Early access for innovative oncology medicines: a different story in each nation

References

• Sung H, Ferlay J, Siegel RL, et al. Global Cancer Statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2021;71(3):209–249. doi: 10.3322/caac.21660.
   PubMed Web of Science ®Google Scholar
• Cancer statistics at a glance; 2022. Available from: https://cancer.ca/en/research/cancer-statistics/cancer-statistics-at-a-glance
   Google Scholar
• Hanna TP, King WD, Thibodeau S, et al. Mortality due to cancer treatment delay: systematic review and meta-analysis. BMJ. 2020;371:m4087. doi: 10.1136/bmj.m4087.
   PubMed Web of Science ®Google Scholar
• Luo Q, O’Connell DL, Yu XQ, et al. Cancer incidence and mortality in Australia from 2020 to 2044 and an exploratory analysis of the potential effect of treatment delays during the COVID-19 pandemic: a statistical modelling study. Lancet Public Health. 2022;7(6):e537–e548. doi: 10.1016/S2468-2667(22)00090-1.
   PubMed Web of Science ®Google Scholar
• Wells C, Galvani AP. Impact of the COVID-19 pandemic on cancer incidence and mortality. Lancet Public Health. 2022;7(6):e490–e491. doi: 10.1016/S2468-2667(22)00111-6.
   PubMed Web of Science ®Google Scholar
• The global use of medicines 2022: outlook to 2026; 2022. Available from: iqviainstitute.org
   Google Scholar
• Global oncology trends 2022: outlook to 2026; 2022. Available from: iqviainstitute.org
   Google Scholar
• Hwang TJ, Kesselheim AS, Tibau A, et al. Clinical benefit and expedited approval of cancer drugs in the United States, European Union, Switzerland, Japan, Canada, and Australia. JCO Oncol Pract. 2022;18(9):e1522–e1532. doi: 10.1200/OP.21.00909.
   PubMedGoogle Scholar
• National Institute of Health and Care Excellence (NICE); 2022 [cited 2022 Aug 5]. Available from: https://www.nice.org.uk/
   Google Scholar
• Haute Autorite de Sante (HAS); 2022 [cited 2022 Aug 5]. Available from: https://www.has-sante.fr/jcms/pprd_2986129/en/home
   Google Scholar
• Gemeinsamer Bundesausschuss: G-BA; 2022 [cited 2022 Aug 5]. Available from: https://www.g-ba.de/english/
   Google Scholar
• Early access and off-label use. Italian Medicines Agency; 2022 [cited 2022 Aug 5]. Available from: https://www.aifa.gov.it/en/web/guest/accesso-precoce-uso-off-label
   Google Scholar
• PBAC. Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory Committee (PBAC); 2016 [cited 2022 Aug 5]. Available from: https://pbac.pbs.gov.au/home.html
   Google Scholar
• The Institut national d’excellence en santé et en services sociaux: INESSS; 2022 [cited 2022 Aug 5]. Available from: https://www.inesss.qc.ca/en/about-us/about-the-institut.html
   Google Scholar
• IQWIG. The Independent Institute for Quality and Efficiency in Health Care; 2022 [cited 2022 Aug 5]. Available from: https://www.iqwig.de/en/
   Google Scholar
• CADTH: Canadian Agency for Drugs and Technologies in Health; 2022 [cited 2022 Aug 5]. Available from: https://www.cadth.ca/
   Google Scholar
• 2017–2027 Quebec Life Sciences Strategy: innovation comes to life. Montreal: Numerique; 2017. Available from: https://numerique.banq.qc.ca/patrimoine/details/52327/2977378
   Google Scholar
• Wang SV, Pottegard A, Crown W, et al. HARmonized protocol template to enhance reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: a good practices report of a Joint ISPE/ISPOR Task Force. Pharmacoepidemiol Drug Saf. 2023;32(1):44–55. doi: 10.1002/pds.5507.
