Rare Disease Protocols

Who should use this

• Heads of Regulatory Affairs building submission-ready trial strategies
• Medical Writing Managers drafting protocols for complex designs
• CMOs and senior clinical leaders accountable for study feasibility and rigour
• Clinical development & biostatistics leads shaping design and statistical power

What you will be able to do

➤ Recognise when a traditional trial won’t work for a rare population
➤ Compare adaptive and master protocol designs at a glance
➤ Choose the design that fits your patients, power and budget
➤ Anticipate the statistical and operational gaps to close early
➤ Align your design with FDA, EMA and CTFG expectations
➤ Improve the odds of regulatory acceptance

Overview

In rare diseases, patient populations are small and scattered. That’s why traditional trials turn slow, costly and hard to power. Most orphan drug studies in non-oncology indications fail on recruitment and statistical power, not lack of efficacy. This resource maps the adaptive and master protocol designs that make these trials feasible, and shows how regulators view each one.

What is covered

1. Why rare disease trials fail: patients, recruitment, cost
2. Adaptive protocols: adaptive randomisation and dose groups
3. Master protocols: basket, umbrella and platform designs
4. Key issues to weigh for your own study
5. Regulatory guidance: FDA, EMA and CTFG at a glance

Peter Morgan

Senior medical writer with a PhD in neural stem cell research and two post-doctoral fellowships in cellular neurophysiology. He works across autoimmune disease, endocrinology, oncology and inflammation, and enjoys applying a deep scientific foundation to new therapeutic areas.

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