Patient Organisations in Europe Submit Comments on the First Joint Clinical Assessment Report under the HTA Regulation
Published 30 June 2026
Last update 22 July 2026
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Following the publication of the first Joint Clinical Assessment report under the new Health Technology Assessment (HTA) Regulation, fifteen patient organisations in Europe have issued a joint statement with comments on the assessment results.
The statement concerns the first Joint Clinical Assessment (JCA) report on **tovorafenib (Ojemda)** by Ipsen Pharma, indicated for the treatment of patients with paediatric low-grade glioma associated with BRAF gene alterations.
Patient organisations welcomed the publication of the first report as an important step towards a more consistent and patient-centred HTA system in the European Union.
At the same time, they emphasised that the involvement of patients, caregivers, and patient organisations must be strengthened.
📌 Patient experience should be taken into account earlier
The statement notes that the involvement of patients and caregivers should begin at an early stage of the assessment – when defining the scope, the assessment question, the patient population, the comparator, and the relevant outcomes.
This would allow for better consideration of those treatment outcomes that truly matter to patients and their families.
For rare diseases and paediatric oncology, this is particularly significant: standard clinical endpoints do not always fully reflect the impact of therapy on the patient's daily life, the family's functional status, and the long-term burden of the disease.
🔎 The question of representativeness
Patient organisations also specifically draw attention to the representativeness of patient input.
The opinion of a single patient or a single caregiver has value, but it does not always reflect the experience of the entire patient community.
Therefore, patient organisations should be more actively involved in the process and present a broader perspective on the disease, treatment experience, unmet needs, and real expectations from therapy.
⚖️ Greater transparency is needed
One of the key remarks was the need for greater transparency.
The report indicates that patient input was considered, but it is not always clear how exactly it influenced the selection of outcomes, the interpretation of clinical data, and the final conclusions of the assessment.
For the patient community, it is important to see not only the fact of participation itself, but also how this input was taken into account in the expert decision-making process.
📊 Lack of data on outcomes that matter to patients
The statement also highlights the lack of data on outcomes that are of direct importance to patients and families:
— quality of life
— disease symptoms
— functional status
— fatigue
— treatment burden
— the impact of the disease and therapy on daily life.
Such data should be planned and collected in advance during the development and evaluation of new medicinal products, especially in rare diseases and paediatric oncology.
🌍 Accessibility of information
Special attention is paid to the accessibility of the assessment results.
In the view of patient organisations, plain-language summaries of Joint Clinical Assessment reports should be written in understandable language and published in the official languages of the EU.
This would enable patients and patient organisations in different countries to use the assessment results, participate in national discussions, and better understand the rationale behind future decisions on access to therapy.
📌 Why this matters
Patient organisations supported the very idea of Joint Clinical Assessment, but emphasised that the new HTA system in the EU must be not only methodologically consistent, but also more transparent, inclusive, and focused on outcomes that are meaningful to patients.
For countries developing their own HTA approaches, this example is important also because it demonstrates that patient involvement should not be a formal step in the procedure. It should influence the framing of questions, the selection of outcomes, and the interpretation of the clinical value of a technology.
📎 Patient organisations' joint statement
The statement concerns the first Joint Clinical Assessment (JCA) report on **tovorafenib (Ojemda)** by Ipsen Pharma, indicated for the treatment of patients with paediatric low-grade glioma associated with BRAF gene alterations.
Patient organisations welcomed the publication of the first report as an important step towards a more consistent and patient-centred HTA system in the European Union.
At the same time, they emphasised that the involvement of patients, caregivers, and patient organisations must be strengthened.
📌 Patient experience should be taken into account earlier
The statement notes that the involvement of patients and caregivers should begin at an early stage of the assessment – when defining the scope, the assessment question, the patient population, the comparator, and the relevant outcomes.
This would allow for better consideration of those treatment outcomes that truly matter to patients and their families.
For rare diseases and paediatric oncology, this is particularly significant: standard clinical endpoints do not always fully reflect the impact of therapy on the patient's daily life, the family's functional status, and the long-term burden of the disease.
🔎 The question of representativeness
Patient organisations also specifically draw attention to the representativeness of patient input.
The opinion of a single patient or a single caregiver has value, but it does not always reflect the experience of the entire patient community.
Therefore, patient organisations should be more actively involved in the process and present a broader perspective on the disease, treatment experience, unmet needs, and real expectations from therapy.
⚖️ Greater transparency is needed
One of the key remarks was the need for greater transparency.
The report indicates that patient input was considered, but it is not always clear how exactly it influenced the selection of outcomes, the interpretation of clinical data, and the final conclusions of the assessment.
For the patient community, it is important to see not only the fact of participation itself, but also how this input was taken into account in the expert decision-making process.
📊 Lack of data on outcomes that matter to patients
The statement also highlights the lack of data on outcomes that are of direct importance to patients and families:
— quality of life
— disease symptoms
— functional status
— fatigue
— treatment burden
— the impact of the disease and therapy on daily life.
Such data should be planned and collected in advance during the development and evaluation of new medicinal products, especially in rare diseases and paediatric oncology.
🌍 Accessibility of information
Special attention is paid to the accessibility of the assessment results.
In the view of patient organisations, plain-language summaries of Joint Clinical Assessment reports should be written in understandable language and published in the official languages of the EU.
This would enable patients and patient organisations in different countries to use the assessment results, participate in national discussions, and better understand the rationale behind future decisions on access to therapy.
📌 Why this matters
Patient organisations supported the very idea of Joint Clinical Assessment, but emphasised that the new HTA system in the EU must be not only methodologically consistent, but also more transparent, inclusive, and focused on outcomes that are meaningful to patients.
For countries developing their own HTA approaches, this example is important also because it demonstrates that patient involvement should not be a formal step in the procedure. It should influence the framing of questions, the selection of outcomes, and the interpretation of the clinical value of a technology.
📎 Patient organisations' joint statement
