Abstract
Objectives: This research aims to evaluate the methodological quality of budget impact analyses for orphan drugs and to provide suggestions for future analyses. Methods: Conference abstracts and peer-reviewed literature on budget impact analyses were collected through searches of Pubmed and Embase. ISPOR good practice guidelines were used as a methodological standard for budget impact analyses. Examined parameters encompassed: perspective, target population, data sources, intervention and comparator(s), time horizon, scope of costs, discounting, validation, assumptions and sensitivity analysis. Results: Seventy studies on individual orphan drugs and 21 studies on a combination of orphan drugs analyzing budget impact were identified. Overall, analyses considered a third-party payer perspective, reported periodic budget impacts over a one-to-five-year time horizon, and did not apply discounting. A dynamically fluctuating population and costs beyond drug costs were accounted for in 18.7% and 51.7% of studies, respectively. Input data were retrieved from published literature, clinical trials, registries, claims databases, expert opinions, historical data and market research. Assumptions were mostly made about population size and intervention/comparator(s) market uptake, but these assumptions were rarely justified and their impact was insufficiently explored through sensitivity analyses. Budget impact results were rarely validated. Conclusion: Existing budget impact analyses for orphan drugs are concise, vary greatly and are of substandard methodological quality. To eliminate possible bias in future budget impact analyses, future studies should adhere to national or ISPOR good practice guidelines on budget impact analysis.
Highlights
With health care expenditure trends surpassing economic growth rates globally (OECD, 2019; WHO, 2019), concerns about financial sustainability have surged
Budget Impact and Orphan Drugs was accentuated by incentivization of drug development for rare diseases (FDA, 1983; Official Journal of the European Communities, 2000); in 1999, the European Commission (EC) adopted the Orphan Regulation for drugs used in the prevention, diagnosis and treatment of chronically debilitating and lifethreatening diseases with low prevalence of five in 10,000 (Official Journal of the European Communities, 2000)
We systematically identify budget impact analysis (BIA) of orphan drugs in the literature with the aim of assessing the methodological quality of the reported analyses
Summary
With health care expenditure trends surpassing economic growth rates globally (OECD, 2019; WHO, 2019), concerns about financial sustainability have surged. Orphan drug expenditure grew 16% compared to only 3% growth in total pharmaceutical expenditure from 2001 to 2017 (MestreFerrandiz et al, 2019) This rise in orphan drug expenditure instigated funding challenges revealing inequity in access to orphan drugs amongst lower-income and higher-income EU countries (Szegedi et al, 2018). Public pharmaceutical expenditure on orphan drugs ranged from 2.25% to 6.51% in, respectively, the Czech Republic and Belgium (Szegedi et al, 2018). It has been of political priority for many countries to address the ascending pressure on health care budgets in order to maintain market access of qualitative interventions within their fiscal constraints
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
