請用此 Handle URI 來引用此文件:
http://tdr.lib.ntu.edu.tw/jspui/handle/123456789/62448
完整後設資料紀錄
DC 欄位 | 值 | 語言 |
---|---|---|
dc.contributor.advisor | 劉仁沛 | |
dc.contributor.author | Shih-Ting Chiu | en |
dc.contributor.author | 邱詩婷 | zh_TW |
dc.date.accessioned | 2021-06-16T16:02:39Z | - |
dc.date.available | 2015-07-11 | |
dc.date.copyright | 2013-07-11 | |
dc.date.issued | 2013 | |
dc.date.submitted | 2013-07-04 | |
dc.identifier.citation | 1 Chow S.C., Shao J., and Wang H. (2002). Individual bioequivalence testing under 2x3 design. Statistics in Medicine; 21:629–648.
2 Chow S.C., Shao J., Wang H. (2003) In vitro bioequivalence testing. Statistics in Medicine; 22: 55–68. 3 Chow S.C., and J.P. (2010) Design and Analysis of Bioavailability and Bioequivalence Studies, 3th Ed. , CRC/ Chapman and Hall, Taylor and France, New York. 4 Chow S.C. (2011) Quantitative Evaluation of Bioequivalence/Biosimilarity. J Bioequiv Availab S1:doi:10.4172/jbb.S1-002. 5 Chow S.C. L. Endreny, P. A Lachenbruch, Yang L.Y., and Chi, Eric (2011) Scientific Factors for Assessing Biosimilarity and Drug Interchangeability of follow-on biologics. Biosimilars : 1 13-26. 6 Congress of The United States Budget Congressional Office (2010). The Long-Term Budget Outlook. A CBO report. Congress of The United States Budget Congressional Office. 7 Department of Health, Republic of China (2010). Guidelines for the Review Drug registration inspection - inspection and registration of biological similarity drugs. The Department of Health, Taipei, Taiwan. 8 Falconer, D. S., and Mackay, T. F. C. (1996). Introduction to Quantitative Genetics, Ed. 4, Longman Scientific and Technical, Harlow, Essex, UK. 9 Graybill, F., and Wang, C. M. (1980). Confidence intervals on nonnegative linear combinations ofvariances. Journal of the American Statistical Association; 75, 869–873. 10 Howe, W. G. (1974). Approximate confidence limits on the mean of X + Y where X and Y are two tabled independent random variables. Journal of the American Statistical Association; 69, 789–794. 11 Hsiao, C. F., Hsu, Y. Y., and Liu, J. P. (2007). Use of prior information for Bayesian evaluation of bridging studies. Journal of Biopharmaceutical Statistics; 17: 109-121. 12 Hyslop T., Hsuan F., Holder D.J.. (2000) A small sample confidence interval approach to assessing individual bioequivalence. Statistics in Medicine ; 19: 2885–2897. 13 Lee Y.H., Shao J., Chow S.C. (2004) Modified large-sample confidence intervals for linear combinations of variance components: extension, theory, and application. Journal of the American Statistical Association; 99, 467-478 14 Liao C.T., Lin C.Y., Liu J.P.. (2007) Non-inferiority tests based on concordance correlation coefficient for assessment of agreement for gene expression data from microarray experiments. Journal of Biopharmaceutical Statistics; 17: 309–327. 15 Lu L.T., (2008). Statistical Inference for Functions of Variance Components under Two-Way Crossed or Nested Random-Effects Models with Applications to Heritability and Reproducibility of Assay Validation. Doctoral Disseration. National Taiwan University. 16 Quirz J. (2004) Assessment of equivalence using a concordance correlation coefficient in a repeated measurement design. Journal of Biopharmaceutical Statistics; 15: 913–928. 17 Sokal, R. R. and Rohlf, F. J. (1995). Biometry: the principles and practice of statistics in biological research, 3rd ed. New York : Freeman. 