OBJECTIVES:

Approaches to identify and encourage new innovative, breakthrough drugs vary greatly among countries. The United States (US) Food and Drug Administration (FDA) “breakthrough-therapy” designation created in 2012 expedites testing and approval. In France, Improvement of the Medical Benefit (ASMR) valued from I-III by the French National Authority for Health (HAS) qualify a drug as innovative and influence pricing. We aimed to evaluate the consistency of FDA and HAS designations and frequency of discordant decisions.

METHODS:

We conducted a retrospective analysis of approved drug innovation classifications. We developed a dataset of 2013-2018 approvals designated as “breakthrough-therapy” by the FDA and/or innovative by HAS. New indications were excluded. All data sources are publically available from government agencies. Variables included approval year, indication, market authorization type, FDA breakthrough designation, ASMR value obtained, and other FDA programs (orphan drug, fast track, and accelerated).

RESULTS:

We identified 57 drug approvals meeting study inclusion/exclusion criteria. More than half (n=30) were indicated for cancer. In the subset of drugs (n=37) classified by FDA and assigned an ASMR value by HAS (n = 37), an innovative designation was more common in the US (78% vs 49% in France). Yet, there was substantial discordance regarding which drugs were defined as innovative. Two of every three breakthrough-therapies approved in the US were not considered as innovative in France. In French review, 44% considered innovative were not classified as breakthrough-therapy in the US. In the sample, 73% had discordant designations in FDA versus HAS.

CONCLUSIONS:

Classification of the innovativeness of new drugs led to substantially different conclusions in France compared to the US. As pharmaceutical companies are incentivized to seek breakthrough and innovative designations during market access, these mixed signals may affect their investments and thus, ultimately, both innovation and access to medicines.