ACCELERATE and European Medicine Agency Paediatric Strategy
Forum for medicinal product development for mature B-cell
malignancies in children

J 2019

ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children

PEARSON, Andrew D. J., Nicole SCOBIE, Koenraad NORGA, Franca LIGAS, Davy CHIODIN et. al.

Basic information

Original name

ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children

Authors

PEARSON, Andrew D. J. (840 United States of America, guarantor), Nicole SCOBIE (756 Switzerland), Koenraad NORGA (56 Belgium), Franca LIGAS (826 United Kingdom of Great Britain and Northern Ireland), Davy CHIODIN (840 United States of America), Amos BURKE (826 United Kingdom of Great Britain and Northern Ireland), Veronique MINARD-COLIN (840 United States of America), Peter ADAMSON (826 United Kingdom of Great Britain and Northern Ireland), Lynley V. MARSHALL (840 United States of America), Arun BALAKUMARAN (840 United States of America), Bouchra BENETTAIB (840 United States of America), Pankaj BHARGAVA (826 United Kingdom of Great Britain and Northern Ireland), Catherine M. BOLLARD (840 United States of America), Ellen BOLOTIN (840 United States of America), Simon BOMKEN (826 United Kingdom of Great Britain and Northern Ireland), Jochen BUECHNER (578 Norway), Birgit BURKHARDT (276 Germany), Hubert CARON (756 Switzerland), Christopher COPLAND (756 Switzerland), Pierre DEMOLIS (250 France), Anton EGOROV (250 France), Mahdi FARHAN (756 Switzerland), Gerhard ZUGMAIER (276 Germany), Thomas GROSS (840 United States of America), Danielle HORTON-TAYLOR (826 United Kingdom of Great Britain and Northern Ireland), Wolfram KLAPPER (276 Germany), Giovanni LESA (826 United Kingdom of Great Britain and Northern Ireland), Robert MARCUS (826 United Kingdom of Great Britain and Northern Ireland), Rodney R. MILES (840 United States of America), Kerri NOTTAGE (840 United States of America), Lida PACAUD (840 United States of America), Rosanna RICAFORT (840 United States of America), Martin SCHRAPPE (276 Germany), Jaroslav ŠTĚRBA (203 Czech Republic, belonging to the institution), Remus VEZAN (840 United States of America), Susan WEINER (840 United States of America), Su Young KIM (840 United States of America), Gregory REAMAN (840 United States of America) and Gilles VASSAL (250 France)

Edition

European Journal of Cancer, Oxford, Elsevier Science Inc. 2019, 0959-8049

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30204 Oncology

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 7.275

RIV identification code

RIV/00216224:14110/19:00112994

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1016/j.ejca.2019.01.013

UT WoS

000460123000012

Keywords in English

Paediatric oncology; Mature B-cell malignancies; Medicinal product development

Abstract

V originále

Paediatric Strategy Forums have been created by the multistakeholder organisation, ACCELERATE, and the European Medicines Agency to facilitate dialogue between all relevant stakeholders and suggest strategies in critical areas of paediatric oncology drug development. As there are many medicines being developed for B-cell malignancies in adults but comparatively few in children with these malignancies, a Paediatric Strategy Forum was held to discuss the best approach to develop these products for children. It was concluded that as current frontline therapy is highly successful, despite associated acute toxicity, de-escalation of this or substitution of presently used drugs with new medicines can only be undertaken when there is an effective salvage regimen, which is currently not available. Therefore priority should be given to developing treatment for patients with relapsed and refractory mature B-cell lymphomas. The consensus of the clinicians attending the meeting was that CAR T-cells, T-cell engagers and antibody drug conjugates (excluding those with a vinca alkaloidelike drug) presently have the greatest probability of providing benefit in relapse in view of their mechanism of action. However, as producing autologous CAR T-cells currently takes at least 4 weeks, they are not products which could be quickly employed initially at relapse in rapidly progressing mature B-cell malignancies but only for the consolidation phase of the treatment. Global, industry-supported, academic-sponsored studies testing compounds from different pharmaceutical companies simultaneously should be considered in rare populations, and it was proposed that an international working group be formed to develop an overarching clinical trials strategy for these disease groups. Future Forums are planned for other relevant paediatric oncologic diseases with a high unmet medical need and relevant molecular targets. (C) 2019 Elsevier Ltd. All rights reserved.

Links

LM2015090, research and development project

Name: Český národní uzel Evropské sítě infrastruktur klinického výzkumu (Acronym: CZECRIN)

Investor: Ministry of Education, Youth and Sports of the CR

MUNI/A/1586/2018, interní kód MU

Name: Personalizovaná léčba v dětské onkologii: na cestě k "liquid dynamic medecine" a "N-of-1 clinical trials" (Acronym: Personalizovaná léčba v dětské onkologii)

Investor: Masaryk University, Category A

Citovat

PEARSON, Andrew D. J., Nicole SCOBIE, Koenraad NORGA, Franca LIGAS, Davy CHIODIN, Amos BURKE, Veronique MINARD-COLIN, Peter ADAMSON, Lynley V. MARSHALL, Arun BALAKUMARAN, Bouchra BENETTAIB, Pankaj BHARGAVA, Catherine M. BOLLARD, Ellen BOLOTIN, Simon BOMKEN, Jochen BUECHNER, Birgit BURKHARDT, Hubert CARON, Christopher COPLAND, Pierre DEMOLIS, Anton EGOROV, Mahdi FARHAN, Gerhard ZUGMAIER, Thomas GROSS, Danielle HORTON-TAYLOR, Wolfram KLAPPER, Giovanni LESA, Robert MARCUS, Rodney R. MILES, Kerri NOTTAGE, Lida PACAUD, Rosanna RICAFORT, Martin SCHRAPPE, Jaroslav ŠTĚRBA, Remus VEZAN, Susan WEINER, Su Young KIM, Gregory REAMAN and Gilles VASSAL. ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children. European Journal of Cancer. Oxford: Elsevier Science Inc., 2019, vol. 110, 1879-0852, p. 74-85. ISSN 0959-8049. Available from: https://dx.doi.org/10.1016/j.ejca.2019.01.013.

@article{1624117,
   author = {Pearson, Andrew D. J. and Scobie, Nicole and Norga, Koenraad and Ligas, Franca and Chiodin, Davy and Burke, Amos and MinardandColin, Veronique and Adamson, Peter and Marshall, Lynley V. and Balakumaran, Arun and Benettaib, Bouchra and Bhargava, Pankaj and Bollard, Catherine M. and Bolotin, Ellen and Bomken, Simon and Buechner, Jochen and Burkhardt, Birgit and Caron, Hubert and Copland, Christopher and Demolis, Pierre and Egorov, Anton and Farhan, Mahdi and Zugmaier, Gerhard and Gross, Thomas and HortonandTaylor, Danielle and Klapper, Wolfram and Lesa, Giovanni and Marcus, Robert and Miles, Rodney R. and Nottage, Kerri and Pacaud, Lida and Ricafort, Rosanna and Schrappe, Martin and Štěrba, Jaroslav and Vezan, Remus and Weiner, Susan and Kim, Su Young and Reaman, Gregory and Vassal, Gilles},
   article_location = {Oxford},
   article_number = {1879-0852},
   doi = {http://dx.doi.org/10.1016/j.ejca.2019.01.013},
   keywords = {Paediatric oncology; Mature B-cell malignancies; Medicinal product development},
   language = {eng},
   issn = {0959-8049},
   journal = {European Journal of Cancer},
   title = {ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children},
   url = {http://dx.doi.org/10.1016/j.ejca.2019.01.013},
   volume = {110},
   year = {2019}
}

