References
- Marsden G, Towse A, Pearson SD, et al. Gene therapy: understanding the science, assessing the evidence, and paying for value. 2017 [cited 2017 Apr 4]. Available from: https://www.ohe.org/publications/gene-therapy-understanding-science-assessing-evidence-and-paying-value
- Abou-El-Enein M, Grainger DW, Kili S. Registry contributions to strengthen cell and gene therapeutic evidence. Mol Ther. 2018;26(5):1172–13.
- The EuropeanParliament and the Council of the European Union. Regulation (EC) No 1394/2007 of the European parliament and of the council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004. Brussels, Belgium: Official Journal of the European Union; 2007.
- European Medicines Agency (EMA). Guideline on safety and efficacy follow-up and risk management of Advanced Therapy Medicinal Products – Draft. 2018 [cited 2018 Sep 17]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2018/02/WC500242959.pdf
- European Medicines Agency (EMA). Assessment report - YESCARTA (EMA/481168/2018). 2018 [cited 2018 Sep 17]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/004480/WC500254960.pdf
- Sabine Grimm MS, Brennan A, Wailoo A. framework for analysing risk in health technology assessments and its application to managed entry agreements. 2016. Available from: http://scharr.dept.shef.ac.uk/nicedsu/wp-content/uploads/sites/7/2016/03/DSU-Managed-Access-report-FINAL.pdf
- Ferrario A, Kanavos P. Managed entry agreements for pharmaceuticals: the European experience. 2013 [cited 2018 Sep 26]. 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
- Garrison, L.P., 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;16:703–719.
- 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.
- Carlson JJ, Chen S, Garrison LP. Performance-based risk-sharing arrangements: an updated international review. PharmacoEconomics. 2017;35(10):1063–1072.
- Carlson, J.J., Gries, K.S., 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.
- Klemp M., Fronsdal KB., Facey K. What principles should govern the use of managed entry agreements? Int J Technol Assess Health Care. 2011;27(1):77–83.
- 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.
- Bouvy JC, Sapede C, Garner S. Managed entry agreements for pharmaceuticals in the context of adaptive pathways in Europe. Front Pharmacol. 2018;9:280.
- Estonian Health Insurance Fund. Overview of Estonian experience on inclusion of medicines in benefits package and pricing. 2017 [cited 2019 Jan 8]. Available from: https://www.haigekassa.ee/sites/default/files/pressile/20170421_washingtondc_estonia.pdf
- Montilla, S., Xoxi, E., Russo, P., et al. Monitoring registries at italian medicines agency: fostering access, guaranteeing sustainability. Int J Technol Assess Health Care. 2015;31(4):210–213.
- Hettle, R., Corbett, M., Hinde, S., et al. The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal. Health Technol Assess. 2017;21(7):1–204.
- Kefalas, P., Jørgensen, J., Merryfield, N., et al. Establishing the cost of implementing a performance-based, managed entry agreement for a hypothetical CAR T-cell therapy. J Mark Access Health Policy. 2018;6(1):1511679.
- National Cancer Registry and Analysis Service (NCRAS) at Public Health England. Systemic Anti-Cancer Therapy (SACT) dataset. 2018. Available from: http://www.chemodataset.nhs.uk/
- Alliance for Regenerative Medicine (ARM). Quarterly data reports. 2018 [cited 2018 Sep 21]. Available from: http://alliancerm.wpengine.com/publications-presentations/
- EU Clinical Trials Register. 2018 [cited 2018 Sep 21]. Available from: https://www.clinicaltrialsregister.eu
- Phacilitate & Biotech and Money. Advanced therapies investment report 2017. 2017 [cited 2018 Sep 6]. Available from: https://www.phacilitate.co.uk/sites/default/files/clarion_phacilitate/pdfs/advanced_therapies_investment_report_phacilitate.pdf
- The UKMI Horizon Scanning and Medicines Evaluation (HSME) Service. New medicines. 2018 [cited 2018 Sep 21]. Available from: https://www.sps.nhs.uk/category/new-medicines/
- National Institute for Health Research (NIHR). Innovation observatory. 2018 [cited 2018 Sep 21]. Available from: http://www.io.nihr.ac.uk/
- de Wilde, S., Guchelaar, H.J., Zandvliet, M.L., et al. Clinical development of gene- and cell-based therapies: overview of the European landscape. Mol Ther Methods Clin Dev. 2016;3:16073.
- 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.
- Hanna, E. Gene therapies development: slow progress and promising prospect. J Mark Access Health Policy. 2017;5(1):1265293.
- Towse A, Cole A, Zamora B The debate on indication-based pricing in the U.S. and five major european countries. 2018 [cited 2018 Sep 26]. Available from: https://www.ohe.org/publications/debate-indication-based-pricing-us-and-five-major-european-countries
- Euroean Society for Bone and Marrow Transplantation (EBMT). Cell therapy registry and pharma collaboration. 2018 [cited 2018 Oct 3]. Available from: https://www.ebmt.org/ebmt/news/cell-therapy-registry-and-pharma-collaboration
- European Medicines Agency (EMA). Summary of opinion (initial authorisation) - Luxturna (voretigene neparvovec). 2018 [cited 2018 Oct 2]. Available from: https://www.ema.europa.eu/documents/smop-initial/chmp-summary-positive-opinion-luxturna_en.pdf
- European Medicines Agency (EMA). Adaptive pathways. 2018 [cited 2018 Sep 26]. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000601.jsp&mid=WC0b01ac05807d58ce
- European Medicines Agency (EMA). PRIME: priority medicines. 2018 [cited 2018 Sep 26]. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000660.jsp&mid=WC0b01ac05809f8439
- McConaghie A England’s ‘world-first’ cancer database – can it deliver on its promise? 2016 [cited 2018 Sep 24]. Available from: https://pharmaphorum.com/views-and-analysis/englands-world-first-cancer-database-can-deliver-promise/
- 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
- Jönsson, B., Hampson, G., Michaels, J., et al. Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. Eur J Health Econ. 2018. DOI:10.1007/s10198-018-1007-x. Epub ahead of print.