   PubMed Web of Science ®Google Scholar
• NICE health technology evaluations: the manual London: National Institute for Health and Care Excellence (NICE); 2022.
   Google Scholar
• Henry AD, Hill SR, Harris A. Drug prices and value for money: the Australian Pharmaceutical Benefits Scheme. JAMA. 2005;294(20):2630–2632. doi: 10.1001/jama.294.20.2630.
   PubMed Web of Science ®Google Scholar
• Public summary document – March 2021 PBAC meeting: venetoclax; 2021.
   Google Scholar
• Increasing access to innovative medicines. Innovative Medicines Canada; 2022 [cited 2023 Jun 23]. Available from: https://innovativemedicines.ca/wp-content/uploads/2022/10/20221007_FINAL_PreBudget_Consultation-1.pdf
   Google Scholar
• Hoskyn S. IMC’s explaining public reimbursement delays for new medicines for Canadian patients. Ottawa: Innovative Medicines Canada; 2020 [cited 2023 Jun 23]. Available from: https://innovativemedicines.ca/wp-content/uploads/2020/07/CADTH-TTL-8.5x11-EN-Final.pdf
   Google Scholar
• Managed access. London: National Institute for Health and Care Excellence (NICE); 2022 [cited 2022 Aug 5]. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/managed-access
   Google Scholar
• Bee C, Hawksworth C, Wakefield L, et al. What goes in must come out: an analysis of NICE recommendations for drugs exiting managed (early) access in England. Value in Health. 2022;25(7):S478.
   Google Scholar
• Real-World Evidence Registry: ISPOR; 2023 [cited 2023 Jun 23]. Available from: https://www.ispor.org/strategic-initiatives/real-world-evidence/real-world-evidence-registry
   Google Scholar
• The ASSESS Project Overview: the ASSESS Project; 2023 [cited 2023 Jun 23]. Available from: https://static1.squarespace.com/static/617739c64fac81568f019217/t/64763ee17f0eea47d6f912fe/1685470957572/The+ASSESS+Project+Overview.pdf
   Google Scholar
• Gazzetta Ufficiale Della Republica Italiana. LEGGE 23 luglio 2021, n. 106. Rome: Republica Italiana; 2021.
   Google Scholar
• Galafold – Maladie de Fabry Avis transmis au ministre en octobre 2018. Institut national d’excellence en santé et en services sociaux (INESSS); 2018 [cited 2023 Mar 28]. Available from: https://www.inesss.qc.ca/fileadmin/doc/INESSS/Inscription_medicaments/Avis_au_ministre/Novembre_2018/Galafold_2018_10.pdf
   Google Scholar
• Authorisation for early access to medicinal products: HAS assessment doctrine. French National Authority for Health; 2021.
   Google Scholar
• Accès précoce des médicaments: accompagnement des laboratoires; 2021.
   Google Scholar
• Beley L, Vooren BV, Bogaert P. New early access and off-label use rules in France Inside EU Life Sciences. Covington; 2021 [updated 2021 Jul 6; cited 2022 Aug 5]. Available from: https://www.insideeulifesciences.com/2021/07/06/new-early-access-and-off-label-use-rules-in-france/
   Google Scholar
• Rodwin M. What can the United States learn from pharmaceutical spending controls in France? The Commonwealth Fund; 2019 [cited 2022 Dec 5]. Available from: https://www.commonwealthfund.org/publications/issue-briefs/2019/nov/what-can-united-states-learn-drug-spending-controls-france
   Google Scholar
• "Compassionate Use" Programmes. Federal institute for drugs and medical devices (BfArM); 2022.
