François Houÿez: Improving how new treatments are assessed across Europe
Novembro 2024
We believe that patients should have a strong voice in decisions about new treatments. Earlier this summer, we provided feedback on a set of rules being developed to guide how new treatments are evaluated across Europe.
These evaluations, called Joint Clinical Assessments (JCA), help determine whether new medicines and medical devices are more, equally, or less effective than an already existing technology and should be made available to patients. Here’s what we think could be improved to make this process work better for everyone.
Ensuring state-of-the-art clinical assessments
One of the most important parts of these assessments is defining what will be studied – things like which patients the treatment is for, what the treatment will be compared to, and what outcomes will be measured. This is known as the PICO (Population, Intervention, Comparator, and Outcomes) framework. We believe it’s crucial that these assessments are based on the most up-to-date medical knowledge. However, we also suggest not overwhelming the process with too many questions, as this could make it difficult to complete the assessment on time and with the quality needed. It’s important that the questions posed to developers are both scientifically grounded and realistic, considering the existing standards of care across different Member States.
Balancing timelines with quality outcomes
We acknowledge the challenges posed by the current timelines for JCAs, particularly for Health Technology Developers (HTDs) who must submit extensive data within a constrained timeframe. The risk here is that the pressure to meet deadlines could lead to incomplete or low-quality submissions, ultimately undermining the credibility of the JCA process. We suggest giving these developers more realistic timelines, aligning their work with other processes like those of the European Medicines Agency (EMA). This adjustment would provide HTDs with a more realistic preparation window, thus enhancing the overall quality of the data submitted.
The role of patients and healthcare professionals
Patients and healthcare professionals offer unique insights that are crucial to these assessments. We strongly support their active and meaningful involvement from the beginning to the end of the process. They should help define what will be studied (also known as the assessment scope, the PICO), contribute during the assessment, and review the final reports. It’s also important that their contributions are clearly acknowledged and made prominent in the final reports, so everyone knows how patient input has contributed to the final decisions.
Facilitating a structured consultation process
We believe that patient organisations should be allowed to provide feedback on broader issues beyond just the specific disease or treatment being assessed. They can offer valuable insights into patient experiences and preferences. To support patients throughout the process, we recommend that they be paired with a mentor from a relevant patient organisation, ensuring they feel confident and prepared at every stage.
Addressing the complexity of medical devices in JCAs
The draft Act currently lacks specific provisions for assessing medicinal products linked to medical devices, such as companion diagnostics – a test used to help match a patient to a specific drug or therapy. For example, a companion diagnostic test may identify whether a patient’s tumour has a specific gene change or biomarker that is targeted by the drug. This helps determine if the patient should receive the drug or not.
These devices are regulated differently from medicines, and the current rules don’t fully address how they should be evaluated together. We recommend that the new rules provide clear guidance on how to handle these cases, ensuring that both the medicine and the device are properly assessed.
Improving expert selection and involvement
Choosing the right experts, including patients, is key to a successful assessment. While we appreciate that the rules suggest using a wide range of sources to find these experts, such as EURORDIS and other patient organisations that are members of the HTA Stakeholder Network, the European Reference Networks (ERNs) and Orphanet, the selection process is currently quite complex, and the timelines are tight. If the chosen experts are not approved, there might not be enough time to find replacements. We suggest creating a special role for experts who might have a conflict of interest, allowing them to provide input without fully participating in the discussions – thus maintaining the integrity of the process.
Protecting confidential information
The current rules include obligations to keep certain information confidential. However, the language used implies that all patient experts are professionals, which isn’t always the case. We suggest changing the term “professional secrecy” to “confidentiality obligations” to reflect the reality that many patient experts are not professionals. Additionally, confidentiality should end once the assessment report is published, so that everyone knows when their obligation to keep information private is over.
Making timelines clearer
Some parts of the current rules are a bit vague, particularly around when patients might be asked for their input. We believe it’s important to have more predictability, ensuring that patients are consulted throughout the process. This ensures that their feedback is considered thoroughly, not just as an afterthought.
Recommendations for annexes
Finally, in our review of the annexes, we identified several areas where additional clarity and detail are needed, particularly concerning orphan medicinal products and the integration of information from previous joint scientific consultations. The current templates for JCA reports and summaries are too simplistic. We suggest forming a working group that includes patient organisations to create more comprehensive and user-friendly templates. This will make the reports more useful and easier to understand for everyone involved.
By François Houÿez, Director of Treatment Information and Access
Disclaimer: As a Staff Blog, the opinions – including possible policy recommendations – expressed in this article are those of the author and do not necessarily represent the views or opinions of EURORDIS. The publication of this article on the EURORDIS website does not equate to endorsement.