Main Article Content
rare diseases, orphan drugs, health technology assessment, price negotiations, formulary listing, Canada
A previous assessment of submissions for rare disorder drugs made to the Canadian Agency for Drugs and Technologies in Health (CADTH) found that, from 2012, all positive recommendations included criteria advocating a price reduction. Since 2016, CADTH and the pan-Canadian Pharmaceutical Alliance (pCPA), which conducts drug price negotiations with manufacturers for all public drug programs, have aligned their processes. This analysis examined drugs for rare and ultra-rare disorders (DRDs and DURDs)—prevalence of ?20 to >2 and ?2 per 100,000, respectively—with a completed pCPA negotiation or no negotiation between 2014 and 2018, together with their reimbursement recommendations and listings in public drug programs. A positive recommendation led to a successful price negotiation for 81.8% and 78.6% of the DRD and DURD submissions and a negative recommendation to no negotiation for 100.0% and 66.7%. Less than half the recommendations for DURDs reported before 2016 mentioned the need for a substantial price reduction, but this increased to 80% in those reported from 2016 onwards. A successful price negotiation led to listing in the majority of the public drug programs and a negative recommendation usually led to no listing. The CADTH-pCPA alignment is working for the governments who own and fund public drug programs but has yet to lead to coverage for all appropriate patients in all provinces. There is still a way to go to ensure that patients with unmet needs can access high-cost innovative medicines that alleviate suffering, prevent premature death, and/
or significantly improve their quality of life.
2. Rocchi A, Miller E, Hopkins RB, Goeree R. Common drug review recommendations: An evi-dence base for expectations? Pharmacoeconomics. 2012;30:229–46. https://doi.org/10.2165/115930 30-000000000-00000
3. Griffiths EA, Hendrich JK, Stoddart SDR, Walsh SCM. Acceptance of health technology assess-ment submissions with incremental cost-effective-ness ratios above the cost-effectiveness threshold. Clinicoecon Outcomes Res. 2015;7:463–76. https://doi.org/10.2147/CEOR.S87462
4. Rawson NSB. Drug safety: Problems, pitfalls and solutions in identifying and evaluating risk. Victoria, BC: Friesen Press, 2016.
5. Rawson NSB. Health technology assessment of new drugs for rare disorders in Canada: Impact of disease prevalence and cost. Orphanet J Rare Dis. 2017;12:59. https://doi.org/10.1186/s13023-017-0611-7
6. The pan-Canadian Pharmaceutical Alliance [Internet]. 2019 [cited 2020 Jan 22]. Available from: http://www.canadaspremiers.ca/pan-canadian-pharmaceutical-alliance
7. Husereau D, Dempster W, Blanchard A, Chambers J. Evolution of drug reimbursement in Canada: The pan-Canadian Pharmaceutical Alliance for new drugs. Value Health. 2014;17:888–94. https:// doi.org/10.1016/j.jval.2014.08.2673
8. Rawson NSB. Pan-Canadian pharmaceutical alli-ance: Another hurdle for Canadian patients to access new drugs? Can Health Policy [Internet]. Toronto: Canadian Health Policy Institute; 2016 [cited 2020 Jan 22]. Available from: https://www. canadianhealthpolicy.com/products/pan-canadi-an-pharmaceutical-alliance--another-hurdle-for-ca-nadian-patients-to-access-new-drugs-.html
9. pCPA Brand Process Guidelines [Internet]. 2018 [cited 2020 Jan 22]. Available from: http://www.can-adaspremiers.ca/wp-content/uploads/2018/11/ pCPA_Brand_Process_Guidelines.pdf
10. Rawson NSB. Pan-Canadian Pharmaceutical Alliance negotiations 2014–2018 [Internet]. Can Health Policy. Toronto: Canadian Health Policy Institute; 2019 [cited 2020 Jan 22]. Available from: https://www.canadianhealthpolicy.com/products/ pan-canadian-pharmaceutical-alliance-negotia-tions-2014-2018-.html
11. Orphanet: The portal for rare diseases and orphan drugs [Internet]. 2020 [cited 2020 Jan 22]. Available from: https://www.orpha.net/consor/ cgi-bin/Disease.php?lng=EN
12. Lee DK, Wong B. An Orphan Drug Framework (ODF) for Canada. J Popul Ther Clin Pharmacol. 2014;21:e42–6.
13. CADTH Common Drug Review (CDR) reports [Internet]. 2020 [cited 2020 Jan 22]. Available from: https://www.cadth.ca/about-cadth/what-we-do/ products-services/cdr/reports
14. Find a review (pCODR) [Internet]. 2020 [cited 2020 Jan 22]. Available from: https://www.cadth. ca/pcodr/find-a-review
15. The Canadian cystic fibrosis registry: 2017 annual data report [Internet]. 2018 [cited 2020 Jan 22]. Available from: https://www.cysticfibrosis.ca/ uploads/Registry%20Report%202017/2017%20 Registry%20Annual%20Data%20Report.pdf
16. CADTH Canadian Drug Expert Committee final recommendation [Internet]. Elosulfase alfa resub-mission. 2016 [cited 2020 Jan 22]. Available from: https://www.cadth.ca/sites/default/files/cdr/com-plete/SR0456_complete_Vimizim_May-26_16.pdf
17. CADTH Canadian Drug Expert Committee rec-ommendation [Internet]. Nusinersen. 2017 [cited 2020 Jan 22]. Available from: https://www.cadth. ca/sites/default/files/cdr/complete/SR0525_ Spinraza_complete_Dec_22_17.pdf
18. Rawson NSB. Canadian, European and United States new drug approval times now relatively similar. Regul Toxicol Pharmacol. 2018;96:121–6. https://doi.org/10.1016/j.yrtph.2018.05.002
19. Rawson NSB, Adams J. Do reimbursement recom-mendation processes used by government drug plans in Canada adhere to good governance princi-ples? Clinicoecon Outcomes Res. 2017;9:721–30. https://doi.org/10.2147/CEOR.S144695
20. Kovacs Burns K. Medicine delayed is medicine denied [Internet]. Toronto: Globe and Mail; 2003 [cited 2020 Jan 22]. Available from: https://www. theglobeandmail.com/opinion/medicine-de-layed-is-medicine-denied/article751859/
21. Woman with rare disorder appeals for coverage of costly life-saving drug [Internet]. Saint John, NB: CBC; 2019 [cited 2020 Jan 22]. Available from: https://www.cbc.ca/news/canada/new-brunswick/ spinal-muscular-atrophy-spinraza-drug-cover-age-1.5091148
22. Nagase FNI, Stafinski T, Sun J, Jhangri G, Menon D. Factors associated with positive and negative recommendations for cancer and non-cancer drugs for rare diseases in Canada. Orphanet J Rare Dis. 2019;14:127. https://doi.org/10.1186/ s13023-019-1104-7
23. Loirat C, Frémeaux-Bacchi V. Atypical hemolytic uremic syndrome. Orphanet J Rare Dis. 2011;6:60. https://doi.org/10.1186/1750-1172-6-60
24. CDEC final recommendation [Internet]. Eculizumab; 2013 [cited 2020 Jan 22]. Available from: https://www.cadth.ca/sites/default/files/cdr/ complete/cdr_complete_Soliris-aHUS_July-23-13.pdf
25. Eculizumab for treating atypical haemolytic ure-mic syndrome [Internet]. 2015 [cited 2020 Jan 22]. Available from: https://www.nice.org.uk/guidance/ hst1/chapter/5-Consideration-of-the-evidence
26. Remarks from the Honourable Jane Philpott, Minister of Health, to the Economic Club of Canada – May 16, 2017 [Internet]. 2017 [cited 2020 Jan 22]. Available from: https://www.canada. ca/en/health-canada/news/2017/05/economic_ club_ofcanada-may162017.html
27. Government of Canada announces changes to lower drug prices and lay the foundation for national pharmacare [Internet]. 2019 [cited 2020 Jan 22]. Available from: https://www.canada.ca/ en/health-canada/news/2019/08/government-of-canada-announces-changes-to-lower-drug-pric-es-and-lay-the-foundation-for-national-phar-macare.html
