Regulatory approval and public drug plan listing of new drugs for rare disorders in Canada and New Zealand
Main Article Content
Keywords
Canada, drugs, formulary listing, New Zealand, rare disorders, regulatory approval
Abstract
A previous assessment of the alignment of health technology assessments and price negotiations for new drugs for rare disorders in Canada completed between 2014 and 2018 demonstrated that it is working for governments but has yet to lead to improved access in a timely manner for all appropriate patients in all provinces. In this analysis, drugs for rare and ultra-rare disorders with a completed price negotiation or no negotiation between 2014 and 2018 in Canada, and their reimbursement recommendations and listings in Canadian public drug programs are compared with their regulatory approval in New Zealand and listing in the New Zealand National Formulary. The results show that pharmaceutical manufacturers generally seek regulatory approval for rare disorder drugs in Canada before New Zealand, and fewer rare disorder medicines receive regulatory approval in New Zealand. One reason for this difference might be New Zealand’s smaller population. However, another reason is likely the restrictive drug formulary in New Zealand. Drugs not given coverage in New Zealand are frequently made unavailable by the manufacturer. Planned changes to Canada’s pricing regulations and guidelines will significantly diminish the country’s attractiveness as a place in which pharmaceutical companies want to do business, which has the potential to negatively impact the health of all Canadians irrespective of whether they have private or public drug coverage.
References
1. Remarks from the Honourable Jane Philpott, Minister of Health, to the Economic Club of Canada – May 16, 2017. Ottawa: Government of Canada; 2017 [cited 2020 Apr 16]. Available from: https:// www.canada.ca/en/health-canada/news/2017/05/ economic_club_ofcanada-may162017.html
2. Patented Medicine Prices Review Board. Mandate and jurisdiction. Ottawa: Government of Canada, 2018 [cited 2020 Apr 16]. Available from: http:// pmprb-cepmb.gc.ca/about-us/mandate-and-jurisdiction
3. Patented Medicine Prices Review Board. PMPRB Guidelines 2019. Ottawa: Government of Canada, 2019 [cited 2020 Apr 16]. Available from: https:// www.canada.ca/content/dam/pmprb-cepmb/docu-ments/consultations/draft-guidelines/draft-guide-lines-en.pdf
4. Government of Canada. Regulations amending the patented medicines regulations (additional fac-tors and information reporting requirements): SOR/2019-298. Canada Gazette 2019 [cited 2020 Apr 16];II;153. Available from: http://www.gazette. gc.ca/rp-pr/p2/2019/2019-08-21/html/sor-dors298-eng.html
5. Rawson NSB. Alignment of health technology assessments and price negotiations for new drugs for rare disorders in Canada: Does it lead to improved patient access? J Popul Ther Clin Pharmacol. 2020 [cited 2020 Apr 16];27:e48–64. Available from: https://jptcp.com/index.php/ jptcp/article/view/658/618
6. Rawson NSB. Regulatory, reimbursement and pricing barriers to accessing drugs for rare disor-ders in Canada. Vancouver: Fraser Institute; 2018 [cited 2020 Apr 16]. Available from: https://www.fraserinstitute.org/sites/default/files/ barriers-to-accessing-drugs-for-rare-disorders-in-canada.pdf
7. Taylor C, Wonder M. Exploring the implications of a fixed budget for medicines: A study of reim-bursement for medicines in Australia and New Zealand. Aust Health Rev. 2015;39:455–61. https://doi.org/10.1071/AH14122
8. Comparison of access and reimbursement envi-ronments 4. Deakin, ACT: Medicines Australia; 2018 [cited 2020 Apr 16]. Available from: https:// medicinesaustralia.com.au/wp-content/uploads/ sites/52/2018/10/MA_Compare-final.pdf (accessed April 16, 2020)
9. Rawson NSB. Canadian, European and United States new drug approval times now relatively sim-ilar. Regul Toxicol Pharmacol. 2018;96:121–6. https://doi.org/10.1016/j.yrtph.2018.05.002
