in study design and the availability of evidence for value demonstration. Systematic reviews and retrospective database studies investigating the efficacy and safety of existing therapies or supportive care, which allow for qualitative comparisons against the new therapy, were generally expected by most of the respondents. Cost-effectiveness analysis was required in some of the countries, with the rest expecting only a budget-impact analysis based on local epidemiological data. Findings were similar for therapies for diseases with low prevalence but without orphan drug designation. CONCLUSIONS: Unmet needs in rare diseases are high, and effective new therapies are welcomed and valued by payers in these key reimbursed markets in Asia. Decision makers are willing to show a degree of flexibility in their evidence requirements for these kinds of products.
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