https://www.selleckchem.com/pr....oducts/gsk2830371.ht
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
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