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McMillan and Campbell believe that Canada needs a regulatory framework for the introduction of orphan drugs for rare disorders (DRDs) [1]. We agree. However, the need is unlikely to be filled in the foreseeable future because, in October 2017, the present federal government deleted, without notice or consultation, all references to the previous government’s 2012 Orphan Drug Regulatory Framework [2].
Access to a new DRD not only depends on its regulatory approval but also on the drug successfully passing the Canadian Agency for Drugs and Technologies in Health’s (CADTH’s) Common Drug Review (CDR) process, negotiations regarding price between the pan-Canadian Pharmaceutical Alliance (pCPA) and the manufacturer, the Patented Medicine Prices Review Board price assessment process, and individual drug plan evaluations and negotiations with the manufacturer.
Although CADTH has considered establishing a focused review process for DRDs, it chose to integrate their review into the usual CDR process. Nevertheless, Richter et al. from CADTH recently analyzed CDR recommendations for DRDs made between 2004 and 2016 and suggested that it may be inappropriate to apply the same appraisal standards to drugs for ultra-rare disorders (those with a prevalence of ≤1 per 100,000) [3].
A different appraisal standard is appropriate for all DRDs, but the likelihood of one being created by CADTH is low. CADTH’s earlier lack of interest in establishing a separate DRD review process and the recent integration of CADTH and the pCPA in which they attend each other’s meetings and share confidential information about manufacturers’ submissions [4], which leads to a negative reimbursement recommendation resulting in no pCPA negotiation and a positive one setting up negotiating factors (usually a substantial price reduction) [5], indicate that the government public drug plans which own, fund and manage CADTH and the pCPA [6] are not in favour of a separate DRD appraisal standard.
Canadian government officials are instead focused on a mantra of “affordability, accessibility and appropriate use of prescription drugs” [7]. The federal government is expanding the already extensive deterrents to the introduction of new drugs into Canada with a planned 77% increase in the regulatory review fee for new drugs [8] and sweeping changes to the Patented Medicines Regulations [9,10], which will delay or deny patient access to new drugs, especially DRDs. Rather than providing hope to patients needing DRDs, Canadian governments appear to be moving towards a basic “pharmacare” system built on a formulary of inexpensive genericized drugs and a relatively small, restricted access list of expensive drugs, including DRDs, available from manufacturers willing to negotiate substantial price reductions [11,12].
Canadian governments and their associated organizations should instead be developing inventive and coherent nationwide policies to balance timely and fair access to DRDs with appropriately competitive pricing negotiations so that drugs are accessible to Canadians who need them. Affordability and accessibility should be implemented together, not as one or the other. Simply erecting more barriers to deter or delay pharmaceutical manufacturers from bringing new DRDs to Canada fails to improve the lives of patients and is not the solution to high drug prices.
References
1. McMillan HJ, Campbell C. We need a “made in Canada” orphan drug framework. CMAJ 2017; 189:E1274-5.
2. Forrest M. Health Canada gives ‘kiss of death’ to planned policy for rare-disease drugs. National Post 2017 Oct 16. Available http://nationalpost.com/news/politics/health-canada-gives-kiss-of-death-... (accessed 1 Mar 2018).
3. Richter T, Janoudi G, Amegatse W, Nester-Parr S. Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR. Orphanet J Rare Dis 2018; 13:15.
4. CDR Update – Issue 119. Ottawa: CADTH, 2016. Available https://www.cadth.ca/cdr-update-issue-119 (accessed 1 Mar 2018).
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.
6. Rawson NSB, Adams J. Do reimbursement recommendation processes used by government drug plans in Canada adhere to good governance principles? Clinicoecon Outcomes Res 2017; 9:721-30.
7. Remarks from the Honourable Jane Philpott, Minister of Health, to the Economic Club of Canada – May 16, 2017. Ottawa: Government of Canada, 2017. Available https://www.canada.ca/en/health-canada/news/2017/05/economic_club_ofcana... (accessed 1 Mar 2018).
8. Fee proposal for drugs and medical devices (for consultation). Ottawa: Government of Canada, 2017. Available https://www.canada.ca/en/health-canada/programs/consultation-fee-proposa... (accessed 1 Mar 2018).
9. Regulations amending the Patented Medicines Regulations. Ottawa: Government of Canada, 2017. Available http://canadagazette.gc.ca/rp-pr/p1/2017/2017-12-02/html/reg2-eng.html (accessed 1 Mar 2018).
10. Rawson NSB. Ottawa’s revisions to drug-pricing review board will delay or prevent access to new drugs. Fraser Institute, Vancouver, 2018. Available https://www.fraserinstitute.org/article/ottawas-revisions-to-drug-pricin... (accessed 1 Mar 2018).
11. Rawson NSB. What does the new Ontario pharmacare plan offer children and young adults with rare disorders? J Popul Ther Clin Pharmacol 2017; 24:90-8.
12. Harris K. Pharmacare won’t be free for all Canadians, Bill Morneau says. CBC News 2018 Feb 28. Available http://www.cbc.ca/news/politics/morneau-budget-2018-pharmacare-1.4555186 (accessed 1 Mar 2018).