Hostname: page-component-cd9895bd7-mkpzs Total loading time: 0 Render date: 2024-12-25T19:31:00.910Z Has data issue: false hasContentIssue false

International experience with performance-based risk-sharing arrangements: implications for the Chinese innovative pharmaceutical market

Published online by Cambridge University Press:  23 September 2020

Weiwei Xu
Affiliation:
GongJing Healthcare Ltd., Nanjing, China
Jing Wu
Affiliation:
School of Pharmaceutical Science and Technology, Tianjin University, Tianjin, China

Abstract

Objectives

Various forms of outcomes-based or risk-sharing agreements have been implemented since early 2000s as a way of access to innovative medicinal products. This study aims to summarize the international experience of performance-based risk-sharing arrangements (PBRSAs) and identify the preconditions for a successful implementation of such schemes. Their implications for the Chinese healthcare market are discussed.

Methods

A systematic literature review (in PubMed) was conducted to review the evidence on the nature and performance of PBRSAs in the past 10 years. Grey literature was searched for reports in government websites of the countries in scope.

Results

The search identifies 463 records from PubMed and 3 additional records from other sources. Thirty-one publications are included in the final review. The following preconditions were identified to support a successful implementation of PBRSAs: (1) Identify meaningful and feasible outcome measurements; (2) Establish an effective and efficient data collection infrastructure; (3) Control of the implementation costs; (4) Develop governance and administrative infrastructure to allow delisting and rebate/refund; (5) Clarify personal data protection issues.

Conclusions

The implementation of PBRSAs has proven to be challenging. Although the Chinese healthcare system is not yet well equipped to implement such schemes, some recent changes may pave the way to successful PBRSAs for particular innovative products.

Type
Assessment
Copyright
Copyright © The Author(s), 2020. Published by Cambridge University Press

