The new EU regulation highlights the importance of patient voices in HTA processes because they provide unique perspectives. Their insights might reveal real-world benefits that clinical trials often overlook. Patients and families provide firsthand information about how experimental treatments impact their quality of life, daily functioning, and mental health.
The HTA Stakeholder Network, established by the European Commission in 2023, aims to ensure that patients’ experiences inform HTA evaluations. The network is made up of all the stakeholders involved in HTA procedures such as national HTA bodies, patient organizations, payer bodies, and professional societies – all of whom play a role in deciding whether a treatment should be approved and funded. Key patient organisations representing HD in the network are the European Federation of Neurological Associations (EFNA) and EURORDIS, which represent neurological and rare diseases.
In the context of HTAs, input from patients who represent rare diseases is particularly vital. Due to limited data and evidence gaps, real-world insights are invaluable, since highlighting the challenges faced by rare disease communities can encourage decision-making bodies to adopt policies that prioritize these groups.
To get involved, patients, especially those who have participated in clinical trials, can share their experiences through surveys, consultations, or advocacy groups to amplify their voices. By actively engaging, individuals with HD and family members can:
- Influence the criteria used to evaluate therapies by emphasizing quality of life and relevant disease progression markers.
- Encourage decision-makers to prioritize equitable access to therapies for rare diseases.
- Build trust in the HTA process through transparency and collaboration.
In general, patient involvement aims to build a more inclusive and effective healthcare system.
