Scientific and political changes will make adaptive pathways the preferred approach to making new treatments available to patients, according to a leading group of scientists.
The concept of adaptive pathways – previously known at the European Medicines Agency (EMA) as adaptive licensing – is based on 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 (MA) to expand access to broader patient populations, based on data gathered from its use and additional studies.
Writing in the journal Clinical Pharmacology and Therapeutics, the scientists analyse the key drivers of adaptive licensing. These include: 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 respond better 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 more targeted use of medicines to increase their therapeutic value; and the pressure on industry to make the development of medicines sustainable, in particular for chronic diseases. Development programmes targeting smaller, better-defined populations would lower the threshold for financing a drug’s development and allow for more medicines to be brought forward, say the authors, who are part of the New Drug Development Paradigm (NEWDIGS) and include EMA members.
In March 2014, EMA launched a pilot project on what was then known as adaptive licensing to explore this approach with real medicines in development. As of last month, the pilot had received and assessed 29 applications, nine of which had been selected for discussion with the applicant.
Stage I of the pilot will close at end-February 2015. The Agency will then focus on Stage II, which will include in-depth, face-to-face meetings with applicants for the projects selected. EMA will also still consider new applications for Stage II face-to-face meetings if they are well-developed.
EMA says it has changed the name of its pilot project from adaptive licensing to adaptive pathways “to better reflect the idea of a lifespan approach to bring new medicines to patients with clinical drug development, licensing, reimbursement and utilisation in clinical practice, and monitoring viewed as a continuum.”