For Industry

CMA Pilot 1 Evaluation and Pilot 2 Preliminary Evaluation Studies -- Final Report Text


May 10, 2006




1. Executive summary

2. Background and overview

3. Methodology Analysis Overview

3.1 Hypotheses/Metrics Development

3.2 Data Collection

4. Pilot 1

4.1 Sponsor Motivations for Participation

4.2.1 Program Outcomes

4.2.2 RU Interdependencies

4.2.3 Timing of Reviewable Unit Submissions

4.2.4 Early Review Analysis

4.3 Incremental Pilot 1 Effort for Sponsors and FDA

4.3.1 Sponsor Effort

4.3.2 FDA Effort

4.4 Summary/Recommendations

5. Pilot 2

5.1 Program Implementation

5.2 Potential Effort/Benefits

5.3 Summary/Takeaways


Exhibit 2–1. Comparison of the Fast-Track/Rolling Review and CMA Pilot 1 Programs

Exhibit 2–2. Illustrative Differences between a Fast-Track IND and a CMA Pilot 2 Product

Exhibit 2–3. Status of the Pilot 1, Pilot 2 and Comparison Cohort Products

Exhibit 3–1. Evaluation Approach

Exhibit 3–2. Hypothesis Generation Approach

Exhibit 3–3. Data Sources and Level of Contribution

Exhibit 4–1. Sponsor Motivations for Pilot 1 Participation

Exhibit 4–2. First-Cycle Approval Rate for Pilot 1 Products, the Fast-Track/Rolling Review Comparison Cohort, and Priority NMEs (FY02-FY04)

Exhibit 4–3. Products with Amendments Requiring Goal Extensions

Exhibit 4–4. Number of Issues Identified for Pilot Products Versus the Comparison Cohort As An Indicator of Application Quality

Exhibit 4–5. FDA Met Pilot 1 Six-month Feedback Expectations

Exhibit 4–6. Average Number of Meetings and Communications for Pilot 1 Products Versus the Comparison Cohort

Exhibit 4–7. Reviewable Unit Interdependencies

Exhibit 4–8. Limitations of Submitting Early Reviewable Units

Exhibit 4–9. Timing of Reviewable Units in Relation to the Complete Submission of the Pilot 1 NDA/BLA

Exhibit 4–10. Factors Beyond the Pilot 1 Process Effecting First-cycle Review Outcome

Exhibit 4–11. Multi-cycle Pilot 1 Products Would Have Had The Same Outcome Regardless of Pilot Status

Exhibit 4–12. Some Fast-Track/Rolling Products Received Early Review and Feedback

Exhibit 4–13. Fast-Track/Rolling Products With Limited Evidence of Early Review

Exhibit 4–14. Major Fast-Track Activities Versus Additional Pilot-specific Activities Reported by Participating Sponsors

Exhibit 4–15. Major FDA Fast-Track Review Activities Versus Additional Incremental Pilot 1-specific Activities

Exhibit 4–16. Incremental Effort for FDA of Pilot-Specific Activities per Application (1 cycle)

Exhibit 4–17. Incremental Effort of Pilot-Specific Activities by Function (1 cycle)

Exhibit 4–18. Extrapolation of the Incremental Pilot Effort to the Average Number of Fast-Track Products Submitted to Divisions with Pilot 1 Experience

Exhibit 4–19. Three Year Total Fast-Track Submissions by Divisions (FY2003-2005)

Exhibit 4–20. Scenarios on Potential FDA Re-Work

Exhibit 5–1. Details of the Meeting Schedule Agreement – Fixed vs. Trigger

Exhibit 5–2. Analysis of the Fixed Schedule Method – Communication Increase and FDA Time Effort

Exhibit 5–3. Sponsor Motivations for Program Participation and Perceived Benefits

Exhibit 5–4. Early Observations on the Pilot 2 Program


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