Upgrading the SACT dataset and EBMT registry to enable outcomes-based reimbursement in oncology in England: a gap analysis and top-level cost estimate

References

• Cell and Gene Therapy Catapult. The cell and gene therapy catapult UK clinical trials database. 2019 [cited 2019 Apr 10]. Available from: https://ct.catapult.org.uk/resources/cell-and-gene-therapy-catapult-uk-clinical-trials-database
   Google Scholar
• European Medicines Agency (EMA). Kymriah (tisagenlecleucel) EMA/595919/2018. 2018 [cited 2019 Feb 22]. Available from: https://www.ema.europa.eu/documents/overview/kymriah-epar-medicine-overview_en.pdf
   Google Scholar
• European medicines Agency (EMA). Yescarta (axicabtagene ciloleucel) EMA/495475/2018. 2018 [cited 2019 Feb 22]. Available from: https://www.ema.europa.eu/documents/overview/yescarta-epar-medicine-overview_en.pdf
   Google Scholar
• England NHS. NHS England announces groundbreaking new personalised therapy for children with cancer. 2018 [cited 2018 Sep 24]. Available from: https://www.england.nhs.uk/2018/09/nhs-england-announces-groundbreaking-new-personalised-therapy-for-children-with-cancer/
   Google Scholar
• England NHS. NHS England strikes deal for ground breaking cancer treatment in a new European first. 2018 [cited 2019 Feb 22]. Available from: https://www.england.nhs.uk/2018/10/nhs-england-strikes-deal-for-ground-breaking-cancer-treatment-in-a-new-european-first/
   Google Scholar
• Staines R NHS to reimburse gilead’s CAR-T in adults. 2018 [cited 2019 Jun 4]. Available from: https://pharmaphorum.com/news/nhs-to-reimburse-gileads-car-t-in-adults/
   Google Scholar
• Marsden G, Towse A, Pearson SD, et al. Gene therapy: understanding the science, assessing the evidence, and paying for value. 2017 [cited 2019 Jun 4]. Available from: https://www.ohe.org/publications/gene-therapy-understanding-science-assessing-evidence-and-paying-value
   Google Scholar
• Jørgensen J, Kefalas P. Annuity payments can increase patient access to innovative cell and gene therapies under England’s net budget impact test. J Mark Access Health Policy. 2017;5(1):1355203.
   PubMedGoogle Scholar
• Garrison LP, Towse A, Briggs A, et al. Performance-based risk-sharing arrangements-good practices for design, implementation, and evaluation: report of the ISPOR good practices for performance-based risk-sharing arrangements task force. Value Health. 2013;16:703–14.
   PubMed Web of Science ®Google Scholar
• Adamski, J, Godman, B, Ofierska-Sujkowska, G, et al. Risk sharing arrangements for pharmaceuticals: potential considerations and recommendations for European payers. BMC Health Serv Res. 2010;10:153.
   PubMed Web of Science ®Google Scholar
• Carlson JJ, Gries KS, Yeung K, et al. Current status and trends in performance-based risk-sharing arrangements between healthcare payers and medical product manufacturers. Appl Health Econ Health Policy. 2014;12(3):231–238.
   Google Scholar
• Ferrario A, Kanavos P Managed entry agreements for pharmaceuticals: the European experience. 2013 [cited 2019 Jun 4]. Available from: http://eprints.lse.ac.uk/50513/1/__Libfile_repository_Content_Ferrario%2C%20A_Ferrario_Managed_%20entry_%20agreements_2013_Ferrario_Managed_%20entry_%20agreements_2013.pdf
   Google Scholar
• Morel T, Arickx F, Befrits G, et al. Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries. Orphanet J Rare Dis. 2013;8(1):198.
   PubMed Web of Science ®Google Scholar
• Cole A, Cubi-Molla P, Pollard J, et al. Making outcome-based payment a reality in the NHS. 2019 [cited 2019 Feb 21]. Available from: https://www.cancerresearchuk.org/sites/default/files/obp_final_report_pdf.pdf
   Google Scholar
• Carlson JJ, Chen S, Garrison LP. Performance-based risk-sharing arrangements: an updated international review. PharmacoEconomics. 2017;35(10):1063–1072.
   PubMed Web of Science ®Google Scholar
• Bouvy JC, Sapede C, Garner S. Managed entry agreements for pharmaceuticals in the context of adaptive pathways in Europe. Front Pharmacol. 2018;9:280.
   PubMed Web of Science ®Google Scholar
• Jørgensen J, Mungapen L, Kefalas P. Data collection infrastructure for patient outcomes in the UK – opportunities and challenges for cell and gene therapies launching. J Mark Access Health Policy. 2019;7(1):1573164.
   PubMedGoogle Scholar
• de Wilde S, Guchelaar H-J, Zandvliet ML, et al. Clinical development of gene- and cell-based therapies: overview of the European landscape. Mol Ther Methods Clin Dev. 2016;3:16073.
