Selective Safety Data Collection in Clinical Studies of Oncology Drugs for Marketing Approval in the United States

Nobuyuki Sekine, Atsushi Aruga

Abstract


Optimization of data collection is a key issue in clinical studies of oncology drugs because it affects the workload and financial burden of the clinical infrastructure. Focusing on oncology drugs, which produce many low-grade as well as serious adverse reactions, we investigated the use of selective safety data collection in the pivotal clinical studies for marketing approval in the United States.

 

Drug labels were examined to find clinical studies that evaluated adverse events with limited data, and found ten drugs approved between 2004 and 2015 whose pivotal studies used selective rather than comprehensive safety data collection. Only three were in accordance with the 2001 FDA Guidance for Industry, "Cancer Drug and Biological Products -- Clinical Data in Marketing Applications", which suggests such selectivity when targeting a similar population to the initial approval. Two selective studies were applied to a drug's initial approval. Three adopted the Guidance criteria for safety data collection of only noting grade 4-5 hematologic and 3-5 non-hematologic toxicities.

 

No major problems caused by this approach were found in the description of medical reviews, approval letters and post-marketing revisions to the boxed warnings on labels issued by the FDA. Selective safety data collection can be an efficient approach to streamlining the procedure of clinical studies and should be considered for use in pivotal clinical studies for oncology drugs.


Keywords


oncology drug, safety data collection, Food and Drug Administration, guidance for industry, clinical study, adverse event

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References


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