ICON’s Center for Rare disease, in collaboration with CISCRP (Center for Information & Study on Clinical Research Participation), join industry experts for a webinar to discuss how the Patient-Centric Trial Development Toolkit for rare diseases can incorporate and elevate patient voice in your company’s drug development programme.
In a live conversational format, panelists will discuss the toolkit which provides a framework for systematically and iteratively incorporating patient perspective from earliest stage of development and throughout the drug development lifecycle for any rare disease clinical program. The toolkit includes:
- Patient-centric protocol risk assessment tool
- Rapid participation burden survey tool
- Patient involvement value dossier
- “What to ask when you are interested in a clinical trial: A guide for rare disease patients & caregivers”
Attendees will learn:
- How this free toolkit can be used throughout the rare disease drug development lifecycle to effectively identify and mitigate barriers for intended participants which often translate to risks of inefficiencies like screen failures, high drop-out rates, protocol amendments and timeline delays
- How Patient Advocacy professionals and champions of patient-centricity elevate patient voice within drug development by implementing these tools
- How the toolkit can be used to empower rare disease patients and caregivers to advocate for their needs in clinical trial participation
Amy Raymond, PhD, PMP
Senior Director of Therapeutic Expertise - Scientific Affairs & Therapeutic Expertise, Center for Rare Diseases, ICON
Amy brings a comprehensive understanding of drug discovery and drug development from the research laboratory to patients’ bedside from 20+ years of experience in academic institutions, biotech companies, and CROs. Originally trained as a molecular biologist, she transitioned into drug development and came up through clinical operations before joining the Center for Rare Diseases in 2017. She has contributed to discovery and development of treatments for many rare disease patient communities, from rare cancers to rare neurodegenerative disorders, including development of a gene therapy treatment now commercially available to rare disease patients.
Amy leads the Gene Therapy Think Tank and is a Subject Matter Expert in the Center for Immuno-Oncology, Cellular, and Gene Therapy. She provides full life cycle support for gene therapy programs outside of oncology, from study design through study close out. This includes close collaboration with sponsor teams, study physicians, clinical operations throughout the program, as well as supporting integration of patient partnership into clinical operations. She is passionate about enhancing the efficiency of clinical trials through cooperative productivity and patient-centered design – key elements of “Market Aware” clinical development protocols, and a proven pathway to commercial success.
Laura Iliescu, MSc
Associate Director, Patient Advocacy Strategy, Center for Rare Diseases, ICON
Laura Iliescu, MSc, has 20 years of experience in development and commercialization of therapies, and patient-centered healthcare services. She has held strategic development roles in rare and complex conditions in leading pharmaceutical, biotechnology, CRO and clinical homecare organizations. Her varied experience comprises strategic development throughout every phase of the product lifecycle from pre-clinical to beyond patent expiry in North America and Europe. She has deep experience in rare metabolic, rare neuromuscular an rare respiratory indications among others. Her background in human factors engineering and user-centered service design methods informs her passion for patient-centered development and for evolving the clinical development paradigm around patients as key stakeholders.
In her current role within ICON’s Center for Rare Diseases, Laura advises product development companies on patient-centered clinical strategy to optimize both study efficiency and the experience of rare disease patients and caregivers. She leads the Center’s Patient Advocacy Strategy team, who support collaboration between companies and rare disease Patient Advocacy Organizations through study design and conduct.
Laura grew up in Toronto, Canada, after which her career (and love of travel) have taken her to more than 40 countries in North America, Europe and Asia. She currently lives in Toronto, Canada with her partner and has both family and friends who are affected by rare conditions. Laura holds a Masters of Human Factors and Ergonomics from the University of Nottingham.
Senior Manager of Patient Advocacy, Harmony Biosciences
Kelli Wright is the Senior Manager of Patient Advocacy for Harmony Biosciences working to keep Patients at the Heart of everything we do at Harmony. It’s critical to include the patient voice in drug development efforts and Kelli is honored work with patient communities, people living with rare neurological diseases, caregivers, and patient advocacy organizations to share their lived experiences across functions at Harmony.
With a 15+ year non-profit career, Kelli has a deep understanding of the patient needs and experiences. Most recently Kelli was the National Director of Strategic Initiatives for CureSearch for Children’s Cancer, driving the strategic program of CureSearch by staying in tune with the barriers in drug development. Kelli learned from innovators in the industry, academic and regulatory space, as well as patients and disease-foundations, to address obstacles and develop action plans to close the communication gaps and reduce duplication of efforts.
Kendall Davis, MPH
Patient Advocacy Lead, Early Development, Spark Therapeutics
Kendall is a driven health advocacy professional with strong community outreach, patient association, health care provider, rare disease and chronic illness expertise. She specializes in patient advocacy throughout rare disease clinical drug development. She currently serves as the Patient Advocacy Lead, Early Development , at Spark Therapeutics. In this role she supports Sparks preclinical and early clinical development programs. Previously, Kendall served as a Patient Advocacy Strategy Lead at PRA Health Sciences and as the Director of Strategic Alliances at Global Genes, a leading rare disease patient advocacy organization. She has held key roles in patient advocacy and patient education in both the nonprofit and biopharmaceutical industries. Kendall’s passion is to improve the health outcomes of individuals living with rare diseases while integrating advocacy strategy into clinical drug development. Kendall specializes in cultivating partnerships with patient advocacy organizations, identifying and partnering with Key Opinion and Community Leaders, identifying business needs, developing customized programs to achieve key outcomes and balancing a global focus with national, regional and local patient centric partnerships. Kendall works with leaders in the rare disease nonprofit community & biotechnology space to advance progress in rare disease drug development. Kendall also serves on the board of directors of Team Telomere and as a strategic advisor to the Rare and Undiagnosed Network. She holds a Master’s Degree of Public Health from Michigan State University as well as a Bachelor’s of Science in Psychology from Loyola University, Chicago. Kendall currently lives in Pittsburgh, PA with her husband, son and dachshund.