Book contents
- Alzheimer’s Disease Drug Development
- Alzheimer’s Disease Drug Development
- Copyright page
- Dedication
- Contents
- Contributors
- Foreword
- Acknowledgments
- Section 1 Advancing Alzheimer’s Disease Therapies in a Collaborative Science Ecosystem
- Section 2 Non-clinical Assessment of Alzheimer’s Disease Candidate Drugs
- Section 3 Alzheimer’s Disease Clinical Trials
- 11 Phase 1 Trials in Alzheimer’s Disease Drug Development
- 12 The Importance of Phase 2 in Drug Development for Alzheimer’s Disease
- 13 Alzheimer’s Disease Drug Development in Pharmaceutical Companies
- 14 Trial Site Infrastructure and Management: Importance to Alzheimer’s Disease Drug Development
- 15 ATRI and ACTC: Academic Programs to Accelerate Alzheimer’s Disease Drug Development
- 16 The European Prevention of Alzheimer’s Disease Program: A Public–Private Partnership to Facilitate the Secondary Prevention of Alzheimer’s Disease Dementia
- 17 The Global Alzheimer’s Platform Foundation®: Delivering New Medicines Faster by Accelerating Clinical Trials
- 18 Clinical Trial Development in Frontotemporal Lobar Degeneration
- 19 Statistical Considerations in the Design and Analysis of Alzheimer’s Disease Clinical Trials
- 20 Alzheimer’s Disease Trial Recruitment and Diversifying Trial Populations
- 21 The Role of Online Registries in Accelerating Alzheimer’s Disease Drug Development
- 22 Data Safety Monitoring Boards in Alzheimer’s Disease Trials
- 23 Globalization of Alzheimer’s Disease Clinical Trials
- 24 The Use and Development of Clinical Measures of Alzheimer’s Disease Trials
- 25 Tele-Trials, Remote Monitoring, and Trial Technology for Alzheimer’s Disease Clinical Trials
- 26 Expanded Access and Compassionate Use in Alzheimer’s Disease Drug Development
- 27 The Role of the Contract Research Organization in Alzheimer’s Disease: The Vital Link in the Clinical Drug-Development Program
- 28 The Role of Regulatory Agencies in Alzheimer’s Disease Drug Development
- 29 Alzheimer’s Disease Clinical Trial Study Partners
- 30 From Trials to Practice: Are We Ready for a Disease-Modifying Treatment?
- 31 Best Practices for Clinical Trials during COVID-19
- Section 4 Imaging and Biomarker Development in Alzheimer’s Disease Drug Discovery
- Section 5 Academic Drug-Development Programs
- Section 6 Public–Private Partnerships in Alzheimer’s Disease Drug Development
- Section 7 Funding and Financing Alzheimer’s Disease Drug Development
- Index
- References
28 - The Role of Regulatory Agencies in Alzheimer’s Disease Drug Development
from Section 3 - Alzheimer’s Disease Clinical Trials
Published online by Cambridge University Press: 03 March 2022
Book contents
- Alzheimer’s Disease Drug Development
- Alzheimer’s Disease Drug Development
- Copyright page
- Dedication
- Contents
- Contributors
- Foreword
- Acknowledgments
- Section 1 Advancing Alzheimer’s Disease Therapies in a Collaborative Science Ecosystem
- Section 2 Non-clinical Assessment of Alzheimer’s Disease Candidate Drugs
- Section 3 Alzheimer’s Disease Clinical Trials
- 11 Phase 1 Trials in Alzheimer’s Disease Drug Development
- 12 The Importance of Phase 2 in Drug Development for Alzheimer’s Disease
- 13 Alzheimer’s Disease Drug Development in Pharmaceutical Companies
- 14 Trial Site Infrastructure and Management: Importance to Alzheimer’s Disease Drug Development
- 15 ATRI and ACTC: Academic Programs to Accelerate Alzheimer’s Disease Drug Development
- 16 The European Prevention of Alzheimer’s Disease Program: A Public–Private Partnership to Facilitate the Secondary Prevention of Alzheimer’s Disease Dementia
- 17 The Global Alzheimer’s Platform Foundation®: Delivering New Medicines Faster by Accelerating Clinical Trials
- 18 Clinical Trial Development in Frontotemporal Lobar Degeneration
- 19 Statistical Considerations in the Design and Analysis of Alzheimer’s Disease Clinical Trials
- 20 Alzheimer’s Disease Trial Recruitment and Diversifying Trial Populations
- 21 The Role of Online Registries in Accelerating Alzheimer’s Disease Drug Development
- 22 Data Safety Monitoring Boards in Alzheimer’s Disease Trials
- 23 Globalization of Alzheimer’s Disease Clinical Trials
- 24 The Use and Development of Clinical Measures of Alzheimer’s Disease Trials
- 25 Tele-Trials, Remote Monitoring, and Trial Technology for Alzheimer’s Disease Clinical Trials
- 26 Expanded Access and Compassionate Use in Alzheimer’s Disease Drug Development
- 27 The Role of the Contract Research Organization in Alzheimer’s Disease: The Vital Link in the Clinical Drug-Development Program
- 28 The Role of Regulatory Agencies in Alzheimer’s Disease Drug Development
- 29 Alzheimer’s Disease Clinical Trial Study Partners
- 30 From Trials to Practice: Are We Ready for a Disease-Modifying Treatment?
