CRAP Regulatory Strategy & Submission Management 2 — Questions and Answers
Question 1: A company wants to market a drug in the EU and US simultaneously. Which regulatory strategy best supports parallel submissions?
- Submit to EMA first, then FDA after approval
- Use ICH CTD format for both agencies and align clinical data packages (Correct answer)
- Submit NDAs to both agencies using country-specific formats
- Wait for FDA approval before engaging EMA
Correct answer: Use ICH CTD format for both agencies and align clinical data packages
The ICH Common Technical Document (CTD) format is accepted by both FDA and EMA, enabling parallel submissions with a harmonized data package.
Question 2: Which FDA submission pathway is most appropriate for a drug with a condition of use in a small, well-defined rare disease population?
- Standard NDA under 505(b)(1)
- Orphan Drug designation with NDA or BLA (Correct answer)
- Section 505(b)(2) NDA referencing existing approved drug
- Abbreviated New Drug Application (ANDA)
Correct answer: Orphan Drug designation with NDA or BLA
Orphan Drug designation is designed for conditions affecting fewer than 200,000 people in the US, providing incentives including market exclusivity.
Question 3: A regulatory submission has been placed on clinical hold by the FDA. What is the sponsor's primary next step?
- File a formal dispute resolution immediately
- Respond in writing to address all FDA concerns within the specified timeframe (Correct answer)
- Withdraw the IND and refile with new data
- Request a Type A meeting to discuss the hold
Correct answer: Respond in writing to address all FDA concerns within the specified timeframe
The sponsor must submit a complete response addressing all clinical hold issues raised by FDA before clinical trials can resume.
Question 4: What is the primary purpose of a Pre-Submission (Q-Sub) meeting with FDA for a medical device?
- To receive binding approval commitments before submission
- To obtain FDA feedback on testing, study design, or submission requirements prior to formal filing (Correct answer)
- To negotiate user fees and review timelines
- To submit draft labeling for pre-approval
Correct answer: To obtain FDA feedback on testing, study design, or submission requirements prior to formal filing
Pre-Submission meetings allow device sponsors to get FDA feedback on regulatory strategy, study design, and data requirements before filing.
Question 5: A company is developing a product that combines a drug with a diagnostic device. Under US regulations, how should the regulatory strategy be approached?
- File separate NDA and 510(k) with each agency independently
- Develop a combination product strategy and determine the lead FDA center via RFD (Correct answer)
- Proceed only under CDER jurisdiction as the drug component dominates
- Submit only a PMA since the device component requires it
Correct answer: Develop a combination product strategy and determine the lead FDA center via RFD
A Request for Designation (RFD) is submitted to FDA's Office of Combination Products to determine the lead center and regulatory pathway for combination products.
Question 6: Which section of the eCTD module contains the Summary of Clinical Safety for an NDA?
- Module 2.4
- Module 2.7.4 (Correct answer)
- Module 5.3.5
- Module 3.2.P
Correct answer: Module 2.7.4
Module 2.7.4 of the ICH CTD contains the Summary of Clinical Safety, providing an integrated safety analysis across trials.
Question 7: A sponsor receives a Refuse to File (RTF) letter from FDA after NDA submission. What does this indicate?
- FDA has identified safety concerns requiring additional studies
- The NDA is incomplete or does not meet threshold requirements for substantive review (Correct answer)
- The NDA has been approved with labeling conditions
- FDA requires additional user fees before review begins
Correct answer: The NDA is incomplete or does not meet threshold requirements for substantive review
An RTF letter means FDA determined the application does not meet minimum completeness standards to allow a substantive review to begin.
CRAP Regulatory Strategy & Submission Management 2
A company wants to market a drug in the EU and US simultaneously.
Which regulatory strategy best supports parallel submissions?