Rare Disease Trials and Gene Therapy: Understanding the Patient Journey

Life Sciences, Clinical Trials, Patient Recruitment & Retention,
  • Tuesday, July 18, 2023

Gene therapies continue to represent an exciting treatment paradigm for patients. With an increase in regulatory approvals in the past few years, there is an expectation for this trend to continue. For rare disease patients, gene therapy trials provide a very viable path toward developing transformational therapies that could have substantial impact on halting disease progression. With any new and emerging therapy, there are specific obstacles that must be considered, especially as they relate to rare disease trials. Some of these obstacles include, but are not limited to, accessing global patient populations, site training and activation, manufacturing and regulatory considerations and minimal industry standardization of data platforms.

Understanding patient challenges and needs in rare disease trials and incorporating their feedback into protocol design is critical to advancing these therapies as part of a complex patient journey.

Join this webinar to gain insights into the challenges of accessing global patient populations, manufacturing, regulations and patient-centered protocol design in rare disease trials.


Meagan Spychala, PPD, part of Thermo Fisher Scientific

Meagan Spychala, DrPH, RAC, Executive Director, Project Management, PPD, part of Thermo Fisher Scientific

Dr. Meagan Spychala is an Executive Director of Project Management at PPD, part of Thermo Fisher Scientific, with almost 20 years of experience in the clinical research industry. Her depth of experience overseeing the operations of clinical trials has focused on rare disease clinical trials and trials utilizing gene therapies, cellular therapies and gene targeting therapies. Dr. Spychala is the co-chair of PPD’s Gene Therapy Operational Center of Excellence, ensuring that project teams successfully support the patient, site and sponsor in gene therapy research. Dr. Spychala holds a bachelor’s degree in mathematics from Washington and Lee University and an MS and DrPH in biostatistics from the University of North Carolina at Chapel Hill.

Message Presenter
Kim Watanabe, PPD, part of Thermo Fisher Scientific

Kim Watanabe, PhD, Executive Director, Cell and Gene Therapy, PPD, part of Thermo Fisher Scientific

Dr. Kim Watanabe is an Executive Director of Cell and Gene Therapy where she leads business and operational strategy development and deployment in hematology and oncology clinical development services. She has 20 years of experience in R&D, business and operations, of which the last 14 years have been focused on cell and gene therapies. She was a postdoctoral scholar at University of California, San Diego, School of Medicine and received her PhD in microbiology from University of Virginia, School of Medicine. She holds a BSc in cell and structural biology from University of Illinois at Urbana-Champaign.

Message Presenter
Dr. Panteli Theocharous, PPD, part of Thermo Fisher Scientific

Dr. Panteli Theocharous, FIBMS, MS, PhD, FRCPath, Global Vice President, Cell and Gene Therapy, PPD, part of Thermo Fisher Scientific

Dr. Panteli Theocharous is Global Vice President, Cell and Gene Therapy Strategy Lead at PPD Clinical Research Business. As a senior-level industry executive in prior CMO roles and a cancer immunotherapy expert, Dr. Theocharous has led multiple hematology/oncology assets in clinical development including innovative immuno-oncology technologies (1st generation clinical CAR T cell applications in hematologic malignancies, and AAV gene-directed therapies in benign disorders) and successful life cycle management, MAA/NDA and HTA submissions. Dr. Theocharous’ background comprises an MS in applied clinical hematology from the University of London; and cancer medicine related higher degrees from the Royal Free and University College Medical School at the University of London where he was clinical director for stem cell transplantation and cell and gene therapies.

Message Presenter

Who Should Attend?

  • Industry: Biotech and biopharma companies involved in site and patient recruitment for clinical research
  • Healthcare providers and clinicians: Involved in the management of clinical research patients for rare disease and gene therapy trials
  • Clinical research investigators and site directors: Exploring options to reduce patient burden for clinical trial participation as part of an expert multidisciplinary approach
  • Patient Advocacy and Study Consortia groups: Bridging the gap between the community and the clinical research ecosystems to develop patient-centric trials

What You Will Learn

Attendees will broaden their knowledge on:

  • What is gene therapy and why is it important to rare disease patients
  • Addressing challenges to operationalizing a gene therapy trial such as manufacturing, patient recruitment, site selection and activation and regulatory considerations
  • How decentralized solutions and patient concierge can help reduce site and patient/caregiver burden
  • Incorporating feedback from patient advocacy groups and regulatory agency consultancy into trial design
  • The importance of long-term follow-up in rare disease gene therapy studies, and the need to consider optimized digital capabilities

Xtalks Partner


The PPD clinical research business of Thermo Fisher Scientific Inc., the world leader in serving science, enables customers to accelerate innovation and increase drug development productivity. Utilizing patient-centered strategies and data analytics, its capabilities cover multiple therapeutic areas and include early development, all phases of clinical development, peri- and post-approval, novel approaches to patient recruitment and investigator sites, and comprehensive laboratory services. Recognized as a global industry leader in accelerating promising medicines from early development through regulatory approval and market access, PPD clinical research serves serve pharma, biotech, medical device and government organizations with custom-tailored solutions, including full-service partnerships and functional service partnerships. As a strategic partner in clinical development and analytical services, the business applies cutting-edge technologies, therapeutic expertise and a firm commitment to quality to help customers deliver life-changing therapies.

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