NCPE Completes Givinostat Review: What It Means for Duchenne Families
In November, MDI, in collaboration with Duchenne families, launched a survey to capture the lived experience of families living with Duchenne muscular dystrophy to support the patient submission for the reimbursement of givinostat. We would like to say a heartfelt thank you to everyone who took the time to participate.
We received 167 responses from parents, adults living with DMD, extended family members, family friends, and health and education professionals. The strong response clearly demonstrated the urgency and need for treatments such as givinostat to address the core unmet need of slowing disease progression. Everyone’s contributions played an important role in ensuring the real-life impact of Duchenne was clearly reflected in the submission and considered as part of the assessment.
On Tuesday, 3 March, the NCPE shared the outcome of their Health Technology Assessment (HTA). An HTA is a review of a new medicine’s benefits, clinical evidence and cost to help decide whether it should be funded by the HSE.
In their assessment of givinostat, the NCPE recommended that the cost-effectiveness of the treatment needs to be improved. In practice, this means the medicine is not considered cost-effective at its current price and the next step is for the pharmaceutical company and the HSE to enter price negotiations. For many rare disease medicines, this type of recommendation is part of the normal process, and the pharmaceutical company has indicated they are willing to enter price negotiations with the HSE.
We recognise that language about “cost-effectiveness” can be upsetting to read. It relates to how the price of a medicine compares with the clinical evidence of benefit within the healthcare system, rather than the value of the lives of those living with Duchenne.
The next step is for the pharmaceutical company to enter price negotiations with the HSE Corporate Pharmaceutical Unit (CPU). Following price negotiations, the application will then move to the HSE Drugs Group, where the full application will be considered, including the clinical evidence and the patient submission.
We recognise that this outcome may feel disappointing or disheartening for families. However, the strong advocacy from families in Ireland seeking timely access to this treatment has not gone unnoticed. The speed at which the application progressed through the NCPE, and the continued political engagement to maintain momentum through the next stages, are encouraging.
As the process now moves forward within the HSE, we will continue to work alongside families and key stakeholders to advocate for timely access to this treatment.