Assessment and value of drugs: report of the focus groups from the XXII National Conference on Pharmaceuticals
DOI:
https://doi.org/10.33393/grhta.2025.3469Keywords:
Access, Health Technology Assessment, HTA regulation, InnovativenessAbstract
The XXII National Conference on Pharmaceuticals, held in Catania from 29 February to 1 March 2024, involved the participation of representatives from more than forty pharmaceutical industries, national authorities, academia, clinicians and clinical pharmacists. The 22nd edition represented a key forum for the analysis of value assessment of medicines, focusing on the impact of new European and National regulations, including the Italian Medicines Agency (AIFA) reform and the HTA regulation. This point of view summarizes insights from focus groups discussions that took place during the Conference, highlighting the pillar role of the new HTA regulation for homogenizing the evaluation across Europe, and the importance to strengthen the collaboration among the parties involved from an early phase, and implementing adaptive and flexible assessment, especially for orphan medicines. The re-evaluation of the innovativeness criteria and framework of the Pricing and Reimbursement (P&R) dossier are points raised among the different focus groups, especially for aligning these tools to the upcoming HTA regulation. The proposals emerged could be useful for AIFA, which is facing a reorganization aimed to optimizing the reimbursement process in Italy.
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References
AIFA. Decreto 8 gennaio 2024, n.3, Regolamento recante modifiche al regolamento sull’organizzazione e sul funzionamento dell’Agenzia Italiana del Farmaco, GU Serie Generale n. 11 del 15 gennaio 2024. (Accessed January 2025) Online
Regolamento (UE) 2021/2282 del Parlamento Europeo e del Consiglio del 15 dicembre 2021 relativa alla valutazione delle tecnologie sanitarie e che modifica la Direttiva 2011/24/UE. (Accessed January 2025) Online
Gazzetta Ufficiale della Repubblica Italiana. Determina 1535/2017 dell’Agenzia Italiana del Farmaco “criteri per la classificazione dei farmaci innovativi, e dei farmaci oncologici innovativi, ai sensi dell’articolo 1, comma 402 della legge 11 dicembre 2016, n. 232, GU n. 218 del 18 settembre 2017”. (Accessed January 2025) Online
AIFA. Modulo per la richiesta del riconoscimento dell’innovatività. (Accessed January 2025) Online
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(1):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
Jommi C, Armeni P, Bertolani A, Costa F, Otto M. Il futuro dei Fondi per Farmaci Innovativi: risultati di uno studio basato su Delphi panel. Glob Reg Health Technol Assess. 2021;8:22-28. https://doi.org/10.33393/grhta.2021.2219 PMID:36627867 DOI: https://doi.org/10.33393/grhta.2021.2219
Lobban T CA and Camm. Patient associations as stakeholders: a valuable partner for facilitationg access to therapy. Europace. 2011, 13, ii21-ii24:10. doi: 10.1093/europace/eur086. PMID: 21518744. DOI: https://doi.org/10.1093/europace/eur086
Ocloo J, Matthews R. From tokenism to empowerment: progressing patient and public involvement in healthcare improvement. BMJ Qual Saf. 2016;25(8):626-632. https://doi.org/10.1136/bmjqs-2015-004839 PMID:26993640 DOI: https://doi.org/10.1136/bmjqs-2015-004839
Haerry D, Landgraf C, Warner K, et al. EUPATI and patients in medicines research and development: guidance for patient involvement in regulatory processes. Front Med (Lausanne). 2018;5:230. https://doi.org/10.3389/fmed.2018.00230 PMID:30175100 DOI: https://doi.org/10.3389/fmed.2018.00230
Graffigna G, Barello S, Riva G, et al. Italian Consensus Statement on Patient Engagement in chronic care: process and outcomes. Int J Environ Res Public Health. 2020;17(11):4167. https://doi.org/10.3390/ijerph17114167 PMID:32545278 DOI: https://doi.org/10.3390/ijerph17114167
Miksad RA, Abernethy AP. Harnessing the Power of Real-World Evidence(RWE): a checklist to ensure regulatory-grade data quality. Clin Pharmacol Ther. 2018;103(2):202-205. https://doi.org/10.1002/cpt.946 PMID:29214638 DOI: https://doi.org/10.1002/cpt.946
Beaulieu-Jones BK, Finlayson SG, Yuan W, et al. prasad V, Yu KH. Examining the use of real-World Evidence in the regulatory Process. Clin Pharmacol Ther. 2020;107(4):843-852. https://doi.org/10.1002/cpt.1658 PMID:31562770 DOI: https://doi.org/10.1002/cpt.1658
Prilla S, Groeneveld S, Pacurariu A, et al. Real-World Evidence to Support EU Regulatory Decision Making-Results From a Pilot of Regulatory Use Cases. Clin Pharmacol Ther. 2024;116(5):1188-1197. https://doi.org/10.1002/cpt.3355 PMID:38962830 DOI: https://doi.org/10.1002/cpt.3355
GRADE Welcome to the GRADE working group. From evidence to recommendations-trasparent and sensible. Online www.gradeworkinggroup.org (Accessed January 2025)
Cicchetti A, Gasbarrini A. The healthcare service in Italy: regional variability. Eur Rev Med Pharmacol Sci. 2016;20(1)(suppl):1-3. PMID:28083867 PMID:28083867
Bortolami A, Jommi C, Bresciani F, Piccoli L, Sangiorgi E, Scroccaro G. Regional Formularies in Italy: current state and future perspectives. Glob Reg Health Technol Assess. 2024;11(1):68-74. https://doi.org/10.33393/grhta.2024.2677 PMID:38504973 DOI: https://doi.org/10.33393/grhta.2024.2677
Wolters S, Jansman FGA, Postma MJ. Differences in evidentiary rewuirements between European Medicines Agency and European Health Technology Asessment of Oncology drugs-Can allignment be enhanced? Value Health. 2022;25(12):1958-1966. https://doi.org/10.1016/j.jval.2022.05.006 PMID:35752535 DOI: https://doi.org/10.1016/j.jval.2022.05.006
Gentilini A, Miraldo M. The role of patient organisations in research and development: evidence from rare diseases. Soc Sci Med. 2023;338:116332. https://doi.org/10.1016/j.socscimed.2023.116332 PMID:37866173 DOI: https://doi.org/10.1016/j.socscimed.2023.116332
Shakhnenko I, Husson O, Chuter D, van der Graaf W. Elements of successful patient involvement in clinical cancer trials: a review of the literature. ESMO Open. 2024;9(4):102947. https://doi.org/10.1016/j.esmoop.2024.102947 PMID:38492274 DOI: https://doi.org/10.1016/j.esmoop.2024.102947
Decreto legge 13 settembre 2012, n. 158, articolo 12, comma 5 bis. “Disposizioni urgenti per promuovere lo sviluppo del Paese mediante un più alto livello di tutela della salute”. (Accessed January 2025) Online
AIFA. Decreto 2 agosto 2019. “Criteri e modalità con cui l’Agenzia Italiana del Farmaco determina, mediante negoziazione, i prezzi dei farmaci rimborsati dal Servizio sanitario nazionale. GU Serie Generale, n. 185 del 24 luglio 2020”. (Accessed January 2025) Online
Patarnello F. Regolamento HTA: come si sta muovendo l’Italia? Glob Reg Health Technol Assess. 2024;11:51-54. https://doi.org/10.33393/grhta.2024.3025 PMID:38449560 DOI: https://doi.org/10.33393/grhta.2024.3025
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This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
Accepted 2025-02-15
Published 2025-02-25
