En route to Adaptive Pathways

Drug development is an increasingly expensive and time-consuming business. More and more data are needed to support a successful approval application. But despite these efforts, the rate of newly approved, innovative drugs and support of patients with unmet medical needs remains flat, says Hans-Georg Eichler. Therefore, the Senior Medical Officer of the European Medicines Agency (EMA) no longer believes in the single “magic moment” between nonapproval and approval, when suddenly enough efficacy and safety data are gathered to allow a drug’s marketing.

This is, in brief, why Eichler initiated EMA’s “Medicines Adaptive Pathways to Patients” (MAPPs) pilot project with the aim to allow a “progressive management and reduction of uncertainty.” Drug candidates addressing urgent medical needs could gain an early, conditional approval based on preliminary phase II randomised clinical trial (RCT) data, but need to provide additional post-approval, so called “real-world” data about any treated patients – either via additional RCTs or via observational studies based on registers. The MAPPs conditional approval is restricted to a biomarker or otherwise defined small patient population. Any further enlargement of this treatment group requires additional data. 

So far, since March 2014, 59 applications have been filed for MAPPs, 11 were selected for the ongoing pilot phase. Public details about the projects and applicants are rare. The EMA has a so-called “safe harbour”, confidentiality policy to ensure fruitful discussions between companies, regulatory authorities and Health Technology Assessment Institutions (HTA). Nevertheless, it’s already public that five projects target orphan diseases, three address cancer. At least four applicants are small biotech companies, whereas it is uncertain, whether any bigger pharmaceutical companies are among the applicants – although Paul McCleverty, Senior Director and Head of Regulatory Oncology at Belgian Janssen Pharmaceuticals publicly spoke about his experiences with MAPPs, recently. 

Read the full background on the EMA’s accelerated route to approval in our print magazine!

• What is the EMA’s “Medicines Adaptive Pathways to Patients”?
• Which treatments qualify?
• Past experiences with conditional approval
• Expert opinion