OBJECTIVES:

Understand how different health technology assessment (HTA) bodies perceive and evaluate real world evidence (RWE) in oncology HTA submissions, and identify drivers of RWE acceptance today and in the future.

METHODS:

All 881 HTA reports in oncology from Germany (IQWiG and G-BA, n=321), England (NICE, n=126), France (HAS, n=251), Sweden (TLV, n=71), and Canada (pCODR, n=112) published between 1 January 2018 and 30 September 2021 were screened to identify submissions where RWE was used to support clinical effectiveness and led to a positive HTA outcome. Of 51 identified reports, 20 were selected based on a qualitative assessment of RWE positively impacting the HTA outcome. An in-depth analysis identified drivers of RWE acceptance and impact on decision-making. Interviews with ten national ex-payers were conducted to validate findings and explore future dynamics.

RESULTS:

Among 20 case studies with impactful RWE, a greater number were identified in England (n=10), Canada (n=5) and Sweden (n=4), compared to France (n=1) and Germany (n=0). Most case studies were defined by the relevant HTA body to have high unmet need (n=18), and 7/20 concerned orphan drugs. RWE most frequently impacted HTA outcomes when used to provide external comparator data (n=15), followed by RWE to provide additional data on intervention effectiveness (n=5). A diverse range of Real World Data (RWD) sources was accepted across all case studies.

CONCLUSIONS:

Examples where RWE has a tangible impact on HTA outcomes remain rare. HTA bodies consider in their evaluation the relevance and quality of the RWD, the evidence gap that is filled by RWE, and the nature of the oncology indication. Looking forwards, collaboration and transparency among the HTA bodies in the use of RWE in HTA assessments could increase its impact and help accelerate patient access to innovative medicines.