Abstract
Currently, there is an increasing focus on the implementation of pharmacokinetic-pharmacodynamic (PK-PD) studies and modelling as essential tools for drug development. Strategies involving specifically the population approach, which are based on relatively recent statistical methodology (e.g. nonlinear mixed effects modelling, NONMEM) have been advocated for investigating pharmacokinetic and pharmacodynamic variability as well as dose-concentration-effect relationships. The present article outlines this approach, and discusses how it can be implemented within the framework of the studies currently performed as part of the clinical phases of new drug development. It also considers study design and performance, based on real-life experiences. Population approaches, if designed carefully and early, as part of the planning of the drug development programme, are expected to play a significant role at every phase of the programme and to contribute to providing information that is valuable for registration purposes. Statistical methodology and software are now widely available. However, practical issues such as integration of the population approach within existing protocols, quality control of the data, timing of laboratory and statistical analyses, as well as resource allocation, remain legitimate concerns to be considered in prospective studies.
MeSH Terms
Clinical Trials, Phase I as Topic
Clinical Trials, Phase II as Topic
Clinical Trials, Phase III as Topic
Cohort Studies
Computer Simulation
Drug Design
Guidelines as Topic
Humans
Multicenter Studies as Topic
Pharmacokinetics
Statistics as Topic
Authors & Affiliations
7 authors, click to expand affiliations / ORCID
Vozeh S
Intercantonal Office for the Control of Medicines, Bern, Switzerland.
Steimer J L
Rowland M
Morselli P
Mentre F
Balant L P
Aarons L
References (21)
21 references, click to expand
-
Results and validation of a population pharmacodynamic model for cognitive effects in Alzheimer patients treated with tacrine.
Proc Natl Acad Sci U S A. 1992 Dec 1;89(23):11471-5
PMID: 1454836
-
Population pharmacokinetic/pharmacodynamic methodology and applications: a bibliography.
Biometrics. 1994 Jun;50(2):566-75
PMID: 8068854
-
Pharmacodynamics in cancer therapy.
J Clin Oncol. 1990 Oct;8(10):1739-53
PMID: 2213109
-
Population dose versus response of betaxolol and atenolol: a comparison of potency and variability.
Clin Pharmacol Ther. 1991 Jan;49(1):24-31
PMID: 1988237
-
A two-step iterative algorithm for estimation in nonlinear mixed-effect models with an evaluation in population pharmacokinetics.
J Biopharm Stat. 1995 Jul;5(2):141-58
PMID: 7581424
-
Estimation of population characteristics of pharmacokinetic parameters from routine clinical data.
J Pharmacokinet Biopharm. 1977 Oct;5(5):445-79
PMID: 925881
-
Study designs for dose-ranging.
Clin Pharmacol Ther. 1989 Jul;46(1):63-77
PMID: 2743708
-
Pharmacodynamic modelling. Application to new drug development.
Clin Pharmacokinet. 1991 Feb;20(2):91-8
PMID: 2029808
-
Minimal modeling, partition analysis, and the estimation of insulin sensitivity.
Fed Proc. 1980 Jan;39(1):110-5
PMID: 6985867
-
Practical experience and issues in designing and performing population pharmacokinetic/pharmacodynamic studies.
Eur J Clin Pharmacol. 1996;49(4):251-4
PMID: 8857068
-
Opportunities for integration of pharmacokinetics, pharmacodynamics, and toxicokinetics in rational drug development.
Clin Pharmacol Ther. 1992 Apr;51(4):465-73
PMID: 1563216
-
The use of kinetic-dynamic interactions in the evaluation of drugs.
Psychopharmacology (Berl). 1990;100(4):433-50
PMID: 2181524
-
Methodologic aspects of a population pharmacodynamic model for cognitive effects in Alzheimer patients treated with tacrine.
Proc Natl Acad Sci U S A. 1992 Dec 1;89(23):11466-70
PMID: 1454835
-
Modelling of individual pharmacokinetics for computer-aided drug dosage.
Comput Biomed Res. 1972 Oct;5(5):411-59
PMID: 4634367
-
Clinical pharmacokinetics in the drug regulatory process.
Clin Pharmacokinet. 1990 Mar;18(3):177-83
PMID: 2323153
-
The randomized concentration-controlled trial: an evaluation of its sample size efficiency.
Control Clin Trials. 1991 Dec;12(6):780-94
PMID: 1665119
-
Nonlinear mixed effects models for repeated measures data.
Biometrics. 1990 Sep;46(3):673-87
PMID: 2242409
-
Population pharmacokinetics/dynamics.
Annu Rev Pharmacol Toxicol. 1992;32:185-209
PMID: 1605567
-
Clinical pharmacokinetics of zidovudine: inter and intraindividual variability and relationship to long term efficacy and toxicity.
Eur J Clin Pharmacol. 1993;45(5):397-407
PMID: 8112367
-
Population approaches in drug development. Report on an expert meeting to discuss population pharmacokinetic/pharmacodynamic software.
Eur J Clin Pharmacol. 1994;46(5):389-91
PMID: 7957530
-
New strategies in drug development and clinical evaluation: the population approach. Commentary on an action for co-operative research.
Eur J Clin Pharmacol. 1993;45(2):93-4
PMID: 8223846