International Harmonization of Expedited Programs: Challenges and Opportunities for Increasing Patient Access to Innovative Therapies

Regulatory agencies across the world, including the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Pharmaceuticals and Medical Devices Agency (PMDA) have implemented regulatory pathways to expedite the development and review of promising new therapies intended to treat serious conditions and address unmet medical need. Although these various expedited programs share common goals and certain key features, they differ in several aspects, including their respective eligibility requirements, application and decision timelines, as well as their evidentiary requirements. These differences reflect a variety of factors, including differing legal and regulatory frameworks, institutional approaches to benefit-risk assessment, and population health needs. However, these disparities may present challenges for drug sponsors seeking marketing approval across multiple jurisdictions. As regulators move toward greater harmonization in an increasingly complex and interconnected global market, it will be important to examine these expedited programs in order to determine whether variations in regulatory processes and evidentiary requirements may be inhibiting innovation or patient access to new therapies, and determine how best to address these issues.

To support further progress in this area, the Duke-Margolis Center for Health Policy convened this half-day expert workshop in order to 1) highlight stakeholder experiences with these programs 2) identify   differences across regulatory agencies that may lead to delays drug approval, and 3) discuss potential solutions for addressing these barriers.  

Funding for this conference was made possible in part by a cooperative agreement with the FDA’s Center for Drug Evaluation and Research. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.