December 12th, 2014

European Medicines Agency

Scientists argue that scientific and political changes will make adaptive pathways the preferred approach to make new treatments available

“Adaptive pathways should be the preferred approach in the near future to bring new medicines to patients.” A number of scientists, including members of the European Medicines Agency (EMA) and its scientific Committees take this position in a co-authored article published in Clinical Pharmacology and Therapeutics.

The concept of adaptive pathways foresees an early approval of a medicine for a restricted patient population based on small initial clinical studies. The first approval is followed by progressive adaptations of the marketing authorisation to expand access to the medicine to broader patient populations based on data gathered from its use and additional studies.

Under the header ‘From adaptive licensing to adaptive pathways: delivering a flexible life-span approach to bring new drugs to patients‘, the authors, who are part of the New Drug Development Paradigm (NEWDIGS) initiative, analyse the key drivers of adaptive licensing. These include:

  • The patients’ demand for timely access to medicines, in particular where there are unmet medical needs. With adaptive licensing, new treatments would be made available to some patients earlier, on a smaller evidence base, if efficacy has been observed in this patient population.
  • A better understanding of pathologies which has led to the identification of subgroups of patients who are likely to better respond to certain medicines than others. For many of these subgroups, a progressive approach to licensing while learning from real-world experience may become the only viable access route to new treatments.
  • The growing financial pressure on healthcare systems and a call for a more targeted use of medicines to increase their therapeutic value.
  • The pressure on industry to make the development of medicines, in particular for chronic diseases, sustainable. Development programs targeting smaller, better defined populations would lower the threshold for financing a drug’s development and allow for more medicines to be brought forward.

A number of recent developments are fostering the transition from a traditional approach, which implies large trials and a marketing authorisation for broad groups of patients, to an adaptive approach. These include the development of innovative clinical trial designs, learning healthcare systems and the inclusion of patients in decision-making processes to better understand what level of uncertainty they are willing to accept.

EMA adaptive pathways pilot project

EMA launched a pilot project on adaptive pathways (formerly known as adaptive licensing) in March 2014 to explore this approach with real medicines in development.

As of November 2014, the Agency had received and assessed 29 applications as part of the pilot, nine of which had been selected for discussion with the applicant.

Stage I of the pilot project will close at the end of February 2015. The Agency will then focus on stage II of the project. This will include in-depth, face-to-face meetings with the applicants for the applications selected.

After 28 February 2015, EMA will still consider new applications for stage II face-to-face meetings if they are well-developed. Applicants are invited to contact EMA at for advice on the content and suitability of their request to be considered for stage II of the pilot.

EMA is planning to publish a report on initial experience gained as part of the pilot project by the end of 2014.

EMA recently changed the name of its pilot project from adaptive licensing to adaptive pathways to better reflect the idea of a life-span approach to bring new medicines to patients with clinical drug development, licensing, reimbursement, and utilisation in clinical practice, and monitoring viewed as a continuum.