Data Science with Real World Data in Pharma: Which Therapy Demonstrates the Strongest Real-World Evidence Impact on HTA Recommendations Globally?

Discover which therapy (Yescarta, Evrysdi, Luxturna, Kymriah, Zolgensma, Polivy, Tecartus, or Rozlytrek) leverages real-world evidence most effectively for HTA approval across countries.

Question

Select the therapy with the best impact of real world evidence in most for the countries’ HTA recommendations

A. Yescarta
B. Evrysdi
C. Luxturna
D. Kymriah
E. Zolgensma
F. Polivy
G. Tecartus
H. Rozlytrek

Answer

E. Zolgensma

Explanation

The therapy with the strongest demonstrated impact of real-world evidence (RWE) on Health Technology Assessment (HTA) recommendations across multiple countries is E. Zolgensma (onasemnogene abeparvovec).

Key Analysis

Evrysdi (B) and Zolgensma (E) were the only therapies with explicit positive mentions of RWE influencing HTA decisions in final reports2. For example:

• Zolgensma’s RWE from the SMART study demonstrated maintained motor milestones in older SMA patients, reinforcing its clinical benefit.
• HTA bodies like NICE (UK) and CADTH (Canada) highlighted Zolgensma’s RWE for addressing uncertainties in long-term outcomes.

Yescarta (A), Kymriah (D), and Tecartus (G) required post-approval registries (e.g., France’s CAR-T registry) to collect RWE, but direct HTA endorsement of this evidence was limited. Outcomes were often tied to conditional reimbursement rather than decisive RWE-driven recommendations.

Luxturna (C) faced challenges in HTA appraisals due to insufficient patient-reported outcomes (PROs) and questions about clinical endpoints, despite some RWE use.

Polivy (F) and Rozlytrek (H) lacked significant RWE citations in HTA reports, as per the Economist Impact study.

Why Zolgensma?

• Multi-country acceptance: Zolgensma’s RWE addressed critical gaps in clinical trial data (e.g., long-term efficacy in older/heavier patients), leading to favorable appraisals in France, Germany, and Canada.
• Methodological robustness: Studies like SMART provided validated, disease-specific outcomes (e.g., HFMSE scores), aligning with HTA agencies’ demands for clinically relevant endpoints.
• Regulatory alignment: Zolgensma’s RWE collection was proactively integrated into post-launch monitoring, reducing evidence uncertainties and enabling faster HTA reassessments.

While Evrysdi also received positive RWE mentions, Zolgensma’s broader geographic impact and stronger methodological validation make it the optimal choice.

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