Background and Objective
Managed entry agreements (MEAs) consist of a set of instruments to reduce the uncertainty and the budget impact of new high-priced medicines; however, there are concerns. There is a need to critically appraise MEAs with their planned introduction in Brazil. Accordingly, the objective of this article is to identify and appraise key attributes and concerns with MEAs among payers and their advisers, with the findings providing critical considerations for Brazil and other high- and middle-income countries.
An integrative review approach was adopted. This involved a review of MEAs across countries. The review question was ‘What are the health technology MEAs that have been applied around the world?’ This review was supplemented with studies not retrieved in the search known to the senior-level co-authors including key South American markets. It also involved senior-level decision makers and advisers providing guidance on the potential advantages and disadvantages of MEAs and ways forward.
Twenty-five studies were included in the review. Most MEAs included medicines (96.8%), focused on financial arrangements (43%) and included mostly antineoplastic medicines. Most countries kept key information confidential including discounts or had not published such data. Few details were found in the literature regarding South America. Our findings and inputs resulted in both advantages including reimbursement and disadvantages including concerns with data collection for outcome-based schemes.
We are likely to see a growth in MEAs with the continual launch of new high-priced and often complex treatments, coupled with increasing demands on resources. Whilst outcome-based MEAs could be an important tool to improve access to new innovative medicines, there are critical issues to address. Comparing knowledge, experiences, and practices across countries is crucial to guide high- and middle-income countries when designing their future MEAs.
This is a preview of subscription content, access via your institution.
Buy single article
Instant access to the full article PDF.
Price includes VAT (USA)
Tax calculation will be finalised during checkout.
Availability of Data and Material
Further data will be available on request.
Lieven A, Francis A, Olivier B, Kris B, Bogaert M, Stefaan C, et al. A call to make valuable innovative medicines accessible in the European Union. 2010. http://www.reesfrance.com/en/IMG/pdf/PlenaryII-Making-Innovative-Medicine-Accessible-in-the-EU.pdf. Accessed 2 May 2019.
INAMI. Outcomes based pricing and reimbursement of innovative medicines with budgetary limitations: discussion document for the multistakeholders meeting on pharmaceuticals (12th September 2017). https://www.inami.fgov.be/SiteCollectionDocuments/innovative_medicines_with_budgetary_limitations.docx. Accessed 9 Jul 2020.
UN. Sustainable Development Goal 3: ensure healthy lives and promote well-being for all at all ages. 2019. https://sustainabledevelopment.un.org/sdg3. Accessed 9 Jul 2020.
Morton S, Pencheon D, Squires N. Sustainable Development Goals (SDGs), and their implementation: a national global framework for health, development and equity needs a systems approach at every level. Br Med Bull. 2017;124(1):81–90.
WHO. Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research. http://www.euro.who.int/__data/assets/pdf_file/0008/306179/Access-new-medicines-TR-PIO-collaboration-research.pdf?ua=1. Accessed 9 Jul 2020.
Garattini S, Bertele V, Godman B, Haycox A, Wettermark B, Gustafsson LL. Enhancing the rational use of new medicines across European health care systems. Eur J Clin Pharmacol. 2008;64(12):1137–8.
Malmstrom RE, Godman BB, Diogene E, Baumgartel C, Bennie M, Bishop I, et al. Dabigatran: a case history demonstrating the need for comprehensive approaches to optimize the use of new drugs. Front Pharmacol. 2013;4:39.
Godman B, Bucsics A, Vella Bonanno P, Oortwijn W, Rothe CC, Ferrario A, et al. Barriers for access to new medicines: searching for the balance between rising costs and limited budgets. Front Public Health. 2018;6:328.
KCE. How to improve the Belgian process for managed entry agreements? 2017. https://kce.fgov.be/sites/default/files/atoms/files/Download%20the%20synthesis%20in%20English%20%2840%20p.%29.pdf. Accessed 9 Jul 2020.
Lopes G, Vulto A, Wilking N, van Harten W, Meier K, Simoens S. Potential solutions for sustaining the costs of cancer drugs. Eur Oncol Haematol. 2017;13(2):102–7.
OECD. Health at a glance 2017. https://www.oecd-ilibrary.org/docserver/health_glance-2017-en.pdf?expires=1531413926&id=id&accname=guest&checksum=656327F799B10217DD2D80F463DAB8732017. Accessed 9 Jul 2020.
Schwarzer R, Rochau U, Saverno K, Jahn B, Bornschein B, Muehlberger N, et al. Systematic overview of cost-effectiveness thresholds in ten countries across four continents. J Comp Eff Res. 2015;4(5):485–504.
Eder C, Wild C. Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption. J Mark Access Health Policy. 2019;7(1):1600939.
Jorgensen 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.
Baumgart DC, Misery L, Naeyaert S, Taylor PC. Biological therapies in immune-mediated inflammatory diseases: can biosimilars reduce access inequities? Front Pharmacol. 2019;10:279.
Oliveira MA, Luiza VL, Tavares NU, Mengue SS, Arrais PS, Farias MR, et al. Access to medicines for chronic diseases in Brazil: a multidimensional approach. Rev Saude Publica. 2016;50(Suppl. 2):6s.
Richards M, Thorlby R, Fisher R, Turton C. Unfinished business: an assessment of the national approach to improving cancer services in England 1995–2015. https://www.health.org.uk/sites/default/files/upload/publications/2018/Unfinished-business-an-assessment-of-the-national-approach-to-improving-cancer-services-in-england-1995-2015.pdf. Accessed 9 Jul 2020.
Babar ZU, Gammie T, Seyfoddin A, Hasan SS, Curley LE. Patient access to medicines in two countries with similar health systems and differing medicines policies: implications from a comprehensive literature review. Res Social Adm Pharm. 2019;15(3):231–43.
Pink GH, Brown AD, Studer ML, Reiter KL, Leatt P. Pay-for-performance in publicly financed healthcare: some international experience and considerations for Canada. Healthc Pap. 2006;6(4):8–26.
Kwon H-Y, Kim H, Godman B. Availability and affordability of drugs with a conditional approval by the European Medicines Agency; comparison of Korea with other countries and the implications. Front Pharmacol. 2018;9:938.
IQVIA. EFPIA patient W.A.I.T. indicator 2018 survey. 2019. Available from: https://www.efpia.eu/media/412747/efpia-patient-wait-indicator-study-2018-results-030419.pdf. Accessed 9 Jul 2020.
Van Herck P, De Smedt D, Annemans L, Remmen R, Rosenthal MB, Sermeus W. Systematic review: effects, design choices, and context of pay-for-performance in health care. BMC Health Serv Res. 2010;10:247.
