What happens when a disease is too rare for a large clinical trial, too complex for a single natural history study, and too urgent for patients to wait years for answers? Most often, development just stops. About half of the clinical trial pipelines in both the U.S. and Europe are focused on developing treatments for rare diseases. Despite such a large number of pipeline opportunities, fewer than 5% of those conditions have been transformed into approved therapies. This is largely due to the fact that most of the affected patient populations are either too small or geographically dispersed for the use of traditional trial designs. Real-world evidence is emerging as the common thread connecting diagnosis, drug development, and access.
To address these challenges, industry experts at Ingenious e-Brain have drafted an insightful whitepaper entitled “The Clinical Trial Alone Isn’t Enough: Why Rare Disease Development Needs Real-World Evidence” as part of our commitment to providing insights to our clients. In addition to explaining what RWE is and why it’s important in drug development for rare diseases, this whitepaper will also provide a template for sponsors to develop their RWE strategy throughout the entire life cycle of rare disease drug development, including early product development planning, late-stage product development, regulatory approval, and long-term post-launch surveillance.
Key Highlights in the Whitepaper
- A modular framework for evidence generation in rare diseases.
- How clinical trials for rare diseases differ from standard clinical trials.
- Real-world case studies from Sanofi, Takeda, Alexion, BioMarin, and more.
- The structural roadblocks limiting RWE adoption in rare diseases.
- How RDEP and CED/MEA pathways are shaping rare disease market access.
- How IeB supports with evidence strategy, study design, patient registry development, and HTA dossier preparation for rare diseases.
For companies navigating rare disease development, the difference between a stalled program and a successful launch often comes down to evidence strategy.
This whitepaper on rare diseases is published specifically to support Market Access & HEOR leaders, RWE/ Data Strategy Directors, Clinical Development Leaders, Regulatory Affairs Directors, Medical Affairs Executives, Publications & Scientific Communications Managers, and Rare Disease Program Executives.
Learn how a modular RWE framework, combined with the proper advisory support, can enable you to build the evidence base your rare disease program requires, from early-stage development through post-launch value demonstration.
Download the full whitepaper and get instant access by filling out the form!
