Design of experiments (DoE) in pharmaceutical development

References

• Hald AA. History of mathematical statistics. New York: Wiley; 1998.
   Google Scholar
• Fisher RA. Statistical methods for research workers. Edinburgh: Oliver and Boyd; 1925.
   Google Scholar
• Fisher RA. The arrangement of field experiments. J Min Agric Gr Br 1926;33:503–13.
   Google Scholar
• Fischer RA. The design of experiments. 1st edn. New York: Hafner Publishing Company; 1935.
   Google Scholar
• ICH Q8(R2), Pharmaceutical Development, Guideline, Available from: http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Quality/Q8_R1/Step4/Q8_R2_Guideline.pdf [last accessed 4 Nov 2016].
   Google Scholar
• Juran JM. Quality by design, the new steps for planning quality into goods and services. US: The Free Press; 1992.
   Google Scholar
• Deming WE Institute, Available from: https://deming.org/theman/theories/profoundknowledge [last accessed 4 Nov 2016].
   Google Scholar
• Shewhart WE. Economic control of quality of manufactured product. New York: D. Van Nostrand Company; 1931.
   Google Scholar
• Box GEP, Wilson KB. On the experimental attainment of optimum conditions. J R Stat Soc Ser B Methods 1951;13:1–45.
   Web of Science ®Google Scholar
• Eriksson L, Byrne T, Johansson E, et al. Process Analytical Technology (PAT) and Quality by Design (QbD). In: Eriksson L, Byrne T, Johansson E, Trygg J, Vikstrom C, eds. Multi and megavariate data analysis, basic principles and applications. Malmo: Umetrics Academy.
   Google Scholar
• Marlow E, Shangraw RF. Dissolution of sodium salicylate from tablet matrices prepared by wet granulation and direct compression. J Pharm Sci 1967;56:498–504.
   PubMed Web of Science ®Google Scholar
• Singh B, Kumar R, Ahuja N. Optimizing drug delivery systems using systematic “Design of Experiments” Part I: fundamental aspects. Crit Rev Ther Drug Carrier Syst 2004;22:27–105.
   Web of Science ®Google Scholar
• Singh B, Dahiya M, Saharan V, Ahuja N. Optimizing drug delivery systems using systematic “Design of Experiments” Part II: retrospect and prospects”. Crit Rev Ther Drug Carrier Syst 2005;22:215–93.
   PubMed Web of Science ®Google Scholar
• Available from: www.scopus.com
   Google Scholar
• Bhote KR. World class quality: using design of experiments to make it happen. New York: Amacon, American Management Association; 1991.
   Google Scholar
• Yu LX, Amidon G, Khan MA, et al. Understanding pharmaceutical quality by design. AAPS J 2014;16:771–83.
   PubMed Web of Science ®Google Scholar
• Korakianiti ES, Rekkas DM. Statistical thinking and knowledge management for quality-driven design and manufacturing in pharmaceuticals, expert review. Pharm Res 2011;28:1465–79.
   PubMed Web of Science ®Google Scholar
• Zhang L, Mao S. Application of quality by design in the current drug development. Asian J Pharm Sci 2017;12:1–8.
   PubMed Web of Science ®Google Scholar
• Gabrielsson J, Lindberg NO, Pålsson M, et al. Multivariate methods in the development of a new tablet formulation. Drug Dev Ind Pharm 2003;29:1053–75.
   PubMed Web of Science ®Google Scholar
• Gabrielsson J, Lindberg NO, Pålsson M, et al. Multivariate methods in the development of a new tablet formulation: optimization and validation. Drug Dev Ind Pharm 2004;30:1037–49.
   PubMed Web of Science ®Google Scholar
• Claycamp HG, Kona R, Fahmy R, Hoag SW. Quality-by-Design II: application of quantitative risk analysis to the formulation of ciprofloxacin tablets. AAPS PharmSciTech 2016;17:233–44.
   PubMed Web of Science ®Google Scholar
• Chavez PF, Lebrun P, Sacré PY, et al. Optimization of a pharmaceutical tablet formulation based on a design space approach and using vibrational spectroscopy as PAT tool. Int J Pharm 2015;486:13–20.
   PubMed Web of Science ®Google Scholar
• Charoo NA, Shamsher AAA, Zidan AS, Rahman Z. Quality by design approach for formulation development: a case study of dispersible tablets. Int J Pharm 2012;423:167–78.
   PubMed Web of Science ®Google Scholar
• Kushner J, Langdon BA, Hicks I, et al. A quality-by-design study for an immediate-release tablet platform: examining the relative impact of active pharmaceutical ingredient properties, processing methods, and excipient variability on drug product quality attributes. J Pharm Sci 2014;103:527–38.
   PubMed Web of Science ®Google Scholar
• Karatzas AA, Politis SN, Rekkas DM. Development of rapidly dissolving pellets within the Quality by Design Approach. Drug Dev Ind Pharm 2016. [Epub ahead of print]. doi: http://dx.doi.org/10.1080/03639045.2016.1220576.