Putrik P, Ramiro S, Kvien TK, Sokka T, Pavlova M, Uhlig T, et al. Inequities in access to biologic and synthetic DMARDs across 46 European countries. Ann Rheum Dis. 2014;73(1):198–206.
Kostic M, Djakovic L, Sujic R, Godman B, Jankovic SM. Inflammatory bowel diseases (Crohn’s disease and ulcerative colitis): cost of treatment in Serbia and the implications. Appl Health Econ Health Policy. 2017;15(1):85–93.
Wilking N, Bucsics A, Kandolf Sekulovic L, Kobelt G, Laslop A, Makaroff L, et al. Achieving equal and timely access to innovative anticancer drugs in the European Union (EU): summary of a multidisciplinary CECOG-driven roundtable discussion with a focus on Eastern and South-Eastern EU countries. ESMO Open. 2019;4(6):e000550.
EURODIS. Breaking the access deadlock to leave no one behind: a contribution by EURORDIS and its members on possibilities for patients’ full and equitable access to rare disease therapies in Europe. 2018. http://download2.eurordis.org.s3.amazonaws.com/positionpapers/eurordis_access_position_paper_final_4122017.pdf. Accessed 9 Jul 2020.
WHO. Pricing of cancer medicines and its impacts. Geneva: World Health Organization; 2018: licence: CC BY-NC-SA 3.0 IGO. https://apps.who.int/iris/bitstream/handle/10665/277190/9789241515115-eng.pdf?sequence=1&isAllowed=y. Accessed 9 Jul 2020.
Godman B, Allocati E, Moorkens E. Ever-evolving landscape of biosimilars in Canada; findings and implications from a global perspective. GABI J. 2019;8(3):93–7.
Sagonowsky E. AbbVie’s massive Humira discounts are stifling Netherlands biosimilars: report. 2019. https://www.fiercepharma.com/pharma/abbvie-stifling-humira-biosim-competition-massive-discounting-dutch-report. Accessed 9 Jul 2020.
Davio K. After biosimilar deals, UK spending on adalimumab will drop by 75%. 2018. https://www.centerforbiosimilars.com/news/after-biosimilar-deals-uk-spending-on-adalimumab-will-drop-by-75. Accessed 9 Jul 2020.
Derbyshire M, Shina S. Patent expiry dates for biologicals: 2017 update. GaBI J. 2018;7(1):29–34.
Godman B, Hill A, Simoens S, Kurdi A, Gulbinovič J, Martin AP, et al. Pricing of oral generic cancer medicines in 25 European countries; findings and implications. GaBI J. 2019;8(2):49–70.
Ferrario A, Dedet G, Humbert T, Vogler S, Suleman F, Pedersen HB. Strategies to achieve fairer prices for generic and biosimilar medicines. BMJ. 2020;368:l5444.
Pegram MD, Bondarenko I, Zorzetto MMC, Hingmire S, Iwase H, Krivorotko PV, et al. PF-05280014 (a trastuzumab biosimilar) plus paclitaxel compared with reference trastuzumab plus paclitaxel for HER2-positive metastatic breast cancer: a randomised, double-blind study. Br J Cancer. 2019;120(2):172–82.
Blackwell K, Gligorov J, Jacobs I, Twelves C. The global need for a trastuzumab biosimilar for patients with HER2-positive breast cancer. Clin Breast Cancer. 2018;18(2):95–113.
World Health Organization. Global status report on noncommunicable diseases. 2014. http://apps.who.int/iris/bitstream/handle/10665/148114/9789241564854_eng.pdf;jsessionid=89D7CBFF5B60AE5394BEAB3AFD48A501?sequence=1. Accessed 9 Jul 2020.
Gyasi RM, Phillips DR. Aging and the rising burden of noncommunicable diseases in sub-Saharan Africa and other low- and middle-income countries: a call for holistic action. Gerontologist. 2019. https://doi.org/10.1093/geront/gnz102.
GBD 2016 Disease and Injury Incidence and Prevalence Collaborators. Global, regional, and national incidence, prevalence, and years lived with disability for 328 diseases and injuries for 195 countries, 1990-2016: a systematic analysis for the Global Burden of Disease Study 2016. Lancet. 2017;390(10100):1211–59.
Howard DH, Bach P, Berndt ER, Conti RM. Pricing in the market for anticancer drugs. J Econ Perspect. 2015;29(1):139–62.
Luzzatto L, Hyry HI, Schieppati A, Costa E, Simoens S, Schaefer F, et al. Outrageous prices of orphan drugs: a call for collaboration. Lancet. 2018;392(10149):791–4.
Bach PB, Saltz LB. Raising the dose and raising the cost: the case of pembrolizumab in lung cancer. J Natl Cancer Inst. 2017. https://doi.org/10.1093/jnci/djx125.
Bray F, Ferlay J, Soerjomataram I, Siegel RL, Torre LA, Jemal A. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018;68(6):394–424.
IMS Institute for Healthcare Informatics. Global oncology trend report a review of 2015 and outlook to 2020. June 2016. https://www.scribd.com/document/323179495/IMSH-Institute-Global-Oncology-Trend-2015-2020-Report. Accessed 9 Jul 2020.
IQVIA Institute for Human Data Science. Global oncology trends 2018. https://www.iqvia.com/institute/reports/global-oncology-trends-2018. Accessed 9 Jul 2020.
Hofmarcher T, Brådvik G, Svedman C, Lindgren P, Jönsson B, Wilking N. Comparator report on cancer in Europe 2019: disease burden, costs and access to medicines. Lund: IHE Report 2019:7. https://www.efpia.eu/media/413449/comparator-report-on-cancer-in-europe-2019.pdf. Accessed 9 Jul 2020.
van Harten WH, Wind A, de Paoli P, Saghatchian M, Oberst S. Actual costs of cancer drugs in 15 European countries. Lancet Oncol. 2016;17(1):18–20.
Simoens S, van Harten W, Lopes G, Vulto A, Meier K, Wilking N. What happens when the cost of cancer care becomes unsustainable. Eur Oncol Haematol. 2017;13(2):108–13.
Wilking N, Lopes G, Meier K, Simoens S, van Harten W, Vulto A. Can we continue to afford access to cancer treatment? Eur Oncol Haematol. 2017;13(2):114–9.
Brau R, Tzeng I. Orphan drug commercial models. https://www.lifescienceleader.com/doc/orphan-drug-commercial-models-0001. Accessed 9 Jul 2020.
