Rare Disease Registries: Practical Ways to Build Trust with Patient Advocacy Groups

Patient advocacy groups (PAGs) are key allies in the development of rare disease registries to fully describe these illnesses and develop new treatments. With the right planning and understanding, collaboration between drug development companies and PAGs can be mutually beneficial in a number of key areas, including:

• Patient engagement — PAGs are key representatives of patient concerns. Patients are very interested in learning about their disease and helping new therapies to become available. They experience research fatigue because of the high burden of their illness. The PAGs surface these concerns and provide mitigating solutions to keep patients involved in long-term rare disease registries.
• Data accessibility — PAGs focus on data ownership and ensuring data reaches the widest possible audience. They are looking for solutions for making the rare disease registry studies they sponsor, or participate in, more accessible to all stakeholders, including patients, providers and industry partners.
• Research sustainability — PAGs maintain societal concern about their disease despite other competing health concerns. At the same time, diagnosis and treatments for rare diseases change over time so rare disease registries need to evolve to stay current. There is a natural partnership between PAG and pharmaceutical sponsors to ensure that the most relevant information is collected in a rare disease registry.

Register for this webinar for guidance on developing rare disease registries that satisfy the needs of PAGs, patients and pharma. Join featured speakers to learn about:

• Practical ways to build PAG and pharma trust and collaboration
• PAG concerns regarding data sharing and ownership
• Building a relevant and sustainable research tool
• Practical strategies for patient engagement, collaboration, multi-sponsor support, data sharing and ownership, including case study examples

Speakers

William C. Maier, MPH, PHD, Epidemiology, Vice President, Rare Disease, Drug Development Sciences, ICON

Dr. Maier has over 25 years of experience in drug development and commercialisation at pharmaceutical companies in Europe, Canada, the United States and Asia. In his current role, he works with product development companies throughout the world to provide regulatory, strategic and scientific guidance on product development and commercialisation. He has experience in rare disease, respiratory, neurology, psychiatry, autoimmune, oncology, endocrinology, dermatology, cardiovascular, cerebrovascular, urology, opioid dependency, vaccine and anti-infective therapy areas.

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Juliane Mills, MPH, MS, Senior Director of Therapeutic Expertise, Rare Diseases, ICON

Ms. Mills has over 20 years of experience in various scientific roles at CROs, pharmaceutical companies, and academic institutions, giving her a comprehensive understanding of the drug development process from discovery to market approval and post-marketing strategy. She has extensive experience developing patient-focused scientific and operational strategies for various study types, such as disease registries, natural history studies, non-interventional studies, biomarker studies, and retrospective chart reviews. Ms. Mills is a trained epidemiologist with a focus on primary and clinical research in autoimmune diseases, including asthma, Crohn’s disease, and several rare indications. In her current role, she provides project teams with strategic support on the use of data and technology to maximise study efficiency and minimise patient and investigator burden.

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