Clinical Research Focus. 50th Edition

FDA Signals Shift Toward a One-Trial Default for Drug Approval

The FDA has announced a shift from the traditional two trial expectation toward one robust pivotal study supported by confirmatory evidence. While the standard for substantial evidence remains unchanged, this evolution places greater emphasis on trial quality, statistical rigor, and early regulatory alignment in drug development.

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EMA Recommends Withdrawal of Levamisole Across the EU

The EMA has recommended the withdrawal of levamisole-containing medicines after confirming a rare but serious risk of leukoencephalopathy, which can occur even after a single dose and with delayed onset. Given the availability of alternative treatments and the unpredictability of the neurological risk, EMA concluded that the benefits no longer outweigh the risks. The decision underscores the impact of ongoing pharmacovigilance and reinforces the EU’s tightening safety standards for marketed medicines.

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Why SCOPE 2026 Felt Different for Clinical Research

Nicole Brenna, US Business Development Director at Cromos Pharma, shares her perspective on the key shifts emerging from SCOPE 2026. From AI moving into real workflows to digital protocols and stronger site enablement, this year’s summit reflected a clear transition from experimentation to practical execution, with regulatory readiness and operational efficiency now at the center of clinical trial strategy.

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FDA Launches Framework to Accelerate Individualized Therapies

The FDA has introduced a new Plausible Mechanism Framework to support approval of individualized genome editing and RNA-based therapies for ultra-rare diseases when traditional trials are not feasible. By allowing greater reliance on mechanistic evidence and small but robust datasets, the guidance marks a significant shift in how highly personalized treatments may reach patients.

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World Cancer Day 2026: What Cancer Vaccines Have Already Achieved in Real Clinical Practice

On World Cancer Day, progress is measured by real outcomes. Preventive vaccines against HBV and HPV have already reduced cancer incidence at population scale, while therapeutic cancer vaccines are delivering durable benefit in selected clinical settings. Cancer vaccines are no longer only a promise of the future but an established part of modern oncology care.

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NIH Halts Stroke Trial Arm After Safety Review

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FDA Rejects Regenxbio’s Hunter Syndrome Gene Therapy: A Turning Point for the AAV Field?

The FDA’s rejection of Regenxbio’s AAV gene therapy for Hunter syndrome reflects a broader tightening of regulatory expectations. Durability of effect, meaningful clinical outcomes, safety, and manufacturing robustness now carry greater weight than unmet need alone. For gene therapy developers, regulatory discipline is becoming as critical as scientific innovation.

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Protara Reports 66% Complete Response in Bladder Cancer Trial

Protara Therapeutics reported that its investigational cell therapy TARA-002 achieved a 68% complete response rate at six months in patients with non-muscle invasive bladder cancer, demonstrating encouraging efficacy and a favorable safety profile in a difficult-to-treat population.

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Rare Disease Day 2026: A New Economic Reality for Pharma

In 2025, half of all new FDA approvals carried orphan designation, a clear signal that rare diseases are no longer regulatory exceptions but a strategic pillar of pharma innovation. Beyond scientific progress, this shift is reshaping portfolio strategy, regulatory pathways, and commercial infrastructure, as companies increasingly balance high volume chronic markets with high value precision therapies. In 2026, rare disease programs are not niche bets but a deliberate economic strategy.

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