Real-World Evidence Shows Positive Impact on HTA Drug Approvals, Analysis Reveals

• Analysis of 1,840 Health Technology Assessment decisions reveals that submissions including real-world evidence achieved 77% approval rate compared to 67% without RWE.
• Case studies from Australia, Scotland, and France demonstrate how real-world evidence helped secure positive reimbursement decisions for drugs like Yervoy, Zaltrap, and Myozyme.
• Despite proven benefits, real-world evidence was only utilized in 6% of HTA evaluations, suggesting significant untapped potential for both pharmaceutical companies and assessment agencies.

A comprehensive analysis of Health Technology Assessment (HTA) decisions has revealed that incorporating real-world evidence (RWE) significantly increases the likelihood of positive drug reimbursement recommendations, despite its current limited utilization in the evaluation process.

The study examined 1,840 HTA decisions across major agencies including the UK's NICE, Scotland's SMC, Canada's CADTH, Australia's PBAC, France's HAS, and Germany's G-BA. Among these decisions, only 106 (6%) incorporated observational data, but those that did showed notably better outcomes. Submissions including RWE achieved a 77% positive decision rate, compared to 67% for those without RWE (p=.025).

Impact of RWE on Specific Drug Approvals

Several case studies demonstrate how RWE has directly influenced positive reimbursement decisions. In Australia, Bristol-Myers Squibb secured approval for Yervoy (ipilimumab) in melanoma treatment after two failed attempts by presenting new real-world post-registration data that demonstrated increased survival rates and durable treatment effects.

Similarly, Sanofi's Zaltrap (aflibercept) gained Scottish approval for metastatic colorectal cancer treatment after including open-label studies that provided crucial quality-of-life data. In France, Myozyme's effectiveness in treating late-onset Pompe disease was confirmed through registry data and observational studies, leading to a positive recommendation.

Strategic Value of Real-World Evidence

RWE serves multiple crucial functions in the drug evaluation process:

• Complementing randomized controlled trial (RCT) data
• Supporting efficient clinical trial design
• Enhancing understanding of benefit/risk profiles
• Strengthening economic model building
• Informing market access strategies

According to the International Society for Pharmaeconomics Outcomes Research (ISPOR), these real-world data sources are "essential for sound coverage, payment, and reimbursement decisions."

Current Limitations and Future Potential

Despite its demonstrated benefits, RWE's limited 6% inclusion rate in HTA evaluations suggests significant untapped potential. The data indicates that both pharmaceutical companies and HTA agencies could benefit from increased investment in RWE sources:

• For pharmaceutical companies: RWE can help overcome reimbursement hurdles and strengthen value propositions
• For HTA agencies: RWE provides valuable insights into real-world drug effectiveness and patient-reported outcomes

Practical Applications of RWE

Real-world evidence has proven particularly valuable in three key areas:

1. Confirming RCT results in actual clinical settings
2. Validating utility estimates in economic models
3. Demonstrating long-term treatment effects

As healthcare systems increasingly focus on value-based care, the role of RWE in HTA decisions is likely to grow, potentially reshaping how drug effectiveness and value are evaluated in the future.