Mestre-Ferrandiz J, Palaska C, Kelly T, Hutchings A, Parnaby A. An analysis of orphan medicine expenditure in Europe: is it sustainable? Orphanet J Rare Dis. 2019;14(1):287.
Haycox A. Why cancer? Pharmacoeconomics. 2016;34(7):625–7.
Simoens S, Picavet E, Dooms M, Cassiman D, Morel T. Cost-effectiveness assessment of orphan drugs: a scientific and political conundrum. Appl Health Econ Health Policy. 2013;11(1):1–3.
Kantarjian HM, Fojo T, Mathisen M, Zwelling LA. Cancer drugs in the United States: justum pretium: the just price. J Clin Oncol. 2013;31(28):3600–4.
Cohen JP, Felix A. Are payers treating orphan drugs differently? J Market Access Health Policy. 2014;2(1):23513.
Cohen D. Cancer drugs: high price, uncertain value. BMJ (Clin Res Ed). 2017;359:j4543.
Godman B, Wild C, Haycox A. Patent expiry and costs for anti-cancer medicines for clinical use. Generics Biosimilars Initiative J. 2017;6(3):105–6.
Garattini L, Freemantle N. Comment on: ‘NICE, in confidence: an assessment of redaction to obscure confidential information in single technology appraisals by the National Institute for Health and Care Excellence’. Pharmacoeconomics. 2020;38(1):121–2.
Sullivan R, Pramesh CS, Booth CM. Look beyond technology in cancer care. Nature. 2017;549:325–8.
Hanna E, Toumi M, Dussart C, Borissov B, Dabbous O, Badora K, et al. Funding breakthrough therapies: a systematic review and recommendation. Health Policy. 2018;122(3):217–29.
Yu TTL, Gupta P, Ronfard V, Vertès AA, Bayon Y. Recent progress in European advanced therapy medicinal products and beyond. Front Bioeng Biotechnol. 2018;6:130.
Jönsson B, Hampson G, Michaels J, Towse A, von der Schulenburg JMG, Wong O. Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. Eur J Health Econ. 2019;20(3):427–38.
Barlow JF, Yang M, Teagarden JR. Are payers ready, willing, and able to provide access to new durable gene therapies? Value Health. 2019;22(6):642–7.
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.
Hampson G, Towse A, Pearson SD, Dreitlein WB, Henshall C. Gene therapy: evidence, value and affordability in the US health care system. J Comp Eff Res. 2018;7(1):15–28.
MIT NEWDIGS FoCUS Project. Designing financial solutions to ensure affordable access to cures: an overview of the MIT FoCUS project. 2018. https://newdigs.mit.edu/sites/default/files/NEWDIGS%20FoCUS%20Frameworks%2020180823.pdf. Accessed 9 Jul 2020.
Davis C, Naci H, Gurpinar E, Poplavska E, Pinto A, Aggarwal A. Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13. BMJ (Clin Res Ed). 2017;359:j4530.
Ferrario A, Kanavos P. Dealing with uncertainty and high prices of new medicines: a comparative analysis of the use of managed entry agreements in Belgium, England, the Netherlands and Sweden. Soc Sci Med. 2015;124:39–47.
Morel T, Arickx F, Befrits G, Siviero P, van der 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;8:198.
Robinson MF, Mihalopoulos C, Merlin T, Roughead E. Characteristics of managed entry agreements in Australia. Int J Technol Assess Health Care. 2018;34(1):46–55.
Grignolo A, Pretorius S. Phase III trial failures, costly but preventable. Appl Clin Trials. 2016. https://www.parexel.com/application/files_previous/5014/7274/5573/ACT_Article.pdf. Accessed 9 Jul 2020.
Amiri-Kordestani L, Fojo T. Why do phase III clinical trials in oncology fail so often? J Natl Cancer Inst. 2012;104(8):568–9.
Gyawali B, Addeo A. Negative phase 3 randomized controlled trials: why cancer drugs fail the last barrier? Int J Cancer. 2018;143(8):2079–81.
Walter RB, Appelbaum FR, Tallman MS, Weiss NS, Larson RA, Estey EH. Shortcomings in the clinical evaluation of new drugs: acute myeloid leukemia as paradigm. Blood. 2010;116(14):2420–8.
Jonker DJ, Nott L, Yoshino T, Gill S, Shapiro J, Ohtsu A, et al. Napabucasin versus placebo in refractory advanced colorectal cancer: a randomised phase 3 trial. Lancet Gastroenterol Hepatol. 2018;3(4):263–70.
Pontes C, Zara C, Torrent-Farnell J, Obach M, Nadal C, Vella-Bonanno P, et al. Time to review authorisation and funding for new cancer medicines in Europe? Inferences from the case of olaratumab. Appl Health Econ Health Policy. 2020;18(1):5–16.
Frisk P, Aggefors K, Cars T, Feltelius N, Loov SA, Wettermark B, et al. Introduction of the second-generation direct-acting antivirals (DAAs) in chronic hepatitis C: a register-based study in Sweden. Eur J Clin Pharmacol. 2018;74(7):971–8.
Randomised trial of cholesterol lowering in 4444 patients with coronary heart disease: the Scandinavian Simvastatin Survival Study (4S). Lancet. 1994;344(8934):1383–9.
Heart Protection Study Collaborative Group. MRC/BHF Heart Protection Study of cholesterol lowering with simvastatin in 20,536 high-risk individuals: a randomised placebo-controlled trial. Lancet. 2002;360(9326):7–22.
Liao JM, Fischer MA. Restrictions of hepatitis C treatment for substance-using Medicaid patients: cost versus ethics. Am J Public Health. 2017;107(6):893–9.
Douglass CH, Pedrana A, Lazarus JV, ‘t Hoen EFM, Hammad R, Leite RB, et al. Pathways to ensure universal and affordable access to hepatitis C treatment. BMC Med. 2018;16(1):175.
Kesselheim AS, Myers JA, Solomon DH, Winkelmayer WC, Levin R, Avorn J. The prevalence and cost of unapproved uses of top-selling orphan drugs. PLoS One. 2012;7(2):e31894.
Godman B, Bucsics A, Burkhardt T, Haycox A, Seyfried H, Wieninger P. Insight into recent reforms and initiatives in Austria: implications for key stakeholders. Expert Rev Pharmacoecon Outcomes Res. 2008;8(4):357–71.
Godman B, Wettermark B, van Woerkom M, Fraeyman J, Alvarez-Madrazo S, Berg C, et al. Multiple policies to enhance prescribing efficiency for established medicines in Europe with a particular focus on demand-side measures: findings and future implications. Front Pharmacol. 2014;5:106.
