Post-Launch Real-World Evidence and Outcomes Assessment for Rare Disease Therapy
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Following launch, uncovering how the therapy was performing in the real world relative to trial expectations and
competitor products.
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Needed evidence to support ongoing payer positioning and quantify the therapy’s real-world clinical and
economic value.
- Identified downstream drivers of comorbidities, mortality, and cost burden in the target population.
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Analyzed specialty pharmacy data to assess real-world use of the therapy, including titration, adverse events,
and discontinuation patterns.
- Compare post-launch utilization / outcomes against trial data and competitors.
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Conducted additional claims data analysis using machine learning methods to identify drivers of comorbidities,
mortality, and healthcare costs.
- Used findings to support understanding of the potential cost offsets associated with the client’s therapy.
- Generated a clearer view of real-world therapy performance and competitive positioning post-launch.
- Identified key clinical and economic drivers within the patient population.
- Built evidence to support future payer value demonstration and broader post-launch medical affairs strategy.
