4th Paediatric Strategy Forum: Acute Myeloid Leukaemia in Children and Adolescents

ACCELERATE in collaboration with the European Medicines Agency (EMA) with participation of the Food and Drug Administration (FDA)

Paediatric Strategy Forum for Medicinal Product Development for Acute Myeloid Leukaemia in Children and Adolescents

11-12 April 2019

Rotterdam, Netherlands

| Background and objectives | 

The fourth multi-stakeholder Paediatric Strategy Forum will focus on medicinal product development for acute myeloid leukaemia in children and adolescents.

Paediatric Strategy Forums have been created to facilitate dialogue and provide an opportunity for constructive interactions between relevant stakeholders (parents /patients representatives, clinicians, academics, pharmaceutical companies and regulators) on topics requiring an open discussion on the development of medicines in the best interests of children and adolescents with cancer.  The goal of this meeting is to share information, in a pre-competitive setting, to facilitate the developments of innovative medicines and ultimately their introduction into the standard-of-care of children with malignancies.

The first three Paediatric Strategy Forums were on anaplastic lymphoma kinase inhibition held in January 2017, on mature B-cell malignancies in November 2017 and immune checkpoint inhibitor combinations in September 2018

There are a number of challenges in developing new medicinal products for children and adolescents with acute myeloid leukaemia. These include the relatively small population of children with acute myeloid leukaemia compared to adults; the different therapeutic approach and better outcomes in paediatrics compared to AML in adults; the various molecular sub-types, and the number of new medicines in some classes.  An additional challenge is that there is attrition with some drugs, which may be effective in children, due to a different risk-benefit profile than in the elderly where they are often first explored.  These challenges become even more complex in the context of different regulatory frameworks.

Within this context, some of the key questions addressed in the Paediatric Strategy Forum will include:

  • The challenges of clinical trials of new drugs for children with acute myeloid leukaemia.
  • How to make the best choices of innovative medicines?
  • How can extrapolation of data from adults to children be best employed?
  • How to coordinate paediatric development plans of novel agents to accommodate global development plans in the context of international academic clinical trial networks?
  • When is it appropriate to evaluate new drugs in children with acute myeloid leukaemia in first relapse, second relapse and/or front line?

The epidemiology, biology, standard therapy, therapeutic needs and future therapeutic plans in North America and Europe for acute myeloid leukemia in children and adolescents will be discussed at this Forum.  This will be followed by presentations of non-clinical and clinical information on medicinal products being developed by pharmaceutical companies.

The output will be a published summary and manuscript from all participants addressing the challenges and documenting the conclusions.

| The deadline for the Expression of Interest was 20 January 2019 |

| Short Programme |

Click here for the Short Programme

| Summary |

Click here for the Summary of the Forum


Supported by Sohn Conference Foundation
The 4th Paediatric Strategy Forum is supported by Sohn Monaco via the Sohn Conference Foundation, which is dedicated to the treatment and cure of pediatric cancer. The Sohn Conference Foundation supports cutting-edge medical research, state-of-the-art research equipment, and innovative programs to ensure that children with cancer survive and thrive.


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