EMA Proposes the Use of Data from Adults in Pediatric Drugs

The European Medicines Agency (EMA) has published a draft reflection paper on the extrapolation of clinical data from adults to children for the authorization of pediatric drugs. The paper outlines an approach to extrapolation of scientifically sound and reliable data from adults and other pediatric populations to support the authorization of a drug. This approach is proposed “to optimize the involvement of children in clinical studies …  by predicting how a medicine may work in children and adolescents on the basis of studies conducted in adults or other pediatric populations.”

The extrapolation framework presented in the paper includes the following:

• An extrapolation concept consisting of “a systematic synthesis of all available data, including the use of modeling and simulation approaches, which aims to predict the differences with regard to pharmacokinetics/pharmacodynamics, disease progression, and clinical response to treatment between adults and children.”

• An extrapolation plan that “aims to propose optimal studies in the pediatric population in line with predictions identified by the extrapolation concept.”

• A confirmation and extrapolation phase “to confirm the extrapolation concept on the basis of the data collected in children and adults. If the extrapolation concept cannot be fully confirmed, it should be updated and the extrapolation plan revised accordingly.”

• A plan to mitigate uncertainty and risk-“the limited data generated in the pediatric population may not be sufficient to resolve all uncertainties and assumptions of the extrapolation concept by the time of marketing authorization. Additional follow-up data may be necessary to address uncertainties and to further evaluate assumptions. Measures to generate these data need to be proposed.”

An EMA workshop on the extrapolation of data will be held May 17–18, 2016 to gather expert and stakeholder views. The draft reflection paper is scheduled to be released for public consultation at the end of July 2016.

Source: EMA