DUPILUMAB

The PBAC recalled that it previously recommended dupilumab for severe atopic dermatitis in patients aged less than 12 years at its March 2022 meeting. The PBAC noted that the supplying company had not taken the steps needed proceed to PBS listing after that meeting. This submission sought PBAC consideration of updated use estimates and a revised risk sharing arrangement for the expanded listing. The PBAC decided not to revise its previous recommendation to expand the PBS listing of dupilumab to include treatment of atopic dermatitis in patients aged less than 12 years on the basis that the proposal did not support a cost-effective listing for dupilumab. 

The PBAC welcomed inputs from consumers, health care professionals and organisations. The PBAC acknowledged that there is a high clinical need for treatments for severe atopic dermatitis in children, noting the disease has a significant impact on many aspects of quality of life and broader impacts on development for younger patients. The PBAC acknowledged the importance of expanding eligibility to enable equitable access to younger patients. The PBAC noted that clinical guidelines state that dupilumab is the preferred treatment for patients with severe atopic dermatitis, who have not responded to daily topical therapy (corticosteroid/calcineurin inhibitor) and that few other treatments are available for patients aged <12 years.

The submission presented revised estimates of how many patients would use dupilumab and an updated Risk Sharing Arrangement proposal. The submission did not propose any additional restriction criteria for the <12 years population to restrict treatment to the most severely affected paediatric population with the highest need. The PBAC noted that the submission proposed a price per month for paediatric patients that was higher than the current price in the adolescent/adult population (aged 12+). The PBAC maintained its advice that dupilumab would be cost-effective at the same price per month for children as for the adolescent/adult population. The PBAC considered that there was no clear evidence provided to support or quantify any additional benefits for paediatric patients and that at the higher proposed price, use of dupilumab in children <12 years was not acceptably cost-effective. 

The PBAC considered that the RSA proposed in the submission did not address uncertainty in use estimates and did not adequately address the potential for use outside the intended population, or the potential continuing use in patients who do not have adequate response or where patients’ disease has resolved and no longer requires treatment.

Overall, the PBAC considered that the price proposed, the changes to the financial estimates, and the proposed parameters for the RSA were not acceptable. Thus, the PBAC advised that its March 2022 recommendation for dupilumab remained unchanged.

The PBAC considered that if the supplying company is unable to progress the intent of the March 2022 PBAC recommendation, an alternative approach may be to explore mechanisms to limit treatment to children with the highest need. The PBAC noted that, depending on the approach, the way forward could include either: 
• Reduce the price, substantially reduce the use estimates, and revise the RSA proposal, so that the listing is cost-effective and the economic and financial risks associated with the listing are adequately managed; or
• Propose revisions to the restriction criteria, allowed prescriber types, restriction authority level, and RSA caps for the <12 years population reflecting a more limited population and requirements for trial of stopping treatment. If a higher cost per month is requested for the <12 years population compared with the adolescent/adult population, a justification for the price would be required.

Sponsor's comment:
Sanofi is disappointed by the PBAC's recent decision, particularly as Dupixent remains the only TGA-approved biologic for uncontrolled severe atopic dermatitis (AD) in children under 12. Sanofi has supported hundreds of children with a high unmet clinical need through compassionate access, in addition to leading Australian children's hospitals also funding therapy for severe cases. We deeply appreciate the medical and patient community for their advocacy throughout this journey, including the many individual letters sent to the PBAC during the public consultation period, highlighting the need for advanced therapy such as Dupixent to treat severe AD and reduce the significant burden this chronic skin condition has on the health and quality of life of young children and their families. We remain committed to working collaboratively toward a viable PBS listing, so that dermatologists and immunologists can prescribe an effective advanced therapy for young patients with uncontrolled severe AD.