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

OECD. Improving forecasting of pharmaceutical spending-insights from 23 OECD and EU countries analytical report [Internet]. 2019 [cited 2019 Jun 3]. Available from: https://ec.europa.eu/health/sites/health/files/policies/docs/pharmaceutical-expenditure-analytical-report-april-2019_en.pdfGoogle Scholar
Grimm, S, Strong, M, Brennan, A, Waillo, A. Framework for analysing risk in health technology assessments and its application to managed entry agreements [Internet]. 2016 [cited 2019 May 20]. Available from: http://nicedsu.org.uk/wp-content/uploads/2018/05/DSU-Managed-Access-report-FINAL.pdfGoogle Scholar
Campillo-Artero, C, del Llano, J, Poveda, JL. Risk sharing agreements: With orphan drugs? Farm Hosp. 2012;36:455–63.Google ScholarPubMed
Espín, J, Rovira, J, García, L. Experiences and impact of European risk-sharing schemes focusing on oncology medicines [Internet]. 2011 [cited 2019 May 20]. Available from: http://ec.europa.eu/DocsRoom/documents/7608/attachments/1/translations/en/renditions/pdfGoogle Scholar
Nazareth, T, Ko, J, Sasane, R, Frois, C, Carpenter, S, Demean, S. Outcomes-based contracting experience: Research findings from U.S. and European stakeholders. J Manag Care Spec Pharm. 2017;23:1018–26.Google ScholarPubMed
Driscoll, D, Farnia, S, Kefalas, P, Maziarz, R. Concise review: The high cost of high tech medicine: Planning ahead for market access. Stem Cells Transl Med. 2017;6:1723–9.CrossRefGoogle ScholarPubMed
European Medicines Agency. Final report on the adaptive pathways pilot. London; 2016.Google Scholar
amp health Europe. Bluebird's Zynteglo becomes world's second most expensive therapy at EUR 1,575 million [Internet]. 2019 [cited 2020 May 18]. Available from: https://www.apmhealtheurope.com/freestory/0/64348/bluebird-s-zynteglo-becomes-world-s-second-most-expensive-therapy-at-e1.575-millionGoogle Scholar
Hutton, J, Trueman, P, Henshall, C. Coverage with evidence development: An examination of conceptual and policy issues. Int J Technol Assess Health Care. 2007;23:425–32.CrossRefGoogle ScholarPubMed
Garber, AM, McClellan, MB. Satisfaction guaranteed – “Payment by Results” for biologic agents. N Engl J Med. 2007;357:1575–7.10.1056/NEJMp078204CrossRefGoogle ScholarPubMed
Cook, JP, Vernon, JA, Manning, R. Pharmaceutical risk-sharing agreements. Pharmacoeconomics. 2008;26:551–6.CrossRefGoogle ScholarPubMed
McCabe C, J, Stafinski, T, Edlin, R, Menon, D. Access with evidence development schemes. Pharmacoeconomics. 2010;28:143–52.CrossRefGoogle ScholarPubMed
Towse, A, Garrison, LP. Can't get no satisfaction? Will pay for performance help? Pharmacoeconomics. 2010;28:93102.CrossRefGoogle ScholarPubMed
Garrison, L, Towse, A, Briggs, A, Pouvourville, G. 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 Heal. 2013;16:703–19.CrossRefGoogle ScholarPubMed
Kefalas, P, Ali, O, Jørgensen, J, Merryfield, N, Richardson, T, Meads, A. Establishing the cost of implementing a performance-based, managed entry agreement for a hypothetical CAR T-cell therapy. J Mark Access Heal Policy. 2018;6:18.Google ScholarPubMed
PRISMA. PRISMA flow diagram [Internet]. 2009 [cited 2019 Apr 10]. Available from: http://prisma-statement.org/PRISMAStatement/FlowDiagram.aspxGoogle Scholar
Gonçalves, F, Santos, S, Silva, C, Sousa, G. Risk-sharing agreements, present and future. Ecancermedicalscience. 2018;12:823.CrossRefGoogle ScholarPubMed
Dentzer, S. Value-based contracting: Creating the terms of engagement around high-cost cancer therapies. Am J Manag Care. 2018;24:SP14950.Google ScholarPubMed
Klemp, M, Frønsdal, KB, Facey, K. What principles should govern the use of managed entry agreements? Int J Technol Assess Health Care. 2011;27:7783.10.1017/S0266462310001297CrossRefGoogle ScholarPubMed
Garrison, LP, Carlson, JJ, Bajaj, PS, Towse, A, Neumann, PJ, Sullivan, SD et al. Private sector risk-sharing agreements in the United States: Trends, barriers, and prospects. Am J Manag Care. 2015;21:632–40.Google ScholarPubMed
Piatkiewicz, T, Traulsen, J, Holm-Larsen, T. Risk-sharing agreements in the EU: A systematic review of major trends. PharmacoEconomics – Open. 2018;2:109–23.CrossRefGoogle ScholarPubMed
Dunlop, W, Staufer, A, Levy, P, Edwards, G. Innovative pharmaceutical pricing agreements in five European markets: A survey of stakeholder attitudes and experience. Health Policy (New York). 2018;122:528–32.10.1016/j.healthpol.2018.02.008CrossRefGoogle ScholarPubMed
Morel, T, Arickx, F, Befrits, G, Silverio, P, Meijden, C, Xoxi, E 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;8:198213.CrossRefGoogle ScholarPubMed
Pauwels, K, Huys, I, Vogler, S, Casteels, M, Simoens, S. Managed entry agreements for oncology drugs: Lessons from the European experience to inform the future. Front Pharmacol. 2017;8:171.10.3389/fphar.2017.00171CrossRefGoogle ScholarPubMed
Agenzia italiana del farmaco (AIFA). Determinazione 24 luglio 2006. Regime di rimborsabilita e prezzo di vendita della specialita medicinale Tarceva (erlotinib). 2006.Google Scholar
Navarria, A, Drago, V, Gozzo, L, Longo, L, Mansueto, S, Pignataro, G et al. Do the current performance-based schemes in Italy really work? “Success fee”: A novel measure for cost-containment of drug expenditure. Value Heal. 2015;18:131–6.10.1016/j.jval.2014.09.007CrossRefGoogle ScholarPubMed
