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Diaz Mora Prameyllawati on Going Beyond the Price Tag for Gene Therapies in Hemophilia
Dec 30, 2025, 08:22

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

Diaz Mora Prameyllawati on Going Beyond the Price Tag for Gene Therapies in Hemophilia

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bleeding disorders Caroline M.A. Mussert Diaz M. Prameyllawati Diaz Mora Prameyllawati Frank W. G. Leebeek Gene therapy Health Hematology Hemophilia Hemostasis Hemostasis Today Hester F. Lingsma Karina Meijer Marjon H. Cnossen Martijn A.H. Oude Voshaar Medicine Michiel Coppens Paul R. van der Valk Renske M.T. ten Ham SYMPHONY
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