The current state of Bayesian methods in nonclinical pharmaceutical statistics: Survey results and recommendations from the DIA/ASA-BIOP Nonclinical Bayesian Working Group.


Journal

Pharmaceutical statistics
ISSN: 1539-1612
Titre abrégé: Pharm Stat
Pays: England
ID NLM: 101201192

Informations de publication

Date de publication:
03 2021
Historique:
received: 21 02 2020
revised: 26 08 2020
accepted: 06 09 2020
pubmed: 8 10 2020
medline: 26 11 2021
entrez: 7 10 2020
Statut: ppublish

Résumé

The use of Bayesian methods to support pharmaceutical product development has grown in recent years. In clinical statistics, the drive to provide faster access for patients to medical treatments has led to a heightened focus by industry and regulatory authorities on innovative clinical trial designs, including those that apply Bayesian methods. In nonclinical statistics, Bayesian applications have also made advances. However, they have been embraced far more slowly in the nonclinical area than in the clinical counterpart. In this article, we explore some of the reasons for this slower rate of adoption. We also present the results of a survey conducted for the purpose of understanding the current state of Bayesian application in nonclinical areas and for identifying areas of priority for the DIA/ASA-BIOP Nonclinical Bayesian Working Group. The survey explored current usage, hurdles, perceptions, and training needs for Bayesian methods among nonclinical statisticians. Based on the survey results, a set of recommendations is provided to help guide the future advancement of Bayesian applications in nonclinical pharmaceutical statistics.

Identifiants

pubmed: 33025743
doi: 10.1002/pst.2072
doi:

Substances chimiques

Pharmaceutical Preparations 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

245-255

Informations de copyright

© 2020 John Wiley & Sons Ltd.

