Premier Research

Achieving Uncommon Success in Rare and Ultra-Rare Disease Studies [Webinar]

In Clinical Development by Freya Smale

Premier ResearchThis blogpost is provided by Premier Research.

You can achieve uncommon success in rare and ultra-rare disease studies. Discover how by attending a live webinar from Premier Research and Alexion Pharmaceuticals on Wednesday, March 11, 11am EDT (NA) / 3pm GMT (UK) / 4pm CET (EU-Central).

Don’t miss this exceptional opportunity to hear a study sponsor and a leading contract research organization examine practical considerations for every stage of the development process. Topics they’ll address will include:

Program design — understanding natural history, reducing sources of bias, and validating endpoints.

Protocol design — reducing the need for protocol amendments.

Patient recruitment and retention — determining the number of study sites, managing patient travel, incorporating international patients, and knowing what questions to expect.

Preparing for site inspections by the FDA.

You’ll hear from these experts:

Angi Robinson, Executive Director and Scientific Account Leader for Premier Research. Angi’s extensive experience includes oversight of global studies in pediatrics and rare diseases.

Patricia Nowowieski, Head of Global Clinical Operations for Alexion Pharmaceuticals. Patricia’s work in global clinical trials involves specialized populations and complex logistical and regulatory challenges.

Mallory Bissett, Associate Director of Global Clinical Operations for Alexion Pharmaceuticals. Mallory focuses solely on rare diseases, including late-stage, ultra-rare disease products.

Register today.

Premier Research has conducted more than 100 rare disease studies over a recent five-year period, and finding rare disease clinical studies solutions is something we work toward every day.  Around the world, more than a thousand Premier Research professionals help innovative biopharmaceutical and medical device companies advance life-changing developments from concept through clinical development to market. It’s what we do. Best.

We’re glad to have Premier Research as a Platinum Sponsor of the World Orphan Drug Congress USA, taking place April 22-24 in Washington, DC.