Cost-of-illness studies in rare diseases: a scoping review

Objective The aim of this scoping review was to overview the cost-of-illness studies conducted in rare diseases. Methods We searched papers published in English in PubMed from January 2007 to December 2018. We selected cost-of-illness studies on rare diseases defined as those with prevalence lower than 5 per 10,000 cases. Studies were selected by one researcher and verified by a second researcher. Methodological characteristics were extracted to develop a narrative synthesis. Results We included 63 cost-of-illness studies on 42 rare diseases conducted in 25 countries, and 9 systematic reviews. Most studies (94%) adopted a prevalence-based estimation, where the predominant design was cross-sectional with a bottom-up approach. Only four studies adopted an incidence-based estimation. Most studies used questionnaires to patients or caregivers to collect resource utilisation data (67%) although an important number of studies used databases or registries as a source of data (48%). Costs of lost productivity, non-medical costs and informal care costs were included in 68%, 60% and 43% of studies, respectively. Conclusion This review found a paucity of cost-of-illness studies in rare diseases. However, the analysis shows that the cost-of-illness studies of rare diseases are feasible, although the main issue is the lack of primary and/or aggregated data that often prevents a reliable estimation of the economic burden.

A Structured Review to Assess the Current Status of Cost-Based Burns Research in Nepal

The management of burns is costly and complex. The problem is compounded in low and middle income countries (LMICs) where the incidence of burn injuries is high but infrastructure and funding for management and prevention is limited. Cost of illness studies allows for quantification of the costs associated with public health problems. Without cost quantification, focus and allocation of funding is challenging. The authors explored the availability of cost-focused burns research data in a target LMIC. The focus of their research was Nepal. A structured literature review including published papers, Ministry of Health (MOH) and World Health Organization (WHO) statistics was conducted to identify cost of illness studies or evidence relating to burn-related resource and costs. Gaps in the evidence base were highlighted. Research methodologies from other LMICs were reviewed. We found 32 papers related to burn injury in Nepal, one key MOH document and one relevant WHO data source. Most research focused on the epidemiology and etiology of burns in Nepal. Of the papers, only 14 reported any type of burn-related resource use and only 1 paper directly reported (limited) cost data. No studies attempted an overall quantification of the cost of burns. MOH statistics provided no additional insight into costs. Our study found an almost complete lack of cost-focused burns research in Nepal. Primary research is needed to quantify the cost of burns in Nepal. Initial focus could usefully be on the cost of care in tertiary hospitals. A full cost of burns for Nepal remains some way off.

Cost of Nine Pediatric Infectious Illnesses in Low- and Middle-Income Countries: A Systematic Review of Cost-of-Illness Studies

Background Cost-of-illness data from empirical studies provide insights into the use of healthcare resources including both expenditures and the opportunity cost related to receiving treatment. Objective The objective of this systematic review was to gather cost data and relevant parameters for hepatitis B, pneumonia, meningitis, encephalitis caused by Japanese encephalitis, rubella, yellow fever, measles, influenza, and acute gastroenteritis in children in low- and middle-income countries. Data Sources Peer-reviewed studies published in public health, medical, and economic journals indexed in PubMed (MEDLINE), Embase, and EconLit. Study Eligibility Criteria, Participants, and Interventions Studies must (1) be peer reviewed, (2) be published in 2000–2016, (3) provide cost data for one of the nine diseases in children aged under 5 years in low- and middle-income countries, and (4) generated from primary data collection. Limitations We cannot exclude missing a few articles in our review. Measures were taken to reduce this risk. Several articles published since 2016 are omitted from the systematic review results, these articles are included in the discussion. Conclusions and Implications of Key Findings The review yielded 37 articles and 267 sets of cost estimates. We found no cost-of-illness studies with cost estimates for hepatitis B, measles, rubella, or yellow fever from primary data. Most estimates were from countries in Gavi preparatory (28%) and accelerated (28%) transition, followed by those who are initiating self-financing (22%) and those not eligible for Gavi support (19%). Thirteen articles compared household expenses to manage illnesses with income and two articles with other household expenses, such as food, clothing, and rent. An episode of illness represented 1–75% of the household’s monthly income or 10–83% of its monthly expenses. Articles that presented both household and government perspectives showed that most often governments incurred greater costs than households, including non-medical and indirect costs, across countries of all income statuses, with a few notable exceptions. Although limited for low- and middle-income country settings, cost estimates generated from primary data collection provided a ‘real-world’ estimate of the economic burden of vaccine-preventable diseases. Additional information on whether common situations preventing the application of official clinical guidelines (such as medication stock-outs) occurred would help reveal deficiencies in the health system. Improving the availability of cost-of-illness evidence can inform the public policy agenda about healthcare priorities and can help to operationalize the healthcare budget in local health systems to respond adequately to the burden of illness in the community.