10. Rawson NSB. Clinical trials in Canada decrease: A sign of uncertainty regarding changes to the PMPRB? Can Health Policy. Toronto: Canadian Health Policy Institute; 2020 [cited 2020 Apr 16]. Available from: https://www.canadianhealthpol-icy.com/products/clinical-trials-in-canada-de-crease--a-sign-of-uncertainty-regarding-changes-to-the-pmprb-.html
11. Rawson NSB. Fewer new drug approvals in Canada: Early indications of unintended conse-quences from new patented medicines regulations. Can Health Policy. Toronto: Canadian Health Policy Institute; 2020 [cited 2020 Apr 16]. Available from: https://www.canadianhealthpol-icy.com/products/fewer-new-drug-approv-als-in-canada--early-indications-of-unintend-ed-consequences-from-new-pmprb-regs.html
12. Rawson NSB, Lawrence D. New patented medi-cine regulations in Canada: Updated case study of a manufacturer’s decision-making about regu-latory submission for a rare disorder treatment. Can Health Policy. Toronto: Canadian Health Policy Institute; 2020 [cited 2020 Apr 16]. Available from: https://www.canadianhealthpol-icy.com/products/new-patented-medicine-regula-tions-in-canada--updated-case-study-.html
13. Kelley LT, Tenbensel T, Johnson A. Ontario and New Zealand pharmaceuticals: Cost and coverage. Healthc Policy. 2018;13:23–34. https://doi.org/ 10.12927/hcpol.2018.25496
14. Morgan S, Hanley G, McMahon M, Barer M. Influencing drug prices through formulary-based policies: Lessons from New Zealand. Healthc Policy. 2007;3:e121–40. https://doi.org/10.12927/ hcpol.2007.19097
15. PHARMAC. Our place in the health system. Wellington: New Zealand Government; 2017 [cited 2020 Apr 16]. Available from: https://www. pharmac.govt.nz/about/your-guide-to-pharmac/ factsheet-03-our-place-in-the-health-system
16. Rawson NSB. Pan-Canadian Pharmaceutical Alliance negotiations 2014–2018. Can Health Policy. Toronto: Canadian Health Policy Institute; 2019 [cited 2020 Apr 16]. Available from: https:// www.canadianhealthpolicy.com/products/pan-ca-nadian-pharmaceutical-alliance-negotia-tions-2014-2018-.html
17. CADTH Common Drug Review (CDR) reports. Ottawa: CADTH; 2019 [cited 2020 Apr 16]. Available from: https://www.cadth.ca/about-cadth/ what-we-do/products-services/cdr/reports
18. Find a review (pCODR). Ottawa: CADTH; 2019 [cited 2020 Apr 16]. Available from: https://www. cadth.ca/pcodr/find-a-review
19. Medsafe. Medicines: Product/application search. Wellington: New Zealand Government; 2018 [cited 2020 Apr 16]. Available from: http://www. medsafe.govt.nz/regulatory/DbSearch.asp
20. Medsafe. Medicines: Registration situation. Wellington: New Zealand Government; 2013 [cited 2020 Apr 16]. Available from: http://www.medsafe. govt.nz/Medicines/registration-situation.asp
21. PHARMAC. Pharmaceutical schedule. Wellington: New Zealand Government; 2018 [cited 2020 Apr 16]. Available from: https://www.pharmac.govt.nz/ tools-resources/pharmaceutical-schedule
22. Rawson NSB. Comparison of numbers and timing of new medication regulatory approvals in Canada and New Zealand. Regul Toxicol Pharmacol. 2019;101:24–8. https://doi.org/10.1016/ j.yrtph.2018.10.019
23. Rawson NSB. What does the new Ontario phar-macare plan offer children and young adults with rare disorders? J Popul Ther Clin Pharmacol. 2017;24:90–8. https://doi.org/10.22374/1710-6222. 24.3.8
24. Gauld R. Ahead of its time? Reflecting on New Zealand’s Pharmac following its 20th anniversary. Pharmacoeconomics. 2014;32:937–42. https://doi. org/10.1007/s40273-014-0178-2
25. Ragupathy R, Aaltonen K, Tordoff J, Norris P, Reith D. A 3-dimensional view of access to licensed and subsidized medicines under single-payer systems in the US, the UK, Australia and New Zealand. Pharmacoeconomics. 2012;30:1051–65. https:// doi.org/10.2165/11595270-000000000-00000
26. Burks M, Stickel S, Galiè N. Pulmonary arterial hypertension: combination therapy in practice. Am J Cardiovasc Drugs. 2018;18:249–57. https:// doi.org/10.1007/s40256-018-0272-5