Références

Lakshminarayanan M, Natanegara F. Bayesian Applications in Pharmaceutical Development. Boca Raton, FL: CRC Press; 2019.
Lesaffre E, Baio G, Boulanger B. Bayesian Methods in Pharmaceutical Research. Boca Raton, FL: CRC Press; 2020.
FDA. Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials, Center for Devices and Radiological Health. 2010. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/guidance-use-bayesian-statistics-medical-device-clinical-trials. Accessed January 7, 2020.
FDA. Complex Innovative Trial Design Pilot Program. 2018. https://www.fda.gov/drugs/development-resources/complex-innovative-trial-designs-pilot-program. Accessed January 7, 2020.
FDA. Draft Guidance for Meta-Analyses of Randomized Controlled Clinical Trials to Evaluate the Safety of Human Drugs or Biological Products. 2018. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/meta-analyses-randomized-controlled-clinical-trials-evaluate-safety-human-drugs-or-biological. Accessed February 3, 2020.
FDA. Guidance Adaptive Designs for Clinical Trials of Drugs and Biologics. 2018. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/adaptive-design-clinical-trials-drugs-and-biologics-guidance-industry. Accessed February 3, 2020.
FDA. Draft guidance on Master Protocols for Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics. 2018. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/master-protocols-efficient-clinical-trial-design-strategies-expedite-development-oncology-drugs-and. Accessed February 3, 2020.
Zhang L, Su C. Introduction to Nonclinical Statistics for Pharmaceutical and Biotechnology Industries. In: Zhang L. (Ed.), Nonclinical Statistics for Pharmaceutical and Biotechnology Industries. 1. Cham, Switzerland: Springer; 2016:3-17.
Peterson JJ, Snee RD, McAllister PR, Schofield TL, Carella AJ. Statistics in pharmaceutical development and manufacturing. J Qual Technol. 2009;41(2):111-134.
Peterson J, Altan S. Overview of Drug Development and Statistical Tools for Manufacturing and Testing. In: Zhang L. (Ed.), Nonclinical Statistics for Pharmaceutical and Biotechnology Industries. Cham, Switzerland: Springer; 2016:383-414.
Burdick RK, LeBlond DJ, Pfahler LB, et al. Statistical Applications for Chemistry, Manufacturing and Controls (CMC) in the Pharmaceutical Industry. New York, NY: Springer; 2017.
Yang H. Emerging Non-Clinical Biostatistics in Biopharmaceutical Development and Manufacturing. New York: Chapman and Hall/CRC; 2016.
Faya P, Seaman JW Jr, Stamey JD. Bayesian assurance and sample size determination in the process validation life-cycle. J Biopharm Stat. 2017;27(1):159-174.
Peterson JJ, Kramer TT, Hofer JD, Atkins G. Opportunities and challenges for statisticians in advanced pharmaceutical manufacturing. Stat Biopharm Res. 2019;11(2):152-161.
FDA. Innovation or Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products. 2004. https://www.fda.gov/science-research/science-and-research-special-topics/critical-path-initiative. Accessed January 7, 2020.
Irony T, Huang L. Bayesian Approaches in the Regulation of Medical Products. In: Lakshminarayanan M, Natanegara F. (Eds.), Bayesian Applications in Pharmaceutical Development. Boca Raton, FL: CRC Press; 2019:307-327.
ICH E9 document. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/e9-statistical-principles-clinical-trials. Accessed January 6, 2020.
Rahman MA, Shen M, Dong XC, Lin KK, Tsong Y. Regulatory nonclinical statistics. In: Zhang L. (Ed.), Nonclinical Statistics for Pharmaceutical and Biotechnology Industries. Cham, Switzerland: Springer; 2016:19-31.
Peers IS, Ceuppens PR, Harbron C. In search of preclinical robustness. Nat Rev. 2012;11:733-774.
Tabora JE, Gonzalez FL, Tom JW. Bayesian probabilistic modeling in pharmaceutical process development. AIChE J. 2019;65(11).
Miller G, Inkret WC, Schillaci ME, Martz HF, Little TT. Analyzing bioassay data using Bayesian methods-a primer. Health Phys. 2000;78(6):598-613.
Gelman A, Chew GL, Shnaidman M. Bayesian analysis of serial dilution assays. Biometrics. 2004;60(2):407-417.
Sivaganesan M, Seifring S, Varma M, Haugland RA, Shanks OC. A Bayesian method for calculating real-time quantitative PCR calibration curves using absolute plasmid DNA standards. BMC Bioinformatics. 2008;9(1):120.
Peterson JJ, Yahyah M. A Bayesian design space approach to robustness and system suitability for pharmaceutical assays and other processes. Stat Biopharm Res. 2009;1(4):441-449.
Feng F, Sales AP, Kepler TB. A Bayesian approach for estimating calibration curves and unknown concentrations in immunoassays. Bioinformatics. 2011;27(5):707-712.
Rozet E, Govaerts B, Lebrun P, et al. Evaluating the reliability of analytical results using a probability criterion: a Bayesian perspective. Anal Chim Acta. 2011;705(1):193-206.
Novick SJ, Yang H, Peterson JJ. A Bayesian approach to parallelism testing in bioassay. Stat Biopharm Res. 2012;4(4):357-374.
Lebrun P, Boulanger B, Debrus B, Lambert P, Hubert P. A Bayesian design space for analytical methods based on multivariate models and predictions. J Biopharm Stat. 2013;23(6):1330-1351.
