- Global Pharma News & Resources
09-Mar-2023 - 09-Mar-2023

How to Make Your Rare Disease Trials More Patient and Site-Centric

Join our webinar!
How to Make Your Rare Disease Trials More Patient and Site-Centric

Thursday March 9, 2023 11:00am EDT | 4:00pm BST | 5:00pm CEST 


Register now



Rare disease patients and caregivers often face numerous obstacles when participating in clinical trials, and these obstacles are usually the result of a 'one size fits all' approach. In recent years, industry professionals are increasingly deploying innovative approaches to both help identify rare disease patients and enable them to actively engage and participate in clinical research.


To help gain further insight into the perspective of rare disease patients and investigative sites - as well as enhance their experience participating in clinical research - Advanced Clinical partnered with Know Rare, a company dedicated to connecting patients with clinical research. The partnership conducted a global survey to capture the opinions of rare disease patients, caregivers, and health care professionals (HCP) regarding how to best implement the voice of the patient into rare disease trials.


In this panel discussion, Rebecca Starkie, Senior Global Patient Engagement Director at Advanced Clinical will moderate a panel discussion with industry experts around these survey results, offering insights into effective patient engagement strategies and how new approaches can add value for sponsors, site staff, regulators and - ultimately - patients in need of new treatments and care regimes.

Key Learning Objectives:

  • What are the gaps faced by HCP providers in clinical research and recruitment, and how are HCPs made aware of clinical research studies as a potential treatment option for rare disease patients?
  • Do patients and caregivers in the rare disease community have an interest in clinical research and do they wish to participate? Do they feel supported?
  • How can we integrate diversity efforts in rare disease clinical research to ensure the optimal participation pool?
  • What are some reported obstacles in converting a patient and caregiver from a 'practice patient' into a 'clinical trial patient'?
  • What are some real-life experiences of patients and caregivers who have participated in clinical research?


  • Nina Wachsman | Founder & President, Know Rare
  • Dr. Pushpa Narayanaswami | Neuromuscular Neurologist, Associate Professor of Neurology and Vice-Chair of Clinical Operations, Department of Neurology Associate Professor of Neurology at Harvard Medical School Beth Israel Deaconess Medical Center, Boston, MA
  • Maria Joshi | Former Director, Clinical Operations, Arena Pharmaceuticals Inc


  • Rebecca Starkie | Senior Global Patient Engagement Director, Advanced Clinical


Register now