Design and regulation of clinical trials; from the laboratory to the pharmacy

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DOI: 10.21522/TIJCR.2014.03.01.Art012

Authors : Young E E, Okafor Chinyere Nkiruka


The development of new drugs and medical devices takes many years and a lot of scientific research. After identifying a new molecule, which is likely to be beneficial in human disease, it must undergo rigorous trials to ensure its efficacy and safety. Regulatory bodies ensure that clinical trials are carried out in a uniformly acceptable manner, and that the results are reliable. Their main aim is to protect the individual from harm. The aim of this review article is to describe the process of drug development and clinical trials as well as the role(s) of the regulatory agencies involved. A MEDLINE search was conducted using relevant keywords to access published information on clinical trials. Other sources of information include relevant websites and publications of regulatory agencies and major textbooks. The development of a new drug or device starts from identification of the molecule in the laboratory, where it is postulated to have useful characteristics. Following rigorous animal tests, the drug is then entered into clinical trials using human subjects. Clinical trials are conducted in four phases. Regulatory agencies are involved at every stage to ensure compliance with guidelines. There are various designs of clinical trials that may be used and the appropriate design has to be chosen to produce reliable results. The appropriate statistics must also be chosen and the submission to regulatory bodies for approval must be done in a systematic and scientific manner. The International Conference on Harmonisation was convened in 1990 and has drafted good clinical practice (GCP) guidelines for clinical trials. Submission of the application for a new drug involves proper regulatory writing procedures and protocols.

Keywords: clinical trial, regulatory agencies, ICH


[1.]   Bellary S, Krishnankutty B, Latha MS. Basics of case report form designing in clinical research. Perspect. Clin Res 2014; 5(4): 159-166.

[2.]   Bhatt A Quality of clinical trials: a moving target. Perspect. Clin Res 2011 ;2(4): 124-128.

[3.]   Chernick M R, Friis R H. Introductory biostatistics for the health sciences. Modern applications including bootstrap. John Wiley & Sons Inc. (2003).

[4.]   Chin, R. Y., & Lee, B. Y. (2008). Principles and practice of clinical trial medicine. Amsterdam: Elsevier/Academic Press.

[5.]   Code of Federal Regulations Annual Edition. Available from Accessed 25thMarch, 2016.

[6.]   Data and Safety Monitoring Board Guidelines. Available from

[7.]   Eypasch E, Lefering R, Kum CK, Troidl H. Probability of adverse events that have not yet occurred: a statistical reminder. BMJ 1995; 311(7005): 619–620.

[8.]   Fleming T R. Current issues in non-inferiority trials. Stat Med 2008; 27(3): 317-332.

[9.]   Fontanarosa PB, Rennie D, DeAngelis CD. Post-marketing surveillance–lack of vigilance, lack of trust. JAMA. 2004; 292(21): 2647–2650.

[10.]   Golafshani M. Understanding reliability and validity in qualitative research. The Qualitative Report 2003; 8(4): 597 – 606.

[11.]   Guidance for industry E6 Good Clinical Practice: Consolidated Guidance. Available from Accessed 25th March, 2016.


[13.]   Hulley SB, Cummings SR, Browner WS, Brady DG, Newman TB. Designing clinical research. Wolters Kluwer.

[14.]   ICH Topic E3. Structure and content of clinical study reports. Available from

[15.]   Investigational New Drug (IND) Application. Available from Accessed on 25thJune, 2016.

[16.]   Johnson JJ, Gupta NV. Recent advances in quality management of clinical trials Int J Pharm PharmSci 2013:5(3): 34-38.

[17.]   Montgomery AA, Peters TJ, Little P. Design, analysis and presentation of factorial randomised controlled trials. BMC Med Res Method. 2003 DOI: 10.1186/1471-2288-3-26.

[18.]   Prentice R L. Surrogate endpoints in clinical trials: definition and operational criteria. Stat Med 1989; 8(4): 431-440.

[19.]   Sibbald B, Roland M Understanding controlled trials; Why are randomised controlled trials important? BMJ 1998; 316(7126): 201.

[20.]   The ICH harmonised tripartite guidelines. Available from

[21.]   The Investigational Medicinal Products Dossier (IMPD). Available from

[22.]   Tobin John J. Walsh G. Medical product regulatory affairs. Pharmaceuticals, Diagnostics, Medical devices.Wiley-VchVerlag GmbH & Co. KGaA (2008).

[23.]   Umscheid CA, Margolis DJ, Grossman CE. Key concepts of clinical trials; a narrative review. Postgrad Med (2011) Vol 123, No 5, pp 194-204.

[24.]   Walker E. Nowacki AS. Understanding equivalence and non-inferiority testing. J Gen Intern Med 2011; 26(2): 192-196.

[25.]   Yang LJS, Chang KWC, Chung K C. Methodology rigors in clinical research. Plast Reconstr Surg 2010; 129(6): 979e-988e.