RAC Regulatory Submissions and Approval 3 — Questions and Answers
Question 1: What does a 505(b)(2) NDA pathway allow a sponsor to do?
- Submit without any clinical data
- Rely in part on published literature or FDA's prior findings for a previously approved drug (Correct answer)
- Bypass FDA review entirely for OTC products
- Submit a biosimilar application
Correct answer: Rely in part on published literature or FDA's prior findings for a previously approved drug
The 505(b)(2) pathway allows sponsors to rely on published studies or FDA's prior approval findings, reducing the amount of new clinical data needed.
Question 2: Which ICH guideline specifically addresses the structure and content of clinical study reports for regulatory submissions?
- ICH E3 (Correct answer)
- ICH E6
- ICH E8
- ICH E9
Correct answer: ICH E3
ICH E3 provides guidance on the structure and content of clinical study reports submitted to regulatory authorities.
Question 3: Under FDA's 21 CFR 312.23, which of the following is NOT a required section of an IND application?
- Investigator's Brochure
- Clinical protocols
- Chemistry, Manufacturing, and Controls information
- Post-marketing surveillance plan (Correct answer)
Correct answer: Post-marketing surveillance plan
A post-marketing surveillance plan is a post-approval requirement, not a component of an initial IND application.
Question 4: What is the significance of the 'refuse to file' (RTF) decision in NDA/BLA review?
- FDA rejects the application permanently
- FDA determines the application is not sufficiently complete to permit substantive review (Correct answer)
- FDA requests additional clinical trials be conducted
- FDA approves the product conditionally
Correct answer: FDA determines the application is not sufficiently complete to permit substantive review
An RTF decision means FDA found the application administratively or scientifically incomplete and will not begin the 10- or 6-month review clock.
Question 5: Which of the following best describes a Risk Evaluation and Mitigation Strategy (REMS)?
- A post-market study commitment required for all new drugs
- A program to ensure a drug's benefits outweigh its risks through specific safety measures (Correct answer)
- A voluntary industry program to reduce counterfeit medications
- An FDA advisory committee review process
Correct answer: A program to ensure a drug's benefits outweigh its risks through specific safety measures
REMS are FDA-required programs for drugs with serious safety concerns that mandate specific interventions to ensure safe use.
Question 6: When must a sponsor submit a Safety Report (IND Safety Report) to the FDA during clinical trials?
- Only at the end of the trial
- Within 7 or 15 calendar days for serious unexpected suspected adverse reactions (SUSARs) (Correct answer)
- Annually, with the IND Annual Report
- Only when the FDA requests it
Correct answer: Within 7 or 15 calendar days for serious unexpected suspected adverse reactions (SUSARs)
Fatal or life-threatening unexpected SUSARs must be reported within 7 calendar days; other serious unexpected SUSARs within 15 calendar days.
Question 7: What is the purpose of a Pediatric Study Plan (PSP) under PREA in the US?
- To waive all pediatric studies for a new drug
- To agree with FDA on the studies needed to assess a drug's safety and efficacy in children (Correct answer)
- To extend market exclusivity by 5 years
- To register the product in pediatric hospitals
Correct answer: To agree with FDA on the studies needed to assess a drug's safety and efficacy in children
An agreed PSP under PREA (Pediatric Research Equity Act) establishes the pediatric studies required and the timeline for completion before or after approval.
RAC Regulatory Submissions and Approval 3
What does a 505(b)(2) NDA pathway allow a sponsor to do?