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Erschienen in: Investigational New Drugs 4/2018

17.04.2018 | PHASE III STUDIES

Unique characteristics of regulatory approval and pivotal studies of orphan anticancer drugs in Japan

verfasst von: Hiroki Nakayama, Katsura Tsukamoto

Erschienen in: Investigational New Drugs | Ausgabe 4/2018

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Summary

The approval of orphan anticancer drugs has increased, with the number exceeding that of non-orphan drugs in Japan in recent years. Although orphan anticancer drugs may have unique characteristics due to their rarity, these have not been fully characterized. We investigated anticancer drugs approved in Japan between April 2004 and November 2017 to reveal the characteristics of regulatory approval and pivotal studies on orphan anticancer drugs compared to non-orphan drugs. The median regulatory review time and number of patients in pivotal studies on orphan anticancer drugs (281.0 days [interquartile range, 263.3–336.0]; 222.5 patients [66.0–454.3]) were significantly lower than those on non-orphan drugs (353.0 days [277.0–535.5]; 521.0 patients [303.5–814.5], respectively) (P < 0.001). Phase II, non-randomized and non-controlled designs were more frequently used in pivotal studies on orphan anticancer drugs (45.9%, 41.9% and 43.2%) than non-orphan drugs (17.2%, 14.1% and 14.1%, respectively). Response rate was more commonly used as a primary endpoint in pivotal studies on orphan anticancer drugs (48.6%) than non-orphan drugs (17.2%). Indications limited by molecular features, second or later treatment line, and accelerated approval in the United States were associated with the use of response rate in orphan anticancer drug studies. In conclusion, we demonstrated that orphan anticancer drugs in Japan have unique characteristics compared to non-orphan drugs: shorter regulatory review and pivotal studies frequently using phase II, non-randomized, or non-controlled designs and response rate as a primary endpoint, with fewer patients.
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Metadaten
Titel
Unique characteristics of regulatory approval and pivotal studies of orphan anticancer drugs in Japan
verfasst von
Hiroki Nakayama
Katsura Tsukamoto
Publikationsdatum
17.04.2018
Verlag
Springer US
Erschienen in
Investigational New Drugs / Ausgabe 4/2018
Print ISSN: 0167-6997
Elektronische ISSN: 1573-0646
DOI
https://doi.org/10.1007/s10637-018-0603-z

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