Despite progress, 95% of rare diseases do not have disease-specific therapy (1). The unique complexities of rare disease research—including small, dispersed patient populations and ethical challenges with traditional trial designs—create significant hurdles for drug development. To overcome these barriers, the industry must implement modern study designs and strategically incorporate patient-centric approaches.
Your Guide to Modern Study Design
Our white paper, Optimizing Evidence Generation in Rare Disease Studies with Patient-Centric Strategies, explores how RWD and RWE can be leveraged as strategic tools from the earliest stages of product development.
The white paper details modern study designs and the essential use of RWD/RWE for:
- Optimizing patient identification, recruitment & retention
- Incorporating decentralized study elements
- Demonstrating value support regulatory decision-making
- Planning your RWE strategy
The Value of Early Planning
Success in rare disease research hinges on strategic foresight—a principle emphasized by UBC’s leading experts. The paper features critical insights from Executive Director of Real-World Study Solutions Judy Lytle and Executive Director of Clinical Research Bruce Smith, who discuss how early strategic planning of evidence generation is pivotal for rare disease studies.
Transform Your Evidence Strategy
Download the paper to learn how a patient-centric RWE approach can:
Solve Recruitment Challenges
Leverage RWD (including claims databases) and advocacy group collaboration to identify and engage hard-to-find patient cohorts.
Minimize Patient Burden
Utilize decentralized study elements (e.g., remote monitoring, in-home nursing, telehealth) to dramatically expand the addressable patient population beyond geographic limits.
Accelerate Regulatory Pathways
Understand how RWE is increasingly used to support regulatory decision-making, including supporting safety and efficacy, particularly through the use of external controls in single-arm trials.
Build Foundational Knowledge
Learn how RWD informs natural history studies, helping to define disease progression, treatment guidelines, and appropriate clinical outcome assessments.
Don’t Let Rare Disease Challenges Stall Your Progress
Download the white paper now to gain UBC’s expert strategies on RWD/RWE and ensure early strategic planning is your key to study success.
References
- NORD. “Rare Disease Facts & Statistics.” Accessed 06/14/2023. https://rarediseases.org/understanding-rare-disease/rare-diseasefacts-and-statistics/
