for which vaccines are available or in development. The search was limited to studies performed in Europe, North America, Australia, and New Zealand and published between January 1, 1990, and July 31, 2013. Searches were conducted in Scopus and PsycINFO, and reference lists were manually searched. Results are reported using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. RESULTS: Of 6410 titles and abstracts and 174 full-text articles reviewed, 17 full-text articles were included for data extraction. The largest number of PRO studies was carried out in patients with anogenital warts and rotavirus gastroenteritis. No PRO studies were identified for nine conditions. A total of 24 PRO measures (12 generic and 12 disease-specific) were used in the studies reviewed. Most of the instruments used were of high quality. Proxy responses were occasionally obtained when self-report would have been feasible. No validated disease-specific instruments for children with any of the conditions studied were found. CONCLUSIONS: The paucity of studies and PRO instruments to assess pediatric health status in vaccine-preventable infectious diseases, and the lack of a standardized approach to measurement, makes it difficult to capture the impact of disease and the benefit of vaccination and could potentially hinder decision making. Guidelines from relevant bodies to steer research in this area would be useful.