- 31 Best Practices for Clinical Trials during COVID-19
- Section 4 Imaging and Biomarker Development in Alzheimer’s Disease Drug Discovery
- Section 5 Academic Drug-Development Programs
- Section 6 Public–Private Partnerships in Alzheimer’s Disease Drug Development
- Section 7 Funding and Financing Alzheimer’s Disease Drug Development
- Index
- References
Summary
All agents approved for marketing must meet the regulatory standards of the country in which they will be marketed, and drug development programs are designed to meet these regulatory requirements. There are frequent interactions between regulators and sponsors during the development and trial process. The FDA in the USA, the EMA in Europe, the NMPA in China, and the Japanese PMDA and other national regulatory bodies communicate regularly and share information. Regulators promote drug development for serious diseases with unmet needs such as Alzheimer’s disease (AD) and can use regulatory tools to facilitate the drug development process. Regulatory agencies oversee the manufacture of treatments (drugs, monoclonal antibodies) in addition to supervising the development process through clinical trials. Regulatory science and regulatory specifications evolve in concert with new knowledge. Agencies now recognize mild cognitive impairment and preclinical AD as populations where therapeutic indications could apply. Agencies have integrated biomarkers into their concepts of drug development and have specified approaches to biomarker qualification.
Keywords
- Type
- Chapter
- Information
- Alzheimer's Disease Drug DevelopmentResearch and Development Ecosystem, pp. 319 - 332Publisher: Cambridge University PressPrint publication year: 2022
References
Food and Drug Administration. Peripheral and Central Nervous System Drugs Advisory Committee meeting. Available at: www.fda.gov/advisory-committees/human-drug-advisory-committees/peripheral-and-central-nervous-system-drugs-advisory-committee (accessed January 2021).Google Scholar
European Medicines Agency. Ebixa (memantine). Available at: www.ema.europa.eu/en/medicines/human/EPAR/ebixa (accessed January 2021).Google Scholar
Chinese National Medical Products Agency. Treatment for Alzheimer’s available by end of 2019. Available at: http://english.nmpa.gov.cn/2019-11/04/c_422037.htm (accessed January 2021).Google Scholar
Food and Drug Administration. Alzheimer’s disease: developing drugs for treatment. Guidance for industry. 2018. Available at: www.fda.gov/regulatory-information/search-fda-guidance-documents/alzheimers-disease-developing-drugs-treatment-guidance-industy (accessed January 2021).Google Scholar
European Medicines Agency. Guideline on the clinical investigation of medicines to treat Alzheimer’s disease, 2018. Available at: www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-investigation-medicines-treatment-alzheimers-disease-revision-2_en.pdf (accessed January 2021).Google Scholar
The University of Tokyo Hospital. Project to promote the development of innovative pharmaceuticals, medical devices, and regenerative medical products (Ministry of Health, Labour, and Welfare) regulatory science research for the establishment of criteria for clinical evaluation of drugs for Alzheimer’s disease, issues to consider in the clinical evaluation and development of drugs for Alzheimer’s disease. Available at: www.pmda.go.jp/files/000221585.pdf (accessed January 2021).Google Scholar
Jack, CR, Bennett, DA, Blennow, K, et al. NIA–AA Research Framework: toward a biological definition of Alzheimer’s disease. Alzheimers Dement 2018; 14: 535–62.CrossRefGoogle Scholar
European Medicines Agency. Connecting the dots towards global knowledge of the international medicine regulatory landscape: mapping of international initiatives. Available at: www.ema.europa.eu/en/documents/leaflet/connecting-dots-towards-global-knowledge-international-medicine-regulatory-landscape-mapping_en.pdf (accessed January 2021).Google Scholar
