Paediatric Strategy Forum

Medicinal Product Development for Mature B cell Malignancies in Children


November 13 and 14, 2017

EMA London, UK


Later deadline for industry application.


The second multi-stakeholder Paediatric Strategy Forum, jointly organized by ACCELERATE and the EMA, will focus on mature B cell malignancies (lymphoma and leukaemia) in children. In this scientific meeting there will be interaction between all stakeholders (patients/patient representatives, clinicians, academics, pharmaceutical companies, and regulators) with an interest in drug development for children and adolescents with cancer. The goal of this meeting is to share information, in a pre-competitive setting, which will inform a paediatric drug development strategy and subsequent decisions and facilitate the developments of innovative medicines for the treatment of children with mature B cell malignancies.

The summary of the first Paediatric Strategy Forum held in January 2017 on ALK inhibition in paediatric malignancies can be found here.

The current therapy for high-risk mature B-cell malignancies with multi-agent chemotherapy and rituximab was evaluated in an international trial in Europe, the US and Asia (Inter B NHL Ritux 2010). The trial resulted in one year event free survival (EFS) rates (95%CI) of 94.2% (88.5% – 97.2 %), which is similar to other first-line protocols. Chemotherapy alone results in greater than 96% EFS for patients with a standard-risk disease. The acute toxicity is significant but most survivors have no or mild long term toxicity.

Therefore the current unmet therapeutic needs for mature B cell malignancies in children are:

i) to reduce the high acute toxicity of current therapy; however further reduction of intensive therapy leads to reduced rates of cure;

ii) to develop innovative treatments for patients remaining incurable.

There are a number of medicines being developed for B cell malignancies in adults; however most of the malignancies in adults differ from those in children. Therefore the challenges are:

i) To identify which of the many potential new drugs will have a) the optimal probability of improving cure rates in those patients who currently have chemo resistant disease and b) the optimal success in reducing toxicity in the majority of patients

ii) To design and execute scientifically sound studies in very small populations with refractory or relapsed mature B cell malignancies

iii) To design and execute scientifically sound and ethical studies in populations to reduce toxicity with a very high rate of cure.

In the Forum the epidemiology, clinical features, biology, similarities and differences compared to adult B-cell malignancies, current international standard approaches and therapeutic needs of mature B cell malignancies will be presented. The medicines for B cell malignancies in development, relevant pre-clinical data and data from all paediatric clinical trials completed or in progress, sponsored by industry or academia, will be reviewed. The output will be a published summary from all participants addressing the challenges and documenting the conclusions.

The number of participants is restricted and parties who wish to participate (patients/parent-representatives, academia, clinicians, industry) are asked to express their interest to participate*.

For clinicians/researchers priority will be given who have expertise and experience in clinical trials for mature B cell malignancies in children or intend to design studies in the future. Participation in the Forum will be on invitation only.


If you have any questions or comments please contact us: or visit EMA webpage.

*Please note that deadline for Expression of Interest was 30th June 2017.

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