Fundamentals of Pharmaceutical and Biologics Regulations: A Global Perspective
Second Edition
16 Regulatory Affairs Professionals Society
References (continued)
3. Food and Drug Administration. Exclusivity and generic drugs – What does it
mean? Date of last update unknown. https://www.fda.gov/files/drugs/
published/Exclusivity-and-Generic-Drugs--What-Does-It-Mean-.pdf
4. Food and Drug Administration. Determining whether to submit an ANDA or
505(b)(2) application [Guidance document]. Issued May 2019. Last updated
3 March 2020. https://www.fda.gov/regulatory-information/search-fda-
guidance-documents/determining-whether-submit-anda-or-505b2-application
5. Official Journal of the EU. Directive 2001/83/EC of the European Parliament
and of the Council of 6 November 2001. Last Updated 1 January 2025. https://
eur-lex.europa.eu/legal-content/en/TXT/?uri=CELEX%3A32001L0083
6. Food and Drug Administration. Generic drugs: Questions and answers.
Updated 16 March 2021. https://www.fda.gov/drugs/frequently-asked-
questions-popular-topics/generic-drugs-questions-answers
7. European Medicines Agency. Generic and hybrid medicines. https://www.
ema.europa.eu/en/human-regulatory/marketing-authorisation/generic-hybrid-
medicines
8. European Medicines Agency. Biosimilar medicines: Overview. https://www.
ema.europa.eu/en/human-regulatory/overview/biosimilar-medicines-overview
9. Food and Drug Administration. Biosimilars and interchangeable biologics:
More treatment choices. Current as of 17 August 2023. https://www.fda.
gov/consumers/consumer-updates/biosimilar-and-interchangeable-biologics-
more-treatment-choices
10. European Medicines Agency. Omnitrope. Last updated 20 September 2023.
https://www.ema.europa.eu/en/medicines/human/EPAR/omnitrope#
authorisation-details-section
11. Drugs@FDA. BLA 125553 Approval Letter, 6 March 2015. https://www.
accessdata.fda.gov/drugsatfda_docs/appletter/2015/125553Orig1s000ltr.pdf
12. Quartz. Viagra’s famously surprising origin story is actually a pretty common
way to find new drugs. Last updated 20 July 2022. https://qz.com/1070732/
viagras-famously-surprising-origin-story-is-actually-a-pretty-common-way-to-
find-new-drugs
13. Kim JH, Scialli AR. Thalidomide: The tragedy of birth defects and the
effective treatment of disease. Toxicol Sci. 2011 Jul 122(1):1-6. 19 April 2011.
Erratum in: Toxicol Sci. 2012 Feb 125(2):613. https://doi.org/10.1093/
toxsci/kfr088
14. Huang R, Southall N, Wang Y, Yasgar A, Shinn P, Jadhav A, Nguyen DT,
Austin CP. The NCGC pharmaceutical collection: A comprehensive resource
of clinically approved drugs enabling repurposing and chemical genomics.
Science Translational Medicine. 2011 3(80):80ps16. https://doi.org/10.1126/
scitranslmed.3001862
15. Drugs@FDA. BLA 125514. https://www.accessdata.fda.gov/scripts/cder/daf/
index.cfm?event=overview.process&ApplNo=125514
16. Rudrapa M et al. Drug repurposing (DR): An emerging approach in drug
discovery. In Badria AF, ed. Drug Repurposing – Hypothesis, Molecular
Aspects and Therapeutic Applications. https://www.intechopen.com/
chapters/72744
17. BIO/QLS Advisors/Informa UK Ltd. Clinical development success rates and
contributing factors 2011-2020. https://www.bio.org/clinical-development-
success-rates-and-contributing-factors-2011-2020
18. Hughes JP, Rees S, Kalindjian SB, Philpott KL. Principles of early drug
discovery. Br J Pharmacol. 2011 Mar 126(6):1239-49. https://bpspubs.
