EMA reports on its adaptive pathways pilot project

• The Agency will organise a workshop in December to further explore the concept


• Adaptive pathways are a planned, progressive approach to bringing a new medicine to patients


• The programme needs to involve patients more in assisting in the selection of drug candidates

The European Medicines Agency (EMA) has published a final report on its experience from its pilot project on adaptive pathways.

The majority of the 62 applications for the pilot were rejected, and the Agency said some aspects of the programme need to be further developed.

Adaptive pathways are a planned, progressive approach to bringing a new medicine to patients.

They are not a new route to marketing authorisation, but make use of existing approval tools.

Using this approach, a drug will first be authorised in a small group of patients who will benefit most from it.

Additional evidence will then be gathered over time, resulting in licensing adaptations to extend or restrict the previously authorised indications of the drug to increasingly wider patient populations.

The EMA said this concept of medicine development and data gathering is not meant to apply to all medicines, but only to those that are likely to address an unmet medical need.

This concept of medicine development and data gathering is not meant to apply to all medicines

Going forward, the Agency said the adaptive pathways programme needs to involve patients more in assisting in the selection of drug candidates.

It also needs to have 'methodologically-sound strategies of real-world evidence collection' to support the assessment of the efficacy and effectiveness of the medicine.

The Agency said it will 'further explore adaptive pathways in the context of its parallel advice with HTA bodies, which provides a framework to include additional stakeholders (e.g. patients and, if relevant, payers)'.

Drug developers who are interested in following the adaptive pathways approach may still submit a proposal to EMA and an updated guidance document outlines the steps to follow.

EMA launched the pilot in March 2014 to explore the practical implications of the adaptive pathways concept with medicines already under development.

To gather the views and proposals from interested parties on the adaptive pathways approach, the Agency will organise a workshop on 8 December.