GUSELKUMAB

The PBAC recommended subsidy of guselkumab for the treatment of adults with severe Crohn’s disease (CD) through the PBS. 

The PBAC welcomed input from individuals, health professionals and organisations. The input received highlighted the significant quality of life impact severe CD has on patients and the importance of having additional treatment options available, particularly medicines with different mechanisms of action to treat CD. 

The PBAC noted that guselkumab had similar effectiveness to ustekinumab for achieving clinical remission and response. Guselkumab was likely more effective than ustekinumab when treatment responses were measured using endoscopic outcomes during maintenance treatment. However, the PBAC noted the difference in the proportion of patients achieving an endoscopic remission after 48 weeks of treatment was modest (9-15%). Furthermore, the additional evidence provided by the supplying company to support claims that improvement in endoscopic outcomes would translate to better long-term outcomes for patients showed only very modest potential benefit. Evidence that endoscopic remission translated to better clinical remission was weak and evidence that it translated to improved quality of life was inconsistent. The PBAC considered there may be a small potential reduction in surgery/ hospitalisation for patients who achieve endoscopic outcomes, but that this was uncertain and could not be reliably estimated with the available data. 

To support its requested price, the company claimed in its economic model that guselkumab would provide benefits which were not supported by the available data presented in the submission. It did not enable a reliable estimate of how much benefit guselkumab would deliver for the cost. 

In context of the potential increase in the proportion of patients achieving a response measured by endoscopic outcomes and the clinical need for additional treatment options, the PBAC considered guselkumab would be cost effective with a cost per patient over two years that is higher than for ustekinumab. 

Noting the higher cost per patient compared to all other biologic or targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs), the PBAC considered it would be appropriate for the subsidy of guselkumab to be restricted to patients who had not responded to at least one other PBS listed b/tsDMARDs for severe Crohn’s disease. The PBAC considered it would be appropriate for guselkumab to be listed with a higher level of restriction than other b/tsDMARDs i.e., as an ‘Authority Required – In Writing (full assessment)’ item. The PBAC advised the restriction criteria should be finalised in consultation with the relevant professional bodies prior to listing. For the financial estimates, the PBAC considered the estimated utilisation of guselkumab should be reduced to account for the more restricted patient population.