Moorkens E, Vulto AG, Huys I, Dylst P, Godman B, Keuerleber S, et al. Policies for biosimilar uptake in Europe: an overview. PLoS One. 2017;12(12):e0190147.
AIM. AIM proposes to establish a European drug pricing model for fair and transparent prices for accessible pharmaceutical innovations. https://www.aim-mutual.org/wp-content/uploads/2019/12/AIMs-proposal-for-fair-and-transparent-prices-for-pharmaceuticals.pdf. Accessed 9 Jul 2020.
Moon S, Mariat S, Kamae I, Pedersen HB. Defining the concept of fair pricing for medicines. BMJ. 2020;368:l4726.
Seixas BV, Dionne F, Conte T, Mitton C. Assessing value in health care: using an interpretive classification system to understand existing practices based on a systematic review. BMC Health Serv Res. 2019;19(1):560.
Mitton C, Seixas BV, Peacock S, Burgess M, Bryan S. Health technology assessment as part of a broader process for priority setting and resourceaAllocation. Appl Health Econ Health Policy. 2019;17(5):573–6.
Moreno-Calderón A, Tong TS, Thokala P. Multi-criteria decision analysis software in healthcare priority setting: a systematic review. Pharmacoeconomics. 2020;38(3):269–83.
Uyl-de Groot CA, Lowenberg B. Sustainability and affordability of cancer drugs: a novel pricing model. Nat Rev Clin Oncol. 2018;15(7):405–6.
Lasalvia P, Prieto-Pinto L, Moreno M, Castrillon J, Romano G, Garzon-Orjuela N, et al. International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review. Expert Rev Pharmacoecon Outcomes Res. 2019;19(4):409–20.
Guarga L, Badia X, Obach M, Fontanet M, Prat A, Vallano A, et al. Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut). Orphanet J Rare Dis. 2019;14(1):157.
Ferrario A, Arāja D, Bochenek T, Čatić T, Dankó D, Dimitrova M, et al. The implementation of managed entry agreements in Central and Eastern Europe: findings and implications. Pharmacoeconomics. 2017;35(12):1271–85.
Ferrario A, Kanavos P. Managed entry agreements for pharmaceuticals: the European experience. EMiNet, Brussels, Belgium. 2013. Available from: http://eprints.lse.ac.uk/50513/. Accessed 9 Jul 2020.
Carlson JJ, Chen S, Garrison LP Jr. Performance-based risk-sharing arrangements: an updated international review. Pharmacoeconomics. 2017;35(10):1063–72.
Piatkiewicz TJ, Traulsen JM, Holm-Larsen T. Risk-sharing agreements in the EU: a systematic review of major trends. Pharmacoecon Open. 2018;2(2):109–23.
Adamski J, Godman B, Ofierska-Sujkowska G, Osinska B, Herholz H, Wendykowska K, et al. Risk sharing arrangements for pharmaceuticals: potential considerations and recommendations for European payers. BMC Health Serv Res. 2010;10:153.
Clopes A, Gasol M, Cajal R, Segu L, Crespo R, Mora R, et al. Financial consequences of a payment-by-results scheme in Catalonia: gefitinib in advanced EGFR-mutation positive non-small-cell lung cancer. J Med Econ. 2017;20(1):1–7.
Goble JA, Ung B, van Boemmel-Wegmann S, Navarro RP, Parece A. Performance-based risk-sharing arrangements: U.S. payer experience. J Manage Care Spec Pharm. 2017;23(10):1042–52.
Yoo SL, Kim DJ, Lee SM, Kang WG, Kim SY, Lee JH, et al. Improving patient access to new drugs in South Korea: evaluation of the National Drug Formulary System. Int J Environ Res Public Health. 2019;16(2):288.
Choi MH, Ghosh W, Brooks-Rooney C. The impact of risk-sharing agreements on drug reimbursement decisions in South Korea. Value Health. 2018;21(Suppl. 2):S57.
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 Health. 2018;21(1):33–40.
Milstein R, Schreyoegg J. Pay for performance in the inpatient sector: a review of 34 P4P programs in 14 OECD countries. Health Policy. 2016;120(10):1125–40.
Lu CY, Lupton C, Rakowsky S, Babar ZU, Ross-Degnan D, Wagner AK. Patient access schemes in Asia-pacific markets: current experience and future potential. J Pharm Policy Pract. 2015;8(1):6.
Darba 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(6):743–8.
Nazareth T, Ko JJ, Sasane R, Frois C, Carpenter S, Demean S, et al. Outcomes-based contracting experience: research findings from U.S. and European stakeholders. J Manage Care Spec Pharm. 2017;23(10):1018–26.
Araja D, Kõlves K. Managed entry agreements for new medicines in the Baltic countries. Eurohealth. 2016;22(1):27–30.
Godman B, Oortwijn W, de Waure C, Mosca I, Puggina A, Specchia ML, et al. Links between pharmaceutical R&D models and access to affordable medicines: a study for the ENVI Committee. http://www.europarl.europa.eu/RegData/etudes/STUD/2016/587321/IPOL_STU(2016)587321_EN.pdf. Accessed 9 Jul 2020.
Ghinea H, Kerridge I, Lipworth W. If we don’t talk about value, cancer drugs will become terminal for health systems. http://theconversation.com/if-we-dont-talk-about-value-cancer-drugs-will-become-terminal-for-health-systems-44072. Accessed 9 Jul 2020.
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.
Kim ES, Kim JA, Lee EK. National reimbursement listing determinants of new cancer drugs: a retrospective analysis of 58 cancer treatment appraisals in 2007-2016 in South Korea. Expert Rev Pharmacoecon Outcomes Res. 2017;17(4):401–9.
Van de Vijver I, Quanten A, Knappenberg V, Arickx F, De Ridder R. Success and failure of straightforward versus sophisticated managed entry agreements. Value Health. 2016;19(7):A499.
Quanten A, Van de Vijver I, Knappenberg V, Arickx F, De Ridder R. Insights from 6 years’ managed entry agreement experience in Belgium. Value Health. 2016;19(7):A499.
Campillo-Artero C, del Llano J, Poveda JL. Risk sharing agreements: with orphan drugs? [in Spanish]. Farm Hosp. 2012;36(6):455–63.
Sola-Morales O, Volmer T, Mantovani L. Perspectives to mitigate payer uncertainty in health technology assessment of novel oncology drugs. J Mark Access Health Policy. 2019;7(1):1562861.
