On February 28th, Global Rare Disease Day reminds us of the millions of patients worldwide living with rare conditions—many of whom face challenges due to delayed diagnoses and limited treatment options.
Schain Research is committed to changing this by using Real-World Evidence (RWE) to generate insights to improve patient care and accelerate drug development.
How Nordic Data Makes a Difference
One of the biggest hurdles in rare disease research is identifying patients and understanding their long-term health outcomes. Nordic healthcare systems offer a unique advantage—By linking population-based health registries with highly-detailed quality registers or electronic medical records (EMRs), we can help researchers and pharmaceutical companies:
- Identify patients with rare diseases more accurately, even with no specific diagnostic codes.
- Track long-term health outcomes to understand disease progression and treatment effectiveness.
- Generate high-quality data to support regulatory submissions and payer discussions.
Advancing Rare Disease Research with RWE
Our expertise in real-world data allows us to support pharmaceutical partners in several key areas:
Helping Companies Understand Patient Populations
- We analyze patient data across multiple therapeutic areas, including neurology, maternal-fetal medicine, and hematology.
- By analyzing patient numbers, treatment patterns, and survival rates, we provide insights that help companies make informed decisions about drug development and market access.
Mapping Disease Progression with Natural History Studies
- By assessing treatment patterns, clinical outcomes, and health resource utilization, we provide a clearer picture of how diseases evolve and a disease’s individual and societal burden.
- This data helps inform clinical trial design and regulatory discussions, ensuring that new treatments are tailored to patient needs.
Feasibility Assessments for Research
- Before investing in a full study, we evaluate the availability and quality of rare disease data.
- This includes assessing genetic testing data, laboratory test results, and treatment histories to determine the best approach for a study.
- We offer a broad and reliable evidence base by working across Sweden, Denmark, Finland, and Norway.
Beyond Data: Real Impact for Patients and Healthcare
At Schain Research, we believe data-driven insights have real-world implications. Our findings contribute to:
- Better Clinical Trial Design – ensuring studies target relevant patient populations and include relevant comparators.
- Regulatory & Market Access Discussions – supporting the approval and reimbursement of new treatments by establishing unmet medical needs and generating indirect treatment comparison data.
- Collaborations with Pharma & Healthcare Stakeholders – helping turn research into action.
Looking Ahead
On this Global Rare Disease Day, we reaffirm our commitment to using data and research to drive meaningful change for patients with rare diseases. Combining Nordic data excellence with deep analytical expertise, we help pharma companies, regulators, and healthcare providers make informed decisions that can improve lives.
We invite our partners and industry colleagues to join the conversation—because we can make a difference together.
Author
Dr. Tina Jacob
Schain Research, Real-World Evidence Expert
