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 language | English |
|---|---|
| Pages (from-to) | 189-194 |
| Number of pages | 6 |
| Journal | Haemophilia |
| Volume | 25 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - 1 Mar 2019 |
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Keywords
- challenges
- gene therapy
- global
- haemophilia
- roundtable
- world
Cite this
}
The 1st WFH Gene Therapy Round Table : Understanding the landscape and challenges of gene therapy for haemophilia around the world. / Pierce, Glenn F.; Coffin, Donna; Lillicrap, David; Ozelo, Margareth; Pasi, John; Rotellini, Dawn; Marquez, Safadi; Sannié, Thomas; Srivastava, Alok; den Berg, Marijke; Weill, Alain; Almeida, Antonio; Baumann, Alain; Brooker, Mark; Durante, Luisa; Naccache, Mayss; Robinson, Fiona; Bagley, Lisa; Chapin, John; Diop, Saliou; El Ekiaby, Magdy; Flume, Mathias; George, Lindsey; Grehan, Jack; Hawe, Matt; High, Katherine; Hilger, Anneliese; Jonsson, Bengt; Kim, Joanne; Kimmelman, Jonathan; Kobelt, Gisela; Lee, Mimi; Lillicrap, David; Linares, Adriana; Lozier, Jay; Mahlangu, Johnny; Makris, Michael; Marten, Klaus; Mathew, Prasad; Noone, Declan; O'Hara, Jamie; Ozelo, Margareth; Ohmori, Tsukasa; Page, David; Pasi, John; Perry, Jed; Pierce, Glenn; Hernandez, Preza; Ragni, Margaret; Rakha, Magda.
In: Haemophilia, Vol. 25, No. 2, 01.03.2019, p. 189-194.Research output: Contribution to journal › Review article
TY - JOUR
T1 - The 1st WFH Gene Therapy Round Table
T2 - Understanding the landscape and challenges of gene therapy for haemophilia around the world
AU - Pierce, Glenn F.
AU - Coffin, Donna
AU - Lillicrap, David
AU - Ozelo, Margareth
AU - Pasi, John
AU - Rotellini, Dawn
AU - Marquez, Safadi
AU - Sannié, Thomas
AU - Srivastava, Alok
AU - den Berg, Marijke
AU - Weill, Alain
AU - Almeida, Antonio
AU - Baumann, Alain
AU - Brooker, Mark
AU - Durante, Luisa
AU - Naccache, Mayss
AU - Robinson, Fiona
AU - Bagley, Lisa
AU - Chapin, John
AU - Diop, Saliou
AU - El Ekiaby, Magdy
AU - Flume, Mathias
AU - George, Lindsey
AU - Grehan, Jack
AU - Hawe, Matt
AU - High, Katherine
AU - Hilger, Anneliese
AU - Jonsson, Bengt
AU - Kim, Joanne
AU - Kimmelman, Jonathan
AU - Kobelt, Gisela
AU - Lee, Mimi
AU - Lillicrap, David
AU - Linares, Adriana
AU - Lozier, Jay
AU - Mahlangu, Johnny
AU - Makris, Michael
AU - Marten, Klaus
AU - Mathew, Prasad
AU - Noone, Declan
AU - O'Hara, Jamie
AU - Ozelo, Margareth
AU - Ohmori, Tsukasa
AU - Page, David
AU - Pasi, John
AU - Perry, Jed
AU - Pierce, Glenn
AU - Hernandez, Preza
AU - Ragni, Margaret
AU - Rakha, Magda
PY - 2019/3/1
Y1 - 2019/3/1
N2 - 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.
AB - 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.
KW - challenges
KW - gene therapy
KW - global
KW - haemophilia
KW - roundtable
KW - world
UR - http://www.scopus.com/inward/record.url?scp=85059535623&partnerID=8YFLogxK
U2 - 10.1111/hae.13673
DO - 10.1111/hae.13673
M3 - Review article
VL - 25
SP - 189
EP - 194
JO - Haemophilia
JF - Haemophilia
SN - 1351-8216
IS - 2
ER -