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



Webinar Date/Time: Thu, Mar 9, 2023 11:00 AM EST

In this webinar, Advanced Clinical will explore best practices for incorporating the perspectives of rare disease patients, caregivers, and HCPs–into clinical trials.

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Event Overview:

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?

Who Should Attend
Global professionals at pharmaceutical, biotech and medical device and diagnostic companies who are responsible for and/or have an interest in rare disease patient engagement strategies as part of clinical research study design, including:

  • Clinical Operations
  • Clinical Outsourcing / Procurement
  • Clinical Trial Management
  • Project Management
  • Clinical (Sponsor) Oversight
  • Site Feasibility, Identification and Selection
  • R&D
  • Executive leadership
  • Patient engagement strategists
  • Patient recruitment and retention experts
  • Medical Affairs
  • Safety & Pharmacovigilance


Rebecca Starkie
Senior Global Patient Engagement Director
Advanced Clinical

Rebecca Starkie
, Senior Global Patient Engagement Director, has spent the entirety of her 24-year career in clinical research, which much of this time focused on strategy and running clinical studies. She has experience in managing studies across a variety of rare disease studies and across all phases. Her most recent experience has been specifically around Patient Engagement, supporting both clients and teams alike with strategies for recruiting and engaging patients. She is passionate about bringing patient engagement to the forefront and has an entrepreneurial mindset liking to think outside of the box. Rebecca holds both a BSc(Hons) Biochemistry and a Certificate in Clinical Research from The University of Leeds, England, U.K

Nina Wachsman
Founder & President
Know Rare

Nina Wachsman has experience across many rare therapeutic categories from autoimmune diseases to rare cancers. Her expertise ranges from market research, strategy, to patient and digital marketing. She has experience in developing and leading programs from early-stage clinical trial recruitment and disease positioning through pre-launch and post-launch HCP engagement. Previously Nina founded and led a dedicated patient marketing division for the Interpublic Group.

Dr. Pushpa Narayanaswami
Neuromuscular Neurologist, Associate Professor of Neurology and Vice-Chair of Clinical Operations, Department of Neurology
Harvard Medical School,Beth Israel Deaconess Medical Center, Boston, MA

Pushpa Narayanaswami is a neuromuscular neurologist, Associate Professor of Neurology at Harvard Medical School and Vice-Chair of Clinical Operations, Department of Neurology, Beth Israel Deaconess Medical Center, Boston, MA. Upon graduating from Bangalore Medical College, India in 1984, she completed a neurology residency at the National Institute of Mental Health and Neuro Sciences, Bangalore, India in 1991. After moving to the US in 1995, she completed another neurology residency, and a fellowship in Neuromuscular Diseases at the University of Tennessee, Memphis, TN. A fellow of the American Academy of Neurology (AAN), she served as a member of the Guideline Development sub-committee of the AAN for 13 years.

Maria Joshi
Former Director, Clinical Operations
Arena Pharmaceuticals Inc

Maria Joshi is a clinical operations professional who has worked in the biopharmaceutical industry for more than 14 years. After 10 years of working in Manufacturing and R&D as a chemist, she decided to start a new career in Clinical Operations. Starting as a Clinical Trial Administrator (CTA), Clinical Research Associate (CRA), Trial Manager all the way to a Program Director managing various studies under multiple indications. She has worked both in CRO and biotech companies gaining experience in therapeutic areas such as cancer, hepatic rare diseases, and cardiovascular indications to name a few. She has helped build infrastructure and processes related to SOPs,TMF, vendor oversight and site management.

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