Live Virtual Panel: Partnering with Patients to Transform Rare Disease Research


February 23 - February 23, 2021

Webinar |

Tuesday - February 23, 2021 / 1:00pm ET
Free Event

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.

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

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

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



We are always available for a conversation.


We are always available for a conversation.


Communication Preference

Communication Preference