   PubMed Web of Science ®Google Scholar
• Hanna, E, Rémuzat, C, Auquier, P, et al. Advanced therapy medicinal products: current and future perspectives. J Mark Access Health Policy. 2016;4. doi:10.3402/jmahp.v3404.31036.
   Google Scholar
• Hanna E, Rémuzat C, Auquier P, et al. Gene therapies development: slow progress and promising prospect. J Mark Access Health Policy. 2017;5(1):1265293.
   PubMedGoogle Scholar
• Systemic Anti-Cancer Therapy (SACT) Dataset. SACT dataset website 2018 [cited 2019 Feb 25]. Available from: www.chemodataset.nhs.uk/home
   Google Scholar
• Systemic Anti-Cancer Therapy (SACT) Dataset. Data completeness report: april 2017 – march 2018. 2018 [cited 2019 Feb 25]. Available from: http://www.chemodataset.nhs.uk/view?rid=268
   Google Scholar
• NHS England. Appraisal and funding of cancer drugs from july 2016 (including the new cancer drugs fund) A new deal for patients, taxpayers and industry. 2016 [cited 2019 Feb 25]. Available from: https://www.england.nhs.uk/wp-content/uploads/2013/04/cdf-sop.pdf
   Google Scholar
• Systemic Anti-Cancer Therapy (SACT) Dataset. Systemic anti-cancer therapy data set: user guide – version 3.0.1 final. 2018 [cited 2019 Mar 13]. Available from: http://www.chemodataset.nhs.uk/view?rid=270
   Google Scholar
• The National Cancer Intelligence Network (NCIN). About the national cancer registration and analysis service. 2018 [cited 2019 Feb 26]. Available from: http://www.ncin.org.uk/about_ncin/
   Google Scholar
• Systemic Anti-Cancer Therapy (SACT) Dataset. Systemic anti-cancer therapy data set: technical guidance − version 3.0. 2018 [cited 2019 Mar 13]. Available from: http://www.chemodataset.nhs.uk/view?rid=271
   Google Scholar
• European Society for Blood and Marrow Transplantation (EBMT). Registry function. 2018 [cited 2019 Mar 11]. Available from: https://www.ebmt.org/sites/default/files/2018-08/EBMTRegistryFunction.pdf
   Google Scholar
• European society for Blood and Marrow Transplantation (EBMT). Annual report 2017. 2018. Available from: https://www.ebmt.org/sites/default/files/2018-03/EBMT%20Annual%20Report%202017_0.pdf.
   Google Scholar
• European Society for Blood and Marrow Transplantation (EBMT). Accreditation definitions. 2018 [cited 2019 Mar 11]. Available from: https://www.ebmt.org/accreditation-definitions
   Google Scholar
• British Society of Blood and Marrow Transplantation (BSBMT). UK transplant centre list. 2019 [cited 2019 Mar 13]. Available from: http://bsbmt.org/uk-transplant-centre-list/
   Google Scholar
• European society for Blood and Marrow Transplantation (EBMT). EBMT membership list - country results - UK. 2019 [cited 2019 Mar 13]. Available from: https://www2.clinicalresearch.nl/members/s-ctry2.asp?pais=GB&paislng=UK
   Google Scholar
• England NHS. Clinical commissioning policy: Haematopoietic Stem Cell Transplantation (HSCT) (all ages): revised. 2018 [cited 2019 Mar 14]. Available from: https://www.england.nhs.uk/wp-content/uploads/2018/07/Haematopoietic-stem-cell-transplantation-All-Ages.pdf
   Google Scholar
• The British Society of Blood and Marrow Transplantation (BSBMT). About the registry. 2018 [cited 2019 Mar 11]. Available from: http://bsbmt.org/about-the-registry/
   Google Scholar
• European society for Blood and Marrow Transplantation (EBMT). Data submission. 2018 [cited 2019 Mar 11]. Available from: https://www.ebmt.org/registry/data-submission
   Google Scholar
• European society for Blood and Marrow Transplantation (EBMT). EMA releases their draft qualification opinion on the cellular therapy module of the EBMT registry for public consultation. 2018 [cited 2019 Mar 11]. Available from: https://www.ebmt.org/ebmt/news/ema-releases-their-draft-qualification-opinion-cellular-therapy-module-ebmt-registry
   Google Scholar
• European medicines Agency (EMA). Patient registries 2018 [cited 2019 Mar 11]. Available from: https://www.ema.europa.eu/en/human-regulatory/post-authorisation/patient-registries
   Google Scholar
• European Medicines Agency (EMA). Report on CAR T-cell therapy registries - workshop 9 february 2018 - patient registries initiative. 2018 [cited 2018 Oct 3]. Available from: https://www.ema.europa.eu/documents/report/report-car-t-cell-therapy-registries-workshop_en.pdf
