Medicinal Product Development for Mature B cell Malignancies in Children
13-14 November, 2017
EMAm London, UK
The second multi-stakeholder Paediatric Strategy Forum, jointly organised by ACCELERATE and the EMA, focused on mature B cell malignancies (lymphoma and leukaemia) in children. In this scientific meeting, there was 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 was to share information, in a pre-competitive setting, which would 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.
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 were presented at the Forum. 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, were reviewed. The output will be a published summary from all participants addressing the challenges and documenting the conclusions.
The number of participants was restricted and parties who wish to participate (patients/parent-representatives, academia, clinicians, industry) were asked to express their interest to participate*.
For clinicians/researchers, priority was given to those 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 was on invitation only.
If you have any questions or comments, please contact us: firstname.lastname@example.org or visit the EMA webpage.
*Please note that deadline for Expression of Interest was 30 June 2017.