API, biologics, novel therapy approvals MCQs With Answer

API, Biologics, Novel Therapy Approvals MCQs With Answer have been curated to help M. Pharm students master core regulatory pathways and quality requirements that drive modern drug development. This set covers key frameworks across the US, EU, and India—ranging from CTD/eCTD structure, DMFs, and CEPs for APIs to BLAs, biosimilar pathways, and ATMP classifications for advanced therapies. You will also practice decision-making concepts around expedited programs like Fast Track, Breakthrough Therapy, Priority Review, Accelerated Approval, and RMAT. Questions delve into ICH guidelines (Q1, Q5, Q7, Q11), comparability for biologics, and critical quality attributes relevant to novel modalities such as cell and gene therapies. Each MCQ includes the correct answer to support efficient self-assessment.

Q1. In the CTD, where should the primary information for the drug substance (API) be placed?

• Module 3.2.S – Drug Substance
• Module 3.2.P – Drug Product
• Module 4 – Nonclinical Study Reports
• Module 2 – Quality Overall Summary

Correct Answer: Module 3.2.S – Drug Substance

Q2. Which US DMF type is appropriate for a drug substance (API) and its intermediates?

• Type I – Manufacturing Site and Facilities
• Type II – Drug Substance and Intermediates
• Type III – Packaging Materials
• Type IV – Excipients and Colorants

Correct Answer: Type II – Drug Substance and Intermediates

Q3. What is the primary purpose of a European Pharmacopoeia CEP issued by EDQM?

• It confirms the API’s compliance with a Ph. Eur. monograph, allowing streamlined API sections in EU dossiers
• It replaces GMP certification for the API manufacturing site
• It grants EU marketing authorization for the final drug product
• It covers only medical device packaging compliance

Correct Answer: It confirms the API’s compliance with a Ph. Eur. monograph, allowing streamlined API sections in EU dossiers

Q4. In the United States, original marketing applications for biologics are typically submitted as:

• BLA under PHS Act Section 351(a)
• NDA under FD&C Act Section 505(b)(1)
• ANDA under FD&C Act Section 505(j)
• Premarket notification under 510(k)

Correct Answer: BLA under PHS Act Section 351(a)

Q5. The US pathway for biosimilar approval is:

• 505(b)(2) NDA
• 351(k) BLA
• 505(j) ANDA
• De Novo Classification

Correct Answer: 351(k) BLA

Q6. In India, which bodies are primarily involved in review and approval of recombinant biologics (marketing authorization)?

• CDSCO (DCGI) with scientific oversight involving DBT/RCGM; GEAC for environmental aspects
• FSSAI as the sole agency
• PCI and AICTE jointly
• ISO certification bodies

Correct Answer: CDSCO (DCGI) with scientific oversight involving DBT/RCGM; GEAC for environmental aspects

Q7. Accelerated Approval in the US can be based on:

• Surrogate endpoints reasonably likely to predict clinical benefit
• Nonclinical pharmacology data alone
• Manufacturing scale-up data without clinical evidence
• Historical controls with no biological plausibility

Correct Answer: Surrogate endpoints reasonably likely to predict clinical benefit

Q8. Breakthrough Therapy Designation (BTD) requires:

• Preliminary clinical evidence of substantial improvement over existing therapy on clinically significant endpoints
• Completion of at least one Phase 3 trial
• A granted Orphan Drug Designation
• Only nonclinical evidence of superiority

Correct Answer: Preliminary clinical evidence of substantial improvement over existing therapy on clinically significant endpoints

Q9. Priority Review in the US most directly impacts which aspect?

• The PDUFA review goal timeline, reducing it generally from 10 to 6 months
• The number of patients required for Phase 3
• Postmarketing surveillance obligations
• CMC validation requirements

Correct Answer: The PDUFA review goal timeline, reducing it generally from 10 to 6 months

Q10. A key regulatory benefit unique to Fast Track Designation is:

• Rolling review of completed sections of the marketing application
• Automatic approval upon submission
• Exemption from Phase 3 studies
• Waiver of all user fees

Correct Answer: Rolling review of completed sections of the marketing application

Q11. RMAT designation applies to:

• Regenerative medicine therapies with preliminary clinical evidence, enabling intensive FDA guidance and potential accelerated approval
• Only small-molecule oncology drugs with animal efficacy
• Devices that replace tissue function via mechanical means
• Excipients used in sterile formulations

Correct Answer: Regenerative medicine therapies with preliminary clinical evidence, enabling intensive FDA guidance and potential accelerated approval

Q12. In the EU, Advanced Therapy Medicinal Products (ATMPs) include:

• Gene therapies, somatic cell therapies, and tissue-engineered products
• Only herbal medicinal products
• In vitro diagnostics
• Parenteral nutrition solutions

Correct Answer: Gene therapies, somatic cell therapies, and tissue-engineered products

Q13. The ICH guideline specifically addressing stability testing for biotechnological/biological products is:

• ICH Q5C
• ICH Q1A(R2)
• ICH Q9
• ICH E6(R2)

Correct Answer: ICH Q5C

Q14. Which ICH guideline focuses on development and manufacture of drug substances (including biotech/biological)?

• ICH Q11
• ICH Q7
• ICH Q10
• ICH E8

Correct Answer: ICH Q11

Q15. The key GMP guideline for APIs globally recognized in CTD submissions is:

• ICH Q7
• ICH Q2(R2)
• ICH E14
• ICH M7(R2)

Correct Answer: ICH Q7

Q16. For biologics, demonstrating similarity before and after manufacturing changes is guided by:

• ICH Q5E – Comparability of Biotechnological/Biological Products
• ICH Q1B – Photostability Testing
• ICH E9 – Statistical Principles
• ICH S9 – Nonclinical Evaluation for Anticancer Pharmaceuticals

Correct Answer: ICH Q5E – Comparability of Biotechnological/Biological Products

Q17. In eCTD, which module is region-specific and contains administrative information?

• Module 1
• Module 2
• Module 3
• Module 5

Correct Answer: Module 1

Q18. Within the EMA, which committee is primarily responsible for ATMP classification and scientific recommendations?

• Committee for Advanced Therapies (CAT)
• Committee for Medicinal Products for Human Use (CHMP)
• Pharmacovigilance Risk Assessment Committee (PRAC)
• Coordination Group for Mutual Recognition and Decentralised Procedures – Human (CMDh)

Correct Answer: Committee for Advanced Therapies (CAT)

Q19. In the US, Orphan Drug Designation confers a period of marketing exclusivity of:

• 7 years
• 5 years
• 10 years
• 12 years

Correct Answer: 7 years

Q20. For CAR-T products, which is a commonly used functional potency measure for lot release?

• Cytotoxicity against antigen-positive target cells
• pH of the formulation buffer
• Container closure extractable profile
• Non-specific hemagglutination titer

Correct Answer: Cytotoxicity against antigen-positive target cells

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