Annuity payments can increase patient access to innovative cell and gene therapies under England’s net budget impact test

Journal of Market Access and Health Policy

Volumn 5, Issue 1, 2017, Pages

  Jørgensen, Jesper ^a   Kefalas, Panos ^a  

^a GUY S HOSPITAL   (United Kingdom)

Author keywords

advanced therapy medicinal product (ATMP); Budget impact; cell therapy; gene therapy; high cost; managed entry agreement; patient access; pricing and reimbursement; risk sharing

Indexed keywords

EID: 85056155404     PISSN: None     EISSN: 20016689     Source Type: Journal    
DOI: 10.1080/20016689.2017.1355203     Document Type: Article

References (28)

1. European Commission. 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. 2007. [cited 2017 Jul 4]. Available from: http://ec.europa.eu/health//sites/health/files/files/eudralex/vol-1/reg_2007_1394/reg_2007_1394_en.pdf
2. House of Lords - Science and Technology Committee. First Report - Regenerative medicine. 2013. [cited 2017 Apr 26]. Available from: https://www.publications.parliament.uk/pa/ld201314/ldselect/ldsctech/23/2305.htm
3. House of Commons - Science and Technology Committee. Regenerative medicine - Fifteenth Report of Session 2016–17. 2017. [cited 2017 Mar 7]. Available from: https://www.publications.parliament.uk/pa/cm201617/cmselect/cmsctech/275/275.pdf
4. National Institute for Health and Care Excellence (NICE). Technology Appraisal and Highly Specialised Technologies Programmes - Procedure for varying the funding requirement to take account of net budget impact. 2017. [cited 2017 Apr 3]. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-technology-appraisals/TA-HST-procedure-varying-the-funding-direction.pdf
5. Abou-El-Enein M, Elsanhoury A, Reinke P. Overcoming challenges facing advanced therapies in the EU market. Cell Stem Cell. 2016;19(3):1–10.
6. 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
7. 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
8. Touchot N, Flume M. Early insights from commercialization of gene therapies in Europe. Genes. 2017;8(2):78.
9. 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
10. Agenzia Italiana del Farmaco (AIFA). Pubblicazione schede di monitoraggio Registro HOLOCLAR (01/03/2017). 2017. Available from: http://www.aifa.gov.it/content/pubblicazione-schede-di-monitoraggio-registro-holoclar-01032017
11. Molmed. The European Commission grants a Conditional Marketing Authorisation to Zalmoxis®, the first immunogene therapy for the treatment of adult patients with high-risk haematological malignancies. 2017. [cited 2017 Apr 25]. Available from: http://www.molmed.com/sites/default/files/uploads/press-releases/2907/2907_1471829992.pdf
12. The National Institute for Health and Care Excellence (NICE). Guide to the methods of technology appraisal 2013 [PMG9]. 2013. [cited 2015 Nov 12]. Available from: https://www.nice.org.uk/process/pmg9/resources/guide-to-the-methods-of-technology-appraisal-2013-pdf-2007975843781
13. National Institute for Health and Care Excellence (NICE). NICE gets go-ahead to fast-track more drug approvals. 2017. [cited 2017 Apr 3]. Available from: https://www.nice.org.uk/news/article/nice-gets-go-ahead-to-fast-track-more-drug-approvals
14. National institute for Health and Care Excellence (NICE). Interim Process and Methods of the Highly Specialised Technologies Programme Updated to reflect 2017 changes - Process for the evaluation of highly specialised technologies. 2017. [cited 2017 May 9]. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-highly-specialised-technologies-guidance/HST-interim-methods-process-guide-may-17.pdf
15. York Health Economics Consortium (YHEC). Cost-utility analysis. 2016. [cited 2017 Mar 31]. Available from: http://www.yhec.co.uk/glossary/cost-utility-analysis/
16. York Health Economics Consortium (YHEC). Budget impact analysis. 2016. [cited 2017 Mar 31]. Available from: http://www.yhec.co.uk/glossary/budget-impact-analysis/
17. Health Economics Resource Center (HERC). Budget impact analysis. [cited 2017 Mar 31]. Available from: https://www.herc.research.va.gov/include/page.asp?id=budget-impact-analysis
18. National institute for Health and Care Excellence (NICE). Assessing resource impact process manual: technology appraisals and highly specialised technologies. 2017. [cited 2017 Apr 3]. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/Into-practice/assessing-resource-impact-process-manual-ta-hst.pdf
19. Claxton K, Martin S, Soares M, et al. Methods for the estimation of the National Institute for Health and Care Excellence cost-effectiveness threshold. Health Technol Assess. 2015;19(14):1–503, v-vi.
20. 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
21. Klemp M, Frønsdal KB, Facey K. What principles should govern the use of managed entry agreements? Int J Technol Assess Health Care. 2011;27(1):77–83.
22. 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
23. Department of Health and the Association of the British Pharmaceutical Industry. The pharmaceutical price regulation scheme 2014. 2013. [cited 2017 Apr 13]. Available from: https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/282523/Pharmaceutical_Price_Regulation.pdf
24. European Medicines Agency (EMA). Guideline on follow-up of patients administered with gene therapy medicinal products. 2009. [cited 2017 Apr 13]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/11/WC500013424.pdf
25. National Institute for Health and Care Excellence (NICE). Exploring the assessment and appraisal of regenerative medicines and cell therapy products. 2016. [cited 2017 Jul 3]. Available from: https://www.nice.org.uk/media/default/about/what-we-do/science%20policy%20and%20research/regenerative-medicine-study-march-2016.pdf
26. Culyer AJ. The dictionary of health economics. Cheltenham (UK); Northampton (MA): Edward Elgar; 2005.
27. National Institute for Health and Care Excellence (NICE) and NHS England. Proposals for changes to the arrangements for evaluating and funding drugs and other health technologies appraised through NICE’s technology appraisal and highly specialised technologies programmes. 2016. [cited 2017 Mar 31]. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/our-programmes/technology-appraisals/NICE_NHSE_TA_and_HST_consultation_document.PDF
28. National institute for Health and Care Excellence (NICE). NICE and NHS England consultation on changes to the arrangements for evaluating and funding drugs and other health technologies assessed through NICE’s technology appraisal and highly specialised technologies programmes (Reference: 17/029). 2017. [cited 2017 Mar 31]. Available from: https://www.nice.org.uk/Media/Default/Get-involved/Meetings-In-Public/Public-board-meetings/agenda-and-papers-mar-17.pdf