The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world

Glenn F. Pierce, Donna Coffin, David Lillicrap, Margareth Ozelo, John Pasi, Dawn Rotellini, Safadi Marquez, Thomas Sannié, Alok Srivastava, Marijke den Berg, Alain Weill, Antonio Almeida, Alain Baumann, Mark Brooker, Luisa Durante, Mayss Naccache, Fiona Robinson, Lisa Bagley, John Chapin, Saliou DiopMagdy El Ekiaby, Mathias Flume, Lindsey George, Jack Grehan, Matt Hawe, Katherine High, Anneliese Hilger, Bengt Jonsson, Joanne Kim, Jonathan Kimmelman, Gisela Kobelt, Mimi Lee, David Lillicrap, Adriana Linares, Jay Lozier, Johnny Mahlangu, Michael Makris, Klaus Marten, Prasad Mathew, Declan Noone, Jamie O'Hara, Margareth Ozelo, Tsukasa Ohmori, David Page, John Pasi, Jed Perry, Glenn Pierce, Preza Hernandez, Margaret Ragni, Magda Rakha

Research output: Contribution to journalReview article

12 Citations (Scopus)

Abstract

In this first in a series of round table meetings, the 1st World Federation of Hemophilia Gene Therapy Round Table was convened to initiate a global dialogue on the expected challenges and opportunities that a disruptive therapy, such as gene therapy, will bring to the haemophilia community. Perspectives from key stakeholder groups, including healthcare professionals, regulators, payors, people with hemophilia and pharmaceutical industry representatives, were sought in the identification of the key issues we expect to face. Didactic presentations and open discussion covered the clinical development of gene therapy in haemophilia; regulatory perspectives of gene therapy; making informed decisions; accessibility, affordability and pricing of gene therapy; and ethical issues of gene therapy clinical trials. These were followed by small group work. This manuscript outlines the key issues identified and the path forward.

Original languageEnglish
Pages (from-to)189-194
Number of pages6
JournalHaemophilia
Volume25
Issue number2
DOIs
Publication statusPublished - 1 Mar 2019

Keywords

  • challenges
  • gene therapy
  • global
  • haemophilia
  • roundtable
  • world

Fingerprint Dive into the research topics of 'The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world'. Together they form a unique fingerprint.

Cite this