Morse GN. Pharmaceutical managed entry agreements: lessons learned from Europe, the United States, Canada and Australia. https://morseconsulting.ca/pharmaceutical-managed-entry-agreements-lessons-learned/. Accessed 9 Jul 2020.
Bullement A, Taylor M, McMordie ST, Waters E, Hatswell AJ. NICE, in confidence: an assessment of redaction to obscure confidential information in single technology appraisals by the National Institute for Health and Care Excellence. Pharmacoeconomics. 2019;37(11):1383–90.
Tuffaha HW, Scuffham PA. The Australian managed entry scheme: are we getting it right? Pharmacoeconomics. 2018;36(5):555–65.
NHS Scotland. Patient access scheme (PAS) guidance. November 2018. https://www.scottishmedicines.org.uk/media/3928/nhs-scotland-patient-access-scheme-pas-guidance-v70.pdf. Accessed 9 Jul 2020.
Makady A, van Veelen A, de Boer A, Hillege H, Klungel OH, Goettsch W. Implementing managed entry agreements in practice: the Dutch reality check. Health Policy. 2019;123(3):267–74.
Grimm SE, Strong M, Brennan A, Wailoo AJ. The HTA risk analysis chart: visualising the need for and potential value of managed entry agreements in health technology assessment. Pharmacoeconomics. 2017;35(12):1287–96.
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.
Annemans L, Panie L. Dynamic outcomes based approaches to pricing and reimbursement of innovative medicines. 2017. https://www.eurordis.org/sites/default/files/FIPRA.pdf. Accessed 9 Jul 2020.
Catapult. Cell and gene therapy: enabling outcomes-based reimbursement through a universal platform for outcomes data. 2019. https://ct.catapult.org.uk/case-study/enabling-outcomes-based-reimbursement-through-universal-platform-outcomes-data?_cldee = ZXdhbi5tb3JyaXNvbkBuaHMubmV0&recipientid = contact-64c168729c5ae8118140e0071b6ee581-1b8c25455c8b44c5ba52f2d26432e9bd&utm_source=ClickDimensions&utm_medium=email&utm_campaign=Monthly%20newsletters&esid=c8f55ab4-ebad-e911-a972-000d3a38ad05. Accessed 9 Jul 2020.
Bouvy JC, Sapede C, Garner S. Managed entry agreements for pharmaceuticals in the context of adaptive pathways in Europe. Front Pharmacol. 2018;9:280.
Castro HE, Kumar R, Malpica-Llanos T, et al. Sharing knowledge on innovative medicines for non-communicable disease: a compendium of good practices for sustainable access: promoting a dialogue among payers and manufacturers. 2018. http://pubdocs.worldbank.org/en/792561542818915277/MSH-RTI-GLOHI-Compendium-Final-Version-2-Nov-21-2018.pdf. Accessed 9 Jul 2020.
Van Wilder P, Pirson M, Dupont A. Impact of health technology assessment and managed entry schemes on reimbursement decisions of centrally authorised medicinal products in Belgium. Eur J Clin Pharmacol. 2019;75(7):895–900.
Yeung K, Li M, Carlson JJ. Using performance-based risk-sharing arrangements to address uncertainty in indication-based pricing. J Manag Care Spec Pharm. 2017;23(10):1010–5.
Carlson JJ, Sullivan SD, Garrison LP, Neumann PJ, Veenstra DL. Linking payment to health outcomes: a taxonomy and examination of performance-based reimbursement schemes between healthcare payers and manufacturers. Health Policy. 2010;96(3):179–90.
Antonanzas F, Juarez-Castello C, Lorente R, Rodriguez-Ibeas R. The use of risk-sharing contracts in healthcare: theoretical and empirical assessments. Pharmacoeconomics. 2019;37(12):1469–83.
LaPointe J. Hospitals, Blue Cross NC share risk with new value-based contract. 2019. https://revcycleintelligence.com/news/hospitals-blue-cross-nc-share-risk-with-new-value-based-contract. Accessed 9 Jul 2020.
Garattini L, Curto A. Performance-based agreements in Italy: ‘trendy outcomes’ or mere illusions? Pharmacoeconomics. 2016;34(10):967–9.
Seeley E, Kesselheim AS. Outcomes-based pharmaceutical contracts: an answer to high U.S. drug spending? Issue Brief. 2017; pp/ 1–8.
Toumi M, Jaroslawski S, Sawada T, Kornfeld A. The use of surrogate and patient-relevant endpoints in outcomes-based market access agreements: current debate. Appl Health Econ Health Policy. 2017;15(1):5–11.
Prasad V, Kim C, Burotto M, Vandross A. The strength of association between surrogate end points and survival in oncology: a systematic review of trial-level meta-analyses. JAMA Intern Med. 2015;175(8):1389–98.
Wild C, Grossmann N, Bonanno PV, Bucsics A, Furst J, Garuoliene K, et al. Utilisation of the ESMO-MCBS in practice of HTA. Ann Oncol. 2016;27(11):2134–6.
Paoletti X, Lewsley LA, Daniele G, Cook A, Yanaihara N, Tinker A, et al. Assessment of progression-free survival as a surrogate end point of overall survival in first-line treatment of ovarian cancer: a systematic review and meta-analysis. JAMA Netw Open. 2020;3(1):e1918939.
van de Wetering EJ, van Exel J, Brouwer WB. The challenge of conditional reimbursement: stopping reimbursement can be more difficult than not starting in the first place! Value Health. 2017;20(1):118–25.
Leopold C, Vogler S, Mantel-Teeuwisse AK, de Joncheere K, Leufkens HG, Laing R. Differences in external price referencing in Europe: a descriptive overview. Health Policy. 2012;104(1):50–60.
WHO. Improving the transparency of markets for medicines, vaccines, and other health products (footnote): draft resolution proposed by Andorra, Brazil, Egypt, Eswatini, Greece, India, Italy, Kenya, Luxembourg, Malaysia, Malta, Portugal, Russian Federation, Serbia, Slovenia, South Africa, Spain, Sri Lanka, Uganda. 2019. https://apps.who.int/gb/ebwha/pdf_files/WHA72/A72_ACONF2Rev1-en.pdf. Accessed 9 Jul 2020.
World Health Assembly. Improving the [access to (Germany)]/[transparency [in access (France)]/[of markets (DEL France)] for (DEL Germany)] medicines, vaccines and other health-related [products and (India)] technologies to be discussed at the 72nd session of the WHA to be held on 20-28 May 2019. https://www.healthpolicy-watch.org/wp-content/uploads/2019/05/WHA-Resolution_DRAFT_10May1740.pdf. Accessed 9 Jul 2020.
