Ministry of Health, Labour and Welfare (2024). Statistics on Pharmaceutical and Medical Device Industry Cited from the Japan Pharmaceutical Manufacturers Association DATA book. Japan Pharmaceutical Manufacturers Association. https://www.jpma.or.jp/news_room/issue/databook/en/rs40ob00000015ux-att/DATABOOK2024_E_ALL.pdf. Accessed May 07, 2024.
Wouters OJ, McKee M, Luyten J (2020) Estimated research and development investment needed to bring a new medicine to market, 2009–2018. JAMA 323:844–853
Article PubMed PubMed Central Google Scholar
Brown DG, Wobst HJ, Kapoor A, Kenna LA, Southall N (2022) Clinical development times for innovative drugs. Nat Rev Drug Discov 21:793–794
Article CAS PubMed PubMed Central Google Scholar
EFPIA MID3 Workgroup (2016) Good practices in model-informed drug discovery and development: practice, application, and documentation. CPT Pharmacometr Syst Pharmacol 5:93–122
Miller R, Ewy W, Corrigan BW, Ouellet D, Hermann D, Kowalski KG et al (2005) How modeling and simulation have enhanced decision making in new drug development. J Pharmacokinet Pharmacodyn 32:185–197
Lalonde RL, Kowalski KG, Hutmacher MM, Ewy W, Nichols DJ, Milligan PA et al (2007) Model-based drug development. Clin Pharmacol Ther 82:21–32
Article CAS PubMed Google Scholar
Wang Y, Zhu H, Madabushi R, Liu Q, Huang SM, Zineh I (2019) Model-informed drug development: current US regulatory practice and future considerations. Clin Pharmacol Ther 105:899–911
US Food and Drug Administration (FDA) (2023) Model-informed drug development paired meeting program. https://www.fda.gov/drugs/development-resources/model-informed-drug-development-paired-meeting-program. Accessed December 12, 2023.
Madabushi R, Seo P, Zhao L, Tegenge M, Zhu H (2022) Review: role of model-informed drug development approaches in the lifecycle of drug development and regulatory decision-making. Pharm Res 39:1669–1680
Article CAS PubMed PubMed Central Google Scholar
Ministry of Health, Labour and Welfare (2019) Guideline on population pharmacokinetic and pharmacodynamic analysis. https://www.pmda.go.jp/files/000235608.pdf. Accessed May 27, 2024.
Ministry of Health, Labour and Welfare (2020) Guideline for exposure-response analysis of drugs. https://www.pmda.go.jp/files/000235605.pdf. Accessed May 27, 2024.
Ministry of Health, Labour and Welfare (2020) Guidelines for analysis reports involving physiologically based pharmacokinetic model. https://www.pmda.go.jp/files/000239317.pdf. Accessed May 27, 2024.
Kijima S, Ochiai Y, Ishiguro A (2020) Meeting report. Meeting report: PMDA public workshop on pharmacometrics at Japan. CPT Pharmacometrics Syst Pharmacol 9:550–552
Article CAS PubMed PubMed Central Google Scholar
International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) (2022) Considerations with Respect to Future MIDD Related Guidelines. https://admin.ich.org/sites/default/files/2022-05/ICH_MIDD_Roadmap_2022_0503.pdf. Accessed May 27, 2024.
International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) (2022) Final Concept Paper M15: Model-Informed Drug Development General Principles Guideline. https://database.ich.org/sites/default/files/ICH_M15_ConceptPaper_Final_2022_1102.pdf. Accessed May 27, 2024.
International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) (2022) Final Business Plan, Model-Informed Drug Development General Principles Guideline. https://database.ich.org/sites/default/files/ICH_M15_BusinessPlan_Final_2022_1027.pdf. Accessed May 27, 2024.
International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) (2024) Work plan, model-informed drug development general principles guideline. https://database.ich.org/sites/default/files/ICH_M15_EWG_WorkPlan_2024_0315.pdf. Accessed May 27, 2024.
