Ratanawijitrasin S, Wondemagegegnehu E. Effective drug regulation: a multicountry study. Geneva: World Health Organization; 2002.

Google Scholar 

O’Rourke B, Oortwijn W, Schuller T. The new definition of health technology assessment: a milestone in international collaboration. Int J Technol Assess Health Care. 2020;36(3):187–90. https://doi.org/10.1017/s0266462320000215.

Article  PubMed  Google Scholar 

Bertram M, Dhaene G, Tan-Torres ET. Institutionalizing health technology assessment mechanisms: a how to guide. Geneva: World Health Organization; 2021.

Google Scholar 

Kirwin E, Round J, Bond K, McCabe C. A conceptual framework for life-cycle health technology assessment. Value Health. 2022;25(7):1116–23. https://doi.org/10.1016/j.jval.2021.11.1373.

Article  PubMed  Google Scholar 

EUPATI: Health technology assessment process: fundamentals. https://toolbox.eupati.eu/resources/health-technology-assessment-process-fundamentals/ Accessed 30 June 2023.

Regier DA, Pollard S, McPhail M, Bubela T, Hanna TP, Ho C, et al. A perspective on life-cycle health technology assessment and real-world evidence for precision oncology in Canada. NPJ Precis Oncol. 2022;6(1):76. https://doi.org/10.1038/s41698-022-00316-1.

Article  PubMed  PubMed Central  Google Scholar 

Angelis A, Lange A, Kanavos P. Using health technology assessment to assess the value of new medicines: results of a systematic review and expert consultation across eight European countries. Eur J Health Econ. 2018;19(1):123–52. https://doi.org/10.1007/s10198-017-0871-0.

Article  PubMed  Google Scholar 

Ofori-Asenso R, Hallgreen CE, De Bruin ML. Improving interactions between health technology assessment bodies and regulatory agencies: a systematic review and cross-sectional survey on processes, progress, outcomes, and challenges. Front Med (Lausanne). 2020;7: 582634. https://doi.org/10.3389/fmed.2020.582634.

Article  PubMed  PubMed Central  Google Scholar 

Henshall C, Mardhani-Bayne L, Frønsdal KB, Klemp M. Interactions between health technology assessment, coverage, and regulatory processes: emerging issues, goals, and opportunities. Int J Technol Assess Health Care. 2011;27(3):253–60. https://doi.org/10.1017/s0266462311000262.

Article  PubMed  Google Scholar 

Vreman RA, Naci H, Goettsch WG, Mantel-Teeuwisse AK, Schneeweiss SG, Leufkens HGM, et al. Decision making under uncertainty: comparing regulatory and health technology assessment reviews of medicines in the United States and Europe. Clin Pharmacol Ther. 2020;108(2):350–7. https://doi.org/10.1002/cpt.1835.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Jansen E, Hines PA, Berntgen M, Brand A. Strengthening the interface of evidence-based decision making across European Regulators and Health Technology Assessment Bodies. Value Health. 2022;25(10):1726–35. https://doi.org/10.1016/j.jval.2022.01.026.

Article  PubMed  Google Scholar 

Bloem LT, Vreman RA, Peeters NWL, Hoekman J, van der Elst ME, Leufkens HGM, et al. Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies. Clin Transl Sci. 2021;14(4):1566–77. https://doi.org/10.1111/cts.13027.

Article  PubMed  PubMed Central  Google Scholar 

Pease AM, Krumholz HM, Downing NS, Aminawung JA, Shah ND, Ross JS. Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review. BMJ. 2017;357: j1680. https://doi.org/10.1136/bmj.j1680.

Article  PubMed  PubMed Central  Google Scholar 

Mitra-Majumdar M, Gunter SJ, Kesselheim AS, Brown BL, Joyce KW, Ross M, et al. Analysis of supportive evidence for US Food and Drug Administration approvals of novel drugs in 2020. JAMA Netw Open. 2022;5(5): e2212454. https://doi.org/10.1001/jamanetworkopen.2022.12454.