Shaw B, Mestre-Ferrandiz J. Talkin’ about a resolution: issues in the push for greater transparency of medicine prices. Pharmacoeconomics. 2020;38(2):125–34.
Suleman F, Low M, Moon S, Morgan SG. New business models for research and development with affordability requirements are needed to achieve fair pricing of medicines. BMJ. 2020;368:l4408.
Eldred K. Cigna’s two new value-based vontracts with Pharma for PCSK9 inhibitor cholesterol drugs tie financial terms to improved customer health. 2016. https://www.cigna.com/newsroom/news-releases/2016/cignas-two-new-value-based-contracts-with-pharma-for-pcsk9-inhibitor-cholesterol-drugs-tie-financial-terms-to-improved-customer-health. Accessed 9 Jul 2020.
Cole A, Cubi-Molla P, Pollard J, Sim D, Sullivan R, Sussex J, Lorgelly P. Making outcome-based payment a reality in the NHS. 2019. file:///C:/Users/mail/Downloads/Cole%20et%20al.%20Making%20Outcome-Based%20Payment%20a%20Reality%20in%20the%20NHS.pdf. Accessed 9 Jul 2020.
Bianchetti A, Ranieri P, Margiotta A, Trabucchi M. Pharmacological treatment of Alzheimer’s disease. Aging Clin Exp Res. 2006;18(2):158–62.
Godman B, Malmstrom RE, Diogene E, Jayathissa S, McTaggart S, Cars T, et al. Dabigatran: a continuing exemplar case history demonstrating the need for comprehensive models to optimize the utilization of new drugs. Front Pharmacol. 2014;5:109.
Mueller T, Alvarez-Madrazo S, Robertson C, Wu O, Bennie M. Comparative safety and effectiveness of direct oral anticoagulants in patients with atrial fibrillation in clinical practice in Scotland. Br J Clin Pharmacol. 2019;85(2):422–31.
Maura G, Pariente A, Alla F, Billionnet C. Adherence with direct oral anticoagulants in nonvalvular atrial fibrillation new users and associated factors: a French nationwide cohort study. Pharmacoepidemiol Drug Saf. 2017;26(11):1367–77.
Kemp-Casey A, Pratt N, Ramsay E, Roughead EE. Using post-market utilisation analysis to support medicines pricing policy: an Australian case study of aflibercept and ranibizumab use. Appl Health Econ Health Policy. 2019;17(3):411–7.
Gomes RM, Barbosa WB, Godman B, Costa JO, Ribeiro Junior NG, Simão Filho C, et al. Effectiveness of maintenance immunosuppression therapies in a matched-pair analysis cohort of 16 years of renal transplant in the Brazilian National Health System. Int J Environ Res Public Health. 2020;17(6):1974.
Dos Santos JB, Almeida AM, Acurcio FA, de Oliveira Junior HA, Kakehasi AM, Guerra Junior AA, et al. Comparative effectiveness of adalimumab and etanercept for rheumatoid arthritis in the Brazilian Public Health System. J Comp Eff Res. 2016;5(6):539–49.
Alvarez-Madrazo S, Kavanagh K, Siebert S, Semple Y, Godman B, Maciel Almeida A, et al. Discontinuation, persistence and adherence to subcutaneous biologics delivered via a homecare route to Scottish adults with rheumatic diseases: a retrospective study. BMJ Open. 2019;9(9):e027059.
Raaschou P, Simard JF, Holmqvist M, Askling J. Rheumatoid arthritis, anti-tumour necrosis factor therapy, and risk of malignant melanoma: nationwide population based prospective cohort study from Sweden. BMJ. 2013;346:f1939.
Raaschou P, Simard JF, Asker Hagelberg C, Askling J. Rheumatoid arthritis, anti-tumour necrosis factor treatment, and risk of squamous cell and basal cell skin cancer: cohort study based on nationwide prospectively recorded data from Sweden. BMJ. 2016;352:i262.
Garcia-Doval I, Cohen AD, Cazzaniga S, Feldhamer I, Addis A, Carretero G, et al. Risk of serious infections, cutaneous bacterial infections, and granulomatous infections in patients with psoriasis treated with anti-tumor necrosis factor agents versus classic therapies: prospective meta-analysis of Psonet registries. J Am Acad Dermatol. 2017;76(2):299–308.e16.
Jorgensen J, Kefalas P. Upgrading the SACT dataset and EBMT registry to enable outcomes-based reimbursement in oncology in England: a gap analysis and top-level cost estimate. J Mark Access Health Policy. 2019;7(1):1635842.
OECD. Stat, pharmaceutical market. 2018. https://stats.oecd.org/Index.aspx?DataSetCode=HEALTH_PHMC#. Accessed 9 Jul 2020.
Ministry of Health Columbia. Avances en la estrategia de negociación y compra centralizada de medicamentos para la hepatitis C. 2018. https://www.minsalud.gov.co/sites/rid/Lists/BibliotecaDigital/RIDE/VS/MET/fact-sheet-hepatitisc.pdf. Accessed 9 Jul 2020.
Ministério da Saúde. Secretaria de Ciência, Tecnologia e Insumos Estratégicos. Departamento de Gestão e Incorporação de Tecnologias em Saúde. Diretrizes metodológicas: avaliação de desempenho de tecnologias em saúde [recurso eletrônico]/Ministério da Saúde, Secretaria de Ciência, Tecnologia e Insumos Estratégicos, Departamento de Gestão e Incorporação de Tecnologias em Saúde. Brasília: Ministério da Saúde; 2017;45: p. il.
Lemos LLP, Guerra Junior AA, Santos M, Magliano C, Diniz I, Souza K, et al. The assessment for disinvestment of intramuscular interferon beta for relapsing-remitting multiple sclerosis in Brazil. Pharmacoeconomics. 2018;36(2):161–73.
Ministério da Saúde. Portaria No. 24, 24 April 2019. Torna pública a decisão de incorporar o nusinersena para atrofia muscular espinhal (AME) 5q tipo I, no âmbito do Sistema Único de Saúde-SUS. 25 April 2019. Seção 1, No. 79.
Ministério da Saúde. Portaria No. 1. 297, 11 June 2019. Institui projeto piloto de acordo de compartilhamento de risco para incorporação de tecnologias em saúde, para oferecer acesso ao medicamento Spinraza (Nusinersena) para o tratamento da Atrofia Muscular Espinhal (AME 5q) tipos II e III no âmbito do Sistema Único de Saúde: SUS. Diário Oficial da União Seção 1, ISSN 1677-7042 No. 112, quarta-feira, 12 June 2019.
