Hostname: page-component-586b7cd67f-rcrh6 Total loading time: 0 Render date: 2024-11-20T15:21:18.730Z Has data issue: false hasContentIssue false
  • English
  • Français

LEGITIMACY OF MEDICINES FUNDING IN THE ERA OF ACCELERATED ACCESS

Published online by Cambridge University Press:  12 September 2017

Jessica Pace ,
Sallie-Anne Pearson  and
Wendy Lipworth
Jessica Pace
Affiliation:
Sydney Health Ethics, The University of [email protected]
Sallie-Anne Pearson
Affiliation:
Centre for Big Data Research in Health, University of NSW
Wendy Lipworth
Affiliation:
Sydney Health Ethics, The University of Sydney
Get access Rights & Permissions [Opens in a new window]

Abstract

Objectives: In recent years, numerous frameworks have been developed to enhance the legitimacy of health technology assessment processes. Despite efforts to implement these “legitimacy frameworks,” medicines funding decisions can still be perceived as lacking in legitimacy. We, therefore, sought to examine stakeholder views on factors that they think should be considered when making decisions about the funding of high-cost breast cancer therapies, focusing on those that are not included in current frameworks and processes.

Methods: We analyzed published discourse on the funding of high-cost breast-cancer therapies. Relevant materials were identified by searching the databases Google, Google Scholar, and Factiva in August 2014 and July 2016 and these were analyzed thematically.

Results: We analyzed fifty published materials and found that stakeholders, for the most part, want to be able to access medicines more quickly and at the same time as other patients and for decision makers to be more flexible with regards to evidence requirements and to use a wider range of criteria when evaluating therapies. Many also advocated for existing process to be accelerated or bypassed to improve access to therapies.

Conclusions: Our results illustrate that a stakeholder-derived conceptualization of legitimacy emphasizes principles of accelerated access and is not fully accounted for by existing frameworks and processes aimed at promoting legitimacy. However, further research examining the ethical, political, and clinical implications of the stakeholder claims raised here is needed before firm policy recommendations can be made.

Keywords

Pharmaceutical funding decisionsResource allocationStakeholder engagementBreast cancerAccelerated access
Type
Policies
Information
International Journal of Technology Assessment in Health Care , Volume 33 , Issue 6 , 2017 , pp. 700 - 707
Copyright
Copyright © Cambridge University Press 2017 

