What payers will ask
Is the clinical benefit the kind payers reward? — Clinical effectiveness
The evidence from the CASTOR trial indicates that daratumumab with bortezomib and dexamethasone significantly reduces the risk of disease progression or death by 79% compared to bortezomib with dexamethasone. This substantial clinical benefit is supported by a median follow-up of 50.2 months for progression-free survival and 72.6 months for overall survival, demonstrating both short- and long-term efficacy.
Does the economic case hold at the expected price? — Cost effectiveness
The cost-effectiveness estimates for daratumumab with bortezomib and dexamethasone are reported to be below what NICE considers acceptable for Healthcare resources. The committee acknowledged that the treatment is likely to provide a defensible ICER, although specific figures are confidential.
Is there quality-of-life evidence payers weigh? — Quality of life
Patient experts reported that daratumumab with bortezomib and dexamethasone had very few side effects and significantly improved their quality of life, allowing them to maintain daily routines. This is further supported by a patient survey indicating that 95% of respondents would recommend the treatment, suggesting moderate but meaningful improvements in HRQoL.
Does the safety profile hold up for payers? — Safety and Adverse Effects
The treatment is reported to have a very good safety profile, with patient experts indicating limited side effects. The committee noted that while some adverse events occur, they are mostly mild or moderate, supporting a favorable tolerability profile compared to existing therapies.
Was the drug compared against what payers expect? — Comparator Selection
The evidence primarily compares daratumumab with bortezomib and dexamethasone against bortezomib with dexamethasone, which is appropriate. However, there is no direct evidence comparing it with lenalidomide treatments, which limits the robustness of the comparator selection.
Is the population defined the way payers need it? — Patient Population and Subgroups
The trial population is representative of adults with relapsed or refractory multiple myeloma, and the committee noted that the evidence is applicable to the intended patient population. However, some subgroup analyses could be more comprehensive.
Does the drug fit how care is delivered and paid for? — Care Pathway Integration
The treatment can be integrated into existing care pathways with minor adjustments, as it aligns with current clinical practices for second-line treatment of multiple myeloma. The committee noted that no new infrastructure or extensive training is required.
Are the wider system costs understood? — Resource Use and Cost Implications
The budget impact of implementing daratumumab with bortezomib and dexamethasone is manageable, and the treatment is expected to be resource-efficient, aligning with Healthcare planning. The committee recognized the potential for net savings.
Would the evidence survive payer scrutiny? — Evidence Quality and Robustness
The evidence is derived from a robust Phase 3 trial (CASTOR) with a low risk of bias. Although there are some limitations due to the evolving treatment landscape and the impact of COVID-19, the overall quality of evidence remains strong.
How exposed is the case to uncertainty? — Uncertainty, Sensitivity, and Broader Impacts
There are notable uncertainties regarding the impact of COVID-19 on treatment outcomes and the evolving treatment pathway for multiple myeloma. The committee acknowledged these uncertainties but deemed them manageable within the context of the appraisal.
