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.
EMA ran a pilot project between March 2014 and August 2016 to explore the practical implications of the adaptive pathways concept with medicines under development.
This pilot provided 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.
EMA received 62 applications and selected 18 proposals for face-to-face meetings.
At the end of the pilot, 6 of the applicants had received parallel advice from EMA and HTA bodies and 1 benefited from EMA scientific advice.
EMA published a final report on lessons learned in August 2016:
For more information on the pilot, 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
Follow up of pilot project
EMA is exploring the adaptive pathways concept further in the context of parallel scientific advice with HTA bodies, with the inclusion of additional stakeholders, such as patients and payer organisations.
Additional support is available to eligible development programmes in an additional pre-submission meeting prior to the one foreseen by the parallel scientific advice procedure, allowing companies to discuss their options and ideas before drafting the protocols that will be the subject of scientific advice.
Micro, small and medium-sized enterprises may have two additional pre-submission meetings.
Medicine developers interested in taking this approach may submit a proposal to EMA following the guidance published in August 2016:
Stakeholder workshop (updated)
In December 2016, EMA, in collaboration with the European Commission, hosted a workshop to discuss the adaptive pathways concept with stakeholders in light of practical experience gained during the pilot project and to plan the next steps in exploring the concept.
EMA has published a workshop report, highlighting stakeholder's views and proposals on the adaptive pathways approach.
EMA and the European Commission will explore ways to integrate these proposals and address concerns within the existing regulatory system.
The presentations and video recording are available on the event page.
Product eligibility for adaptive pathways
In parallel to EMA’s adaptive pathways 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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- Adaptive pathways workshop (8/12/2016)
- EMA hosts workshop on adaptive pathways (13/12/2016)
- Adaptive pathways: key learnings and next steps (3/8/2016)
- Accelerated development of appropriate patient therapies: a sustainable, multi-stakeholder approach from research to treatment outcomes (ADAPT SMART project) kick-off meeting (4/9/2015)
- Support for early access