Article  PubMed  PubMed Central  Google Scholar 

Mills M. HTA barriers for conditional approval drugs. Pharmacoeconomics. 2023;41(5):529–45. https://doi.org/10.1007/s40273-023-01248-9.

Article  PubMed  Google Scholar 

Trapani D, Tay-Teo K, Tesch ME, Roitberg F, Sengar M, Altuna SC, et al. Implications of oncology trial design and uncertainties in efficacy-safety data on health technology assessments. Curr Oncol. 2022;29(8):5774–91. https://doi.org/10.3390/curroncol29080455.

Article  PubMed  PubMed Central  Google Scholar 

Downing NS, Aminawung JA, Shah ND, Krumholz HM, Ross JS. Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005–2012. JAMA. 2014;311(4):368–77. https://doi.org/10.1001/jama.2013.282034.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Naci H, Smalley KR, Kesselheim AS. Characteristics of preapproval and postapproval studies for drugs granted accelerated approval by the US Food and Drug Administration. JAMA. 2017;318(7):626–36. https://doi.org/10.1001/jama.2017.9415.

Article  PubMed  PubMed Central  Google Scholar 

Byrne D, Prendergast C, Fahey T, Moriarty F. Clinical study reports published by the European Medicines Agency 2016–2018: a cross-sectional analysis. BMJ Open. 2023;13(5): e068981. https://doi.org/10.1136/bmjopen-2022-068981.

Article  PubMed  PubMed Central  Google Scholar 

Wallerstedt SM, Henriksson M. Balancing early access with uncertainties in evidence for drugs authorized by prospective case series—systematic review of reimbursement decisions. Br J Clin Pharmacol. 2018;84(6):1146–55. https://doi.org/10.1111/bcp.13531.

Article  PubMed  PubMed Central  Google Scholar 

Hatswell AJ, Baio G, Berlin JA, Irs A, Freemantle N. Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999–2014. BMJ Open. 2016;6(6): e011666. https://doi.org/10.1136/bmjopen-2016-011666.

Article  PubMed  PubMed Central  Google Scholar 

Downing NS, Shah ND, Aminawung JA, Pease AM, Zeitoun JD, Krumholz HM, et al. Postmarket safety events among novel therapeutics approved by the US Food and Drug Administration between 2001 and 2010. JAMA. 2017;317(18):1854–63. https://doi.org/10.1001/jama.2017.5150.

Article  PubMed  PubMed Central  Google Scholar 

Herder M. Pharmaceutical drugs of uncertain value, lifecycle regulation at the US Food and Drug Administration, and institutional incumbency. Milbank Q. 2019;97(3):820–57. https://doi.org/10.1111/1468-0009.12413.

Article  PubMed  PubMed Central  Google Scholar 

Pereira VC, Barreto JOM, Neves F. Health technology reassessment in the Brazilian public health system: analysis of the current status. PLoS ONE. 2019;14(7): e0220131. https://doi.org/10.1371/journal.pone.0220131.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Health technology reassessment: an overview of Canadian and international processes (Environmental Scan; no. 85). Ottawa: CADTH; 2019.

Rubio-Tapia A, Herman ML, Ludvigsson JF, Kelly DG, Mangan TF, Wu TT, et al. Severe spruelike enteropathy associated with olmesartan. Mayo Clin Proc. 2012;87(8):732–8. https://doi.org/10.1016/j.mayocp.2012.06.003.

Article  PubMed  PubMed Central  CAS  Google Scholar 

U.S. Food and Drug Administration: FDA drug safety communication: FDA approves label changes to include intestinal problems (sprue-like enteropathy) linked to blood pressure medicine olmesartan medoxomil. https://www.fda.gov/drugs/drug-safety-and-availability/fda-drug-safety-communication-fda-approves-label-changes-include-intestinal-problems-sprue. 2013. Accessed 30 June 2023.