Food and Drug Administration. 21st Century Cures Act. Available at: www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-century-cures-act (accessed January 2021).Google Scholar
European Medicines Agency. EU Medicines Agencies network strategy to 2020, working together to improve health. Available at: www.ema.europa.eu/en/documents/other/eu-medicines-agencies-network-strategy-2020-working-together-improve-health_en.pdf (accessed January 2021).Google Scholar
European Medicines Agency. Regulatory science strategy 2025. Available at: www.ema.europa.eu/en/about-us/how-we-work/regulatory-science-strategy#regulatory-science-strategy-to-2025-section (accessed January 2021).Google Scholar
National Medical Products Administration (NMPA). http://english.nmpa.gov.cn/drugs.html (accessed January 2021).Google Scholar
Engen, S. The improving Japanese pharmaceutical regulatory environment. Available at: Locustwalk.com/the-improving-japanese-pharmaceutical-regulatory-environment/ (accessed January 2021).Google Scholar
Food and Drug Administration. Investigational new drug (IND) application. Available at: www.fda.gov/drugs/types-applications/investigational-new-drug-ind-application (accessed January 2021).Google Scholar
Food and Drug Administration. Formal meetings between the FDA and sponsors or applicants of PDUFA products guidance for industry, December 2017. Available at: www.fda.gov/regulatory-information/search-fda-guidance-documents/formal-meetings-between-fda-and-sponsors-or-applicants-pdufa-products-guidance-industry (accessed January 2021).Google Scholar
Food and Drug Administration. Special protocol assessment guidance for industry, April 2018. Available at: www.fda.gov/regulatory-information/search-fda-guidance-documents/special-protocol-assessment-guidance-industry (accessed January 2021).Google Scholar
Food and Drug Administration. Expedited programs for serious conditions: drugs and biologics, May 2014. Available at: www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics (accessed January 2021).Google Scholar
European Medicines Agency. Clinical trial regulation. Available at: www.ema.europa.eu/en/human-regulatory/research-development/clinical-trials/clinical-trial-regulation (accessed January 2021).Google Scholar
European Medicines Agency. Scientific advice and protocol assistance. Available at: www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance (accessed January 2021).Google Scholar
European Medicines Agency. Adaptive pathways. Available at: www.ema.europa.eu/en/human-regulatory/research-development/adaptive-pathways (accessed January 2021).Google Scholar
European Medicines Agency. PRIME: priority medicines. Available at: www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines (accessed January 2021).Google Scholar
European Medicines Agency. Conditional marketing authorization. Available at: www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation (accessed January 2021).Google Scholar
European Medicines Agency. Exceptional circumstances. Available at: www.ema.europa.eu/en/glossary/exceptional-circumstances (accessed January 2021).Google Scholar
Japan Pharmaceutical Manufacturers Association. Pharmaceutical Administration and Regulations in Japan. Available at: www.jpma.or.jp/english/parj/ (accessed January 2021).Google Scholar
Kajiwara, E, Shikano, M. Considerations and regulatory challenges for innovative medicines in expedited approval programs: breakthrough therapy and Sakigake designation. Ther Innov Regul Sci 2020; 54: 814–20.CrossRefGoogle ScholarPubMed
Kondo, H, Sugita, T, Ida, N, Fukushima, H, Yasuda, N. A comparison of PMDA and EMA consultations for regulatory and scientific matters in drugs and regenerative medicine products. Ther Innov Regul Sci 2017; 51: 355–9.Google Scholar
Zhuxing, Y, Haixue, W. The regulatory requirements and critical points of drug clinical trials registration in China. Available at: www.appliedclinicaltrialsonline.com/view/regulatory-requirements-and-key-points-drug-clinical-trials-registration-china (accessed January 2021).Google Scholar
