Wolfgang Miesbach: Why Patient-Reported Outcomes (PROs) Are Essential for Hemophilia Gene Therapy
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about his recent article published in PubMed, adding:
“Thrilled that this new open-access review, ‘Patient-Reported Outcomes in Haemophilia Gene Therapy,’ is out.
Three AAV gene therapies approved in the West since 2022, yet uptake has been slow — Beqvez discontinued, Roctavian withdrawn.
PRO evidence can help bridge the gap between trial efficacy and real-world adoption by showing what gene therapy means for everyday life.
Bleeding rates capture efficacy, but not what patients experience. Across six trials and 12 instruments, the signal is consistent:
Disease-specific QoL improved 6–15 points, beyond clinically important thresholds.
The new coreHEM-MHO measures what older tools can’t — durability anxiety, disease identity adjustment
Cost-effectiveness rests almost entirely on these QoL gains; life-year gains are negligible
The paradox: instruments built two decades ago can penalise patients who no longer bleed, because ‘not applicable’ isn’t an option.
We’re underestimating the very benefit we’re trying to prove.
Priorities: standardised coreHEM measurement, long-term registries (WFH GTR, EAHAD-GTD), and certified centres equipped for PRO follow-up.
With the FDA and EMA now recognising PROs in regulatory and HTA decisions, and the economic case resting on quality of life, we owe it to patients to measure it properly.”
Title: Overview of Patient-Reported Outcomes in Haemophilia Gene Therapy Open Access
Authors: Wolfgang Miesbach
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