The concept paper is intended to discuss the need and possibility to develop a framework for extrapolation approaches that are considered scientifically valid and reliable to support the authorization of medicines in the EU.

Extrapolation can be defined as using data from studies in a particular patient population to justify use of a drug in another group. The main rationale for the use of extrapolation is ethical, i.e. so as not to carry out unnecessary human trials. But extrapolation also enables drugmakers to carry out fewer clinical trials thus reducing drug costs and time to get to market and this is important in the development of both generics and biosimilars.

EMA wants to set up a framework in order to set out a structured approach to be followed for each extrapolation exercise to improve interactions with stakeholders and to standardize decision making across EMA committees. The framework would require drug developers to follow a systematic approach when applying for extrapolation of data to another indication or patient population and may include:

• Development of an extrapolation concept: this would build on a systematic synthesis of available data (in vitro, preclinical, clinical), and include the use of modelling and simulation approaches, to develop an explicit (quantitative) hypothesis regarding the similarity of the disease and the similarity of response to intervention between source and target populations.
• Extrapolation plan, proposing a reduced set of supportive studies in the target population in accordance with the extrapolation concept.
• Validation of the extrapolation concept by relevant emerging data (clinical data in the target population as well as in vitro, preclinical, or other population data); or, if the concept cannot be validated, update of the extrapolation concept and plan.
• Extrapolation: interpretation of the limited data in the target population in the context of information extrapolated from the source population(s).

In developing the concept paper EMA took into account feedback from industry groups: European Federation of Pharmaceutical Industries and Associations (EFPIA), European Generic medicines Association (EGA), UK’s National Institute for Health and Clinical Excellence (NICE); and biotech company such as Biogen Idec, among others.

EMA is asking for feedback on the concept paper from academics and industry, and is hoping to develop a finalized reflection paper within the next 12 months.

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