A detailed analysis of expedited regulatory review time of marketing authorization applications for new anticancer drugs in the US and EU

The aim of this study was to assess the effect of expedited regulatory approval programs used by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), type of product (small molecule or biotechnology‐derived product) and consulting scientific advisory committees on the regulatory review time of the marketing authorization applications (MAAs) for new anticancer drugs. A dataset composed of 76 new anticancer drugs was constructed. The date of submission of the MAAs in the United States and the European Union were comparable. The typical review time of MAAs was 136 days shorter in the United States (201 days [median]) than in the European Union (337 days [median]). The type of product did not have a high impact on the review time. The review time of the MAAs for drugs undergoing priority review in the United States or accelerated assessment in the European Union at the stage of review of MAA was generally shorter than that for drugs following the standard regulatory pathway. The regulatory pathway using at least one expedited regulatory program at the stages of drug development, review of MAA, and approval of drug in the United States (172 days [median]), and that at the stages of review of MAA and approval of drug in the European Union (183 days [median]) enabled the shortest review time of MAAs. Referral to advisory committee meeting increased the review time of MAAs for drugs undergoing one or more expedited regulatory approval programs in the United States and the European Union close to that for drugs undergoing the standard regulatory approval pathway.

Study Highlights

• WHAT IS THE CURRENT KNOWLEDGE ON THE TOPIC?

The US Food and Drug Administration (FDA) has more expedited regulatory approval programs than the European Medicines Agency (EMA), suggested to result in earlier availability of anticancer drugs in the United States compared to in the European Union.

• WHAT QUESTION DID THIS STUDY ADDRESS?

The effect of expedited regulatory approval programs, type of product, and consulting scientific advisory committees on the regulatory review time of the marketing authorization applications (MAAs) for new anticancer drugs in the United States and the European Union.

• WHAT DOES THIS STUDY ADD TO OUR KNOWLEDGE?

It was shown that the review of MAAs for new anticancer drugs was finalized typically 136 days later and expedited regulatory programs were less frequently employed in the European Union compared to in the United States. The regulatory pathways leading to shortest review time of MAAs for new anticancer drugs were identified.

• HOW MIGHT THIS CHANGE CLINICAL PHARMACOLOGY OR TRANSLATIONAL SCIENCE?

A field of improvement for the regulatory framework in the European Union to enable earlier drug availability was indicated. Insight for the industry into combinations of expedited regulatory approval programs advantageous to apply for was provided.