   Google Scholar
• Drug approval and early benefit assessment in Germany: IQWIG; 2022 [cited 2022 Aug 5]. Available from: https://www.iqwig.de/en/presse/in-the-focus/new-drugs-approval-benefit-assessment-coverage/1-drug-approval-and-early-benefit-assessment-in-germany/
   Google Scholar
• AIFA. Early access and off-label use. Italian Medicines Agency; 2022 [cited 2022 Aug 5]. Available from: https://www.aifa.gov.it/en/web/guest/accesso-precoce-uso-off-label
   Google Scholar
• Lawlor R, Wilsdon T, Darquennes E, et al. Accelerating patient access to oncology medicines with multiple indications in Europe. J Mark Access Health Policy. 2021;9(1):1964791. doi: 10.1080/20016689.2021.1964791.
   PubMedGoogle Scholar
• Selletti S, Putignano M, Tiboni F. Pricing & reimbursement laws and regulations 2022. Italy; 2022.
   Google Scholar
• Dickson M, Hurst J, Jacobzone S. Survey of pharmacoeconomic assessment activity in eleven countries. OECD Health Working Papers; 2003.
   Google Scholar
• Fast track approval pathways. Australian Government Department of Health and Aged Care. Therapeutic Goods Administration; 2022 [cited 2022 Aug 5]. Available from: https://www.tga.gov.au/fast-track-approval-pathways
   Google Scholar
• Provisional approval pathway: prescription medicines. Australian Government, Department of Health and Aged Care - Therapeutic Goods Administration; 2018 [cited 2023 Mar 28]. Available from: https://www.tga.gov.au/provisional-approval-pathway-prescription-medicines
   Google Scholar
• Tuffaha HF, Scuffham PA. The Australian Managed Entry Scheme: are we getting it right? Pharmacoeconomics. 2018;36(5):555–565. doi: 10.1007/s40273-018-0633-6.
   PubMed Web of Science ®Google Scholar
• Kubler P. Fast-tracking of new drugs: getting the balance right. Aust Prescr. 2018;41(4):98–99. doi: 10.18773/austprescr.2018.032.
   PubMed Web of Science ®Google Scholar
• Special Access Programme: Drugs: Government of Canada. Ottawa: Health Canada; 2022 [cited 2023 Mar 28]. Available from: https://www.canada.ca/en/health-canada/services/drugs-health-products/special-access/drugs/special-access-programme-drugs.html
   Google Scholar
• Outcomes-based agreements: Canadian experience and perceptions; 2021. Available from: https://static1.squarespace.com/static/61d708f9587415184afa9452/t/62055c8e7508c21a4b4239f0/1645542729885/RWE%2BOBA%2BWorking%2BGroup_Canadian%2BOBA%2BSurvey%2BResults_Oct-12-2021.pdf
   Google Scholar
• Hoskyn S. Explaining public reimbursement delays for new medicines for Canadian patients. Ottawa (ON); 2020.
   Google Scholar
• Sehgal C, Commissiong G, Miller M. Innovative medicines Canada: data analytics and members’ economic footprint and impact in Canada; 2017.
   Google Scholar
• Birch A. Patient assisting programs: LinkedIn; 2022 [cited 2023 Mar 28]. Available from: https://www.linkedin.com/posts/alan-birch-85b6842b_patient-assistance-programspdf-activity-6927594368677638144-LTEV?utm_source=share&utm_medium=member_desktop
   Google Scholar
• BC Cancer. Manufacturer patient assistance programs; 2022. Available from: http://www.bccancer.bc.ca/systemic-therapy-site/Documents/Policy%20and%20Forms/Patient%20assistance%20programs.pdf
   Google Scholar
• Wang SV, Pinheiro S, Hua W, et al. STaRT-RWE: structured template for planning and reporting on the implementation of real world evidence studies. BMJ. 2021;372:m4856. doi: 10.1136/bmj.m4856.
   PubMed Web of Science ®Google Scholar
• Alkhatib NS, Abraham I. The six Delta platform for outcome-based contracting for pharmaceuticals. J Med Econ. 2020;23(11):1209–1214. doi: 10.1080/13696998.2020.1824161.
