One of Rare Voices Australia’s (RVA) formal research partnerships was chosen for funding under the Medical Research Future Fund (MRFF) Preventive and Public Health Research Initiative 2022 Assessment of High-Cost Gene Treatments and Digital Health Interventions Grant Opportunity. RVA congratulates lead researcher Professor Kirsten Howard, from the University of Sydney, and the incredible team of researchers and other partners on this successful outcome.
This program of work is entitled, Development of a generalisable evaluation framework for high upfront-cost gene therapies: clinical, financial, ethico-legal and cultural considerations. It will address key challenges associated with the assessment of gene therapies for reimbursement, directly informed by stakeholder preferences and ethical and equity considerations. This work directly responds to the following actions under the Care and Support Pillar of the Australian Government’s National Strategic Action Plan for Rare Diseases:
Action 2.4.1
Develop policy that supports people living with a rare disease to have timely and equitable access to new and emerging health technologies.
Action 2.4.3
Ensure people living with a rare disease have equitable access to medicines with demonstrated clinical benefit for a rare disease, including those that are already funded for another condition.
RVA provided a Letter of Support to this grant and has committed to the following roles in this research program:
- Provision of rare disease consumer and policy expertise on the project advisory board
- Support to disseminate results from this research to the rare disease community
- Support with project governance
- Participation in face-to-face Knowledge Exchange Workshops
- Ad hoc policy advice across the two-year duration of this project
More information about RVA’s formal Research Partnerships Program can be found on RVA’s website.
