By Cinzia Doringo
While the traditional function of Rare Disease Patient Organizations is to support patients and their families, in today’s world, these organizations are playing an increasingly active role in rare disease research. Patient centricity in clinical research is more than just incorporating the patient’s voice into a trial; it involves the careful consideration of patients’ and caregivers’ thoughts, values, preferences, strengths and shortcomings.
While patient centricity has become an industry catch phrase, implementation can vary broadly, especially in rare or orphan disease clinical development. The patients’ ability to digest myriad available data and information to make informed decisions about their care is becoming more and more sophisticated.
In The Past Five Years, We Have Conducted:
- >350 projects in rare disease indications including:
- >over 30 real world evidence projects including registries, non-interventional studies and expanded access programs
- ~25% of our rare disease research experience is in the pediatric population, including first in patient studies in ultra-rare indications
- ~20 projects with ATMPs (Advanced Therapy Medicinal Products)
Acknowledging this trend, the European Patients Academy a Therapeutic Innovation (EUPATI) project, supported by the Innovative Medicines Initiative (IMI), will support patient education and training to allow them to act as effective partners in clinical development. The evolution of patients’ awareness, education and involvement in clinical development requires a simultaneous evolution of medicine developers and of all other stakeholders to properly incorporate the patient perspective.
Many pharmaceutical companies have started to organize regular meetings with patient advocacy groups to bring these patient considerations inside their own organization to facilitate incorporation into the development plans. It is important to include regional groups, as patient views may vary from region to region.
Syneos Health is a leading clinical research organization (CRO) focusing on rare or orphan disease clinical development through the Syneos Health Rare Disease Consortium. Our vision is to bring patients’ perspectives to all stakeholders involved in the planning and delivery of rare disease clinical trials and to develop collaborative strategies focused on patient values.
About the Author
Cinzia has more than 18 years of experience in clinical research, 14 of those in international project management. She is leading the Syneos Health Rare Disease Consortium focused on an effective strategic implementation for successful delivery of rare and ultra-rare disease studies. Cinzia has broad experience in rare and ultra-rare disease studies in Metabolic, Endocrine, and Cardiovascular indications. In the context of these studies, she oversaw many pediatric trials including pediatric intensive care studies. Her experience includes pre-clinical, healthy volunteer, and Phase 1 to Phase 4 studies. She has proven experience in successfully delivering these studies through the development of tailored strategies in single geographic regions and in global settings. Cinzia is a neuropharmacologist by background. Her therapeutic areas experience includes Infectious Diseases, Cardiovascular, Metabolic, Endocrinology, Gastrointestinal, Oncology, Respiratory, and CNS.
