Health technologies include medications, devices, and systems designed to address health problems, improve health outcomes, and enhance the quality of care. For Huntington’s Disease (HD), health technologies include potential therapies that, if developed, could profoundly impact patients’ lives. But, in case a therapy is developed, how do governments decide which health technologies are worth adopting? This is where Health Technology Assessments (HTAs) come in.
HTAs are rigorous systematic evaluations of health technologies, assessing their clinical effectiveness, economic value, and impact on society. They aim to answer critical questions, such as: Does the therapy work better than existing options? Is it worth the cost? Can it improve patients’ quality of life? In general, HTAs help ensure healthcare resources are used effectively by guiding decisions on funding and implementation. Therefore, they have a direct impact on access to therapies.
Bringing a new therapy to patients involves two main steps: market approval and reimbursement decisions.
Currently, this process is fragmented across Europe, with each country conducting its own HTAs. This leads to duplicated efforts, inconsistent outcomes, and delays in patient access in most countries. The new EU HTA Regulation, which becomes operative this week, aims to harmonize this process, with the intention of providing faster and more consistent access to therapies for patients across Member States.
The EU HTA Regulation (EU 2021/2282) introduces Joint Clinical Assessments (JCAs), which standardize the evaluation of a therapy’s clinical value across all Member States of the European Union. The JCAs aim to eliminate duplication, reduce delays, and provide a unified framework for decision-making. They will be implemented gradually, initially becoming mandatory for oncology and advanced therapies in 2025, expanding to orphan drugs in 2028, and eventually including all new EMA-approved medicines in 2030. Although HD is a rare disease and therapies under study are considered potential orphan drugs, some of the treatments being researched qualify as advanced therapies due to the technologies they employ.
The goal with this new system is to improve equity in access, by harmonizing assessments to ensure that patients, regardless of where they live in the EU, have more equal opportunities to access innovative therapies. Despite the implementation of JCAs, which will help for all Member States to have access to the same information, reimbursement decisions will remain national responsibilities. Therefore it’s important that the decision-making leaders trust the JCAs, and pave the way for faster and fairer access.
For rare conditions like Huntington’s Disease, HTAs face unique challenges such as high treatment costs and lack of awareness. Therapies for rare diseases, known as orphan drugs, are expensive to develop, and traditional cost-effectiveness models can disfavor these treatments, even when they are life-changing for patients. Rare diseases often lack visibility, meaning the broader impacts on patients and caregivers may be overlooked in reimbursement evaluations. To address these challenges, HTAs are also increasingly taking into account what is known as real world data. information collected outside clinical trials.
Additionally, many HTA bodies struggle with a shortage of experts familiar with rare diseases like HD. This lack of expertise can lead to inconsistent or incomplete evaluations, further delaying access to therapies. To address this, the new EU HTA Regulation includes the creation of professional groups that bring together experts from across Member States. These groups aim to ensure a higher scientific standard for evaluations, offering more consistent and informed assessments of therapies for rare diseases.
If you are interested in this topic and would like to contribute, you can contact us at astri@eurohuntington.org. Stay tuned to our next blog to learn more about why patients and caregivers should be involved in HTAs.
– Article written by Jarelys López
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