   Google Scholar
• Malladi M, Jukanti R. Formulation development and evaluation of a novel bi-dependent clarithromycin gastroretentive drug delivery system using Box–Behnken design. J Drug Deliv Sci Technol 2016;35:134–45.
   Web of Science ®Google Scholar
• Belotti S, Rossi A, Colombo P, et al. Spray dried amikacin powder for inhalation in cystic fibrosis patients: a quality by design approach for product construction. Int J Pharm 2014;471:507–15.
   PubMed Web of Science ®Google Scholar
• Belotti S, Rossi A, Colombo P, et al. Spray-dried amikacin sulphate powder for inhalation in cystic fibrosis patients: the role of ethanol in particle formation. Eur J Pharm Biopharm 2015;93:165–72.
   PubMed Web of Science ®Google Scholar
• Pallagi E, Karimi K, Ambrus R, et al. New aspects of developing a dry powder inhalation formulation applying the quality-by-design approach. Int J Pharm 2016;511:151–60.
   PubMed Web of Science ®Google Scholar
• Cilurzo F, Minghetti P, Gennari CG, et al. Formulation study of a patch containing propranolol by design of experiments. Drug Dev Ind Pharm 2014;40:17–22.
   PubMed Web of Science ®Google Scholar
• Patel N, Jain S, Madan P, Lin S. Application of design of experiments for formulation development and mechanistic evaluation of iontophoretic tacrine hydrochloride delivery. Drug Dev Ind Pharm 2016;42:1894–902.
   PubMed Web of Science ®Google Scholar
• de Mattos CB, Argenta DF, de Lima Melchiades G, et al. Nanoemulsions containing a synthetic chalcone as an alternative for treating cutaneous leshmaniasis: optimization using a full factorial design. Int J Nanomed 2015;10:5529–42.
   PubMed Web of Science ®Google Scholar
• Kumar PM, Ghosh A. Development and evaluation of metronidazole loaded microsponge based gel for superficial surgical wound infections. J Drug Deliv Sci Technol 2015;30:15–29.
   Web of Science ®Google Scholar
• Sánchez-Lópeza E, Egea MA, Cano A, et al. PEGylated PLGA nanospheres optimized by design of experiments for ocular administration of dexibuprofen – in vitro, ex vivo and in vivo characterization. Colloids Surf B: Biointerfaces 2016;145:241–50.
   PubMed Web of Science ®Google Scholar
• Achouri D, Hornebecq V, Piccerelle P, et al. Self-assembled liquid crystalline nanoparticles as an ophthalmic drug delivery system. Part I: influence of process parameters on their preparation studied by experimental design. Drug Dev Ind Pharm 2015;41:109–15.
   PubMed Web of Science ®Google Scholar
• Achouri D, Sergent M, Tonetto A, et al. Self-assembled liquid crystalline nanoparticles as an ophthalmic drug delivery system. Part II: optimization of formulation variables using experimental design. Drug Dev Ind Pharm 2015;41:493–501.
   PubMed Web of Science ®Google Scholar
• Rahali Y, Saulnier P, Benoit JP, Bensouda Y. Incorporating vegetal oils in parenteral nutrition using lipid nanocapsules. J Drug Deliv Sci Technol 2010;20:425–9.
   Web of Science ®Google Scholar
• Elliott P, Billingham S, Bi J, Zhang H. Quality by design for biopharmaceuticals: a historical review and guide for implementation. Pharm Bioprocess 2013;1:105–22.
   Google Scholar
• Rathore AS, Winkle H. Quality by design for biopharmaceuticals. Nat Biotechnol 2009;27:26–34.
   PubMed Web of Science ®Google Scholar
• Kelley B, Cromwell M, Jerkins J. Integration of QbD risk assessment tools and overall risk management. Biologicals 2016;44:341–51.
   PubMed Web of Science ®Google Scholar
• Wurth C, Demeule B, Mahler HC, Adler M. Quality by design approaches to formulation robustness – an antibody case study. J Pharm Sci 2016;105:1667–75.
   PubMed Web of Science ®Google Scholar
• Morales JO, Joks GM, Lamprecht A, et al. A design of experiments to optimize a new manufacturing process for high activity protein-containing submicron particles. Drug Dev Ind Pharm 2013;39:1793–801.
   PubMed Web of Science ®Google Scholar
• Troiano G, Nolan J, Parsons D, et al. A quality by design approach to developing and manufacturing polymeric nanoparticle drug products. AAPS J 2016;18:1354–65.
   PubMed Web of Science ®Google Scholar
• Maretti E, Rustichelli C, Romagnoli M, et al. Solid Lipid Nanoparticle assemblies (SLNas) for an anti-TB inhalation treatment – a Design of Experiments approach to investigate the influence of pre-freezing conditions on the powder respirability. Int J Pharm 2016;511:669–79.
   PubMed Web of Science ®Google Scholar
• Faulhammer E, Fink M, Llusa M, et al. Low-dose capsule filling of inhalation products: critical material attributes and process parameters. Int J Pharm 2014;473:617–26.
   PubMed Web of Science ®Google Scholar
• Shariare MH, de Matas M, York P, Shao Q. The impact of material attributes and process parameters on the micronisation of lactose monohydrate. Int J Pharm 2011;408:58–66.
