Expedited Approval in Oncology: A Study of European Regulators' Perspectives and Trade-Offs

Abstract

The purpose of this study was to describe the perspectives and trade-offs of EU regulators about expedited approvals for treatments of patients with advanced cancer. The study consisted of a cross-sectional survey with assessors and experts from the network of the European Medicines Agency (EMA). First, general attitudes were collected using a 5-point Likert scale. Second, a conjoint analysis was used to assess the compatibility of “conditional approval” for different scenarios of response rate, response duration, and toxicity from a single-arm trial, and timeliness of confirmatory data. The survey was completed by 60 participants out of 351 invited participants. The majority agreed that outstanding activity is indicative of efficacy, that toxicity is a concern, and that confirmatory trials should be ongoing at the time of approval. The majority also agreed that conditional approval is not well understood by patients and doctors. Opinions varied on the usefulness of external comparator studies. Higher antitumour activity could outweigh higher toxicity or longer time to confirmation. Based on confidence with expedited pathways and evidence generation, participants could be categorized as “supportive,” “pragmatic,” and “cautious.” The importance of toxicity and timeliness of confirmatory studies was similar between groups but varied for antitumour activity. This study highlighted different attitudes when balancing speed of access with incomplete evidence in situations of high unmet medical needs. Confidence in expedited pathways and early evidence varied. There is an opportunity for improving communication and better alignment on evidentiary standards for expedited approvals.