Discover which recent FDA drug approvals, including Prograf for lung transplants, utilized real-world evidence (RWE) to demonstrate efficacy and safety.
Table of Contents
Question
Select recent FDA approvals based on real world evidence
A. Prograf
B. Zolgensma
C. Crenezumab
Answer
A. Prograf
Explanation
Explanation of FDA Approvals Based on Real-World Evidence (RWE)
Prograf (tacrolimus)
Approval Context: In July 2021, the FDA approved Prograf for preventing organ rejection in lung transplant patients using real-world data (RWD) from the U.S. Scientific Registry of Transplant Recipients (SRTR). This marked the first FDA approval relying primarily on observational RWE to demonstrate efficacy.
Key Evidence:
- A non-interventional study compared outcomes of lung transplant patients receiving Prograf with historical controls lacking immunosuppressive therapy, showing dramatic survival improvements.
- RWE was supplemented by prior randomized controlled trial (RCT) data from liver, kidney, and heart transplant approvals.
Significance: This approval validated RWE’s role in regulatory decisions when RCTs are impractical (e.g., rare populations like lung transplant recipients).
Zolgensma (onasemnogene abeparvovec)
Approval Context: Zolgensma, a gene therapy for spinal muscular atrophy (SMA), was approved in 2019 based on clinical trials (e.g., START trial).
Evidence Basis: Trials showed improved survival and motor milestones compared to natural disease progression, but no RWE was used for initial approval. Post-approval studies (e.g., SMART trial) later reinforced efficacy in older/heavier children but were not part of the original regulatory submission.
Crenezumab
Outcome: Crenezumab, tested for Alzheimer’s disease (AD), failed to show efficacy in phase 3 trials (CREAD/CREAD2). These trials used RCTs, not RWE, and were discontinued early due to futility.
Why Prograf Is the Correct Answer
Only Prograf’s lung transplant indication explicitly relied on RWE for FDA approval. Zolgensma and Crenezumab were evaluated through traditional RCTs, with the latter failing to gain approval. This distinction highlights the growing role of RWE in addressing unmet needs where conventional trials are challenging.
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