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.


Caprari F, Alfano A, Buzzetti G, et al. [Unmet clinical need and new therapeutic options.]. Recenti Prog Med. 2022;113(3):161-166. PMID:35315445

EFPIA. Let’s discuss the future of Unmet Medical Needs (UMN) in EU Policies. Report from a multi-stakeholder workshop (https://www.efpia.eu/media/fiveaumy/let-s-discuss-the-future-of-unmet-medical-needs.pdf) (Accessed August 2023).

Cleemput I, Devriese S, Christiaens W, Kohn L. Multi-criteria decision analysis for the appraisal of medical needs: a pilot study. Health Services Research (HSR) Brussels: Belgian Health Care Knowledge Centre (KCE). 2016. KCE Reports 272. https://doi.org/10.57598/R272C (Accessed May 2023).

Cleemput I, Devriese S, Kohn L, Westhovens R. A multi-criteria decision approach for ranking unmet needs in healthcare. Health Policy. 2018;122(8):878-884. https://doi.org/10.1016/j.healthpol.2018.06.010 PMID:29983193 DOI: https://doi.org/10.1016/j.healthpol.2018.06.010

Lakdawalla D, Malani A, Reif J. The insurance value of medical innovation. J Public Econ. 2017;145:94-102. https://doi.org/10.1016/j.jpubeco.2016.11.012 DOI: https://doi.org/10.1016/j.jpubeco.2016.11.012

Angelis A, Kanavos P. Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond: The Advance Value Framework. Soc Sci Med. 2017;188:137-156. https://doi.org/10.1016/j.socscimed.2017.06.024 PMID:28772164 DOI: https://doi.org/10.1016/j.socscimed.2017.06.024

Murphy J, Coster G. Issues in patient compliance. Drugs. 1997;54(6):797-800. https://doi.org/10.2165/00003495-199754060-00002 PMID:9421690 DOI: https://doi.org/10.2165/00003495-199754060-00002

OECD. (2021), Health at a Glance 2021: OECD Indicators, OECD Publishing, Paris (https://www.oecd-ilibrary.org/social-issues-migration-health/health-at-a-glance-2021_ae3016b9-en) (Accessed May 2023).

Vreman RA, Heikkinen I, Schuurman A, et al. Unmet Medical Need: An Introduction to Definitions and Stakeholder Perceptions. Value Health. 2019;22(11):1275-1282. https://doi.org/10.1016/j.jval.2019.07.007 PMID:31708064 DOI: https://doi.org/10.1016/j.jval.2019.07.007

World Health Organization. Consitution. Online https://www.who.int/about/governance/constitution (Accessed May 2023).

Zhang K, Kumar G, Skedgel C. Towards a New Understanding of Unmet Medical Need. Appl Health Econ Health Policy. 2021;19(6):785-788. https://doi.org/10.1007/s40258-021-00655-3 PMID:34143420 DOI: https://doi.org/10.1007/s40258-021-00655-3

Kusynová Z, Pauletti GM, van den Ham HA, Leufkens HGM, Mantel-Teeuwisse AK. Unmet Medical Need as a Driver for Pharmaceutical Sciences - A Survey Among Scientists. J Pharm Sci. 2022;111(5):1318-1324. https://doi.org/10.1016/j.xphs.2021.10.002 PMID:34634318 DOI: https://doi.org/10.1016/j.xphs.2021.10.002

Tarantola A, Otto MH, Armeni P, Costa F, Malandrini F, Jommi C. Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case. J Pharm Policy Pract. 2023;16(1):67. https://doi.org/10.1186/s40545-023-00570-z PMID:37198599 DOI: https://doi.org/10.1186/s40545-023-00570-z

Reform of the EU pharmaceutical legislation. Online https://health.ec.europa.eu/medicinal-products/pharmaceutical-strategy-europe/reform-eu-pharmaceutical-legislation_en (Accessed May 2023).

Determina AIFA n. 1535/2017 . Online https://www.aifa.gov.it/farmaci-innovativi) (Accessed May 2023).