Coulton L, Annemans L, Carter R, Herrera MB, Thabrany H, Lim J, et al. Outcomes-based risk-sharing schemes: is there a potential role in the Asia-Pacific markets? Health Outcomes Res Med. 2012;3(4):e205–19.
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(10):1028–40.
Garrison LP Jr, Towse A, Briggs A, de Pouvourville G, Grueger J, Mohr PE, 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(5):703–19.
Godman B, Basu D, Pillay Y, Mwita JC, Rwegerera GM, Anand Paramadhas BD, et al. Review of ongoing activities and challenges to improve the care of patients with type 2 diabetes across Africa and the implications for the future. Front Pharmacol. 2020;11:108.
Godman B, Malmstrom RE, Diogene E, Gray A, Jayathissa S, Timoney A, et al. Are new models needed to optimize the utilization of new medicines to sustain healthcare systems? Expert Rev Clin Pharmacol. 2015;8(1):77–94.
Godman B, Finlayson AE, Cheema PK, Zebedin-Brandl E, Gutierrez-Ibarluzea I, Jones J, et al. Personalizing health care: feasibility and future implications. BMC Med. 2013;11:179.
Vella Bonanno P, Ermisch M, Godman B, Martin AP, Van Den Bergh J, Bezmelnitsyna L, et al. Adaptive pathways: possible next steps for payers in preparation for their potential implementation. Front Pharmacol. 2017;8:497.
Guerra-Junior AA, de Lemos PLL, Godman B, Bennie M, Osorio-de-Castro CGS, Alvares J, et al. Health technology performance assessment: real-world evidence for public healthcare sustainability. Int J Technol Assess Health Care. 2017;33(2):279–87.
Godman B, McCabe H, Leong T, et al. Fixed dose drug combinations: are they pharmacoeconomically sound? Findings and implications especially for lower- and middle-income countries. Expert Rev Pharmacoecon Outcomes Res. 2020;20(1):1–26.
Green S, Higgins JPT, Alderson P, Clarke M, Mulrow CD, Oxman AD. Chapter 1: introduction. In: Higgins JPT, Green S, editors. Cochrane handbook for systematic reviews of interventions. Version 5.1.0 (updated March 2011). The Cochrane Collaboration, 2011. www.handbook.cochrane.org. Accessed 9 Jul 2020.
Vogler S, Paris V, Ferrario A, Wirtz VJ, de Joncheere K, Schneider P, et al. How can pricing and reimbursement policies improve affordable access to medicines? Lessons learned from European countries. Appl Health Econ Health Policy. 2017;15(3):307–21.
Vogler S, Zimmermann N, Ferrario A, Wirtz VJ, de Joncheere K, Pedersen HB, et al. Pharmaceutical policies in a crisis? Challenges and solutions identified at the PPRI Conference. J Pharm Policy Pract. 2016;9:9.
WHO. Fair pricing forum 2017 meeting report. 2017. https://www.who.int/medicines/access/fair_pricing/FairPricingForum2017MeetingReport.pdf?ua=1. Accessed 9 Jul 2020.
Pomorski MK, Matusewicz W, Lipińska A. Types of risk-sharing schemes proposed in reimbursement application received by Aotmit in 2015. Value Health. 2016;19(7):A500.
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. https://www.england.nhs.uk/wp-content/uploads/2013/04/cdf-sop.pdf. Accessed 9 Jul 2020.
Cole A, Towse A, Lorgelly P, Sullivan R. Economics of innovative payment models compared with single pricing of pharmaceuticals. https://www.ohe.org/publications/economics-innovative-payment-models-compared-single-pricing-pharmaceuticals-0. Accessed 9 Jul 2020.
Pearson SD, Dreitlein WB, Towse A, Hampson G, Henshall C. A framework to guide the optimal development and use of real-world evidence for drug coverage and formulary decisions. J Comp Eff Res. 2018;7(12):1145–52.
Editorial. Safety surveillance of bevacizumab biosimilar (Bevax) in Argentina. 2019. http://gabionline.net/Biosimilars/Research/Safety-surveillance-of-bevacizumab-biosimilar-Bevax-in-Argentina. Accessed 9 Jul 2020.
Secretaria do Estado de Minas Gerais. Superintendência de Assistência Farmacêutica. SIGAF. 2019. http://sigaf.saude.mg.gov.br/. Accessed 9 Jul 2020.
Ministério da Saúde. Portaria No. 77, 14 December 2018. Torna pública a decisão de incorporar o eculizumabe para tratamento de pacientes com hemoglobinúria paroxística noturna (HPN) no âmbito do Sistema Único de Saúde: SUS. 17 December 2018. Seção 1, No. 241.
Congreso de la Republica de Colombia. Ministerio de Salud y Protección Social. LEY 1751 DE 2015: estatutaria de salud: regula el Derecho Fundamental a la Salud y se dictan otras disposiciones. Bogotá; 16 February 2015.
Fontrier AM, Gill J, Kanavos P. International impact of external reference pricing: should national policy-makers care? Eur J Health Econ. 2019;20(8):1147–64.
Persson U, Jonsson B. The end of the international reference pricing system? Appl Health Econ Health Policy. 2016;14(1):1–8.
Leopold C, Mantel-Teeuwisse AK, Seyfang L, Vogler S, de Joncheere K, Laing RO, et al. Impact of external price referencing on medicine prices: a price comparison among 14 European countries. South Med Rev. 2012;5(2):34–41.
Houy N, Jelovac I. Drug Launch timing and international reference pricing. Health Econ. 2015;24(8):978–89.
van der Gronde T, Uyl-de Groot CA, Pieters T. Addressing the challenge of high-priced prescription drugs in the era of precision medicine: a systematic review of drug life cycles, therapeutic drug markets and regulatory frameworks. PLoS One. 2017;12(8):e0182613.
O’Mahony JF. Beneluxa: what are the prospects for collective bargaining on pharmaceutical prices given diverse health technology assessment processes? Pharmacoeconomics. 2019;37(5):627–30.
KCE Report 283. Horizon scanning for pharmaceuticals: proposal for the Beneluxa Collaboration. Available from: http://www.beneluxa.org/sites/beneluxa.org/files/2017-07/Horizon%20scanning_ScientificReport_full.pdf. Accessed 9 Jul 2020.
Eatwell E, Swierczyna A. Emerging voluntary cooperation between European healthcare systems: are we facing a new future? Med Access@Point Care. 2019; pp. 1–8.
