A Europe-based pharmaceutical company approached PHARMA-STATS with an urgent regulatory challenge. They had completed their clinical trials and were in the process of seeking approvals from the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA). However, regulatory authorities had raised a query regarding the sample size justification in their submission, a critical factor that could influence the approval process.

Uncovering Gaps and Strengthening the Submission

Upon reviewing the regulatory query, our biostatistics team conducted a thorough audit of the Contract Research Organization (CRO) responsible for the clinical trial execution. We meticulously examined their methodologies, data integrity, and statistical reasoning. Beyond the audit, we searched references from historical dossiers—leveraging previously submitted and approved regulatory documents—to strengthen our client’s response.

Expertise in Action: A Strategic Approach

Using a combination of statistical rigor and regulatory insights, we:

1. Refined the sample size justification with scientifically sound arguments. 
2. Aligned the response with precedents from prior regulatory approvals, ensuring compliance with EMA and MHRA expectations. 
3. Assisted the client in crafting a well-substantiated reply, reducing ambiguity and reinforcing confidence in their submission.

The Outcome: Regulatory Success

With our strategic guidance and statistical expertise, the client successfully addressed the regulators’ concerns, ultimately securing EMA and MHRA approvals for their clinical trial. (From 2019 Diary)

This case underscores PHARMA-STATS’ deep expertise in biostatistics and regulatory strategy—bridging the gap between statistical science and regulatory compliance to help pharmaceutical innovators bring life-changing therapies to market.

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