   PubMed Web of Science ®Google Scholar
• Gotfrit J, Shin JJW, Mallick R, et al. Potential life-years lost: the impact of the cancer drug regulatory and funding process in Canada. Oncologist. 2020;25(1):e130–e137. doi: 10.1634/theoncologist.2019-0314.
   PubMed Web of Science ®Google Scholar
• The Conference Board of Canada. Tomorrow can’t wait; understanding the value of breakthrough cancer treatments for Canadians; 2022 [cited 2022 Oct 31]. Available from: https://www.conferenceboard.ca/focus-areas/health/tomorrow-cant-wait
   Google Scholar
• NHS. Thousands of patients to benefit as NHS marks 100th fast-tracked cancer drug. NHS.UK; 2022 [cited 2022 Sep 18]. Available from: https://www.england.nhs.uk/2022/07/thousands-of-patients-to-benefit-as-nhs-marks-100th-fast-tracked-cancer-drug/
   Google Scholar
• NHS commercial framework for new medicines; 2022 [cited 2022 Aug 5]. Available from: https://www.england.nhs.uk/wp-content/uploads/2021/02/B1688-nhs-commercial-framework-for-new-medicines-june-22.pdf
   Google Scholar
• Gandjour A. Willingness to pay for new medicines: a step towards narrowing the gap between NICE and IQWiG. BMC Health Serv Res. 2020;20(1):343. doi: 10.1186/s12913-020-5050-9.
   PubMed Web of Science ®Google Scholar
• NICE. Technology appraisal data: appraisal recommendations; 2022 [cited 2022 Nov 16]. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data/appraisal-recommendations
   Google Scholar
• Armoiry X, Spath H, Clarke A, et al. Comparison of health technology assessment for new medicines in France and England: an example based on ixazomib for patients with relapsed or refractory multiple myeloma. J Mark Access Health Policy. 2019;7(1):1648971. doi: 10.1080/20016689.2019.1648971.
   PubMedGoogle Scholar
• Government of France. Code de la sécurité sociale-version en vigueur du 07 juin 2018 au 28 août 2020; 2018 [cited 2023 Mar 28]. Available from: https://www.legifrance.gouv.fr/codes/article_lc/LEGIARTI000037008543/2018-06-07
   Google Scholar
• Haute Autorite de Sante (HAS). Décision n°2013.0111/DC/SEESP du 18 septembre 2013 du collège de la Haute Autorité de santé relatif à l’impact significatif sur les dépenses de l’assurance maladie déclenchant l’évaluation médico-économique des produits revendiquant une ASMR ou une ASA de niveaux I, II ou III; 2013 [cited 2023 Mar 28]. Available from: https://www.has-sante.fr/upload/docs/application/pdf/2013-09/c_2013_0111_definition_impact_significatif.pdf
   Google Scholar
• Haute Autorite de Sante (HAS). Décision n°2022.0212/DC/SED/SEM du 23 juin 2022 du collège de la Haute Autorité de santé relative à l’impact significatif sur les dépenses de l’assurance maladie déclenchant l’évaluation médico-économique des produits de santé revendiquant une ASMR ou une ASA de niveaux I, II ou III; 2022 [cited 2023 Mar 28]. Available from: https://www.has-sante.fr/upload/docs/application/pdf/2022-08/decision_n2022.0212_dc_sed_sem_du_23_juin_2022_du_college_de_la_haute_autorite_de_sante_relative_a_limpact_significatif_sur_.pdf
   Google Scholar
• HAS. Decision No. 2018.0233/DC/SEESP of 5 December 2018 of the College of the High Authority for Health amending Decision No. 2013.0111/DC/SEESP of 18 September 2013 on the significant impact on health insurance expenditure triggering the medico-economic evaluation of products claiming ASMR or ASA levels I, II or III; 2018 [cited 2022 Oct 31]. Available from: https://www.has-sante.fr/jcms/c_2896885/fr/decision-n-2018-0233/dc/seesp-du-5-decembre-2018-du-college-de-la-haute-autorite-de-sante-modifiant-la-decision-n-2013-0111/dc/seesp-du-18-septembre-2013-relative-a-l-impact-significatif-sur-les-depenses-de-l-assurance-maladie-declenchant-l-evaluation-medico-economique-des-produits-revendiquant-une-asmr-ou-une-asa-de-niveaux-i-ii-ou-iii
   Google Scholar
• Covington. New German government plans significant changes with impact on the healthcare, life sciences and food sector; 2021 [cited 2022 Sep 19]. Available from: https://www.insideeulifesciences.com/2021/11/29/new-german-government-plans-significant-changes-with-impact-on-the-healthcare-life-sciences-and-food-sector/
   Google Scholar
• Epstein D, Espín J. Evaluation of new medicines in Spain and comparison with other European countries. Gac Sanit. 2020;34(2):133–140. doi: 10.1016/j.gaceta.2019.02.009.
