Diaz Mora Prameyllawati on Going Beyond the Price Tag for Gene Therapies in Hemophilia
Diaz Mora Prameyllawati, PhD Candidate at Erasmus MC, shared on LinkedIn:
”Beyond the price tag of gene therapy
Gene therapies are often discussed in terms of their high price, which can range from €1 to €3.5 million per individual.
In our latest study on gene therapy for hemophilia, we went beyond the price tag and focused on what it actually takes to deliver these therapies in clinical practice.
To support successful and sustainable implementation, we developed:
- A detailed process map covering all care activities from site preparation to follow-up.
- Comprehensive cost catalogs capturing every component of care, enabling other hospitals and countries to estimate their own delivery costs.
These tools aim to inform economic evaluations, reimbursement discussions, and the real-world implementation of gene therapy for hemophilia.
More details in our latest publication.
This work was made possible through the support of the SYMPHONY consortium.
I sincerely thank all participating healthcare professionals for their openness and valuable insights during the interviews, as well as my supervisors and co-authors for their guidance and collaboration throughout the study.”
Read the full article here.
Article: Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice
Authors: Diaz M. Prameyllawati, Caroline M.A. Mussert, Martijn A.H. Oude Voshaar, Hester F. Lingsma, Marjon H. Cnossen, Michiel Coppens, Karina Meijer, Paul R. van der Valk, Frank W.G. Leebeek, Renske M.T. ten Ham

Stay updated on all scientific advances with Hemostasis Today.
-
May 29, 2026, 03:19Building Safer Systems for Anticoagulation Management – ISTH
-
May 29, 2026, 03:07Early Diagnosis Can Change the Lives of Women With Haemophilia – EHC
-
May 29, 2026, 02:41Ifeanyichukwu Ifechidere: The Biggest DOAC Myth in Coagulation – ‘No Monitoring Required’
-
May 29, 2026, 02:35Mariam Swidan: Why Is LDH the ‘Tattletale’ of the Lab?
-
May 29, 2026, 02:20Yuntian Wang: Multi-View Learning for Predicting Avatrombopag Response in Pediatric Immune Thrombocytopenia
-
May 29, 2026, 01:57Giuseppina Caligiuri: Pathophysiological Challenges and Emerging Therapeutic Strategies in Acute Stroke
-
May 28, 2026, 23:47Hussnain Javaid: The Importance of Targeted Examination in Iron Deficiency Anaemia
-
May 28, 2026, 23:47ASH Heads to ASCO26 with Career Opportunities and Global Hematology Leadership
-
May 28, 2026, 23:46Armenia Takes Center Stage in Global Hemophilia Initiative – Yeolyan Hematology and Oncology Center