Regulatory divergence can impact healthcare, particularly in the context of economic evaluations. The differences between the FDA and EMA in clinical trial submissions highlight the challenges in developing global value dossiers and the value story of new medical innovations. The FDA and EMA, while aligned in their goals, often have differing requirements for clinical trials. This divergence can lead to variations in sample sizes and endpoint values in regulatory submissions, impacting the economic valuation of innovation healthcare technologies.
Key Analysis from a Recent Study
A recent study published in JAMA examined 15 cell and gene therapy products approved by both the FDA and EMA. The study found that 65% of trials had differing sample sizes, with the EMA often receiving larger samples. Also, 68.4% of trials reported different efficacy outcomes. These discrepancies can significantly influence health economic studies. Larger sample sizes may provide more robust data, affecting the pharmaceutical pricing strategy and outcomes-based pricing models. Variations in trial data may also delay market access, increasing costs and prolonging time-to-market.
Points to Ponder
The JAMA study highlights critical challenges in the regulatory landscape that can impact healthcare economics and patient access to new therapies. By addressing these discrepancies and promoting regulatory harmonization, stakeholders can enhance the efficiency of the drug approval process and deliver greater value to healthcare systems and patients. Ongoing collaboration and innovation will be key to overcoming these challenges.
Further Reading
- Elsallab M, Gillner S, Bourgeois FT. Comparison of Clinical Evidence Submitted to the FDA and EMA for Cell and Gene Therapies. JAMA Internal Medicine (2025) doi: 10.1001/jamainternmed.2024.7569
- In-depth look at the differences between EMA and FDA. Mabion https://www.mabion.eu/science-hub/articles/similar-but-not-the-same-an-in-depth-look-at-the-differences-between-ema-and-fda/ [Accessed February 5, 2025]
- Read “Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities” at NAP.edu. doi: 10.17226/27968
- Study: Sponsors often submit different clinical evidence to FDA, EMA, for cell and gene therapies. https://www.raps.org/News-and-Articles/News-Articles/2025/2/Study-Sponsors-often-submit-different-clinical-evi [Accessed February 5, 2025]
- FDA, Sponsors Advance Reliance on Real-World Data & Evidence. Applied Clinical Trials (2021) https://www.appliedclinicaltrialsonline.com/view/fda-sponsors-advance-reliance-on-real-world-data-evidence [Accessed February 5, 2025]
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