onlinelibrary.wiley.com/doi/10.1111/j.1476-5381.2010.01127.x
19. Food and Drug Administration. Content and format of investigational new
drug applications (INDs) for Phase 1 studies of drugs, including well-
characterized, therapeutic, biotechnology-derived products [Guidance
document]. Current as of 6 May 2020. https://www.fda.gov/regulatory-
information/search-fda-guidance-documents/content-and-format-
investigational-new-drug-applications-inds-phase-1-studies-drugs-including-well
20. National Institutes of Health. NIH’s definition of a clinical trial. Last updated
18 September 2024. https://grants.nih.gov/policy/clinical-trials/definition.htm
21. International Council for Harmonisation. General considerations for clinical
studies E8(R1) [guideline]. Adopted 6 October 2021. https://database.ich.org/
sites/default/files/ICH_E8-R1_Guideline_Step4_2021_1006.pdf
22. International Council for Harmonisation (ICH). Guideline on good clinical
practice E6(R3). Adopted 6 January 2025. https://database.ich.org/sites/
default/files/ICH_E6%28R3%29_Step4_FinalGuideline_2025_0106.pdf
23. National Institutes of Health. NIH clinical research trials and you: The basics.
Last reviewed 24 April 2025. https://www.nih.gov/health-information/nih-
clinical-research-trials-you/basics
24. Burt T, et al. Phase 0/microdosing approaches: Time for mainstream application
in drug development? Nat Rev Drug Discov. 2020 19(11):801-818. https://doi.
org/10.1038/s41573-020-0080-x
25. Food and Drug Administration. Vioxx (rofecoxib) Questions and answers.
Current as of 6 April 2016. https://www.fda.gov/drugs/postmarket-drug-
safety-information-patients-and-providers/vioxx-rofecoxib-questions-and-
answers
26. Pharmaguideline. International health regulatory bodies. https://www.
pharmaguideline.com/2011/02/international-regulatory-bodies.html
27. International Council for Harmonisation. History. https://www.ich.org/
page/history
28. Biomapas. eCTD implementation across MENA region: What is the current
status? https://www.biomapas.com/ectd-implementation-across-mena-region-
what-is-the-current-status/
29. Food and Drug Administration. Electronic common technical document
(eCTD) v4.0. Last updated 19 September 2024. https://www.fda.gov/drugs/
electronic-regulatory-submission-and-review/electronic-common-technical-
document-ectd-v40
30. International Council for Harmonisation. CTD. https://www.ich.org/page/ctd
31. European Agency for the Evaluation of Medicinal Products. Work programme
2023. Adopted 19 December 2002. https://www.ema.europa.eu/en/documents/
work-programme/work-programme-european-agency-evaluation-medicinal-
products-2003_en.pdf
32. European Medicines Agency. EMEA implementation of electronic-only
submissions and eCTD submissions: Practical guidelines relating to non-
eCTD electronic submissions. December 2008 EMEA/6339/2008 V1.
https://www.ema.europa.eu/en/documents/regulatory-procedural-
guideline/emea-implementation-electronic-only-submission-ectd-submission-
practical-guidelines-relating-non_en.pdf
33. European Medicines Agency. Regulatory information – Transitioning to
mandatory use of electronic application forms. 25 February 2015. https://www.
ema.europa.eu/en/news/regulatory-information-transitioning-mandatory-use-
electronic-application-forms
34. FDA Guidance for Industry, Providing regulatory submissions in electronic
format – Certain human pharmaceutical product applications and related
submissions using the eCTD specifications. September 2024. https://www.fda.
gov/media/135373/download
35. Pharmaceuticals and Medical Devices Agency. ICH electronic common
technical document (eCTD) v4.0 implementation guide in Japan v1.20.
Provisional Translation (as of December 2017). https://www.pmda.go.jp/
files/000222267.pdf
36. Government of Canada. Notice – Mandatory requirements for using the
Common Electronic Submissions Gateway (CESG). https://www.canada.
