Drugs price and reimbursement regulation: comparators, endpoints and role of the cost-effectiveness
Keywords:Comparators, Cost-effectiveness, Endpoints, 648 List, Price and reimbursement
This document illustrates the results of a discussion of two multi-disciplinary expert panels on pricing and reimbursement of medicines. Experts work in different organizations. The discussion focused on comparator(s), endpoint(s), negotiation of prices of new medicines and/or indications to include in the List 648, as well as the role of cost-effectiveness in the price and reimbursement negotiation. The debate took place during the fourth edition of the Seminari di Mogliano, organized on the 30th of September/1st of October, 2021.
The two panels agreed on a general need to enhance interaction among the different stakeholders, in the early assessment and negotiation phases, and to increase the transparency/reproducibility of the decisions taken. The experts have also emphasized the need (i) to improve clarity in the evaluation of additional therapeutic value and the place in therapy with respect to comparators and how comparators are identified; (ii) to create work groups to identify the most appropriate endpoint(s), for each therapeutic area and level of unmet needs; (iii) to provide for a systematic use of cost-effectiveness when an added therapeutic value is delivered by a new medicine. With regard to the 648 List, the experts advocated for an overall reorganization of the current rules governing the special uses of drugs.
Decreto Ministeriale 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 24-07-2020. Online https://www.gazzettaufficiale.it/eli/id/2020/07/24/20A03810/sg. Accessed June, 10, 2022.
AIFA. Linee-Guida per la compilazione del dossier a supporto della domanda di rimborsabilità e prezzo di un medicinale. Versione 1.0, 2020. Online https://www.aifa.gov.it/documents/20142/1283800/Linee_guida_dossier_domanda_rimborsabilita.pdf. Accessed June, 10, 2022.
AIFA. Report tecnico-scientifici per specialità medicinale. Online https://www.aifa.gov.it/report-tecnico-scientifici. Accessed June, 10, 2022.
HAS (Haute Autorité de Santé). Authorisation for early access to medicinal products: HAS assessment doctrine. Online https://www.has-sante.fr/upload/docs/application/pdf/2021-08/authorisation_for_early_access_to_medicinal_products_has_assessment_doctrine.pdf. Accessed June, 10, 2022.
AIFA. Accesso precoce al farmaco e uso off-label. Online https://www.aifa.gov.it/accesso-precoce-uso-off-label. Accessed June, 10, 2022.
Newton M, Scott K, Troein P. VP. EFPIA Patients W.A.I.T. Indicator 2021 Survey (April 2022). Online https://www.efpia.eu/media/636821/efpia-patients-wait-indicator-final.pdf. Accessed June, 10, 2022.
Husereau D, Drummond M, Augustovski F, et al. Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 Explanation and Elaboration: A Report of the ISPOR CHEERS II Good Practices Task Force. Value Health. 2022;25(1):10-31. https://doi.org/10.1016/j.jval.2021.10.008 PMID:35031088 DOI: https://doi.org/10.1016/j.jval.2021.10.008
How to Cite
Copyright (c) 2022 The authors
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
Authors contributing to Global & Regional Health Technology Assessment agree to publish their articles under the CC-BY-NC 4.0 license, which allows third parties to re-use the work without permission as long as the work is properly referenced and the use is non-commercial.