What payers will ask
Is the clinical benefit the kind payers reward? — Clinical effectiveness
The clinical trial evidence indicates that teduglutide reduces the number of days of parenteral support needed by patients with short bowel syndrome compared to placebo. However, the extent of this reduction is uncertain due to the trial design not fully reflecting Healthcare practice. The evidence is moderate, showing a benefit but with limitations in generalizability.
Does the economic case hold at the expected price? — Cost effectiveness
The cost-effectiveness estimates for teduglutide are below what NICE considers acceptable, even when accounting for uncertainties. The updated analyses reflect the committee’s preferences and show that teduglutide is likely to be cost-effective for both children and adults.
Is there quality-of-life evidence payers weigh? — Quality of life
The evidence regarding HRQoL improvements is mixed, with the company using alternative utility values instead of trial data due to inconsistencies. While a reduction in parenteral support is expected to improve quality of life, the trial data did not show statistically significant differences in HRQoL between teduglutide and standard care.
Does the safety profile hold up for payers? — Safety and Adverse Effects
The safety profile of teduglutide is acceptable, with adverse events being broadly similar to placebo. The most common adverse events were manageable and did not significantly undermine the treatment’s benefits.
Was the drug compared against what payers expect? — Comparator Selection
Teduglutide was compared against the appropriate standard of care, which is parenteral support combined with best supportive care. This selection aligns with clinical practice and reflects the treatment pathway for patients with short bowel syndrome.
Is the population defined the way payers need it? — Patient Population and Subgroups
The trial population is generally representative of the intended patient population, with some subgroup analyses conducted. However, there are limitations in the data for children, which were not included in the economic model.
Does the drug fit how care is delivered and paid for? — Care Pathway Integration
Teduglutide can be integrated into existing care pathways with manageable adjustments. The treatment is positioned appropriately within the treatment pathway for patients who are stable on parenteral support.
Are the wider system costs understood? — Resource Use and Cost Implications
The resource implications of teduglutide are manageable, with the potential for cost savings associated with reduced parenteral support needs. The economic model reflects realistic resource use in Healthcare practice.
Would the evidence survive payer scrutiny? — Evidence Quality and Robustness
The evidence base is primarily derived from a phase 3 trial with good methodological quality. However, there are some concerns regarding the generalizability of the results due to the trial design and the use of alternative data sources.
How exposed is the case to uncertainty? — Uncertainty, Sensitivity, and Broader Impacts
There are notable uncertainties regarding the clinical effectiveness and cost-effectiveness estimates, particularly related to the generalizability of trial results. While some uncertainties have been addressed, residual concerns remain.