Singh RSP, Toussi SS, Hackman F, Chan PL, Rao R, Allen R et al (2022) Innovative randomized Phase I study and dosing regimen selection to accelerate and inform pivotal COVID-19 trial of nirmatrelvir. Clin Pharmacol Ther 112:101–111
Article CAS PubMed Google Scholar
Bi Y, Liu J, Li L, Yu J, Bhattaram A, Bewernitz M et al (2019) Role of model-informed drug development in pediatric drug development, regulatory evaluation, and labeling. J Clin Pharmacol 59(Supplement 1):S104–S111
Haber G, Conway KM, Paramsothy P, Roy A, Rogers H, Ling X et al (2021) Association of genetic mutations and loss of ambulation in childhood-onset dystrophinopathy. Muscle Nerve 63:181–191
Article CAS PubMed Google Scholar
Kijima S, Yoshida S, Ochiai Y (2022) Activity and perspective on quantitative modeling and simulation in Japan: Update from the Pharmaceuticals and Medical Devices Agency. CPT Pharmacometrics Syst Pharmacol 11:1552–1555
Article CAS PubMed PubMed Central Google Scholar
Kaddi CD, Niesner B, Baek R, Jasper P, Pappas J, Tolsma J et al (2018) Quantitative systems pharmacology modeling of acid sphingomyelinase deficiency and the enzyme replacement therapy Olipudase Alfa is an innovative Tool for linking pathophysiology and pharmacology. CPT Pharmacometrics Syst Pharmacol 7:442–452
Article CAS PubMed PubMed Central Google Scholar
Gadkar K, Kirouac D, Parrott N, Ramanujan S (2016) Quantitative systems pharmacology: a promising approach for translational pharmacology. Drug Discov Today Technol 21–22:57–65
Ramakrishnan V, Friedrich C, Witt C, Sheehan R, Pryor M, Atwal JK et al (2023) Quantitative systems pharmacology model of the amyloid pathway in Alzheimer’s disease: insights into the therapeutic mechanisms of clinical candidates. CPT Pharmacometrics Syst Pharmacol 12:62–73
Article CAS PubMed Google Scholar
Kuemmel C, Yang Y, Zhang X, Florian J, Zhu H, Tegenge M et al (2020) Consideration of a credibility assessment framework in model-informed drug development: Potential application to physiologically-based pharmacokinetic modeling and simulation. CPT Pharmacometrics Syst Pharmacol 9:21–28
Article CAS PubMed Google Scholar
Musuamba FT, Skottheim Rusten IS, Lesage R, Russo G, Bursi R, Emili L et al (2021) Scientific and regulatory evaluation of mechanistic in silico drug and disease models in drug development: Building model credibility. CPT Pharmacometrics Syst Pharmacol 10:804–825
Article CAS PubMed PubMed Central Google Scholar
Rusten IS, Musuamba FT (2021) Scientific and regulatory evaluation of empirical pharmacometric models: an application of the risk informed credibility assessment framework. CPT Pharmacometr Syst Pharmacol 10:1281–1296
Kerbusch T (2011) Phase 2b dose selection, leveraging comparator data through multidisciplinary modeling & simulation. EMA-EFPIA Modelling and Simulation Workshop, London
Patsalos PN, Fröscher W, Pisani F, van Rijn CM (2002) The importance of drug interactions in epilepsy therapy. Epilepsia 43:365–385
Article CAS PubMed Google Scholar
Hoffelt C, Gross T (2016) A review of significant pharmacokinetic drug interactions with antidepressants and their management. Ment Health Clin 6:35–41
Article PubMed PubMed Central Google Scholar
US Food and Drug Administration (FDA) (2018) Burosumab, Multidiscipline review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2018/761068Orig1s000MultidisciplineR.pdf. Accessed December 13, 2023.
US Food and Drug Administration (FDA) (2021) Fosdenopterin, Integrated review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214018Orig1s000IntegratedR.pdf. Accessed December 13, 2023.
Perrone DR, Moukassi MS, Romero K, Czerwiec FS, Chapman AB, Gitomer BY et al (2017) A drug development tool for trial enrichment in patients with autosomal dominant polycystic kidney disease. Kidney Int Rep 21:451–460
Li RJ, Ma L, Li F, Li L, Bi Y, Yuan Y et al (2022) Model-informed approach supporting drug development and regulatory evaluation for rare diseases. J Clin Pharmacol 62(Supplement 2):S27-37
Mitra A, Lee JB, Steinbach D, Hazra A, Krishna R (2023) Rare oncology therapeutics: review of clinical pharmacology package of drug approvals (2019–2023) by US FDA, best practices and recommendations. J Pharmacokinet Pharmacodyn 50:475–493
Tanaka M, Idei M, Sakaguchi H, Kato R, Sato D, Sawanobori K et al (2021) Evolving landscape of new drug approval in Japan and lags from international birth dates: retrospective regulatory analysis. Clin Pharmacol Ther 109:1265–1273
International council for harmonisation of technical requirements for pharmaceuticals for human use (ICH) (2018). Ethnic factors in the acceptance of foreign clinical data E5 (R1). https://database.ich.org/sites/default/files/E5_R1__Guideline.pdf. Accessed May 27, 2024.
International council for harmonisation of technical requirements for pharmaceuticals for human use (ICH) (2017) General principles for the planning and design of multi-regional clinical trials E17. https://database.ich.org/sites/default/files/E17EWG_Step4_2017_1116.pdf. Accessed May 27, 2024.
Iwasa T, de Almeida C, Fauchet F, Winchell GA, de Greef R, Hasegawa C et al (2023) Model-informed dose justifications of posaconazole in Japanese patients for prophylaxis and treatment against fungal infection. J Clin Pharmacol 63:421–434
留言 (0)