Long-term real-world evidence

What years of registry data add to the trials

Randomized trials test an effect over months; observational cohorts show what happens over years. For EPP, several cohorts have followed afamelanotide patients well beyond the trial windows, and this unit summarizes what they found.

The recurring themes are quality-of-life gains, high continuation rates, and a contested hint of liver benefit, which one cohort reported and another did not find.