CROSS-AGENCY APPRAISAL OF REAL-WORLD EVIDENCE IN ONCOLOGY AND RARE DISEASE DRUG-INDICATION HTA APPRAISALS ACROSS SELECT APAC MARKETS AND ENGLAND: A COMPARATIVE HTA APPRAISAL REVIEW (2021-2026)
Author(s)
Faith T. Wong, MSc1, Smarth Lakhanpal, PhD2.
1Analyst, Avalere Health, Singapore, Singapore, 2Avalere Health, Singapore, Singapore.
1Analyst, Avalere Health, Singapore, Singapore, 2Avalere Health, Singapore, Singapore.
OBJECTIVES: RWE supports oncology and rare disease HTA submissions by validating long-term outcomes, informing extrapolation, and estimating treatment effects when head-to-head RCTs are unavailable. While RWE frameworks have advanced in Europe and North America, APAC agencies' appraisal remains less understood. This study aims to: (1) characterize RWE sources and uses across drug-indication sets (drugs appraised by ≥2 agencies for the same indication); (2) compare payer appraisals; and (3) assess RWE's role in reimbursement decision-making.
METHODS: A structured comparative review used publicly available oncology and rare disease HTA appraisals (2021-2026) from NICE (England), PBAC/MSAC (Australia), ACE (Singapore), and CDE (Taiwan). Eligible appraisals incorporated RWE for: (1) long-term outcome validation; (2) external control arms; or (3) extrapolation to under-represented populations. An extraction template captured drug name and indication, RWE source and use, payer feedback, recommendation, and RWE's role in decision-making. Thematic coding identified cross-agency similarities and differences.
RESULTS: Eleven drug-indication sets (26 submissions) were identified across 4 HTA agencies. Oncology and rare diseases comprised 24 and 2 submissions, respectively. Recommendations were positive (n=19), negative (n=6), or conditional (n=1). RWE influenced decisions in 10 submissions and was primarily used for external control arms (n=21), long-term outcome validation (n=9), and extrapolation to under-represented populations (n=7). Agencies shared concerns regarding selection bias, confounding, heterogeneity, shrinking effect sizes from MAIC, and comparator selection. NICE applied the most technical scrutiny; PBAC/MSAC emphasised evidence maturity; ACE local applicability; and CDE interpretation of indirect comparisons and reference to international precedent. NICE, PBAC/MSAC, CDE were more willing to accept residual RWE uncertainty through managed access agreements, ACE often concluded treatments with uncertain RWE and unfavourable cost-effectiveness precluded subsidy.
CONCLUSIONS: Despite methodological similarities, RWE appraisals vary by agency tolerance for immature data and emphasis on local applicability. Future research should explore RWE appraisal across other disease areas to assess generalisability beyond oncology and rare diseases.
METHODS: A structured comparative review used publicly available oncology and rare disease HTA appraisals (2021-2026) from NICE (England), PBAC/MSAC (Australia), ACE (Singapore), and CDE (Taiwan). Eligible appraisals incorporated RWE for: (1) long-term outcome validation; (2) external control arms; or (3) extrapolation to under-represented populations. An extraction template captured drug name and indication, RWE source and use, payer feedback, recommendation, and RWE's role in decision-making. Thematic coding identified cross-agency similarities and differences.
RESULTS: Eleven drug-indication sets (26 submissions) were identified across 4 HTA agencies. Oncology and rare diseases comprised 24 and 2 submissions, respectively. Recommendations were positive (n=19), negative (n=6), or conditional (n=1). RWE influenced decisions in 10 submissions and was primarily used for external control arms (n=21), long-term outcome validation (n=9), and extrapolation to under-represented populations (n=7). Agencies shared concerns regarding selection bias, confounding, heterogeneity, shrinking effect sizes from MAIC, and comparator selection. NICE applied the most technical scrutiny; PBAC/MSAC emphasised evidence maturity; ACE local applicability; and CDE interpretation of indirect comparisons and reference to international precedent. NICE, PBAC/MSAC, CDE were more willing to accept residual RWE uncertainty through managed access agreements, ACE often concluded treatments with uncertain RWE and unfavourable cost-effectiveness precluded subsidy.
CONCLUSIONS: Despite methodological similarities, RWE appraisals vary by agency tolerance for immature data and emphasis on local applicability. Future research should explore RWE appraisal across other disease areas to assess generalisability beyond oncology and rare diseases.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
RWD22
Topic
Real World Data & Information Systems
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases, STA: Multiple/Other Specialized Treatments