27. Wonder M, Milne R. Access to new medicines in New Zealand compared to Australia. NZ J Med. 2011;124(1346):12–28. [cited 2020 Apr 16]. Available from: https://global-uploads.webflow. com/5e332a62c703f653182faf47/5e332a62c70 3f6366f2fdee1_content.pdf
28. Della Barca C. Funding medicines in New Zealand: Revision of the medicines waiting list 2019. Auckland: SubscRipts Ltd; 2019 [cited 2020 Apr 16]. Available from: https://www.medicinesnz. co.nz/fileadmin/user_upload/Medicines_New_ Z e a l a n d _ Wa i t i n g _ L i s t _ Re p o r t _ t o _ 3 0 _ June_2019_Published_Sept_2019.pdf
29. Impact of PMPRB pricing changes: Final research report. Toronto: Life Sciences Ontario; 2020 [cited 2020 Apr 16]. Available from: https://lifescienc-esontario.ca/wp-content/uploads/2020/02/ Research-Etc.-PMPRB-Survey-02-03-20.pdf
2. Patented Medicine Prices Review Board. Mandate and jurisdiction. Ottawa: Government of Canada, 2018 [cited 2020 Apr 16]. Available from: http:// pmprb-cepmb.gc.ca/about-us/mandate-and-jurisdiction
3. Patented Medicine Prices Review Board. PMPRB Guidelines 2019. Ottawa: Government of Canada, 2019 [cited 2020 Apr 16]. Available from: https:// www.canada.ca/content/dam/pmprb-cepmb/docu-ments/consultations/draft-guidelines/draft-guide-lines-en.pdf
4. Government of Canada. Regulations amending the patented medicines regulations (additional fac-tors and information reporting requirements): SOR/2019-298. Canada Gazette 2019 [cited 2020 Apr 16];II;153. Available from: http://www.gazette. gc.ca/rp-pr/p2/2019/2019-08-21/html/sor-dors298-eng.html
5. Rawson NSB. Alignment of health technology assessments and price negotiations for new drugs for rare disorders in Canada: Does it lead to improved patient access? J Popul Ther Clin Pharmacol. 2020 [cited 2020 Apr 16];27:e48–64. Available from: https://jptcp.com/index.php/ jptcp/article/view/658/618
6. Rawson NSB. Regulatory, reimbursement and pricing barriers to accessing drugs for rare disor-ders in Canada. Vancouver: Fraser Institute; 2018 [cited 2020 Apr 16]. Available from: https://www.fraserinstitute.org/sites/default/files/ barriers-to-accessing-drugs-for-rare-disorders-in-canada.pdf
7. Taylor C, Wonder M. Exploring the implications of a fixed budget for medicines: A study of reim-bursement for medicines in Australia and New Zealand. Aust Health Rev. 2015;39:455–61. https://doi.org/10.1071/AH14122
8. Comparison of access and reimbursement envi-ronments 4. Deakin, ACT: Medicines Australia; 2018 [cited 2020 Apr 16]. Available from: https:// medicinesaustralia.com.au/wp-content/uploads/ sites/52/2018/10/MA_Compare-final.pdf (accessed April 16, 2020)
9. Rawson NSB. Canadian, European and United States new drug approval times now relatively sim-ilar. Regul Toxicol Pharmacol. 2018;96:121–6. https://doi.org/10.1016/j.yrtph.2018.05.002
10. Rawson NSB. Clinical trials in Canada decrease: A sign of uncertainty regarding changes to the PMPRB? Can Health Policy. Toronto: Canadian Health Policy Institute; 2020 [cited 2020 Apr 16]. Available from: https://www.canadianhealthpol-icy.com/products/clinical-trials-in-canada-de-crease--a-sign-of-uncertainty-regarding-changes-to-the-pmprb-.html
11. Rawson NSB. Fewer new drug approvals in Canada: Early indications of unintended conse-quences from new patented medicines regulations. Can Health Policy. Toronto: Canadian Health Policy Institute; 2020 [cited 2020 Apr 16]. Available from: https://www.canadianhealthpol-icy.com/products/fewer-new-drug-approv-als-in-canada--early-indications-of-unintend-ed-consequences-from-new-pmprb-regs.html
12. Rawson NSB, Lawrence D. New patented medi-cine regulations in Canada: Updated case study of a manufacturer’s decision-making about regu-latory submission for a rare disorder treatment. Can Health Policy. Toronto: Canadian Health Policy Institute; 2020 [cited 2020 Apr 16]. Available from: https://www.canadianhealthpol-icy.com/products/new-patented-medicine-regula-tions-in-canada--updated-case-study-.html
13. Kelley LT, Tenbensel T, Johnson A. Ontario and New Zealand pharmaceuticals: Cost and coverage. Healthc Policy. 2018;13:23–34. https://doi.org/ 10.12927/hcpol.2018.25496
