EMA releases paper on using adult medicine data to develop child medicines

The European Medicines Agency (EMA) has released a paper that proposes how pharmaceutical companies and regulators could accept extrapolated data from adult subjects when developing pharmaceuticals targeted at children. Its guidance is designed to 'ensure harmonised and consistent decision making along the product development lifecycle regarding the use of extrapolation in paediatric population,' said the report’s conclusion.

'This should result in more rational, consistent, and more efficient paediatric drug development, and a better targeting of paediatric needs,' it added.

The paper stressed that such extrapolation was important, because the number of children available for clinical trials is often restricted, with many paediatric diseases being rare; tests often focusing on specific age groups; problems in securing study subjects’ consent for participating in tests; and more. But despite this, the report said 'accepting and implementing extrapolation for medicine development is challenging for many reasons.'

For instance, it is difficult to predict age-related differences in drug efficacy and safety. So, pharmaceutical firms and regulators need to develop a systematic inventory and qualification of paediatric medicine data extrapolation tools, it said. These would cover in vitro models, animal models, biomarkers, endpoints, experimental designs, analytical assays, data analysis tools, systems pharmacology approaches, and better in silico tools.

Also needed are criteria to assess the quality of available data; standardised methods and decision criteria for extrapolation; and strategies to manage uncertainty and risk.

'Development of a medicine in adults will present a rich source of data and understanding that can inform the design of a paediatric programme which may potentially allow a reduction in paediatric data requirements…without reducing evidentiary standards,' suggested the paper.