   PubMed Web of Science ®Google Scholar
• Pisano R, Fissore D, Barresi AA, et al. Quality by design: optimization of a freeze-drying cycle via design space in case of heterogeneous drying behavior and influence of the freezing protocol. Pharm Dev Technol 2013;18:280–95.
   PubMed Web of Science ®Google Scholar
• Korhonen K, Poikolainen M, Korhonen O, et al. Systematic evaluation of a spraying method for preparing thin Eudragit-drug films by Design of Experiments. J Drug Deliv Sci Technol 2016;35:241–51.
   Web of Science ®Google Scholar
• Draheim C, de Crécy F, Hansen S, et al. A design of experiment study of nanoprecipitation and nano spray drying as processes to prepare PLGA nano-and microparticles with defined sizes and size distributions. Pharm Res 2015;32:2609–24.
   PubMed Web of Science ®Google Scholar
• Candioti LV, De Zan MM, Cámara MS, Goicoechea HC. Experimental design and multiple response optimization. Using the desirability function in analytical methods development. Talanta 2014;124:123–38.
   PubMed Web of Science ®Google Scholar
• Hibbert DB. Experimental design in chromatography: a tutorial review. J Chromatogr B Analyt Technol Biomed Life Sci 2012;910:2–13.
   PubMed Web of Science ®Google Scholar
• Bezerra MA, Santelli RE, Oliveira EP, et al. Response surface methodology (RSM) as a tool for optimization in analytical chemistry. Talanta 2008;76:965–77.
   PubMed Web of Science ®Google Scholar
• Michaelis M, Leopold CS. A measurement system analysis with design of experiments: investigation of the adhesion performance of a pressure sensitive adhesive with the probe tack test. Int J Pharm 2015;496:448–56.
   PubMed Web of Science ®Google Scholar
• Shah SA, Dickens CJ, Ward DJ, et al. Design of experiments to optimize an in vitro cast to predict human nasal drug deposition. J Aerosol Med Pulm Drug Deliv 2014;27:21–9.
   PubMed Web of Science ®Google Scholar
• Krishnaiah YS, Xu X, Rahman Z, et al. Development of performance matrix for generic product equivalence of acyclovir topical creams. Int J Pharm 2014;475:110–22.
   PubMed Web of Science ®Google Scholar
• de Matas M, De Beer T, Folestad S, et al. Strategic framework for education and training in Quality by Design (QbD) and process analytical technology (PAT). Eur J Pharm Sci 2016;90:2–7.
   PubMed Web of Science ®Google Scholar
• Aksu B, De Beer T, Folestad S, et al. Strategic funding priorities in the pharmaceutical sciences allied to Quality by Design (QbD) and Process Analytical Technology (PAT). Eur J Pharm Sci 2012;47:402–5.
   PubMed Web of Science ®Google Scholar
• Montgomery DC. Introduction to statistical quality control. 6th edn. New York: John Wiley Sons Inc; 2009.
   Google Scholar
• Montgomery DC. Design and analysis of experiments. 8th edn. New York: John Wiley Sons, Inc.; 2013.
   Google Scholar
• Barker TB. Quality by experimental design. New York: Marcel Dekker, Inc.; 1985.
   Google Scholar
• Lewis GA, Mathieu D, Phan-Tan-Luu R. Pharmaceutical experimental design. New York: Marcel Dekker, Inc.; 1999.
   Google Scholar
• NIST Engineering statistics handbook 5. Process improvement, Available from: http://www.itl.nist.gov/div898/handbook/pri/section1/pri1.htm [last accessed 4 Nov 2016].
   Google Scholar
• ICH Q9, Quality Risk Management, Guideline. Available from: http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Quality/Q9/Step4/Q9_Guideline.pdf [last accessed 4 Nov 2016].
   Google Scholar
• ICH Q10, Pharmaceutical Quality System, Guideline, Available from: http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Quality/Q10/Step4/Q10_Guideline.pdf [last accessed 4 Nov 2016].
   Google Scholar
• Piepel GF, Cornell JA. Mixture experiment approaches: examples, discussion and recommendations. J Qual Technol 1994;26:177–96.
   Web of Science ®Google Scholar
• Cornell JA. Experiments with mixtures 3rd edition: designs, models, and the analysis of mixture data. New York: John Wiley Sons; 2002.
   Google Scholar
• Smith WF. Experimental design for formulation. New York: ASA-SIAM Series on Statistics and Applied Probability; 2005.
   Google Scholar
• Margaritis G, Matsagkas E, Taouktsi AN, Rekkas DM. Use of mixture designs for optimizing MDT and DE of fast disintegrating pellets with possible paediatric use. Poster presented at 8th A.It.U.N. Annual Meeting, 6–7 March, Pavia, Italy, 2014.
   Google Scholar
• Αrmstrong NA, James KC. Understanding experimental design and interpretation in pharmaceutics. New York: Ellis Horwood; 1990.
   Google Scholar
• Politis SN, Rekkas DM. The evolution of the manufacturing science and the pharmaceutical industry. Pharm Res 2011;28:1779–81.
   PubMed Web of Science ®Google Scholar