Arai, S, Fanale, M, DeVos, S, Engert, A, Illidge, T, Borchmann, P et al. Defining a Hodgkin lymphoma population for novel therapeutics after relapse from autologous hematopoietic cell transplant. Leuk Lymphoma. 2013;54:2531–3.CrossRefGoogle ScholarPubMed
Garattini, L, Curto, A, van de Vooren, K. Italian risk-sharing agreements on drugs: Are they worthwhile? Eur J Heal Econ. 2015;16:13.CrossRefGoogle ScholarPubMed
Darbà, J, Ascanio, M. The current performance-linked and risk sharing agreement scene in the Spanish region of Catalonia. Expert Rev Pharmacoecon Outcomes Res. 2019;19:734738.CrossRefGoogle ScholarPubMed
Palace, J, Duddy, M, Lawton, M, Bregenzer, T, Zhu, F, Boggild, M et al. Assessing the long-term effectiveness of interferon-beta and glatiramer acetate in multiple sclerosis: Final 10-year results from the UK multiple sclerosis risk-sharing scheme. J Neurol Neurosurg Psychiatry. 2019;90:251–60.10.1136/jnnp-2018-318360CrossRefGoogle ScholarPubMed
van de Vooren, K, Curto, A, Freemantle, N, Garattini, L. Market-access agreements for anti-cancer drugs. J R Soc Med. 2015;108:166–70.10.1177/0141076814559626CrossRefGoogle ScholarPubMed
Barlas, S. Health plans and drug companies dip their toes into value-based pricing. P&T. 2016;41:3943.Google ScholarPubMed
Neumann, PJ, Chambers, JD, Simon, F, Meckley, LM. Risk-sharing arrangements that link payment for drugs to health outcomes are proving hard to implement. Health Aff (Millwood). 2011;30:2329–37.CrossRefGoogle ScholarPubMed
Bouvy, J, Sapede, C, Garner, S. Managed entry agreements for pharmaceuticals in the context of adaptive pathways in Europe. Front Pharmacol. 2018;9:18.10.3389/fphar.2018.00280CrossRefGoogle ScholarPubMed
Brown, JD, Sheer, R, Pasquale, M, Sudharshan, L, Axelsen, K, Subedi, P et al. Payer and pharmaceutical manufacturer considerations for outcomes-based agreements in the United States. Value Heal. 2018;21:3340.CrossRefGoogle ScholarPubMed
Neilson, LM, Swart, ECS, Good, CB, Shrank, WH, Henderson, R, Manolis, C et al. Identifying outcome measures for coronary artery disease value-based contracting using the Delphi method. Cardiol Ther. 2019;8:135–43.CrossRefGoogle ScholarPubMed
Neilson, LM, Swart, ECS, Good, CB, Shrank, WH, Henderson, R, Manolis, C et al. Identifying outcome measures for type 2 diabetes value-based contracting using the Delphi method. J Manag Care Spec Pharm. 2019;25:324–31.Google ScholarPubMed
Blommestein, HM, Franken, MG, Uyl-de Groot, CA. A practical guide for using registry data to inform decisions about the cost effectiveness of new cancer drugs: Lessons learned from the PHAROS registry. Pharmacoeconomics. 2015;33:551–60.10.1007/s40273-015-0260-4CrossRefGoogle ScholarPubMed
Mohseninejad, L, van Gils, C, Uyl-de Groot, CA, Buskens, E, Feenstra, T. Evaluation of patient registries supporting reimbursement decisions: The case of oxaliplatin for treatment of stage III colon cancer. Value Heal. 2015;18:8490.CrossRefGoogle ScholarPubMed
Yu, JS, Chin, L, Oh, J, Farias, J. Performance-based risk-sharing arrangements for pharmaceutical products in the United States: A systematic review. J Manag Care Spec Pharm. 2017;23:1028–40.Google ScholarPubMed
Capozzi, M, De Divitiis, C, Ottaiano, A, Teresa, T, Capuozzo, M, Maiolino, P et al. Funds reimbursement of high-cost drugs in gastrointestinal oncology: An Italian real practice 1 year experience at the National Cancer Institute of Naples. Front Public Heal. 2018;6:291.10.3389/fpubh.2018.00291CrossRefGoogle Scholar
Garattini, L, Curto, A. Performance-based agreements in Italy: “Trendy Outcomes” or mere illusions? Pharmacoeconomics. 2016;34:967–9.CrossRefGoogle ScholarPubMed
Walker, S, Sculpher, M, Claxton, K, Palmer, S. Coverage with evidence development, only in research, risk sharing, or patient access scheme? A framework for coverage decisions. Value Heal. 2012;15:570–9.CrossRefGoogle ScholarPubMed
Garattini, L, Casadei, G. Risk sharing agreements: What lessons from Italy? Int J Technol Assess Health Care. 2011;27:169–72.CrossRefGoogle ScholarPubMed
Fojo, T, Mailankody, S, Lo, A. Unintended consequences of expensive cancer therapeutics—The pursuit of marginal indications and a me-too mentality that stifles innovation and creativity. JAMA Otolaryngol Neck Surg. 2014;140:1225.10.1001/jamaoto.2014.1570CrossRefGoogle Scholar
National Healthcare Security Administration of China. National Healthcare Security Administrative published national pilot technical guideline and grouping plan of DRG [Internet]. 2019 [cited 2019 Nov 22]. Available from: http://www.nhsa.gov.cn/art/2019/10/24/art_14_1874.htmlGoogle Scholar
Hsia, DC, Ahern, CA, Ritchie, BP, Moscoe, LM, Krushat, WM. Medicare reimbursement accuracy under the prospective payment system, 1985 to 1988. JAMA. 1992;268:896–9.CrossRefGoogle ScholarPubMed
Supplementary material: File

Xu and Wu Supplementary Materials

Xu and Wu Supplementary Materials 1

Download Xu and Wu Supplementary Materials(File)
File 19.8 KB
Supplementary material: File

Xu and Wu Supplementary Materials

Xu and Wu Supplementary Materials 2

Download Xu and Wu Supplementary Materials(File)
File 16.8 KB