ca/en/health-canada/services/drugs-health-products/drug-products/
announcements/notice-mandatory-requirements-using-common-electronic-
submissions-gateway.html
37. International Council of Harmonisation. ICH M8 electronic common
technical document (eCTD) v3.2.2 Implementation Status. Date of last update
unknown. https://www.ich.org/page/multidisciplinary-guidelines
38. Food and Drug Administration. FDA: User fees explained. Current as of
22 May 2024. https://www.fda.gov/industry/fda-user-fee-programs/fda-user-fees-
explained
Second Edition
16 Regulatory Affairs Professionals Society
References (continued)
3. Food and Drug Administration. Exclusivity and generic drugs – What does it
mean? Date of last update unknown. https://www.fda.gov/files/drugs/
published/Exclusivity-and-Generic-Drugs--What-Does-It-Mean-.pdf
4. Food and Drug Administration. Determining whether to submit an ANDA or
505(b)(2) application [Guidance document]. Issued May 2019. Last updated
3 March 2020. https://www.fda.gov/regulatory-information/search-fda-
guidance-documents/determining-whether-submit-anda-or-505b2-application
5. Official Journal of the EU. Directive 2001/83/EC of the European Parliament
and of the Council of 6 November 2001. Last Updated 1 January 2025. https://
eur-lex.europa.eu/legal-content/en/TXT/?uri=CELEX%3A32001L0083
6. Food and Drug Administration. Generic drugs: Questions and answers.
Updated 16 March 2021. https://www.fda.gov/drugs/frequently-asked-
questions-popular-topics/generic-drugs-questions-answers
7. European Medicines Agency. Generic and hybrid medicines. https://www.
ema.europa.eu/en/human-regulatory/marketing-authorisation/generic-hybrid-
medicines
8. European Medicines Agency. Biosimilar medicines: Overview. https://www.
ema.europa.eu/en/human-regulatory/overview/biosimilar-medicines-overview
9. Food and Drug Administration. Biosimilars and interchangeable biologics:
More treatment choices. Current as of 17 August 2023. https://www.fda.
gov/consumers/consumer-updates/biosimilar-and-interchangeable-biologics-
more-treatment-choices
10. European Medicines Agency. Omnitrope. Last updated 20 September 2023.
https://www.ema.europa.eu/en/medicines/human/EPAR/omnitrope#
authorisation-details-section
11. Drugs@FDA. BLA 125553 Approval Letter, 6 March 2015. https://www.
accessdata.fda.gov/drugsatfda_docs/appletter/2015/125553Orig1s000ltr.pdf
12. Quartz. Viagra’s famously surprising origin story is actually a pretty common
way to find new drugs. Last updated 20 July 2022. https://qz.com/1070732/
viagras-famously-surprising-origin-story-is-actually-a-pretty-common-way-to-
find-new-drugs
13. Kim JH, Scialli AR. Thalidomide: The tragedy of birth defects and the
effective treatment of disease. Toxicol Sci. 2011 Jul 122(1):1-6. 19 April 2011.
Erratum in: Toxicol Sci. 2012 Feb 125(2):613. https://doi.org/10.1093/
toxsci/kfr088
14. Huang R, Southall N, Wang Y, Yasgar A, Shinn P, Jadhav A, Nguyen DT,
Austin CP. The NCGC pharmaceutical collection: A comprehensive resource
of clinically approved drugs enabling repurposing and chemical genomics.
Science Translational Medicine. 2011 3(80):80ps16. https://doi.org/10.1126/
scitranslmed.3001862
15. Drugs@FDA. BLA 125514. https://www.accessdata.fda.gov/scripts/cder/daf/
index.cfm?event=overview.process&ApplNo=125514
16. Rudrapa M et al. Drug repurposing (DR): An emerging approach in drug
discovery. In Badria AF, ed. Drug Repurposing – Hypothesis, Molecular
Aspects and Therapeutic Applications. https://www.intechopen.com/
chapters/72744
17. BIO/QLS Advisors/Informa UK Ltd. Clinical development success rates and
contributing factors 2011-2020. https://www.bio.org/clinical-development-
success-rates-and-contributing-factors-2011-2020
18. Hughes JP, Rees S, Kalindjian SB, Philpott KL. Principles of early drug
discovery. Br J Pharmacol. 2011 Mar 126(6):1239-49. https://bpspubs.
