Due to the nature of rare diseases, real-world data are often required to characterise and understand the experiences of these patient groups and their families. Rare diseases are often complex and present a number of challenges in terms of diagnosis, management and treatment.
Given the limited therapeutic options, it’s critical to understand the clinical, economic and humanistic burden of rare diseases, including (but not limited to) misdiagnosis, referral delays, burden of illness, health-related quality of life (HR-QoL), impact on work and productivity, treatment access and availability, and caregiver/family burden.
Adelphi Real World’s Disease Specific Programme (DSP) databases are designed to address these needs.
Our DSP datasets generate comprehensive real-world data reflecting the complexities of rare diseases
We recognise and understand the challenges faced when working in rare diseases. Conducting research where true prevalence is unknown, and patients are hard to reach can be difficult.
Through engagement with healthcare professionals, patients and caregivers/family members, we provide real-word data on a range of themes:
Disease Identification and Diagnosis
Understand disease presentation, misdiagnoses, referral patterns, testing, family history, and delayed diagnosis.
Disease Progression and Patient Burden
Understand how the disease has progressed since first presentation and diagnosis, including evolution of symptoms and other manifestations, clinician reported outcomes and impact on patient and caregiver health-related quality of life.
Treatment and Disease Management Strategies
Explore treatment patterns, therapy duration and drivers for treatment choice, switch and discontinuation. In addition, understand the role of other healthcare providers in patient care.
Unmet Needs
Identify critical knowledge gaps, therapeutic barriers, disease management and treatment challenges, and the specific outcomes that matter most to specialists, patients and caregivers.
Rare Disease Specific Programmes (DSPs) Portfolio
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Why Choose Adelphi For Research In Rare Diseases?
Rare Diseases Centre of Excellence
Dedicated expertise in rare disease methodologies, reaching small patient populations and performing appropriate and fit-for-purpose statistical analyses.
Specialist Networks
Direct access to specialists, patients and caregivers treating or living with rare diseases, spanning cardiology, haematology, hepatology, immunology, nephrology, neurology, metabolic, and oncology.
Patient and Caregiver Informed Evidence
Key to ensuring that rare diseases are fully understood is ensuring that patient and caregiver voices are heard. Adelphi actively engage with patients, caregivers and family members to ensure the data collected are relevant and meaningful, which is especially important given the paucity of disease-specific patient reported outcome tools and clinical endpoints.
Cross-Country Comparisons
Cross-country analysis enables understanding of how disease management, treatment approaches and patient experience compares across regions.
Geographies Covered By DSPs In Rare Diseases
Our Partnerships
We work with biopharmaceutical companies, rare disease centres, patient advocacy organisations, research institutions, and healthcare authorities to generate evidence to support the understanding of rare diseases and help advance the development of therapeutic options.
Explore Our Rare Disease Expertise
- Schedule a strategic discussion: Connect with our rare disease specialists to explore how our datasets and methodologies can support with your real-world evidence needs.
- Request customised resources: Interested in our rare disease research capabilities, international scope, specialist networks, or specific clinical applications? Contact our team for tailored information.
Strengthen your rare disease research with Adelphi Real World’s integrated DSP solutions. Let’s collaborate to deepen clinical understanding, drive therapeutic innovation, and ultimately improve outcomes for rare disease patients worldwide.
Contact us today using the form below.
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