18 The European Medicine Agency (2005), Guideline on Similar Biological Medicinal Products. CHMP/437/04. Commitee for medical products for human use. 19 The United States Food and Drug Administration (2001) Guidance on Statistical Approach to Establishing Bioequivalence , Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Rockville, MD. 20 The United States Food and Drug Administration (2003a). Guidance on Bioavailability and Bioequivalence Studies for Orally Administered Drug. Products- General Considerations, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Rockville, MD 21 The United States Food and Drug Administration (2003b). Second Draft Guidance on Bioavailability and Bioequivalence Studies for Nasal Aerosols and Nasal Sprays for Local Action, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Rockville, MD. 22 The United States Food and Drug Administration (2003c). Statistical Information from the June 1999 Draft Guidance and Statistical Information for In Vitro Bioequivalence Data Posted on August 18, 1999, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Rockville, MD. 23 The United States Food and Drug Administration (2012a). Guidance for Industry. Scientific Considerations in Demonstrating Biosimilarity to a Reference Product. Draft guidance. Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration. 24 The United States Food and Drug Administration (2012b). Quality Considerations in Demonstrating Biosimilarity to a Reference Protein Product. Draft guidance. Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration. 25 The United States Food and Drug Administration (2012c). Biosimilars: Questions and Answers Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009. Draft guidance. Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration. 26 Ting, N., Burdick, R. K., Graybill, F. A., Jeyaratnam, S., and Lu, T. F. C. (1990). Confidence intervals on linear combinations of variance components that are unrestricted in sign. Journal of Statistical Computation and Simulation; 35,135-143 27 The United States Federal Law(2009), Biologics Price Competition and Innovation Act of 2009 abbreviated as BPCI Act, United States federal law. Accessed by website : http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/ucm216146.pdf. 28 The United States Federal Law, The Drug Price Competition and Patent Term Restoration Act (1984) informally known as the 'Hatch-Waxman Act' ,Public Law 98-417, United States federal law. 29 The United States National Archives and Records Administration. (2012). Code of federal regulations. Title 21. Volume 5, Part 320, Bioavalibility and bioequivalence requirements. 30 Weerahandi S. (1991) Testing variance components in mixed models with generalized p-values. Journal of the American Statistical Association; 86: 151–153. 31 Weerahandi S. (1993) Generalized confidence intervals. Journal of the American Statistical Association; 88: 899–905. | |
dc.identifier.uri | http://tdr.lib.ntu.edu.tw/jspui/handle/123456789/62448 | - |
dc.description.abstract | 本論文的主要目的是將廣義樞紐量應用於線性化的二次動差評估標準信賴上界的建立及應用,包括:
(1) 體外生物等效性(In vitro Bioequivalence)評估 (2) 遺傳率(Heritability)檢定 (3) 生物相似性(Biosimilar)產品評估 對於一些局部作用的藥品,並不會被吸收到血液中。美國FDA建議利用體外的生物等效性試驗來衡量這些產品兩種藥物產品是否提供相同的治療效果 (2003 U.S. FDA draft guidance)。然而,在美國FDA指南中所建立的生物等效性檢定方法中,生物等效性檢定標準(BE criterion)並未考慮到試驗中不同的變異來源。因此我們考慮在巢式設計之下,利用修正大樣本漸進(modified large-sample)與廣義樞紐量(generalized pivotal quantities)方法,來建立(1-α)100%的信賴上界進行檢定。 遺傳率由各種不同的育種數據組成總變異,因此包含不同的變異來源。因此,也可以利用相同的模式和方法,來進行遺傳率檢定方法的建立。 另外,由於小分子仿製藥和大分子生物製劑產品的一些本質上的區別,小分子藥物的生物等效性的評估方法並不能直接應用到評估生物相似性產品上。在本論文中,考慮在2×3附加對照組的交叉設計之下,同樣利用廣義樞紐量來建立個體生物等效性指標(Individual bioequivalence; IBE)的(1-α)100%信賴上界。 本論文亦進行了模擬數據的研究,以評估上述方法在各種變異參數組合情況下的表現,並計算實例進行比較方法間的優劣,最後進行數據結果與方法表現上的討論。 | zh_TW |
dc.description.abstract | The objectives of this dissertation is to apply the GPQ approach to constructing the upper confidence limit of the linearized criteria based on second moments for evaluation of the in vitro bioequivalence, heritability testing and biosimilar drug product.