14. Morgan S, Hanley G, McMahon M, Barer M. Influencing drug prices through formulary-based policies: Lessons from New Zealand. Healthc Policy. 2007;3:e121–40. https://doi.org/10.12927/ hcpol.2007.19097
15. PHARMAC. Our place in the health system. Wellington: New Zealand Government; 2017 [cited 2020 Apr 16]. Available from: https://www. pharmac.govt.nz/about/your-guide-to-pharmac/ factsheet-03-our-place-in-the-health-system
16. Rawson NSB. Pan-Canadian Pharmaceutical Alliance negotiations 2014–2018. Can Health Policy. Toronto: Canadian Health Policy Institute; 2019 [cited 2020 Apr 16]. Available from: https:// www.canadianhealthpolicy.com/products/pan-ca-nadian-pharmaceutical-alliance-negotia-tions-2014-2018-.html
17. CADTH Common Drug Review (CDR) reports. Ottawa: CADTH; 2019 [cited 2020 Apr 16]. Available from: https://www.cadth.ca/about-cadth/ what-we-do/products-services/cdr/reports
18. Find a review (pCODR). Ottawa: CADTH; 2019 [cited 2020 Apr 16]. Available from: https://www. cadth.ca/pcodr/find-a-review
19. Medsafe. Medicines: Product/application search. Wellington: New Zealand Government; 2018 [cited 2020 Apr 16]. Available from: http://www. medsafe.govt.nz/regulatory/DbSearch.asp
20. Medsafe. Medicines: Registration situation. Wellington: New Zealand Government; 2013 [cited 2020 Apr 16]. Available from: http://www.medsafe. govt.nz/Medicines/registration-situation.asp
21. PHARMAC. Pharmaceutical schedule. Wellington: New Zealand Government; 2018 [cited 2020 Apr 16]. Available from: https://www.pharmac.govt.nz/ tools-resources/pharmaceutical-schedule
22. Rawson NSB. Comparison of numbers and timing of new medication regulatory approvals in Canada and New Zealand. Regul Toxicol Pharmacol. 2019;101:24–8. https://doi.org/10.1016/ j.yrtph.2018.10.019
23. Rawson NSB. What does the new Ontario phar-macare plan offer children and young adults with rare disorders? J Popul Ther Clin Pharmacol. 2017;24:90–8. https://doi.org/10.22374/1710-6222. 24.3.8
24. Gauld R. Ahead of its time? Reflecting on New Zealand’s Pharmac following its 20th anniversary. Pharmacoeconomics. 2014;32:937–42. https://doi. org/10.1007/s40273-014-0178-2
25. Ragupathy R, Aaltonen K, Tordoff J, Norris P, Reith D. A 3-dimensional view of access to licensed and subsidized medicines under single-payer systems in the US, the UK, Australia and New Zealand. Pharmacoeconomics. 2012;30:1051–65. https:// doi.org/10.2165/11595270-000000000-00000
26. Burks M, Stickel S, Galiè N. Pulmonary arterial hypertension: combination therapy in practice. Am J Cardiovasc Drugs. 2018;18:249–57. https:// doi.org/10.1007/s40256-018-0272-5
27. Wonder M, Milne R. Access to new medicines in New Zealand compared to Australia. NZ J Med. 2011;124(1346):12–28. [cited 2020 Apr 16]. Available from: https://global-uploads.webflow. com/5e332a62c703f653182faf47/5e332a62c70 3f6366f2fdee1_content.pdf
28. Della Barca C. Funding medicines in New Zealand: Revision of the medicines waiting list 2019. Auckland: SubscRipts Ltd; 2019 [cited 2020 Apr 16]. Available from: https://www.medicinesnz. co.nz/fileadmin/user_upload/Medicines_New_ Z e a l a n d _ Wa i t i n g _ L i s t _ Re p o r t _ t o _ 3 0 _ June_2019_Published_Sept_2019.pdf
29. Impact of PMPRB pricing changes: Final research report. Toronto: Life Sciences Ontario; 2020 [cited 2020 Apr 16]. Available from: https://lifescienc-esontario.ca/wp-content/uploads/2020/02/ Research-Etc.-PMPRB-Survey-02-03-20.pdf
Article Sidebar
Published
Jun 1, 2020
Article Details
How to Cite
B. Rawson, N. S. (2020). Regulatory approval and public drug plan listing of new drugs for rare disorders in Canada and New Zealand. Journal of Population Therapeutics and Clinical Pharmacology, 27(2), e58-e67. https://doi.org/10.15586/jptcp.v27i2.673
Section
Articles
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
All articles published in JPTCP are licensed under Copyright Creative Commons Attribution-NonCommercial 4.0 International License.