Workshop on adaptive pathways - discussing a concept for development of medicines addressing unmet medical needs

Registration is now open

The European Medicines Agency (EMA) in collaboration with the European Commission is organising a workshop on 8 December 2016 to gather the views of stakeholders on the adaptive pathways approach.

Stakeholders who are interested in participating in this event can register their interest by sending their completed registration form to apworkshop@ema.europa.eu by 1 November 2016. Due to a limited number of spaces, EMA will only be able to accept one participant per organisation to allow attendance of a wide range of stakeholders. The workshop will be broadcast live on the EMA website.

The workshop aims to bring together a broad range of interested parties, including representatives of healthcare professionals, patients, health technology assessment (HTA) bodies, payers, regulators and medicine developers.

In light of the practical experience gained during a pilot project which ran from March 2014 to July 2016, workshop participants will discuss the key aspects of adaptive pathways as well as the next steps to further explore the concept. Topics to be discussed include:

• patients' and healthcare professionals' perspectives on addressing patients' needs through adaptive pathways;
• knowledge generation throughout a medicine's lifespan;
• from bench to bed: involvement of all relevant parties in the delivery of medicines for unmet medical needs.

Adaptive pathways can be defined as a planned, progressive approach to bringing a medicine to patients. Under this approach, the medicine will first be authorised in a small patient population that is likely to benefit most from the medicine. Then, additional evidence is gathered over time resulting in progressive adaptations of marketing authorisation to extend or restrict the previously authorised indications of the medicine. Adaptive pathways can support medicine development in therapeutic areas where evidence generation is challenging, such as infectious diseases, Alzheimer's disease, degenerative diseases, and rare cancers.

Between March 2014 and July 2016, EMA conducted a pilot project to explore the practical implications of the adaptive pathways concept with medicines under development.

The pilot showed that adaptive pathways can bring multiple stakeholders together – regulators, HTA bodies, healthcare professionals and patients – to agree on a prospective plan to generate data on a medicine across its lifespan in areas of unmet medical need. The project also helped to identify a number of aspects for further reflection which are described in the report published in August 2016.

A preliminary agenda for the workshop has been published today.

Adaptive pathways: a scientific concept for the development of medicines

Adaptive pathways is not a new route of marketing authorisation. It makes use of existing approval tools, in particular conditional marketing authorisation which has been in operation in the European Union since 2006. It also builds on the experience gained with strengthened post-marketing monitoring tools introduced by the 2012 pharmacovigilance legislation. As for any medicine, a marketing authorisation will only be granted if the balance of benefits and risks for a defined patient population is found to be positive; the same principles and legal tools apply.

This concept of medicine development and data gathering is meant to apply only to medicines that are likely to address an unmet medical need. The medicine development needs to meet the characteristics of adaptive pathways: a staggered approval from very small, restricted patient populations to increasingly wider populations; a binding plan of post-authorisation evidence gathering; and involvement of key stakeholders in the process.