Fronczyk K, Kottas A. A Bayesian approach to the analysis of quantal bioassay studies using nonparametric mixture models. Biometrics. 2014;70(1):95-102.
Rozet E, Lebrun P, Michiels JF, Sondag P, Scherder T, Boulanger B. Analytical procedure validation and the quality by design paradigm. J Biopharm Stat. 2015;25(2):260-268.
Muleme M, Stenos J, Vincent G, et al. Bayesian validation of the indirect immunofluorescence assay and its superiority to the enzyme-linked immunosorbent assay and the complement fixation test for detecting antibodies against Coxiella burnetii in goat serum. Clin Vaccine Immunol. 2016;23(6):507-514.
Sondag P, Lebrun P, Rozet E, Boulanger B. Assay Validation. In: Zhang L. (Ed.), Nonclinical Statistics for Pharmaceutical and Biotechnology Industries. Cham, Switzerland: Springer; 2016:415-432.
Novick S, Sondag P, Schofield T, Miller K. A novel method for qualification of a potency assay through partial computer simulation. PDA J Pharm Sci Technol. 2018;72(3):249-263.
Yang H, Novick S. Bayesian Analysis with R for Drug Development: Concepts, Algorithms, and Case Studies. New York: Chapman & Hall/CRC; 2019.
Lebrun P, Rozet E. Analytical Method and Assay. In: Lesaffre E, Baio G, Boulanger B. (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Peterson JJ. A posterior predictive approach to multiple response surface optimization. J Qual Technol. 2004;36(2):139-153.
Miro-Quesada G, Del Castillo E, Peterson JJ. A Bayesian approach for multiple response surface optimization in the presence of noise variables. J Appl Stat. 2004;31(3):251-270.
Peterson JJ. A Bayesian approach to the ICH Q8 definition of design space. J Biopharm Stat. 2008;18(5):959-975.
Peterson JJ, Miro-Quesada G, del Castillo E. A Bayesian reliability approach to multiple response optimization with seemingly unrelated regression models. Qual Technol Quant Manag. 2009;6(4):353-369.
Peterson JJ, Lief K. The ICH Q8 definition of design space: a comparison of the overlapping means and the Bayesian predictive approaches. Stat Biopharm Res. 2010;2(2):249-259.
Mockus L, LeBlond D, Basu PK, Shah RB, Khan MA. A QbD case study: Bayesian prediction of lyophilization cycle parameters. AAPS Pharm Sci Tech. 2011;12(1):442-448.
Woodward P. Bayesian Analysis Made Simple: An Excel GUI for WinBUGS. New York: Chapman and Hall/CRC; 2011.
Lebrun P, Krier F, Mantanus J, et al. Design space approach in the optimization of the spray-drying process. Eur J Pharm Biopharm. 2012;80(1):226-234.
Hubert C, Lebrun P, Houari S, Ziemons E, Rozet E, Hubert P. Improvement of a stability-indicating method by quality-by-design versus quality-by-testing: a case of a learning process. J Pharm Biomed Anal. 2014;88:401-409.
Lebrun P, Giacoletti K, Scherder T, Rozet E, Boulanger B. A quality by design approach for longitudinal quality attributes. J Biopharm Stat. 2015;25(2):247-259.
Mockus L, Peterson JJ, Lainez JM, Reklaitis GV. Batch-to-batch variation: a key component for modeling chemical manufacturing processes. Org Process Res Dev. 2015;19(8):908-914.
Lebrun P, Sondag P, Lories X, Michiels JF, Rozet E, Boulanger B. Quality by design applied in formulation development and robustness. Statistics for Biotechnology Process Development. New York: Chapman and Hall/CRC; 2018:89-104.
Sano S, Kadowaki T, Tsuda K, Kimura S. Application of Bayesian optimization for pharmaceutical product development. J Pharm Innov. 2020;15:333-343.
Tabora JE, Lora Gonzalez F, Tom JW. Bayesian probabilistic modeling in pharmaceutical process development. AIChE J. 2019;65(11):e16744.
Boulanger B., Mutsvari T. Product Development and Manufacturing. In: Lesaffre E, Baio G, Boulanger B. (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Yang H. How many batches are needed for process validation under the new FDA guidance? PDA J Pharm Sci Technol. 2013;67(1):53-62.
LeBlond D, Mockus L. The posterior probability of passing a compendial standard, part 1: uniformity of dosage units. Statistics in Biopharmaceutical Research. 2014;6(3):270-286.
Peterson JJ. Process Development and Validation. In: Lesaffre E, Baio G, Boulanger B. (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Mockus L, Laínez JM, Reklaitis G, Kirsch L. A Bayesian approach to pharmaceutical product quality risk quantification. Informatica. 2011;22(4):537-558.
Yang H, Zhang J. A Bayesian approach to residual host cell DNA safety assessment. PDA J Pharm Sci Technol. 2016;70(2):157-162.
Novick SJ, Zhao W, Yang H. Setting alert and action limits in the presence of significant amount of censoring in data. PDA J Pharm Sci Technol. 2017;71(1):20-32.
Faya P, Stamey JD, Seaman JW Jr. A Bayesian approach to determination of F, D, and z values used in steam sterilization validation. PDA J Pharm Sci Technol. 2017;71(2):88-98.
Yu B, Yang H, Ren P. Bayesian tolerance intervals for zero-inflated data with applications in pharmaceutical quality control. J Valid Technol. 2017;23(3).
