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Eur J Cancer. 2019 Mar;110:74-85. doi: 10.1016/j.ejca.2019.01.013. Epub 2019 Feb 14.

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

Author information

1
ACCELERATE. Electronic address: andy1pearson@btinternet.com.
2
Zoé4life, Switzerland.
3
Universitair Ziekenhuis Antwerpen, Belgium.
4
Paediatric Medicines Office, Product Development Scientific Support Department, European Medicines Agency, London, UK.
5
Acerta Pharma, SF, USA.
6
Department of Paediatric Haematology and Oncology, Addenbrooke's Hospital Cambridge, UK.
7
Institute Gustave Roussy, France.
8
Children's Hospital of Philadelphia, PA, USA.
9
Paediatric Drug Development, Children and Young People's Unit, The Royal Marsden NHS Foundation Trust, London, UK; Divisions of Clinical Studies and Cancer Therapeutics, The Institute of Cancer Research, London, UK.
10
Merck & Co, Inc, Kenilworth, NJ, USA.
11
Celgene Corporation, NJ, USA.
12
Gilead Sciences International Limited, Cambridge, UK.
13
Centre for Cancer and Immunology Research, Children's National Health System, The George Washington University, Washington DC, USA.
14
Bayer Healthcare, NJ, USA.
15
Wolfson Childhood Cancer Research Centre, Northern Institute for Cancer Research, Newcastle University, UK.
16
Department of Paediatric Hematology and Oncology, Oslo University Hospital, Norway.
17
Pediatric Hematology, Oncology and BMT, University Hospital Münster, Germany.
18
Hoffman-La Roche Limited, Basel, Switzerland.
19
Unite2cure, UK.
20
ANSM, Saint-Denis, France.
21
Centre for Therapeutic Innovation in Oncology, Servier, France.
22
Debiopharm International SA, Lausanne, Switzerland.
23
Amgen Research, Munich, Germany.
24
Centrer for Global Health, NCI, NIH, USA.
25
Paediatric Oncology Reference Team, UK.
26
Christian Albrechts Universität, Kiel, Germany.
27
HCA Healthcare, London, UK.
28
University of Utah, Department of Pathology, Salt Lake City, UT, USA.
29
Janssen Research & Development, NJ, USA.
30
Novartis, NJ, USA.
31
Oncology Clinical Development, Bristol-Myers Squibb Pharma EEIG, NJ, USA.
32
Universitätsklinikum Schleswig-Holstein, Kiel, Germany.
33
Pediatric Oncology Department, University Hospital Brno, School of Medicine Masaryk University, Brno, Regional Centre for Applied Molecular Oncology, Masaryk Memorial Cancer Institute, ICRC Brno, St. Anna University Hospital Brno, Czech Republic.
34
Clinical Development, Kite Pharma, CA, USA.
35
Children's Cause for Cancer Advocacy, Washington DC, USA.
36
AbbVie Limited, North Chicago, IL, USA.
37
Office of Hematology and Oncology Products, U.S. Food and Drug Administration, MD, USA.
38
Department of Clinical Research, Gustave Roussy, Paris-Sud University, Paris, France.

Abstract

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 alkaloid-like 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.

KEYWORDS:

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

PMID:
30772656
DOI:
10.1016/j.ejca.2019.01.013

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