Valuing rare pediatric drugs: an economics perspective

There is a coming wave of novel genetic therapies aiming to treat rare pediatric disease. A large literature investigates the valuation of new treatments, but the valuation of treatments for rare pediatric illness raises a host of unique issues. In this paper, we review the challenges of applying both the standard economic model and standard approaches to estimating cost-effectiveness using the quality-adjusted life year (QALY) to this case. We argue that there are a large number of special issues that have only been partially addressed by past work and we conclude that more data and the development of new methods are vital as innovators, health technology assessment practitioners and policymakers confront the launch of these new drugs.https://www.nber.org/papers/w27978First author draf

Principles of Economic Evaluation in a Pandemic Setting: An Expert Panel Discussion on Value Assessment During the Coronavirus Disease 2019 Pandemic

As the coronavirus disease 2019 (COVID-19) pandemic continues to generate significant morbidity and mortality as well as economic and societal impacts, the landscape of potential treatments has slowly begun to broaden. In the case of a novel disease with widespread consequences, society is more likely to place significant value on interventions that reduce the outsized economic burden of COVID-19. Treatments for severe disease will have a different value profile to that of large-scale vaccines because of their application in targeted and potentially small subsets of those with symptomatic disease vs broad deployment as a preventative measure. Where vaccines reduce transmissibility of COVID-19, use of therapeutics will target symptoms, up to and including death for infected individuals. This paper describes discussions from a virtual expert panel that met to attempt a consensus on how existing principles of economic evaluation should be applied to therapeutics that emerge in a pandemic setting, with specific focus on severe hospitalised cases of COVID-19. The panel concluded that the core principles of economic evaluation do not need to be drastically overhauled to meet the challenges of a pandemic, but that there are several additional elements of value such as equity, disease severity, insurance value, and scientific and family spillover effects that should be considered when presenting results to decision makers. The panel also highlighted the persistent challenges on how society should value novel therapies, such as the appropriate cost-effectiveness threshold to apply, which are particularly salient during a pandemic

Perspective and Costing in Cost-Effectiveness Analysis, 1974-2018

OBJECTIVE: Our objective was to examine perspective and costing approaches used in cost-effectiveness analyses (CEAs) and the distribution of reported incremental cost-effectiveness ratios (ICERs). METHODS: We analyzed the Tufts Medical Center's CEA and Global Health CEA registries, containing 6907 cost-per-quality-adjusted-life-year (QALY) and 698 cost-per-disability-adjusted-life-year (DALY) studies published through 2018. We examined how often published CEAs included non-health consequences and their impact on ICERs. We also reviewed 45 country-specific guidelines to examine recommended analytic perspectives. RESULTS: Study authors often mis-specified or did not clearly state the perspective used. After re-classification by registry reviewers, a healthcare sector or payer perspective was most prevalent (74%). CEAs rarely included unrelated medical costs and impacts on non-healthcare sectors. The most common non-health consequence included was productivity loss in the cost-per-QALY studies (12%) and patient transportation in the cost-per-DALY studies (21%). Of 19,946 cost-per-QALY ratios, the median ICER was $US26,000/QALY (interquartile range [IQR] 2900-110,000), and 18$US430/DALY (IQR 67-3400), and 8% were cost saving and DALY averting. Based on 16 cost-per-QALY studies (2017-2018) reporting 68 ICERs from both the healthcare sector and societal perspectives, the median ICER from a societal perspective ($US22,710/QALY [IQR 11,991-49,603]) was more favorable than from a healthcare sector perspective ($US30,402/QALY [IQR 10,486-77,179]). Most governmental guidelines (67%) recommended either a healthcare sector or a payer perspective. CONCLUSION: Researchers should justify and be transparent about their choice of perspective and costing approaches. The use of the impact inventory and reporting of disaggregate outcomes can reduce inconsistencies and confusion

Correction to : Perspective and Costing in Cost‑Effectiveness Analysis, 1974-2018

The article Perspective and Costing in Cost-Effectiveness Analysis

Direct Health Care Costs of Crohn’s Disease and Ulcerative Colitis in United States Children and Adults

Data regarding the health care costs of inflammatory bowel disease (IBD) in the United States are limited

Utilization of healthcare resources by U.S. children and adults with inflammatory bowel disease:

The inflammatory bowel diseases (IBDs), Crohn’s disease (CD) and ulcerative colitis (UC) affect over 1 million people in the United States, yet little is known about healthcare utilization by affected individuals

Identifying the need for good practices in Health Technology Assessment : summary of the ISPOR HTA Council Working Group Report on Good Practices in HTA

The systematic use of evidence to inform healthcare decisions, particularly health technology assessment (HTA), has gained increased recognition. HTA has become a standard policy tool for informing decision makers who must manage the entry and use of pharmaceuticals, medical devices, and other technologies (including complex interventions) within health systems, for example, through reimbursement and pricing. Despite increasing attention to HTA activities, there has been no attempt to comprehensively synthesize good practices or emerging good practices to support populationbased decision-making in recent years. After the identification of some good practices through the release of the ISPOR Guidelines Index in 2013, the ISPOR HTA Council identified a need to more thoroughly review existing guidance. The purpose of this effort was to create a basis for capacity building, education, and improved consistency in approaches to HTA-informed decision-making. Our findings suggest that although many good practices have been developed in areas of assessment and some other key aspects of defining HTA processes, there are also many areas where good practices are lacking. This includes good practices in defining the organizational aspects of HTA, the use of deliberative processes, and measuring the impact of HTA. The extent to which these good practices are used and applied by HTA bodies is beyond the scope of this report, but may be of interest to future researchers