onlinelibrary.wiley.com/doi/10.1111/j.1476-5381.2010.01127.x
19. Food and Drug Administration. Content and format of investigational new
drug applications (INDs) for Phase 1 studies of drugs, including well-
characterized, therapeutic, biotechnology-derived products [Guidance
document]. Current as of 6 May 2020. https://www.fda.gov/regulatory-
information/search-fda-guidance-documents/content-and-format-
investigational-new-drug-applications-inds-phase-1-studies-drugs-including-well
20. National Institutes of Health. NIH’s definition of a clinical trial. Last updated
18 September 2024. https://grants.nih.gov/policy/clinical-trials/definition.htm
21. International Council for Harmonisation. General considerations for clinical
studies E8(R1) [guideline]. Adopted 6 October 2021. https://database.ich.org/
sites/default/files/ICH_E8-R1_Guideline_Step4_2021_1006.pdf
22. International Council for Harmonisation (ICH). Guideline on good clinical
practice E6(R3). Adopted 6 January 2025. https://database.ich.org/sites/
default/files/ICH_E6%28R3%29_Step4_FinalGuideline_2025_0106.pdf
23. National Institutes of Health. NIH clinical research trials and you: The basics.
Last reviewed 24 April 2025. https://www.nih.gov/health-information/nih-
clinical-research-trials-you/basics
24. Burt T, et al. Phase 0/microdosing approaches: Time for mainstream application
in drug development? Nat Rev Drug Discov. 2020 19(11):801-818. https://doi.
org/10.1038/s41573-020-0080-x
25. Food and Drug Administration. Vioxx (rofecoxib) Questions and answers.
Current as of 6 April 2016. https://www.fda.gov/drugs/postmarket-drug-
safety-information-patients-and-providers/vioxx-rofecoxib-questions-and-
answers
26. Pharmaguideline. International health regulatory bodies. https://www.
pharmaguideline.com/2011/02/international-regulatory-bodies.html
27. International Council for Harmonisation. History. https://www.ich.org/
page/history
28. Biomapas. eCTD implementation across MENA region: What is the current
status? https://www.biomapas.com/ectd-implementation-across-mena-region-
what-is-the-current-status/
29. Food and Drug Administration. Electronic common technical document
(eCTD) v4.0. Last updated 19 September 2024. https://www.fda.gov/drugs/
electronic-regulatory-submission-and-review/electronic-common-technical-
document-ectd-v40
30. International Council for Harmonisation. CTD. https://www.ich.org/page/ctd
31. European Agency for the Evaluation of Medicinal Products. Work programme
2023. Adopted 19 December 2002. https://www.ema.europa.eu/en/documents/
work-programme/work-programme-european-agency-evaluation-medicinal-
products-2003_en.pdf
32. European Medicines Agency. EMEA implementation of electronic-only
submissions and eCTD submissions: Practical guidelines relating to non-
eCTD electronic submissions. December 2008 EMEA/6339/2008 V1.
https://www.ema.europa.eu/en/documents/regulatory-procedural-
guideline/emea-implementation-electronic-only-submission-ectd-submission-
practical-guidelines-relating-non_en.pdf
33. European Medicines Agency. Regulatory information – Transitioning to
mandatory use of electronic application forms. 25 February 2015. https://www.
ema.europa.eu/en/news/regulatory-information-transitioning-mandatory-use-
electronic-application-forms
34. FDA Guidance for Industry, Providing regulatory submissions in electronic
format – Certain human pharmaceutical product applications and related
submissions using the eCTD specifications. September 2024. https://www.fda.
gov/media/135373/download
35. Pharmaceuticals and Medical Devices Agency. ICH electronic common
technical document (eCTD) v4.0 implementation guide in Japan v1.20.
Provisional Translation (as of December 2017). https://www.pmda.go.jp/
files/000222267.pdf
36. Government of Canada. Notice – Mandatory requirements for using the
Common Electronic Submissions Gateway (CESG). https://www.canada.
ca/en/health-canada/services/drugs-health-products/drug-products/
announcements/notice-mandatory-requirements-using-common-electronic-
submissions-gateway.html
37. International Council of Harmonisation. ICH M8 electronic common
technical document (eCTD) v3.2.2 Implementation Status. Date of last update
unknown. https://www.ich.org/page/multidisciplinary-guidelines
38. Food and Drug Administration. FDA: User fees explained. Current as of
22 May 2024. https://www.fda.gov/industry/fda-user-fee-programs/fda-user-fees-
explained