Jianqing, C. What’s new for conditional approval of drugs in China? Tigermed Insight. Available at: https://tigermedgrp.com/whats-new-for-conditional-approval-of-drugs-in-china/ (accessed January 2021).Google Scholar
Covington and Burling. China promulgates revised drug registration regulation, August 2020. Available at: www.cov.com/-/media/files/corporate/publications/2020/04/china-promulgates-revised-drug-registration-regulation.pdf (accessed January 2021).Google Scholar
Centre for Innovation and Regulatory Science. CIRS RD Briefing 77: new drug approvals in six major authorities, June 2020. Available at: www.cirsci.org/publications/cirs-rd-briefing-77-new-drug-approvals-in-six-major-authorities/ (accessed January 2021).Google Scholar
Xu, W, Yuanyuan, D. New drug approvals in China in 2019. Available at: https://globalforum.diaglobal.org/issue/may-2020/new-drug-approvals-in-china-in-2019/ (accessed January 2021).Google Scholar
Food and Drug Administration. Qualification process for drug development tools guidance for industry and FDA staff, November, 2020. Available at: www.fda.gov/regulatory-information/search-fda-guidance-documents/qualification-process-drug-development-tools-guidance-industry-and-fda-staff (accessed January 2021).Google Scholar
European Medicines Agency. Qualification of novel methodologies for medicine development. Available at: www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance/qualification-novel-methodologies-medicine-development-0 (accessed January 2021).Google Scholar
Food and Drug Administration. About biomarkers and qualification. Available at: www.fda.gov/drugs/biomarker-qualification-program/about-biomarkers-and-qualification (accessed January 2021).Google Scholar
European Medicines Agency. Opinions and letters of support on the qualification of novel methodologies for medicine development. Available at: www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance/novel-methodologies-biomarkers/opinions-letters-support-qualification-novel-methodologies-medicine-development (accessed January 2021).Google Scholar
Arnerić, SP, Batrla-Utermann, R, Beckett, L, et al. Cerebrospinal fluid biomarkers for Alzheimer’s disease: a view of the regulatory science qualification landscape from the Coalition Against Major Diseases CSF Biomarker Team. J Alzheimers Dis 2017; 55: 19–35.Google Scholar
Sauer, JM, Porter, AC; Biomarker Programs, Predictive Safety Testing Consortium. Preclinical biomarker qualification. Exp Biol Med (Maywood) 2018; 243: 222–7.Google Scholar
Pacifici, E, Bain, S (eds.). An Overview of FDA Regulated Products: From Drugs and Cosmetics to Food and Tobacco. Cambridge, MA: Academic Press; 2018.Google Scholar
Van Norman, GA. Drugs and devices: comparison of European and US approval processes. J Am Coll Cardiol Basic Transl Sci 2016; 1: 399–412.Google Scholar
Food and Drug Administration. Benefit–risk assessment in drug regulatory decision making. Draft PDUFA VI implementation plan (FY 2018-2022). Available at: www.fda.gov/files/about%20fda/published/Benefit-Risk-Assessment-in-Drug-Regulatory-Decision-Making.pdf (accessed January 2021).Google Scholar
Juhaeri, J. Benefit–risk evaluation: the past, present and future. Ther Adv Drug Saf 2019; 10: 2042098619871180;DOI: http://doi.org/10.1177/2042098619871180.Google Scholar
European Medicines Agency. Benefit–risk methodology. Available at: www.ema.europa.eu/en/about-us/support-research/benefit-risk-methodology (accessed January 2021).Google Scholar
Morant, AV, Vestergaard, HT, Blædel-Lassen, A, Navikas, V. US, EU, and Japanese regulatory guidelines for development of drugs for treatment of Alzheimer’s disease: implications for global drug development. Clin Transl Sci 2020; 13: 652–64.Google Scholar
Cummings, J, Lee, G, Ritter, A, Sabbagh, M, Zhong, K. Alzheimer’s disease drug development pipeline: 2020. Alzheimers Dement (N Y) 2020; 6: e12050.Google Scholar