In December, Michael Rawlins was appointed head of the U.K.’s Medicines and Healthcare products Regulatory Agency. He recognizes the MHRA’s mutually beneficial relationship with European counterparts and is looking to safeguard synergies with other national regulatory bodies and NICE.

The EU's adaptive licensing pilot project, which was launched in March 2014, is designed to deliver an early centralized marketing license for promising innovative therapies for patients with serious or life-threatening conditions. However, while the benefits are clear, there are a number of challenges that will need to be addressed.

A single-arm trial using a biomarker and animal model could be enough for an accelerated approval, especially in a pediatric rare disease where randomization and placebo controls may not be feasible, the CBER director said.