Sponsored By:
Parexel

Speakers:
Sarah Glass, PhD, Senior Director, Translational Medicine and Head of Rare Diseases, Parexel, and rare parent Lucas Kempf, Vice President, Technical, Parexel, Former interim associate director, FDA Rare Disease Program Nick Sireau, CEO of Alkaptonuria Society Rob Long, Executive Director of Uplifting Athletes and brain cancer survivor Andrea Peet, ALS patient and founder of Team Drea Foundation

Watch On Demand Recording

Rare diseases are, in fact, not so rare. An estimated 400 million people globally are affected by 7,000+ rare diseases, comprising between 4 – 6 % of the total world population.

Despite their heterogeneity, rare diseases share commonalities that necessitate a highly orchestrated approach to developing new treatments and cures – one that integrates the voices of patients, providers, researchers and regulators to fully appreciate and overcome the challenges.

Hear from patients, a rare parent and Parexel rare disease experts on how they’re pushing to achieve this goal.

This event will be moderated by Taren Grom, Co-Founder and Editor, PharmaVOICE.

Discussion topics include:

• Patient-driven protocol designs to reduce the patient burden and expand trial access
  • Incorporating patient perspectives early in trial design
  • Utilizing decentralized clinical trials and hybrid models to reduce patient burden
• Protecting the endpoints during trial delivery to ensure data integrity and market access
• Using real-world data to expedite trial timelines and advance new therapies

On Demand

About the Sponsors

Parexel supports the development of innovative new medicines to improve the health of patients worldwide. We provide services to help life science and biopharmaceutical clients transform scientific discoveries into new treatments. From clinical trials to regulatory and consulting services to commercial and market access, our therapeutic, technical and functional ability is underpinned by a deep conviction in keeping patients at the heart of everything we do.

For more information please visit https://www.parexel.com