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

REFERENCES

1. Littlejohns, P, Weale, A, Chalkidou, K, Faden, R, Teerawattananon, Y. Social values and health policy: A new international research programme. J Health Organ Manag. 2012;26:285–292.Google Scholar
2. Daniels, N, Sabin, JE. Setting limits fairly: Can we learn to share medical resources? New York: Oxford University Press; 2002.CrossRefGoogle Scholar
3. Engelhardt, HT Jr. Confronting moral pluralism in posttraditional western societies: Bioethics critically reassessed. J Med Philos. 2011;36:243–260.Google Scholar
4. Rothstein, B. Creating political legitimacy electoral democracy versus quality of government. Am Behav Sci. 2009;53:311–330.Google Scholar
5. Prasad, V, Mailankody, S. The UK Cancer Drugs Fund Experiment and the US Cancer Drug Cost Problem: Bearing the cost of cancer drugs until it is unbearable. Mayo Clin Proc. 2016;91:707–712.Google Scholar
6. Linley, WG, Hughes, DA. Societal views on nice, cancer drugs fund and value-based pricing criteria for prioritising medicines: A cross-sectional survey of 4118 adults in Great Britain. Health Econ. 2013;22: 948–964.Google Scholar
7. Mayor, S. New “managed access” process for cancer drugs fund to go ahead, NHS England confirms. BMJ. 2016;352:i1208.CrossRefGoogle ScholarPubMed
8. Sibbald, SL, Singer, PA, Upshur, R, Martin, DK. Priority setting: what constitutes success? A conceptual framework for successful priority setting. BMC Health Serv Res. 2009;9:43.Google Scholar
9. Smith, N, Mitton, C, Hall, W, et al. High performance in healthcare priority setting and resource allocation: A literature- and case study-based framework in the Canadian context. Soc Sci Med. 2016;162:185–192.CrossRefGoogle ScholarPubMed
10. Degeling, C, Carter, SM, Rychetnik, L. Which public and why deliberate? – A scoping review of public deliberation in public health and health policy research. Soc Sci Med. 2015;131:114–121.Google Scholar
11. Wortley, S, Tong, A, Howard, K. Preferences for engagement in health technology assessment decision-making: A nominal group technique with members of the public. BMJ Open. 2016;6:e010265.CrossRefGoogle ScholarPubMed
12. Green, C, Gerard, K. Exploring the social value of health-care interventions: A stated preference discrete choice experiment. Health Econ. 2009;18:951–976.Google Scholar
13. NICE Public Involvement Programme. http://www.nice.org.uk/about/nice-communities/public-involvement/public-involvement-programme (accessed April 26, 2017).Google Scholar
14. CADTH Common Drug Review Patient Input. https://www.cadth.ca/about-cadth/what-we-do/products-services/cdr/patient-input (accessed April 26, 2017).Google Scholar
15. Australian Senate Community Affairs Reference Committee. Availability of new, innovative and specialist cancer drugs in Australia. Canberra: Australian Government; 2015.Google Scholar
16. Accelerated Access Review 2016. https://www.gov.uk/government/organisations/accelerated-access-review (accessed April 26, 2017).Google Scholar
17. Gallego, G, Taylor, SJ, Brien, JA. Priority setting for high cost medications (HCMs) in public hospitals in Australia: A case study. Health Policy. 2007;84:58–66.Google Scholar
18. Martin, DK, Giacomini, M, Singer, PA. Fairness, accountability for reasonableness, and the views of priority setting decision-makers. Health Policy. 2002;61:279–290.Google Scholar
19. U.S. Food and Drug Administration. Haematology/oncology (cancer) approvals & safety notifications: Previous news items (updated December 7, 2014). https://www.fda.gov/drugs/informationondrugs/approveddrugs/ucm279177.htm (accessed April 27, 2017).Google Scholar
20. National Cancer Institute. FDA Approval for Pertuzumab (updated October 1, 2013). https://www.cancer.gov/about-cancer/treatment/drugs/fda-pertuzumab (accessed April 26, 2017).Google Scholar
21. Levin, J. FDA approves new treatment for late-stage breast cancer (updated February 22, 2013). http://www.fiercebiotech.com/biotech/fda-approves-new-treatment-for-late-stage-breast-cancer (accessed April 26, 2017).Google Scholar
22. MacKenzie, R, Chapman, S, Salkeld, G, Holding, S. Media influence on Herceptin subsidization in Australia: Application of the rule of rescue? J R Soc Med. 2008;101:305–312.Google Scholar
23. Gabe, J, Chamberlain, K, Norris, P, et al. The debate about the funding of Herceptin: A case study of 'countervailing powers. Soc Sci Med. 2012;75:2353–2361.Google Scholar
24. Fenton, E. Making fair funding decisions for high cost cancer care: The case of herceptin in New Zealand. Public Health Ethics. 2010;3:137–146.Google Scholar
25. Patton, MQ. Qualitative evaluation and research methods. Newbury Park, CA: Sage Publications; 1990.Google Scholar
26. Boon, WPC, Moors, EHM, Meijer, A, Schellekens, H. Conditional approval and approval under exceptional circumstances as regulatory instruments for stimulating responsible drug innovation in Europe. Clin Pharmacol Ther. 2010;88:848–853.Google Scholar
27. U.S. Food and Drug Administration. Fast track, breakthrough therapy, accelerated approval, priority review 2015 (updated September 14, 2015). http://www.fda.gov/forpatients/approvals/fast/ucm20041766.htm (accessed April 26, 2017).Google Scholar
28. Fujiwara, Y. Evolution of frameworks for expediting access to new drugs in Japan. Nat Rev Drug Discov. 2016;15:293–294.Google Scholar
29. Guidance for the Public, Industry, and CMS Staff: Coverage with Evidence Development (updated November 20, 2014). https://www.cms.gov/medicare-coverage-database/details/medicare-coverage-document-details.aspx?MCDId=27 (accessed April 26, 2017).Google Scholar
30. Hints and tips for companies considering a Patient Access Scheme (PAS) proposal in England. https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/217037/PAS-Good-Practice-Guidance.pdf (accessed April 26, 2017).Google Scholar
31. Framework for the introduction of a Managed Entry Scheme for submissions to the Pharmaceutical Benefits Advisory Committee. (updated February 23, 2011). http://www.pbs.gov.au/info/publication/factsheets/shared/framework-for-introduction-of-managed-entry-scheme-for-PBAC-submissions (accessed April 26, 2017).Google Scholar
32. FDA Commissioner announces Avastin decision: Drug not shown to be safe and effective in breast cancer patients (updated November 18, 2011). https://wayback.archive-it.org/7993/20170112232043/http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm280536.htm (accessed April 26, 2017).Google Scholar
33. Kim, C, Prasad, V. Cancer drugs approved on the basis of a surrogate end point and subsequent overall survival: An analysis of 5 years of US Food and Drug Administration Approvals. JAMA Int Med. 2015;175:1992–1994.CrossRefGoogle ScholarPubMed
34. Lewis, JRR, Kerridge, I, Lipworth, W. Coverage with evidence development and managed entry in the funding of personalized medicine: Practical and ethical challenges for oncology. J Clin Oncol. 2015;33:4112–4117.CrossRefGoogle ScholarPubMed
35. Australian Government Department of Health Post-market Review of the Life Saving Drugs Programme (LSDP) (updated May 19, 2015). http://www.pbs.gov.au/info/reviews/life-saving-drugs (accessed April 26, 2017).Google Scholar
Supplementary material: File

Pace et al supplementary material

Figure S1

Download Pace et al supplementary material(File)
File 21.5 KB
Supplementary material: File

Pace et al supplementary material

Table S1

Download Pace et al supplementary material(File)
File 13.9 KB
Supplementary material: File

Pace et al supplementary material

Table S2

Download Pace et al supplementary material(File)
File 17.9 KB