Unmet need: definitions, literature evidence and implications for HTA


  • Claudio Jommi Dipartimento di Scienze del Farmaco, Università del Piemonte Orientale, Novara - Italy https://orcid.org/0000-0002-2862-2990
  • Michela Meregaglia Dipartimento di Scienze del Farmaco, Università del Piemonte Orientale, Novara - Italy and Cergas, SDA Bocconi School of Management, Università Bocconi - Italy https://orcid.org/0000-0003-0092-5970
  • Carmine Pinto Oncologia Medica, Comprehensive Cancer Centre, AUSL-IRCCS Arcispedale di Reggio Emilia - Italy https://orcid.org/0000-0002-3159-5268




Early Access, Health Technology Assessment, Unmet need


The present paper illustrates the definition of unmet need provided by the peer-reviewed literature and the Health Technology Assessment (HTA) authorities across Europe in the assessment and appraisal process and within the early access schemes for medicines.

The analysis relied on a descriptive review of the peer-reviewed literature and HTA documents on the definition of need (disease severity) and the way it is satisfied (existence and validity of alternatives).

HTA agencies were found using (i) a narrow definition of need, focused on the clinical impact and the impact on health-related quality of life of the disease and (ii) a broad definition of comparators, including treatments used off-label in the clinical practice. Most of the contributions of the literature advocated for a broader definition of need, including additional dimensions (for example, the socio-economic impact of the disease) and the effects of existing treatments beyond their risk-benefit profile (for example, acceptability to patients).

On the one hand, these contributions should be considered by HTA agencies, considering its multi-disciplinary and multi-stakeholder nature. On the other one, the explicit inclusion of the unmet need domains, at present disregarded, should depend on the decisions taken on the ground of the assessment.


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How to Cite

Jommi, C., Meregaglia, M., & Pinto, C. (2023). Unmet need: definitions, literature evidence and implications for HTA. Global and Regional Health Technology Assessment, 10(1), 70–78. https://doi.org/10.33393/grhta.2023.2624



Original Research Articles


Received 2023-07-02
Accepted 2023-08-28
Published 2023-10-02


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