TY  - JOUR
ID  - 1624117
AU  - Pearson, Andrew D. J. - Scobie, Nicole - Norga, Koenraad - Ligas, Franca - Chiodin, Davy - Burke, Amos - Minard-Colin, Veronique - Adamson, Peter - Marshall, Lynley V. - Balakumaran, Arun - Benettaib, Bouchra - Bhargava, Pankaj - Bollard, Catherine M. - Bolotin, Ellen - Bomken, Simon - Buechner, Jochen - Burkhardt, Birgit - Caron, Hubert - Copland, Christopher - Demolis, Pierre - Egorov, Anton - Farhan, Mahdi - Zugmaier, Gerhard - Gross, Thomas - Horton-Taylor, Danielle - Klapper, Wolfram - Lesa, Giovanni - Marcus, Robert - Miles, Rodney R. - Nottage, Kerri - Pacaud, Lida - Ricafort, Rosanna - Schrappe, Martin - Štěrba, Jaroslav - Vezan, Remus - Weiner, Susan - Kim, Su Young - Reaman, Gregory - Vassal, Gilles
PY  - 2019
TI  - ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children
JF  - European Journal of Cancer
VL  - 110
IS  - 1879-0852
SP  - 74-85
EP  - 74-85
PB  - Elsevier Science Inc.
SN  - 09598049
KW  - Paediatric oncology
KW  - Mature B-cell malignancies
KW  - Medicinal product development
UR  - http://dx.doi.org/10.1016/j.ejca.2019.01.013
L2  - http://dx.doi.org/10.1016/j.ejca.2019.01.013
N2  - Paediatric Strategy Forums have been created by the multistakeholder organisation, ACCELERATE, and the European Medicines Agency to facilitate dialogue between all relevant stakeholders and suggest strategies in critical areas of paediatric oncology drug development. As there are many medicines being developed for B-cell malignancies in adults but comparatively few in children with these malignancies, a Paediatric Strategy Forum was held to discuss the best approach to develop these products for children. It was concluded that as current frontline therapy is highly successful, despite associated acute toxicity, de-escalation of this or substitution of presently used drugs with new medicines can only be undertaken when there is an effective salvage regimen, which is currently not available. Therefore priority should be given to developing treatment for patients with relapsed and refractory mature B-cell lymphomas. The consensus of the clinicians attending the meeting was that CAR T-cells, T-cell engagers and antibody drug conjugates (excluding those with a vinca alkaloidelike drug) presently have the greatest probability of providing benefit in relapse in view of their mechanism of action. However, as producing autologous CAR T-cells currently takes at least 4 weeks, they are not products which could be quickly employed initially at relapse in rapidly progressing mature B-cell malignancies but only for the consolidation phase of the treatment. Global, industry-supported, academic-sponsored studies testing compounds from different pharmaceutical companies simultaneously should be considered in rare populations, and it was proposed that an international working group be formed to develop an overarching clinical trials strategy for these disease groups. Future Forums are planned for other relevant paediatric oncologic diseases with a high unmet medical need and relevant molecular targets. (C) 2019 Elsevier Ltd. All rights reserved.
ER  -

PEARSON, Andrew D. J., Nicole SCOBIE, Koenraad NORGA, Franca LIGAS, Davy CHIODIN, Amos BURKE, Veronique MINARD-COLIN, Peter ADAMSON, Lynley V. MARSHALL, Arun BALAKUMARAN, Bouchra BENETTAIB, Pankaj BHARGAVA, Catherine M. BOLLARD, Ellen BOLOTIN, Simon BOMKEN, Jochen BUECHNER, Birgit BURKHARDT, Hubert CARON, Christopher COPLAND, Pierre DEMOLIS, Anton EGOROV, Mahdi FARHAN, Gerhard ZUGMAIER, Thomas GROSS, Danielle HORTON-TAYLOR, Wolfram KLAPPER, Giovanni LESA, Robert MARCUS, Rodney R. MILES, Kerri NOTTAGE, Lida PACAUD, Rosanna RICAFORT, Martin SCHRAPPE, Jaroslav ŠTĚRBA, Remus VEZAN, Susan WEINER, Su Young KIM, Gregory REAMAN and Gilles VASSAL. ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children. \textit{European Journal of Cancer}. Oxford: Elsevier Science Inc., 2019, vol.~110, 1879-0852, p.~74-85. ISSN~0959-8049. Available from: https://dx.doi.org/10.1016/j.ejca.2019.01.013.

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