The adaptive pathways approach is part of the European Medicines Agency’s (EMA) efforts to improve timely access for patients to new medicines. Adaptive pathways is a scientific concept for medicine development and data generation which allows for early and progressive patient access to a medicine. The approach makes use of the existing European Union (EU) regulatory framework for medicines.
Adaptive pathways is based on three principles:
- iterative development, which either means:
- approval in stages, beginning with a restricted patient population then expanding to wider patient populations;
- confirming the benefit-risk balance of a product, following a conditional approval based on early data (using surrogate endpoints) considered predictive of important clinical outcomes;
- gathering evidence through real-life use to supplement clinical trial data;
- early involvement of patients and health-technology-assessment bodies in discussions on a medicine’s development.
This concept applies primarily to treatments in areas of high medical need where it is difficult to collect data via traditional routes and where large clinical trials would unnecessarily expose patients who are unlikely to benefit from the medicine.
The approach builds on regulatory processes already in place within the existing EU legal framework. These include:
- scientific advice;
- compassionate use;
- the conditional approval mechanism (for medicines addressing life-threatening conditions);
- patient registries and other pharmacovigilance tools that allow collection of real-life data and development of the risk-management plan for each medicine.
Adaptive pathways does not change the standards for the evaluation of benefits and risks or the requirement to demonstrate a positive benefit-risk balance to obtain marketing authorisation.
Clarifying the adaptive pathways concept (updated)
EMA has provided a comprehensive response to a letter from a group of nine professors and scientists on the adaptive pathways approach, in which the authors make a number of comments and suggestions. EMA's response further clarifies the concept of adaptive pathways:
The Agency values contributions from stakeholders on its initiatives. Feedback and open debate are essential for adapting and fine tuning concepts and approaches and ensuring that they meet stakeholders’ expectations.
In March 2014 EMA launched a pilot project to explore how the adaptive pathways approach might work in the existing regulatory framework with real medicines in development.
This ongoing pilot provides a framework for informal dialogue between stakeholders, including patients and health-technology-assessment bodies, to explore different options in a 'safe harbour' environment and consider detailed technical and scientific questions based on concrete examples.
Scientific advice given on individual medicines remains confidential in this very early stage of product development, as normal.
Status of pilot project
EMA is still accepting applications for an adaptive pathways meeting with health-technology-assessment bodies if they are well-developed.
EMA will publish a final report on the pilot once six products have undergone parallel scientific advice from EMA and health-technology-assessment bodies. The report is expected to be published in the course of 2016.
For guidance on the submission process, criteria and examples, see:
Applicants may contact EMA at email@example.com for advice on the content and suitability of their request to be considered for the pilot.
For more information on the pilot project, see:
- Pilot project on adaptive licensing
- Adaptive Pathways to patients: Report on the initial experience of the pilot project
- Questions and answers following the initial experience of the pilot project
In parallel to EMA’s adaptive pathways pilot project, the Innovative Medicines Initiative (IMI) runs ADAPT-SMART, a project investigating the conceptual framework that could be used in adaptive pathways, including tools and methodologies.
The IMI is a European public-private collaboration bringing together 32 participants for which EMA is the scientific leader.
EMA organised an ADAPT-SMART kick-off meeting in September 2015, which was attended by patient organisations, health-technology-assessment bodies, regulators, payers, academia and industry.
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To initiate a pilot case: