The UK will allow compassionate use of unlicensed drugs under a new program launched in April by the Medicines and Healthcare products Regulatory Agency. The Early Access to Medicines Scheme is similar to the US breakthrough therapy designation in that it is intended to help fast-track drugs for life-threatening or seriously debilitating diseases with no adequate treatment options. But the UK scheme goes one step further than its US counterpart, by allowing doctors to prescribe drugs still in phase 2 or 3 testing if the agency believes there is a positive benefit-risk balance. One major concern over this scheme is lack of funding. With no government support to provide drugs to patients in the National Health Service (NHS) for free, companies will have to make an upfront investment to participate. “Without centrally funded reimbursement the early access scheme risks being under-utilized,” says Steve Bates, CEO of the UK BioIndustry Association. A similar program exists in France, the cohort Authorized Temporary Use program. But the French government pays for compounds used in the program. The UK's scheme begins with companies submitting an application for a Promising Innovative Medicine designation. Once such a designation is obtained, products will be channeled through a new, collaborative appraisal by the National Institute of Health and Care Excellence (NICE) and a new commissioning scheme in the NHS. For small companies, manufacturing the novel drug and meeting demand may be problematic. Early access programs are also risky because a drug might be killed if it is not effective in seriously ill patients or causes serious side effects. But Bates points out that these schemes are aimed at drug developers already operating in challenging areas, such as rare diseases and gene therapy. “I wouldn't expect everyone to be interested.” A few days before the UK scheme was announced, the European Medicines Agency launched a pilot project designed to give early approval to products still in development that address an unmet need in restricted patient groups. The principle behind this adaptive licensing pilot is that early phases of data gathering would eventually allow the license to be expanded to different categories of patients. “Adaptive licensing is part of the [early access scheme] mix,” says Bates. “It goes with the grain of thinking that as you accumulate evidence you get a license to do more trials.”