Banton D, Vacante D, Bulthuis B, Goldstein J, Wineburg M, Schreffler J. The use of Bayesian hierarchical logistic regression in the development of a modular viral inactivation claim. PDA J Pharm Sci Technol. 2019;73(6):552-561.
Overstall AM, Woods DC, Martin KJ. Bayesian prediction for physical models with application to the optimization of the synthesis of pharmaceutical products using chemical kinetics. Comput Stat Data Anal. 2019;132:126-142.
Novick S, Hudson-Curtis B. Content Uniformity Testing. In: Lesaffre E., Baio G, Boulanger B (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Mockus L, LeBlond D. Bayesian Methods for In Vitro Dissolution Drug Testing and Similarity Comparisons. In: Lesaffre E, Baio G, Boulanger B. (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Tonakpon AH, Lebrun P, Rozet E, Scherder T, Giacoletti K. Bayesian Statistics for Manufacturing. In: Lesaffre E, Baio G, Boulanger B. (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Chen J, Zhong J, Nie L. Bayesian hierarchical modeling of drug stability data. Stat Med. 2008;27(13):2361-2380.
Faya P, Seaman JW Jr, Stamey JD. Using accelerated drug stability results to inform long-term studies in shelf life determination. Stat Med. 2018;37(17):2599-2615.
Yu B, Zeng L, Yang H. A Bayesian approach to setting the release limits for critical quality attributes. Stat Biopharm Res. 2018;10(3):158-165.
Montes RO, Burdick RK, Leblond DJ. Simple approach to calculate random effects model tolerance intervals to set release and shelf-life specification limits of pharmaceutical products. PDA J Pharm Sci Technol. 2019;73(1):39-59.
Avohou TH, Lebrun P, Rozet E, Boulanger B. Bayesian Methods for the Design and Analysis of Stability Studies. In: Lesaffre E, Baio G, Boulanger B. (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Novick S, Shen Y, Yang H, Peterson J, LeBlond D, Altan S. Dissolution curve comparisons through the F 2 parameter, a Bayesian extension of the f 2 statistic. J Biopharm Stat. 2015;25(2):351-371.
Zeng L, Novick S, Yu B, Yang H. General framework for equivalence testing over a range of linear outcomes with CMC applications. Stat Biopharm Res. 2019;11(2):182-190.
Ekins S, Reynolds RC, Kim H, et al. Bayesian models leveraging bioactivity and cytotoxicity information for drug discovery. Chem Biol. 2013;20(3):370-378.
Walley R, Sherington J, Rastrick J, Detrait E, Hanon E, Watt G. Using Bayesian analysis in repeated preclinical in vivo studies for a more effective use of animals. Pharm Stat. 2016;15(3):277-285.
Lazic SE, Edmunds N, Pollard CE. Predicting drug safety and communicating risk: benefits of a Bayesian approach. Toxicol Sci. 2018;162(1):89-98.
Novick SJ, Sachsenmeier K, Leow CC, Roskos L, Yang H. A novel Bayesian method for efficacy assessment in animal oncology studies. Stat Biopharm Res. 2018;10(3):151-157.
Li Y, Higgs RE, Hoffman RW, et al. A Bayesian gene network reveals insight into the JAK-STAT pathway in systemic lupus erythematosus. PloS One. 2019;14(12).
Williams DP, Lazic SE, Foster AJ, Semenova E, Morgan P. Predicting drug-induced liver injury with Bayesian machine learning. Chem Res Toxicol. 2020;33(1):239-248.
Claycamp HG. Probability concepts in quality risk management. PDA J Pharm Sci Technol. 2012;66(1):78-89.
Campbell G. FDA Regulatory Acceptance of Bayesian Statistics. In: Lesaffre E, Baio G, Boulanger B. (Eds.), Bayesian Methods in Pharmaceutical Research. New York: Chapman and Hall/CRC; 2020.
Ye J, Travis J. A Bayesian approach for incorporating adult clinical data into pediatric. Paper presented at: 2017 FDA Workshop on Pediatric Trial Design and Modeling; 2017; Silver Spring, MD.
LaVange L. General considerations for other innovative designs. Paper presented at: FDA Workshop on Promoting the Use of Complex Innovative Designs in Clinical Trials; 2018; Silver Springs, MD.
Irony T. The value of Bayesian approaches in the regulatory setting: lessons from the past and perspectives for the future. BSWG KOL Lect Series. January 2018. http://www.bayesianscientific.org/wp-content/uploads/2018/01/Irony_BayesKOL_11918.pdf.
Natanegara F, Neuenschwander B, Seaman JW Jr, et al. The current state of Bayesian methods in medical product development: survey results and recommendations from the DIA Bayesian scientific working group. Pharm Stat. 2014;13(1):3-12.

Auteurs

Paul Faya (P)

Statistics-Discovery/Development, Eli Lilly and Company, Indianapolis, Indiana, USA.

Perceval Sondag (P)

Center for Mathematical Sciences, Merck & Co., Inc, Kenilworth, New Jersey, USA.

Steven Novick (S)

Department of Data Sciences & Quantitative Biology, Discovery Sciences, R&D, AstraZeneca, Gaithersburg, Maryland, USA.

Dwaine Banton (D)

Translational Medicine and Early Development Statistics, Janssen, Raritan, New Jersey, USA.

John W Seaman (JW)

Department of Statistical Science, Baylor University, Waco, Texas, USA.

James D Stamey (JD)

Department of Statistical Science, Baylor University, Waco, Texas, USA.

Bruno Boulanger (B)

PharmaLex Statistical Solutions, Mont-Saint-Guibert, Belgium.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH