Quality of Clinical Evidence Submitted to a National HTA Agency in Ireland: 10 Years of Drugs for Oncology

OBJECTIVES: To review the quality of clinical evidence submitted by applicant companies to a national Health Technology Assessment (HTA) agency in Ireland. To investigate potential associations between the quality of this evidence and the reimbursement recommendations made by the agency.

METHODS: This was a retrospective analysis of the clinical evidence that supported HTAs submitted to the National Centre for Pharmacoeconomics (NCPE) from May 2012 to March 2023 inclusive. NCPE recommendations were classed as ‘Conditional-positive/Positive’ or ‘Conditional-negative/Negative’ for the purposes of this analysis. Only HTAs for oncology drugs with a positive incremental net monetary benefit were included. Data extraction, of key clinical-evidence characteristics, was informed by EUnetHTA guidance. Potential associations between the quality of clinical evidence and reimbursement recommendations were investigated via a hierarchy of chi-squared tests and multivariate regression analysis.

RESULTS: During the study period, 91 HTA submissions were made to the NCPE; 78 were supported by randomised controlled trials (RCTs). Direct evidence with an active comparator was available in 53. Indirect treatment comparisons were included in 73. HTAs supported by RCTs were numerically more likely to be associated with a ‘Conditional-positive/Positive’ recommendation (43.6%), compared to HTAs supported by single-arm trials (23.6%). HTAs supported by RCTs that compared the intervention with an active treatment or placebo resulted in similar proportions of ‘Conditional-positive/Positive’ recommendations. Similarly, there was no statistically significant difference in the proportion of ‘Conditional-positive/Positive’ recommendations for HTAs supported by blinded or open-label trials. Lacking direct evidence with a relevant comparator was associated with a higher relative proportion of ‘Conditional-negative/Negative’ recommendations.

CONCLUSIONS: This study suggests that HTAs supported by RCTs are more likely to receive a ‘Conditional-positive/Positive’ recommendation. A key limitation of this study is that the quality of evidence submitted is only one domain of the HTA evaluation process. No tests were statistically significant; partly explained by small sample sizes.