03/06/2026
Orphan drugs force HTA systems into their hardest trade-off: urgent need vs. limited evidence.
In rare diseases, uncertainty is not a “problem to hide”, it’s a feature of the context. Small samples, short follow-up, and imperfect comparators are often unavoidable. But what is avoidable is letting that uncertainty become vague, unstructured, and therefore easy to dismiss.
The most credible dossiers don’t pretend the data is stronger than it is. They do something far more persuasive: they make uncertainty measurable, explainable, and governable. That’s how you build confidence, not by promising certainty, but by demonstrating decision quality and a credible path to learn fast after access.