- Garattini L, Curto A, van de Vooren K. Italian risk-sharing agreements on drugs: are they worthwhile? Eur J Health Econ. 2015;16(1):1–3.
- European Medicines Agency (EMA). Patient registries workshop, 28 October 2016 - observations and recommendations arising from the workshop. 2017 [cited 2018 Sep 12]. Available from: https://www.ema.europa.eu/documents/report/report-patient-registries-workshop_en.pdf
- Abou-El-Enein M, Elsanhoury A, Reinke P. Overcoming challenges facing advanced therapies in the EU market. Cell Stem Cell. 2016;19(3):293–297.
- European Biotechnology Life Science and Industry Magazine. Uniqure withdraws €1m drug Glybera from market. 2017 [cited 2017 Apr 28]. Available from: http://european-biotechnology.com/up-to-date/latest-news/news/uniqure-withdraws-eur1m-drug-glybera-from-market.html
- National Institute for Health and Care Excellence (NICE). Talimogene laherparepvec for treating unresectable metastatic melanoma - technology appraisal guidance [TA410]. 2016 [cited 2017 Apr 25]. Available from: https://www.nice.org.uk/guidance/ta410/chapter/1-Recommendations
- Touchot N, Flume M. Early insights from commercialization of gene therapies in Europe. Genes (Basel). 2017;8(2):78.
- National Institute for Health and Care Excellence (NICE). Talimogene laherparepvec for treating unresectable metastatic melanoma - technology appraisal guidance. 2016 [cited 2018 Sep 24]. Available from: https://www.nice.org.uk/guidance/ta410/resources/talimogene-laherparepvec-for-treating-unresectable-metastatic-melanoma-pdf-82604596507333
- Haute Autorité de Santé (HAS). Commission de la transparence Avis 20 juillet 2016 - cellules épithéliales cornéennes humaines autologues amplifiées ex vivo contenant des cellules souches. 2016. Available from: http://www.has-sante.fr/portail/upload/docs/evamed/CT-15190_HOLOCLAR_PIC_INS_Avis2_CT15190.pdf
- Agenzia Italiana del Farmaco (AIFA). Pubblicazione schede di monitoraggio Registro HOLOCLAR. [cited 2017 Mar 1]. 2017 Available from: http://www.aifa.gov.it/content/pubblicazione-schede-di-monitoraggio-registro-holoclar-01032017
- Agencia Española de Medicamentos y Productos Sanitarios (AEMPS). Informe de Posicionamiento Terapéutico de células epiteliales corneales humanas autólogas, expandidas ex vivo, entre las que se encuentran células madre (Holoclar®) en el tratamiento de pacientes adultos con deficiencia de células madre limbares debida a quemaduras oculares por agentes físicos o químicos. 2016 [cited 2018 Sep 24]. Available from: https://www.aemps.gob.es/medicamentosUsoHumano/informesPublicos/docs/IPT-celulas-epiteliales-Holoclar-deficiencia-celulas-limbares.pdf
- National Institute for Health and Care Excellence (NICE). Holoclar for treating limbal stem cell deficiency after eye burns - technology appraisal guidance. 2017 [cited 2018 Sep 24]. Available from: https://www.nice.org.uk/guidance/ta467/resources/holoclar-for-treating-limbal-stem-cell-deficiency-after-eye-burns-pdf-82604910595525
- National Institute for Health and Care Excellence (NICE). Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency - highly specialised technologies guidance. 2018 [cited 2018 Sep 24]. Available from: https://www.nice.org.uk/guidance/hst7/resources/strimvelis-for-treating-adenosine-deaminase-deficiencysevere-combined-immunodeficiency-pdf-1394905926085
- Molmed. MolMed: Board of Directors approved Full Year 2017 Results. 2018 [cited 2018 Sep 17]. Available from: https://www.molmed.com/sites/default/files/uploads/press-releases/3265/3265_1520621626.pdf
- CO.DON AG. CO.DON AG: spherox - largest product launch in the company’s history. 2017 [cited 2018 Sep 26]. Available from: http://www.codon.de/news-overview/press-release/read-news/article/codon_ag_spherox_groesster_produktlaunch_der_unternehmensgeschichte.html?L=1&cHash=57889048b5ad69a67bd9b22280a7cddb
- National Institute for Health and Care Excellence (NICE). Autologous chondrocyte implantation using chondrosphere for treating symptomatic articular cartilage defects of the knee. 2018 [cited 2018 Sep 26]. Available from: https://www.nice.org.uk/guidance/ta508
- NHS England. NHS England strikes deal for ground breaking cancer treatment in a new European first. 2018 [cited 2018 Oct 12]. Available from: https://www.england.nhs.uk/2018/10/nhs-england-strikes-deal-for-ground-breaking-cancer-treatment-in-a-new-european-first/
- NHS England. 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/
- Pena A NICE rejects kymriah for England’s NHS to treat aggressive DLBCL. 2018 [cited 2018 Oct 12]. Available from: https://lymphomanewstoday.com/2018/09/25/nice-rejects-kymriah-treating-aggressive-dlbcl-england-national-health-service/