Navigating Expedited Regulatory Pathways in the U.S. and Europe

Breakthrough therapy designation in the U.S. and PRIority MEdicines (PRIME) in the EU are two programs intended to facilitate and expedite development and review of new drugs to address unmet medical needs.

By successfully engaging with regulatory authorities early in the clinical development process, sponsors of eligible investigational drugs can help ensure that their development plans generate the data needed to support expedited development pathways and approval of much-needed therapies for patients.

Innovative companies developing products for a serious disease or condition with unmet medical needs, including rare diseases, may face challenges with the design and execution of early pharmaceutical product development plans. The U.S. Food and Drug Administration (FDA) and European Medicines Agency

(EMA) have created pathways to encourage early engagement with regulatory authorities to support more efficient product development strategies. The ultimate goal of these expedited regulatory pathways is to achieve earlier patient access to important therapies.

In this white paper, we discuss two similar regulatory pathways – breakthrough therapy designation in the U.S. and PRIority MEdicines (PRIME) in the EU – and how sponsors that potentially qualify for these programs can successfully navigate these pathways.

Breakthrough therapy designation

Breakthrough therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious disease or condition. A serious disease or condition is defined as one associated with morbidity that has a substantial impact on day-to-day functioning. Whether a disease or condition is serious is a matter of clinical judgment based on its impact on factors such as survival, daily functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.

To read more, download the complete white paper by submitting the form on this page.