28. Rawson NSB, Lawrence D. New patented med-icine regulations in Canada: Updated case study of a manufacturer’s decision-making about reg-ulatory submission for a rare disorder treat-ment [Internet]. Can Health Policy. Toronto: Canadian Health Policy Institute; 2020 [cited 2020 Jan 22]. Available from: https://www.cana-dianhealthpolicy.com/products/new-patent-ed-medicine-regulations-in-canada--updat-ed-case-study-.html
29. Rawson NSB. Proposed new drug regulations will hurt all Canadians – and Ottawa has been warned [Internet]. Toronto: Financial Post; 2019 [cited 2020 Jan 22]. Available from: https://business.financialpost. com/opinion/proposed-new-drug-regulations-will-hurt-all-canadians-and-ottawa-has-been-warned# (accessed on January 22, 2020).
30. Rawson NSB. Ottawa’s move to cut drug prices may lead to fewer new launches, less R and D [Internet]. Toronto: Globe and Mail; 2019 [cited 2020 Jan 22]. Available from: https://www.theglobeandmail. c o m / o p i n i o n / a r t i c l e - o t t awa s - c h a n g e s -to-drug-pricing-will-hurt-some-canadians/#com-ments
31. Rawson NSB, Adams J. New rules will keep the best medications from desperate Canadians [Internet]. Toronto: Financial Post; 2019 [cited 2020 Jan 22]. Available from: https://business. financialpost.com/opinion/new-rules-will-keep-the-best-medications-from-desperate-canadians (accessed on January 22, 2020).
32. A prescription for Canada: Achieving pharmacare for all – final report of the Advisory Council on the Implementation of National Pharmacare [Internet]. 2019 [cited 2020 Jan 22]. Available from: https:// www.canada.ca/en/health-canada/corporate/about-health-canada/public-engagement/external-adviso-ry-bodies/implementation-national-pharmacare/ final-report.html#4.2.2
33. Rawson NSB. Canada’s national pharmacare plan: Reason for hope or more questions? [Internet]. CMAJ Blogs. 2019 [cited 2020 Jan 22].
Available from: https://cmajblogs.com/canadas-national-pharmacare-plan-reason-for-hope-or-more-questions
34. Rawson NSB, Adams J. Civil servants get the Rolls Royce of pharmacare while party leaders can’t even muster a decent plan [Internet]. Toronto: National Post; 2019 [cited 2020 Jan 22]. Available from: https://nationalpost.com/opinion/ opinion-civil-servants-get-the-rolls-royce-of-pharmacare-while-party-leaders-cant-even-mus-ter-a-decent-plan
35. Rawson NSB. National pharmacare: Equity, equality, affordability? [Internet]. Policy Options. 2019 [cited 2020 Jan 22]. Available from: https:// policyoptions.irpp.org/magazines/january-2019/ national-pharmacare-equity-equality-afford-ability
36. Richter T, Janoudi G, Amegatse W, Nester-Parr S. Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA sub-missions made to the CADTH CDR. Orphanet J Rare Dis. 2018;13:15. https://doi.org/10.1186/ s13023-018-0762-1
37. Wong-Rieger D. Why Canada needs a comprehen-sive rare disease strategy [Internet]. Policy Options. 2019 [cited 2020 Jan 22]. Available from: https:// policyoptions.irpp.org/magazines/may-2019/ why-canada-needs-a-comprehensive-rare-dis-ease-strategy