Charlene Son Rigby
CEO, Global Genes
The patient voice cannot be overlooked, and patients can’t be engaged on a transactional basis. To make real headway in rare disease treatment, we must work together — patients and caregivers, advocacy groups, government, researchers and industry.
For rare disease treatment to progress, patients should be brought into the drug development process as early as possible. Patient-centricity is about patients and advocates being at the table as meaningful partners and being official stakeholders for the success of drug research.
Patient role in rare disease treatment pathway
Global Genes has divided its services into three pillars: support; education; research and development, which focuses on research and data enablement, with platform services to support these critical efforts.
Within the research and development pillar, the RARE-X data collection programme enables rare patient communities to collect robust, secure, patient-reported data that is patient-owned and makes that data widely available for research. Patient-centricity is a key component of this programme, which now includes over 80 patient communities and greater than 4,000 participants collecting and sharing data through the platform.
There has never been more opportunity for patient advocates to get started with new therapies and treatments in the research readiness realm.
Collaborative roadmap for rare diseases
Patients interested in being part of the research and innovation to help find treatments and therapies for their conditions may also see themselves traversing the RARE Disease Research Roadmap.
The roadmap is a comprehensive resource to support advocates to drive research and to work effectively with researchers and industry partners. It was generated from work at last year’s RARE Drug Development Symposium.
Patient participation in research
There has never been more opportunity for patient advocates to get started with new therapies and treatments in the research readiness realm. To support this need, Global Genes introduced a research readiness framework earlier this year that will be one of the tracks at the RARE Advocacy Summit in September.
In short, engagement with the patient communities has evolved, and the opportunity for patients to engage with industry and academic researchers in critical collaborations to create shared benefits has never been greater. Patients are now at the centre of the vital ecosystem that includes shared responsibilities with all stakeholders, including advocates, researchers and industry.
