The role of combination therapies in oncology is growing, becoming standard of care for many cancers. Whilst scientific rationale for this is clear, it is unclear whether Health Technology Assessment processes, designed for the appraisals of single products, can adequately reward the value of combination therapies. This study investigates Single Technology Appraisals (STAs) conducted by the National Institute for Health and Care Excellence (NICE), comparing outcomes for monotherapies and combinations.
STAs of oncology therapies published by NICE from January 2019 to December 2021 were identified, excluding reassessments. Data was segmented into monotherapies, double-branded combinations, and combinations with one branded therapy and a generic backbone, “generic combinations”. The following parameters were compared: success rate, as defined by number of ‘recommended’ or ‘optimised’ appraisals; terminated appraisals; Cancer Drugs Fund (CDF) utilisation; financial agreements and time from final scope to publication of final appraisal document (FAD).
108 STAs were included for analysis: 60 monotherapies, 31 double-branded combinations and 17generic combinations. Success rate was similar for monotherapies and generic combinations (78.3%and 81.3%), but lower for double-branded combinations (71.0%). Similarly, 13.3% of monotherapies and 12.3% of generic combinations had terminated appraisals, but 22.6% of appraisals for double-branded combinations were terminated. Double-branded combinations were more likely to utilise the CDF than monotherapies (45.5% versus 34.0%). Use of financial agreements and time to FAD publication were similar between double-branded combinations and monotherapies.
Double-branded combination therapies have a lower success rate, and manufacturers are almost twice as likely to not submit double-branded combinations to NICE (based off terminated appraisal rate). This leads to proportionally fewer double-branded combinations getting access in the UK. Outcomes for generic combinations more closely resemble that of monotherapies than double-branded combinations. This initial analysis suggests current NICE methods may not adequately reward the value of double-branded combination therapies.
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