Some locally acting products are not intended to be absorbed into the bloodstream. The in vitro bioequivalence studies based on machinery experiments are suggested to measure these products whether the two drug products provide the same therapeutic effect (FDA, 2003). However, the linearized criterion recommended in the draft FDA guidance does not take in consideration different sources of variation. Therefore, we apply the modified large sample (MLS) and generalized pivotal quantities (GPQ) methods to derive the (1-α)100% upper confidence limit for the linearized criterion for evaluation of in vitro bioequivalence with consideration of different sources of variation under the two-stage nested random-effect model. The heritability is defined as the ratio of additive genetic variance to phenotype variance. The hypothesis of interest is to evaluate whether the ratio is greater than some pre-specified value. As results, the same methodology for evaluation of in vitro bioequivalence can be used for assessment of heritability. Due to some fundamental differences between small molecule generics and large molecule biological products, the standard methods for bioequivalence assessment of small molecule drug products cannot be directly applied to assessing biosimilarity of biosimilar products. We proposed to assess biosimilarity by constructing a (1-α)100% upper confidence bound for the Individual bioequivalence (IBE) criterion based on the method of GPQ under a 2×3 extra-reference crossover design. Simulation studies were conducted to evaluate the performance of these proposed methods in terms of size and power under various scenarios. Numerical examples illustrate the application of in vitro bioequivalence, nested random-effect model, modified large sample method proposed methods. | en |
dc.description.provenance | Made available in DSpace on 2021-06-16T16:02:39Z (GMT). No. of bitstreams: 1 ntu-102-D97621201-1.pdf: 9116704 bytes, checksum: 959e5c0bce8e6f6d141bf03ab0a0a54b (MD5) Previous issue date: 2013 | en |
dc.description.tableofcontents | CONTENTS I
LIST OF TABLES IV LIST OF FIGURES VII 中文摘要 VIII ENGLISH ABSTRACT X CHAPTER 1 INTRODUCTION 1 CHAPTER 2 LITERATURE REVIEW 7 2.1 IN VITRO POPULATION BIOEQUIVALENCE (PBE) CRITERION AND HYPOTHESIS 7 2.2 HERITABILITY 10 2.3 BIOSIMILARITY AND INDIVIDUAL BIOEQUIVALENCE (IBE) 12 2.4 BASIC CONCEPTS OF MODIFIED LARGE SAMPLE (MLS) METHOD AND GENERALIZED PIVOTAL QUANTITY (GPQ) 13 CHAPTER 3 ASSESSMENT OF IN-VITRO BIOEQUIVALENCE 17 3.1 ONE-STAGE NESTED RANDOM-EFFECTS MODEL 17 3.1.1 The MLS Approach 18 3.1.2 The GPQ Approach 21 3.2 TWO-STAGE NESTED RANDOM-EFFECTS MODEL 27 3.2.1 The US FDA Naive Approach 27 3.2.2 The MLS Approach 30 3.2.3 The GPQ Approach 32 3.3 NUMERICAL EXAMPLES 38 3.3.1 Numerical Example Under One-stage Nested Random-effects Model 39 3.3.2 Numerical Examples Under Two-stage Nested Random-effects Model 41 CHAPTER 4 HERITABILITY TESTING 54 4.1 THE LINEARIZED CRITERIA OF HERITABILITY 54 4.2 THE MLS APPROACH 56 4.3 THE GPQ APPROACH 57 4.4 NUMERICAL EXAMPLE 58 CHAPTER 5 EVALUAATION OF BIOSIMILAR DRUG PRODUCT 64 5.1 TEST BIOSIMILARITY BASED ON IBE CRITERIA 65 5.2 THE GPQ APPROACH 68 CHAPTER 6 SIMULATION STUDIES 72 6.1 IN-VITRO BIOEQUIVALENCE 72 6.1.1 Simulation Result for One-stage Nested Random Effects Model 72 6.1.2 Simulation Results for Two-stage Nested Random-effects Model 75 6.2 HERITABILITY TESTING 78 6.3 EVALUATION OF BIOSIMILARITY 80 CHAPTER 7 RESULTS AND DISCUSION 134 REFERENCES 139 APPENDIX I DATA LIST OF 30 SAMPLED CANISTERS 145 APPENDIX II DATA LIST OF D50 146 APPENDIX III DATA LIST OF SPAN 149 APPENDIX IV PROGRAMING CODES 152 APPENDIX V PUBLICATION IN JOURNAL OF CHEMOMETRICS 180 | |
dc.language.iso | en | |
dc.title | 廣義樞紐量在生物對等性評估、遺傳率及生物相似性產品檢定方法應用之研究 | zh_TW |
dc.title | A Study on Applications of Generalized Pivotal Quantity Approach to Evaluation of Bioequivalence, Heritability and
Biosimilar Products | en |
dc.type | Thesis | |
dc.date.schoolyear | 101-2 | |
dc.description.degree | 博士 | |
dc.contributor.oralexamcommittee | 周賢忠,季瑋珠,蕭金福,蔡政安,林志榮 | |
dc.subject.keyword | 體外生物等效性,巢式設計模型,大樣本漸進方法,廣義樞紐量,遺傳率,生物相似性,個體生物等效性,2×3附加對照組交叉設計, | zh_TW |
dc.subject.keyword | In vitro bioequivalence,Nested random-effects model,Modified large sample method,Generalized pivotal quantity,Heritability,Individual bioequivalence,2×3 extra-reference crossover design, | en |
dc.relation.page | 188 | |
dc.rights.note | 有償授權 | |
dc.date.accepted | 2013-07-04 | |
dc.contributor.author-college | 生物資源暨農學院 | zh_TW |
dc.contributor.author-dept | 農藝學研究所 | zh_TW |
顯示於系所單位: | 農藝學系 |
文件中的檔案:
檔案 | 大小 | 格式 | |
---|---|---|---|
ntu-102-1.pdf 目前未授權公開取用 | 8.9 MB | Adobe PDF |
系統中的文件,除了特別指名其著作權條款之外,均受到著作權保護,並且保留所有的權利。