4th Paediatric Strategy Forum

ACCELERATE with participation of the 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.


| Expression of Interest |

Deadline: 10 January 2019

Expression of Interest for Clinicians

Expression of Interest for Industry

Expression of Interest for Parents & Patients

Expression of Interest for Regulators


| Draft Programme |

Click here for the Draft Programme



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