As Prescribed

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has announced its intention to introduce sweeping reforms for accelerating rare disease therapy development and approval, with the aim to position the UK as a global leader in the field. The new framework will put in place flexible licensing for rare disease therapies with adaptive trial design and a new “investigational marketing authorization.” It will also ensure a patient-centered approach with an emphasis on transparency, flexible benefit-risk thresholds, and use of real-world evidence.

Drug approval is unequivocally the linchpin to any drug development effort and, given the role of the US market in the global marketplace, the drug approval standard employed by FDA remains a perennial focus of stakeholders across the industry. In light of recent remarks from the FDA commissioner on the development of a new conditional approval pathway, we revisit FDA’s existing approval standard and the broader regulatory and policy context that has, to date, underpinned FDA’s drug approval practices and anchored the agency as the worldwide “gold standard.”