Jommi C, Galeone C. The Evaluation of Drug Innovativeness in Italy: Key Determinants and Internal Consistency. PharmacoEconom Open. 2023;7(3):373-381. https://doi.org/10.1007/s41669-023-00393-3 PMID:36763319 DOI: https://doi.org/10.1007/s41669-023-00393-3

Fortinguerra F, Perna S, Marini R, Dell’Utri A, Trapanese M, Trotta F; Scientific & Technical Committee (Commissione Tecnico-Scientifica, CTS) of Italian Medicines Agency-AIFA. The Assessment of the Innovativeness of a New Medicine in Italy. Front Med (Lausanne). 2021;8:793640. https://doi.org/10.3389/fmed.2021.793640 PMID:34957163 DOI: https://doi.org/10.3389/fmed.2021.793640

Galeone C, Bruzzi P, Jommi C. Key drivers of innovativeness appraisal for medicines: the Italian experience after the adoption of the new ranking system. BMJ Open. 2021;11(1):e041259. https://doi.org/10.1136/bmjopen-2020-041259 PMID:33441356 DOI: https://doi.org/10.1136/bmjopen-2020-041259

AIFA. Linee guida per la compilazione del dossier a supporto della domanda di rimborsabilità e prezzo di un medicinale ai sensi del d.m. 2 agosto 2019 - Versione 1.0 – 2020. (https://www.aifa.gov.it/documents/20142/1283800/Linee_guida_dossier_domanda_rimborsabilita.pdf) (Accessed May 2023).

AIFA. Accesso precoce al farmaco e uso off-label. Online https://www.aifa.gov.it/accesso-precoce-uso-off-label (Accessed May 2023).

Transparency Committee (HAS) doctrine. Principles of medicinal product assessments and appraisal for reimbursement purposes. 2020. Online https://www.has-sante.fr/upload/docs/application/pdf/2019-07/doctrine_de_la_commission_de_la_transparence_-_version_anglaise.pdf (Accessed May 2023).

Transparency Committee (HAS) doctrine. Authorisation for early access to medicinal products: HAS assessment doctrine. 2021. Online https://www.has-sante.fr/upload/docs/application/pdf/2021-08/authorisation_for_early_access_to_medicinal_products_has_assessment_doctrine.pdf) (Accessed May 2023).

UK Government Guidance. Early access to medicines scheme (EAMS): task group and principles. 2016. Online https://www.gov.uk/government/publications/early-access-to-medicines-scheme-eams-how-the-scheme-works/early-access-to-medicines-scheme-eams-task-group-and-principles (Accessed May 2023).

NICE health technology evaluations: the manual. Process and methods (PMG36) 2022. Online https://www.nice.org.uk/process/pmg36/chapter/introduction-to-health-technology-evaluation (Accessed May 2023).

Karen M, Vishnu PW. PD48 Does Unmet Need Influence the Scottish Medicines Consortium Health Technology Assessment Decisions For Rare Disease Conditions? Online https://www.cambridge.org/core/journals/international-journal-of-technology-assessment-in-health-care/article/pd48-does-unmet-need-influence-the-scottish-medicines-consortium-health-technology-assessment-decisions-for-rare-disease-conditions/16EE12B6704F13803FEEAFCFE5656027 (Accessed May 2023).

García CH. Spanish routes for making available medicines to patients before authorisation. Online https://health.ec.europa.eu/system/files/2016-11/stamp_stamp_agenda_point_4_es_aemps_en_0.pdf (Accessed May 2023).

Regolamento UE. 2021/2282 del Parlamento Europeo e del Consiglio del 15 dicembre 2021 relativo alla valutazione delle tecnologie sanitarie e che modifica la direttiva 2011/24/UE. Online https://eur-lex.europa.eu/legal-content/IT/TXT/PDF/?uri=CELEX:32021R2282&from=EN (Accessed May 2023)



How to Cite

Jommi C, Meregaglia M, Pinto C. Unmet need: definitions, literature evidence and implications for HTA. Grhta [Internet]. 2023 Oct. 2 [cited 2023 Dec. 7];10(1):70-8. Available from: https://journals.aboutscience.eu/index.php/grhta/article/view/2624



Original Research Articles


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


Most read articles by the same author(s)

1 2 > >>