Rare Diseases
Build on our experience of more than 245 rare disease studies in 96 countries to fulfill your promise of hope to millions around the world.
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Americas
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EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
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EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more
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Americas
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Asia & Oceania
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Europe
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Middle East & Africa
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Americas
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Asia & Oceania
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Europe
Europe
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EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more -
Middle East & Africa
EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more
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Real World Evidence
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VIEW ROLESArticle
Real World Evidence (RWE) Remains Essential for Rare and Ultra-Rare Disease Drug Development
May 16, 2025
Recent statements from the FDA in support of accelerating the approval pathway for ultra-rare disorders include "plausible mechanisms" rather than traditional randomized controlled trials, which are often impractical or infeasible for ultra rare disease patient populations.
This IQVIA RWS response discusses the importance of acknowledging the existing approved new products that have included real world evidence (RWE) for rare diseases, and explains how successful use cases of real world data (RWD) and RWE will continue to support life sciences and patient advocacy partners in advocating for patients and the accelerated approval of more treatments.
Related solutions
Patient Organizations
Incorporate the patient voice in real world studies to improve outcomes for patients and caregivers