PMID- 35355238
OWN - NLM
STAT- MEDLINE
DCOM- 20220530
LR  - 20220531
IS  - 2168-4804 (Electronic)
IS  - 2168-4790 (Linking)
VI  - 56
IP  - 4
DP  - 2022 Jul
TI  - Estimation of a Suitable Number of Patients for Selective Safety Data Collection 
      (ICH E19 Draft Guideline): When is the Safety Profile of a Drug Well 
      Characterized?
PG  - 587-595
LID - 10.1007/s43441-022-00392-2 [doi]
AB  - PURPOSE: We propose methods to estimate a suitable number of patients for 
      implementing selective safety data collection (SSDC) in clinical investigations 
      based on a confidence interval of the incidence rate or risk difference using 
      Monte Carlo simulation. METHODS: The incidence rates and risk differences of 
      adverse events (AEs) were based on the safety outcome measures. A suitable number 
      of patients for implementing SSDC was estimated based on the probability that the 
      half-width of the two-sided 95% confidence interval of incidence rate or risk 
      difference was equal to or less than a pre-specified cut-off value (0.5-3.0%). 
      Monte Carlo simulation was used to estimate the suitable number of patients at 
      probabilities of 70%, 80%, and 90%. The applicability of our proposed method for 
      estimating a suitable number of patients for SSDC implementation was confirmed 
      based on the incidence rates or risk differences from actual clinical trial data 
      for panitumumab. RESULTS: We demonstrated the performance of our proposed method 
      in estimating a suitable number of patients to implement SSDC in several 
      situations. Furthermore, according to the safety datasets of three phase III 
      clinical trials, the number of suitable patients for implementing SSDC using 
      incidence rates or risk differences of common AEs with panitumumab could confirm 
      the applicability of our proposed method. CONCLUSION: A suitable number of 
      patients estimated based on our proposed method may be one of the foundations for 
      implementing SSDC, as additional data accrual may not impact the precision of the 
      estimates of the frequency of common AEs.
CI  - (c) 2022. The Drug Information Association, Inc.
FAU - Saeki, Hiroyuki
AU  - Saeki H
AUID- ORCID: 0000-0002-2230-5652
AD  - Data Science Expert Committee, Drug Evaluation Committee, Japan Pharmaceutical 
      Manufacturers Association, Tokyo, Japan. hiroyuki.saeki@fujifilm.com.
AD  - FUJIFILM Toyama Chemical Co., Ltd., 2-14-1 Kyobashi, Chuo-ku, Tokyo, 104-0031, 
      Japan. hiroyuki.saeki@fujifilm.com.
FAU - Komeda, Takuji
AU  - Komeda T
AD  - Data Science Expert Committee, Drug Evaluation Committee, Japan Pharmaceutical 
      Manufacturers Association, Tokyo, Japan.
AD  - Shionogi & Co., Ltd., Osaka, Japan.
FAU - Tanaka, Risa
AU  - Tanaka R
AD  - Data Science Expert Committee, Drug Evaluation Committee, Japan Pharmaceutical 
      Manufacturers Association, Tokyo, Japan.
AD  - Asahi Kasei Pharma Corporation, Tokyo, Japan.
FAU - Yamatani, Yuki
AU  - Yamatani Y
AD  - Data Science Expert Committee, Drug Evaluation Committee, Japan Pharmaceutical 
      Manufacturers Association, Tokyo, Japan.
AD  - Kissei Pharmaceutical Co., LTD., Tokyo, Japan.
FAU - Sakai, Hironori
AU  - Sakai H
AD  - Data Science Expert Committee, Drug Evaluation Committee, Japan Pharmaceutical 
      Manufacturers Association, Tokyo, Japan.
AD  - Eisai Co., Ltd., Tokyo, Japan.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20220330
PL  - Switzerland
TA  - Ther Innov Regul Sci
JT  - Therapeutic innovation & regulatory science
JID - 101597411
RN  - 6A901E312A (Panitumumab)
SB  - IM
MH  - Computer Simulation
MH  - Humans
MH  - Incidence
MH  - Monte Carlo Method
MH  - *Panitumumab
MH  - Probability
OTO - NOTNLM
OT  - Half-width of two-sided 95% confidence interval
OT  - Incidence rate
OT  - Risk difference
OT  - Selective safety data collection
OT  - Simulation
OT  - Suitable number of patients
EDAT- 2022/04/01 06:00
MHDA- 2022/05/31 06:00
CRDT- 2022/03/31 05:14
PHST- 2021/12/22 00:00 [received]
PHST- 2022/03/11 00:00 [accepted]
PHST- 2022/04/01 06:00 [pubmed]
PHST- 2022/05/31 06:00 [medline]
PHST- 2022/03/31 05:14 [entrez]
AID - 10.1007/s43441-022-00392-2 [pii]
AID - 10.1007/s43441-022-00392-2 [doi]
PST - ppublish
SO  - Ther Innov Regul Sci. 2022 Jul;56(4):587-595. doi: 10.1007/s43441-022-00392-2. 
      Epub 2022 Mar 30.