Putting Rare Disease Patients at the Center of Our Work
Rare disease patients are the reason ODDIFACT exists. Our mission is to help more treatments emerge by activating underused science and prioritizing opportunities that reflect real unmet need.
Understanding the Realities of Rare Disease
Thousands of rare conditions remain undiagnosed, untreated, or poorly understood.
Patients often face:
These challenges guide how we prioritize the therapeutic opportunities we choose to advance.
Years of diagnostic uncertainty
limited or no approved treatment options
Reliance on off-label therapies
Fragmented care and specialist access
Significant financial and emotional burden
Learning From Patient-Relevant Data and Research
We ground program selection in patient-relevant evidence and clinical reality through:
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input from disease-specific clinicians and specialists
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scientific and clinical advisor review
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published unmet-need analyses, natural history insights, and patient-burden literature
This ensures each program we advance is grounded in meaningful scientific and patient-relevant insight.
Our Commitment to Future Patient Engagement
We aim to develop constructive relationships with patient organizations, caregivers, and advocacy groups to strengthen how we understand:
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quality-of-life impact
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access barriers
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treatment priorities and expectations
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real-world disease challenges
Our goal is to integrate these perspectives more directly into how we prioritize and shape programs over time.
Our mission is to enable the emergence of new treatments for rare diseases by activating underused academic research and transforming it into structured therapeutic opportunities.
As a société à mission, we pursue both financial performance and societal benefit, anchored in a long-term commitment to patients and the healthcare ecosystem.
Our Promise
We will keep building with rigor, urgency, and patient relevance—so more rare disease treatments can move from evidence to real development pathways.