Office of the Deputy Prime Minister and the Ministry for Health of Malta. Valletta Technical Group continues to grow. 2018http://www.livenewsmalta.com/index.php/2018/01/31/valletta-technical-group-continues-to-grow/. Accessed 9 Jul 2020.
Sutton S. Joint pricing and health technology assessment between European countries: is this the future of pricing and market access negotiations? https://remapconsulting.com/joint-pricing-and-health-technology-assessment-between-european-countries-is-this-the-future-of-pricing-and-market-access-negotiations/. Accessed 9 Jul 2020.
The Centre for Biosimilars Staff. On the strength of a discount and biosimilar trastuzumab savings, NICE recommends pertuzumab. 2019. https://www.centerforbiosimilars.com/news/on-the-strength-of-a-discount-and-biosimilar-trastuzumab-savings-nice-recommends-pertuzumab. Accessed 9 Jul 2020.
Sermet C, Andrieu V, Godman B, Van Ganse E, Haycox A, Reynier JP. Ongoing pharmaceutical reforms in France: implications for key stakeholder groups. Appl Health Econ Health Policy. 2010;8(1):7–24.
Bright CJ, Lawton S, Benson S, Bomb M, Dodwell D, Henson KE, et al. Data resource profile: the Systemic Anti-Cancer Therapy (SACT) dataset. Int J Epidemiol. 2019;49(1):15–l51.
Scottish Government. Making medicine more effective: £300,000 for Cancer Medicines Outcome Programme. 2017. https://news.gov.scot/news/making-medicine-more-effective. Accessed 9 Jul 2020.
Mestre-Ferrandiz J, Zozaya N, Alcalá B, Hidalgo-Vega Á. Multi-indication pricing: nice in theory but can it work in practice? Pharmacoeconomics. 2018;36(12):1407–20.
Ferguson JS, Summerhayes M, Masters S, Schey S, Smith IE. New treatments for advanced cancer: an approach to prioritization. Br J Cancer. 2000;83(10):1268–73.
NICE. Procedure for the review of patient access scheme proposals. https://www.nice.org.uk/Media/Default/About/what-we-do/PASLU/PASLU-procedure-guide.pdf. Accessed 9 Jul 2020.
Berchick ER, Hood E, Barnett JC. US Census Bureau: health insurance coverage in the United States. 2017: current population reports. Available from: https://www.census.gov/content/dam/Census/library/publications/2018/demo/p60-264.pdf. Accessed 9 Jul 2020.
Marin LGG, Peñaloza JLL, Saldarriaga JAT, Atehortúa LGV, Medina FC, Pulecio LFT, et al. El Pago por Resultados como una estrategia que aborda los Acuerdos de Riesgo Compartido en Colombia: una opción posible! 2015. https://cuentadealtocosto.org/site/images/Bolet%C3%ADn%20especial%20Riesgo%20compartido.pdf. Accessed 9 Jul 2020.
World Bank. 2019. Uruguay: noncommunicable diseases prevention project. Independent evaluation group, project performance assessment report 131480, Washington, DC. https://ieg.worldbankgroup.org/sites/default/files/Data/reports/ppar_uruguayncdprevention.pdf. Accessed 9 Jul 2020.
Iskrov G, Stefanov R. Prospects of risk-sharing agreements for innovative therapies in a context of deficit spending in bulgaria. Front Public Health. 2015;3:64.
Schlander M, Garattini S, Kolominsky-Rabas P, Nord E, Persson U, Postma M, et al. Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement. J Mark Access Health Policy. 2016;4:1–9.
Oliveira MD, Mataloto I, Kanavos P. Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art. Eur J Health Econ. 2019;20(6):891–918.
Iskrov G, Miteva-Katrandzhieva T, Stefanov R. Multi-criteria decision analysis for assessment and appraisal of orphan drugs. Front Public Health. 2016;4:214.
Baltussen R, Marsh K, Thokala P, Diaby V, Castro H, Cleemput I, et al. Multicriteria decision analysis to support health technology assessment agencies: benefits, limitations, and the way forward. Value Health. 2019;22(11):1283–8.
FDA. FDA approves larotrectinib for solid tumors with NTRK gene fusions. 2018. https://www.fda.gov/drugs/fda-approves-larotrectinib-solid-tumors-ntrk-gene-fusions-0. Accessed 9 Jul 2020.
Costa S, Bentley C, Regier DA, McTaggart-Cowan H, Mitton C, Burgess MM, et al. Public perspectives on disinvestments in drug funding: results from a Canadian deliberative public engagement event on cancer drugs. BMC Public Health. 2019;19(1):977.
Permanand G, Pedersen H. Managing new premium-priced medicines in Europe. J Pharm Policy Pract. 2015;8(Suppl. 1):K2.
Löblová O, Csanadi M, Ozieranski P, Kalo Z, King L, McKee M. Alternative access schemes for pharmaceuticals in Europe: towards an emerging typology. Health Policy. 2019;123(7):630–4.
Conflicts of interest/competing interests
Carolina Zampirolli Dias, Brian Godman, Ludmila Peres Gargano, Pâmela Santos Azevedo, Marina Morgado Garcia, Maurílio de Souza Cazarim, Laís Lessa Neiva Pantuzza, Nelio Gomes Ribeiro Junior, André Luiz Pereira, Marcus Carvalho Borin, Isabella de Figueiredo Zuppo, Roberto Iunes, Tomas Pippo, Renata Curi Hauegen, Carlos Vassalo, Tracey-Lea Laba, Steven Simoens, Sergio Márquez, Carolina Gomez, Luka Voncina, Gisbert W. Selke, Livio Garattini, Hye-Young Kwon, Jolanta Gulbinovic, Aneta Lipinska, Maciej Pomorski, Lindsay McClure, Jurij Fürst, Rosana Gambogi, Carla Hernandez Ortiz, Vânia Canuto, Denizar Vianna Araújo, Vânia Eloisa Araujo, Francisco de Assis Acurcio, Juliana Alvares-Teodoro and Augusto Afonso Guerra Junior have no conflicts of interest that are directly relevant to the content of this article. However, most of the co-authors are employed by health authorities or are advisers to health authorities.
No funding was received to conduct the research or write this paper.
There was no need for informed consent or ethical approval as the senior-level personnel providing advice and contextualising the findings from the literature review were the co-authors.
Electronic supplementary material
Below is the link to the electronic supplementary material.
About this article
Cite this article
Zampirolli Dias, C., Godman, B., Gargano, L.P. et al. Integrative Review of Managed Entry Agreements: Chances and Limitations. PharmacoEconomics 38, 1165–1185 (2020). https://doi.org/10.1007/s40273-020-00943-1