   Google Scholar
• European Society for Blood and Marrow Transplantation (EBMT). Submitting data to the EBMT. 2018 [cited 2019 Mar 11]. Available from: https://www.ebmt.org/sites/default/files/2018-08/Submitting%20data%20to%20the%20EBMT.pdf
   Google Scholar
• European society for Blood and Marrow Transplantation (EBMT). Cell therapy - MED - A form. 2017 [cited 2019 Mar 13]. Available from: https://www.ebmt.org/sites/default/files/migration_legacy_files/document/25%20Cellular%20Therapy%20MED-A.pdf
   Google Scholar
• European Society for Blood and Marrow Transplantation (EBMT). Important change to MED-A forms for cell therapy. 2019 [cited 2019 Mar 13]. Available from: https://www.ebmt.org/ebmt/news/important-change-med-forms-cell-therapy
   Google Scholar
• European Society for Bone and Marrow Transplantation (EBMT). Cell therapy form manual - a guide to the completion of the EBMT cell therapy med-a form. 2019 [cited 2019 Mar 13]. Available from: https://www.ebmt.org/sites/default/files/2019-03/Cellular%20Therapy%20Manual.pdf
   Google Scholar
• European medicines Agency (EMA). Draft qualification opinion on Cellular therapy module of the European Society For Blood & Marrow Transplantation (EBMT) registry. 2018 [cited 2019 Mar 11]. Available from: https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/draft-qualification-opinion-cellular-therapy-module-european-society-blood-marrow-transplantation_en.pdf
   Google Scholar
• European society for Blood and Marrow Transplantation (EBMT). Cell therapy registry and pharma collaboration. 2018 [cited 2019 Mar 11]. Available from: https://www.ebmt.org/ebmt/news/cell-therapy-registry-and-pharma-collaboration
   Google Scholar
• Employers NHS. NHS terms and conditions (AfC) pay scales - annual. 2019 [cited 2019 Jun 7]. Available from: https://www.nhsemployers.org/pay-pensions-and-reward/agenda-for-change/pay-scales/annual
   Google Scholar
• European society for Blood and Marrow Transplantation (EBMT). Cell therapy - MED - A - REGISTRATION - DAY 0. 2019 cited 2019 Mar 14; Available from: https://www.ebmt.org/sites/default/files/2019-03/25_1_Cellular%20Therapy%20MED-A%20Day%200.pdf
   Google Scholar
• European Society for Blood and Marrow Transplantation (EBMT). Cell therapy - MED - A - SECOND REPORT – 100 DAYS AFTER CELL THERAPY. 2019 [cited 2019 Mar 14]. Available from: https://www.ebmt.org/sites/default/files/2019-03/25_2_Cellular%20Therapy%20MED-A%20Day%20100.pdf
   Google Scholar
• European Society for Blood and Marrow Transplantation (EBMT). Cell therapy - MED - A - THIRD AND FOURTH REPORTS - 6 MONTH and ANNUAL FOLLOW UP. 2019 [cited 2019 Mar 14]. Available from: https://www.ebmt.org/sites/default/files/2019-03/25_3_Cellular%20Therapy%20MED-A%206M%20and%20Annual%20Follow%20Up.pdf
   Google Scholar
• Agenzia Italiana del Farmaco (AIFA). Delibera del Consiglio di Amministrazione dell’AIFA n. 37 del 14 ottobre 2014 (11/09/2015). 2014 [cited 2019 Apr 5]. Available from: http://www.aifa.gov.it/sites/default/files/delibera_CDA_AIFA_14102014.pdf
   Google Scholar
• Meyer F, Brühl H, Ormstad SS Session 3: taking a lifecycle approach to EUnetHTA´s work: current and future collaborations between early dialogue and relative effectiveness assessments. 2018 [cited 2019 Apr 9] Available from: https://www.eunethta.eu/wp-content/uploads/2018/05/Session-3-Merged-25-May.pptx
   Google Scholar
• European Network for Health Technology Assessment (EUnetHTA). JA3 work package 5 – lifecycle approach to improve evidence generation. 2018 [cited 2019 Apr 9]. Available from: https://www.eunethta.eu/ja3-archive/work-package-5-life-cycle-approach-to-improve-evidence-generation/
   Google Scholar
• Gimenez E, Espallargues M, Rodrigues J, et al. PHP329 - THE REGISTRY EVALUATION AND QUALITY STANDARDS TOOL (REQUEST) FOR HEALTH TECHNOLOGY ASSESSMENT FROM AN OUTCOMES ASSESSMENT PERSPECTIVE. Value Health. 2018;21:S206.
   Google Scholar
• EY. The missing piece - how EY’s health outcomes platform can create “triple wins” through outcomes-based contracting. 2018 [cited 2019 Apr 11]. Available from: https://assets.ey.com/content/dam/ey-sites/ey-com/en_gl/topics/life-sciences/life-sciences-pdfs/ey-health-outcomes-platform-the-missing-piece.pdf
   Google Scholar