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In our previous article about Health Technology Assessments (HTAs), we explained what HTAs are and why they matter. If you haven’t had the chance to read it, we suggest you take a look and then come back here, since there is valuable information there too.
In today’s article, we want to take a deeper dive into the topic of HTAs and their importance for the HD community.
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.Â
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:
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In general, patient involvement aims to build a more inclusive and effective healthcare system.
An important way patients contribute to HTAs is through Real-World Data (RWD), which provides insights beyond the data collected in clinical trials. RWD refers to data collected outside clinical trials in uncontrolled settings, such as observational studies, and patient-reported experiences. By combining clinical trial data with RWD, HTAs can deliver a more comprehensive evaluation of a therapy’s value, ensuring that it takes into consideration the actual needs of the population it is meant to serve.
Unlike clinical trials, which occur under controlled conditions, RWD reflects how therapies perform in everyday settings, providing insights that are often more relevant to patients. This contrast highlights that while some outcomes measured in clinical trials using standardized scales may seem minor, they can have a significant impact on the person’s quality of life and ability to function better in their daily life. RWD comes from sources such as:
For HD, this can mean collecting information on how a treatment impacts patients’ quality of life, functional abilities, and mental well-being. For example, a therapy might not significantly slow disease progression according to data collected on clinical trials, but could dramatically improve mobility, daily functioning, or caregiver burden, all of which RWD can reveal.Â
When analyzed, RWD is transformed into Real World Evidence (RWE), which are scientific insights that regulators and HTA bodies use to make informed policy and reimbursement decisions. By including RWE, HTAs gain a more holistic view of a therapy’s value, ensuring decisions reflect real-world benefits that matter most to patients and families.
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 HTAs are more than technical evaluations – they drive reimbursement decisions by determining whether a therapy offers good value for its cost and if it should be funded by the government. The new EU regulation aims to ensure that new therapies deliver real value to patients while also being financially sustainable. Sustainability is complex, as it depends not only on the information collected through HTAs, but also on the pricing set by the pharmaceutical companies that develop new drugs and treatments. Also, the cost of new treatments affects each country’s decision-making bodies differently, as they operate within distinct budgetary constraints.
By involving patient organizations and harmonizing HTA processes, we can ensure therapies not only reach patients but also meaningfully improve their lives. For Huntington’s Disease, for which no specific treatment currently exists, it is crucial to stay informed and involved so that regulatory bodies in each country understand what matters most to the community before making decisions. The new EU HTA Regulation promises a more collaborative and equitable approach, and by participating, patients can help drive progress.Â
While the details of Health Technology Assessments and real-world data may seem complex, it’s important to stay informed. The decisions made through these processes affect not only the accessibility of treatments but also their meaningful impact on patients’ lives.Â
Stay tuned for our next article where we will cover the topic of cost-benefit and the analysis decision-making bodies perform before making reimbursement decisions.
If you are interested in this topic and would like to get involved, you can contact us at astri@eurohuntington.org.
– Article written by Jarelys López Delgado