❗Fallacy 13 ❓​

Medicinal products that address the symptoms of a cancer are not subject to EU HTA. Really?​

 

At first glance, the EU HTA guidance seems to support this. Since January 2025, Joint Clinical Assessments are mandatory for new active substances intended for the treatment of cancer. And the scientific specifications explicitly exclude "agents intended to treat cancer associated symptoms (e.g. cancer pain)".​

 

But the guidance draws a clear line. Products that achieve symptom palliation via anti-tumour effects are considered anti-neoplastic treatment. Only supportive and palliative care without anti-tumour effect is excluded, such as analgesics, corticosteroids for pain management or antidepressants.​

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A current JCA shows why this distinction matters: pelabresib.​

 

🔬 The case: ​
🔹 Pelabresib is an oral BET inhibitor, originally developed by MorphoSys and now owned by Novartis.

🔹Indication: treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis. ​

🔹 The JCA started on 1 October 2026.​
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The wording "splenomegaly or symptoms" may suggest a symptom-oriented product. Yet pelabresib is subject to a JCA. Why?​

 

🎯 Three reasons:​
🔹 Myelofibrosis is cancer. Primary myelofibrosis (ICD-O 9961/3), polycythaemia vera (9950/3) and essential thrombocythaemia (9962/3) all carry behaviour code /3 and therefore fall under the EMA definition of cancer. ​
🔹 Splenomegaly is the disease itself. In myelofibrosis, it reflects extramedullary haematopoiesis driven by the neoplastic clone. Reducing spleen volume means reducing disease burden, not masking a symptom. ​
🔹 The mechanism is anti-neoplastic. Pelabresib inhibits BET-mediated transcription of genes that drive myelofibrosis pathogenesis. And there is a precedent: ruxolitinib, approved with exactly the same indication wording, is classified as an antineoplastic agent (ATC L01).​

By the way: the orphan designation is not the trigger here. Orphan medicinal products only come into scope in January 2028. The oncology criterion applies first.​
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📌 Key takeaway:

💡 The indication wording does not determine the scope. The mechanism of action does. Health Technology Developers in oncology should check early whether their product improves symptoms through an anti-tumour effect. If so, the JCA is mandatory, and preparation needs to start well before submission.​
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We support Health Technology Developers from scope assessment and PICO anticipation through to JCA dossier preparation. 🤝​