   PubMed Web of Science ®Google Scholar
• AIFA. Italian Medicines Agency. Authorisation of medicinal products; [cited 2022 Aug 5]. Available from: https://www.aifa.gov.it/en/web/guest/autorizzazione-dei-farmaci
   Google Scholar
• AIFA. Economic evaluations; [cited 2022 Oct 31]. Available from: https://www.aifa.gov.it/en/valutazioni-economiche
   Google Scholar
• Pharmaceutical Benefits Advisory Committee (PBAC). Public summary document – November 2021 PBAC meeting: gilteritinib; 2021 [cited 2023 Mar 28]. Available from: https://www.pbs.gov.au/industry/listing/elements/pbac-meetings/psd/2021-11/files/gilteritinib-psd-nov-2021.pdf
   Google Scholar
• Pharmaceutical Benefits Advisory Committee (PBAC). Public summary document – July 2021 PBAC meeting: Venetoclax; 2021 [cited 2023 Mar 28]. Available from: https://www.pbs.gov.au/industry/listing/elements/pbac-meetings/psd/2021-07/files/venetoclax-psd-july-2021.pdf
   Google Scholar
• CADTH. CADTH, Programs and Services; 2022 [cited 2022 Oct 2]. Available from: https://www.cadth.ca/programs-and-services
   Google Scholar
• INAHTA. INESSS – Institit national d’excellence en sante’ et en services sociaux; 2022 [cited 2022 Oct 31]. Available from: https://www.inahta.org/members/inesss/
   Google Scholar
• NHS UK. NHS to offer new drug that halves the risk of cancer returning; 2021 [cited 2022 Sep 18]. Available from: https://www.england.nhs.uk/2021/05/nhs-to-offer-new-drug-that-halves-the-risk-of-cancer-returning/
   Google Scholar
• Glennie J, Villalba E, Wheatley-Price P. Closing the gaps to timely patient access: perspectives on conditional funding models. Curr Oncol. 2022;29(2):981–988. doi: 10.3390/curroncol29020083.
   PubMed Web of Science ®Google Scholar
• National Institute of Health and Care Excellence (NICE). Over 600 people set to benefit from innovative lung cancer treatment within the Cancer Drugs Fund; 2021 [cited 2022 Nov 21]. Available from: https://www.nice.org.uk/news/article/over-600-people-set-to-benefit-from-innovative-lung-cancer-treatment-within-the-cancer-drugs-fund
   Google Scholar
• Deloitte. A collaborative assessment of access to cancer medicines in Australia; 2017 [cited 2022 Sep 18]. Available from: https://www2.deloitte.com/au/en/pages/economics/articles/access-cancer-medicines-australia.html
   Google Scholar
• Kim H, Comey S, Hausler K, et al. A real world example of coverage with evidence development in Australia – ipilimumab for the treatment of metastatic melanoma. J Pharm Policy Pract. 2018;11:4. doi: 10.1186/s40545-018-0131-4.
   PubMed Web of Science ®Google Scholar