Introduction
Health Economics and Outcomes Research (HEOR) plays a pivotal role in demonstrating the value of new therapies. HEOR not only supports market access and reimbursement decisions but also ensures that patients receive effective and affordable treatments.
Often in Europe a rare disease is defined as a condition that affects less than 1 person in 2,000. With over 7,000 rare diseases affecting millions globally, the need for robust economic evaluations and outcomes research is more critical than ever.
The Importance of HEOR in Rare Diseases
HEOR is essential in rare diseases for a number of reasons:
Challenges of HEOR for Rare Diseases
Recruitment Barriers:
It can be difficult to access individuals with rare diseases to take part in research as healthcare management is often not standardised given the heterogeneous nature of these disorders.
Small sample size:
This can make data generation efforts challenging and once captured can present statistical challenges to power the analyses especially across ultra-rare disease.
Limited secondary data:
For a number of rare diseases ICD-10 codes do not exist which means hospital/claims data may not be able to be utilized to its full potential.
Overcoming Challenges
To address these challenges, companies can employ strategies such as:
Conclusion
HEOR is a vital component providing the necessary evidence to support the development, approval, and reimbursement of new therapies in rare diseases. By adapting and overcoming the unique challenges of rare diseases, HEOR can provide methodologies to demonstrate the impact, preferences and effectiveness of novel treatments, ultimately improving patient access to treatment options that can offer these often-unmet communities with improvements in their quality of life (QoL). As technology and methodologies continue to develop opportunities to advance patient care and support evidence-based decision-making will continue to emerge.
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