Panel Access and Pricing track
How Should Evidence be Assessed in Rare Diseases?
Wed, Nov 11, 2026 · 8:00–9:00 AM CET Hall E1, Level 0, Austria Center Vienna
About this session
Issue: Assessing treatments for rare diseases presents fundamental challenges for HTA. Small populations with systemic, evolving, life-limiting diseases with a range of symptoms make it difficult to plan and execute sufficiently large randomized-controlled trials (RCTs). As a result, evidence is typically generated through small, short-term RCTs or single-arm trials, with outcomes that may not best reflect patient benefit.
Speakers
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Thermo Fisher Scientific
Panelist
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Tufts Center for the Evaluation of Value and Risk in Health
Panelist
