The world’s first pathway for individually designed drugs

The headlines MHRAabout the autumn statement from Jeremy Hunt, the chancellor, this week focused on tax and spending. But tucked away in the written documents was news that could hone the cutting-edge of medicine. The Medicines and Health-care products Regulatory Agency () is working with Genomics England, Oxford University and Mila’s Miracle Foundation, a charity, to develop a regulatory pathway to allow one-off drugs to be designed and approved for use in individual patients in less than a year.Medicines currently travel a well-worn route to market comprising two or three phases of trials in large groups of patients. But this approach is impossible for the vast majority of genetic disorders, which are often vanishingly rare or even unique. The technology to for people with such conditions exists but the regulatory route they must take is rocky.