OECD: Using routinely collected data to inform pharmaceutical policies: analytical report for OECD and EU countries. https://www.oecd.org/health/health-systems/Using-Routinely-Collected-Data-to-Inform-Pharmaceutical-Policies-Analytical-Report-2019.pdf. 2019. Accessed 30 June 2023.

Humbert X, Rabiaza A, Schonbrodt L, Raginel T, Le Bas F. Impact du déremboursement de l’olmésartan chez les patients hypertendus en soins premiers. Ann Cardiol Angeiol. 2020;69(3):139–43. https://doi.org/10.1016/j.ancard.2020.03.018.

Article  CAS  Google Scholar 

Agence nationale de sécurité du médicament et des produits de santé: L'ANS rappelle le risque d'entéropathies graves chez certains patients traités par l'amésartan - Point d'information http://www.pharmacovigilance-tours.fr/tl_files/Documents/PJ%20des%20alertes/PJ%20olmesartan%2001.03.14.pdf. 2014. Accessed 10 Oct 2023.

Haute Autorité de santé: Commission de la transparence avis: olmésartan. https://www.has-sante.fr/upload/docs/evamed/CT-14788_OLMETEC_PIC_RECOURS_R163-13_Avis3_CT14788.pdf. 2015). Accessed 30 June 2023.

Haute Autorité de santé: Déremboursement des spécialités à base d’olmésartan dans le traitement de l’hypertension artérielle. https://www.has-sante.fr/jcms/c_2621700/fr/deremboursement-des-specialites-a-base-d-olmesartan-dans-le-traitement-de-l-hypertension-arterielle. 2016. Accessed 10 Oct 2023.

Chien RN, Lu SN, Pwu RF, Wu GH, Yang WW, Liu CL. Taiwan accelerates its efforts to eliminate hepatitis C. Glob Health Med. 2021;3(5):293–300. https://doi.org/10.35772/ghm.2021.01064.

Article  PubMed  PubMed Central  Google Scholar 

Wu GH, Yang WW, Liu CL, Pwu RF, Chien RN, Lee PC, et al. The epidemiological profile of chronic hepatitis C with advanced hepatic fibrosis regarding virus genotype in Taiwan: a nationwide study. J Formos Med Assoc. 2021;120(7):1444–51. https://doi.org/10.1016/j.jfma.2021.01.005.

Article  PubMed  CAS  Google Scholar 

Meeting minutes for the Clinical Medicine Team of the National Hepatitis C Program. In: National Hepatitis C Program Office of Ministry of Health and Welfare, editor. March, 2017.

Institute of Medicine. The future of drug safety: promoting and protecting the health of the public. Washington, DC: The National Academies Press; 2007.

Google Scholar 

Moseley J, Vamvakas S, Berntgen M, Cave A, Kurz X, Arlett P, et al. Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines. Br J Clin Pharmacol. 2020;86(6):1034–51. https://doi.org/10.1111/bcp.14279.

Article  PubMed  PubMed Central  Google Scholar 

Thokala P, Srivastava T, Smith R, Ren S, Whittington MD, Elvidge J, et al. Living health technology assessment: issues, challenges and opportunities. Pharmacoeconomics. 2023;41(3):227–37. https://doi.org/10.1007/s40273-022-01229-4.

Article  PubMed  PubMed Central  Google Scholar 

Pulini AA, Caetano GM, Clautiaux H, Vergeron L, Pitts PJ, Katz G. Impact of real-world data on market authorization, reimbursement decision & price negotiation. Ther Innov Regul Sci. 2021;55(1):228–38. https://doi.org/10.1007/s43441-020-00208-1.

Article  PubMed  Google Scholar 

Katkade VB, Sanders KN, Zou KH. Real world data: an opportunity to supplement existing evidence for the use of long-established medicines in healt