Pulling the Plug on Orphan Drug Policy

Pulling the Plug on Orphan Drug Policy

“Deeply rooted in this absence of pharmacare coverage in its universal healthcare system, the lack of an effective policy addressing medical treatments for patients afflicted with rare diseases in Canada stems back as far as the 1960s.”

Canada is known internationally for its universal healthcare. It is interesting, then, to consider that many citizens are marginalized from the healthcare system we pride ourselves in. The void created by lack of an effective and universal strategy for prescription drugs has long been considered Canada’s greatest fault, and coverage for rare disease patients is almost entirely nonexistent. As such, the resounding effects of the recent pull of Canada’s 2012 Orphan Drug Regulatory Framework Proposal may be felt much farther than just in the pockets of families affected by rare diseases. Deeply rooted in this absence of pharmacare coverage in its universal healthcare system, the lack of an effective policy addressing medical treatments for patients afflicted with rare diseases in Canada stems back as far as the 1960s.1

In 2005, the Canadian Organization for Rare Diseases (CORD) released a paper regarding its position on the necessity of an effective policy for orphan drugs in Canada, emphasizing that Canadians risked being “the last of the developed countries” to have such legislation in place.2 The skyrocketing costs associated with rare diseases have been a consistent area of concern since the 1990s and was the primary problem the 2012 proposal sought to resolve.

What was the proposal and why was it removed? Health Canada’s Office of Legislative and Regulatory Modernization (OLRM) released its December 2012 draft as a means of acknowledging the need for regulation, but highlighted the multifaceted nature of the issue. Drawing upon already-existing provisions, primarily the Food and Drug Act and its regulations, the proposal sought to weave in new content regarding orphan drug designation, market authorization and overall transparency in regulation. Highlighting that patients’ “perspective on the severity of the disease or unmet medical need in a therapeutic area” would lend greatly to the government’s understanding of regulatory responsibilities, it emphasized the need for proper patient representation. All traces of this proposal were removed from Health Canada’s webpage on October 6, 2017.3

It is unnerving to consider that the commitment to the 2012 Orphan Drug Regulatory Proposal was removed entirely from Health Canada’s regulatory plans almost five years later with no indication of replacement. More alarmingly, the fact that it was to be implemented between late 2017-2019 casts a dark shadow on future endeavours and families struggling with the burden of orphan drugs. In an open letter to the Canadian Government released on October 12, 2017, CORD President Durhane Wong-Rieger re-identified Canada as an “outlier among developed and developing countries in adopting a national approach to rare diseases” and stressed the need for explanation and action.

The pull of the 2012 policy brings to light a whole slew of new questions and holes in Canada’s drug regulatory programs - particularly those pertaining to the National Pharmaceutical Strategy and lack of prescription drug coverage within our universal healthcare system. The fact that much of negotiation regarding pricing is province-driven, where each province is left to negotiate its own terms with major pharmaceutical companies, creates a situation in which patients end up paying hugely inflated prices.4 According to 2016 reports by the Patented Medicine Prices Review Board (PMPRB), Canadians pay a 20% markup on prescription medication due to lack of federal initiative with bulk purchasing.5 As such, 1 in 10 Canadians find themselves unable to fill their prescriptions.1 This underlying issue of common prescription costs raises an even bigger concern - is it possible to implement an effective orphan drug regulation policy in good faith when no solid foundation exists for it to be built upon?

Outside of costs for the consumer, many pharmaceutical companies are found to completely disregard the thought of seeking approval for their treatments in Canada due to a lack of demand. As such, many families afflicted by rare diseases find themselves travelling across the border to seek clinical trials that Canadian healthcare cannot offer or support financially.6 Medical practitioners also find themselves prescribing off-label when dealing with rare diseases.7

Joining the pan-Canadian Pharmaceutical Alliance (pCPA) in 2010 was the federal government’s means of tackling rising prescription drug prices through the universalization of some drugs. With 170 successful joint negotiations with manufacturers for brand-name medications reported on October 11, 2017, patients can receive coverage for these medications under their province’s public health plan.8 However, it does not appear that drugs targeting rare diseases will be in negotiation talks any time soon.

It is interesting to consider the effects the pull has on other outlets for combatting rare diseases - particularly as it applies to rare disease research funding and goals. The Canadian Institutes of Health Research (CIHR) recently committed to supporting a new decade of rare disease research, supporting a recent press release by the International Rare Diseases Research Consortium which called for rare disease patients to receive proper care and medication within a year of diagnosis.9 Rare disease research is also listed as the Genetic Institutes priority topic, which has functioned on $1.2 billion dollars of approved grants since 2004. However, without a tangible framework to implement the results of this research (such as treatment and medications), it is difficult to believe that supporting rare disease research is a prime concern for the federal government.

When the prospect of treatment becomes so daunting, the appeal of effective genetic counselling and even gene editing becomes exponentially more appealing, particularly when addressing potentially detrimental rare diseases. While the current Assisted Human Reproduction Act explicitly states that all editing to a human embryo is prohibited, thoughts on human germline editing have shifted within recent years. In its December 2016 Points of Consideration, the CIHR described less opposition towards human germline editing depending on its goals, such as disease prevention (particularly rare diseases). However, while the thought may manifest as an option, there is still a long way to go before it can become a reality in Canada.


“If Canada hopes to continue embodying the ‘Medicare for everyone’ mentality for which it prides itself on having, taking steps towards resolving this gaping hole in policy regarding pharmaceuticals and orphan drugs must be set as a priority and acted upon immediately.”

At the end of the day, orphan drug regulation and the surging prices of pharmaceuticals overall is an issue for all Canadians, not just those with a rare disease. Without a solidified plan of action to replace its 2012 policy proposal, the federal government can expect that families affected by rare diseases will be alienated further financially. And while there may not be one perfect solution to rectify the situation, as patient advocates and fellow citizens it is important to maintain ongoing conversations surrounding prescription drug pricing and how debilitating the high costs of orphan drugs can be. If Canada hopes to continue embodying the “Medicare for everyone” mentality for which it prides itself on having, taking steps towards resolving this gaping hole in policy regarding pharmaceuticals and orphan drugs must be set as a priority and acted upon immediately.


Works Cited:

1. Morgan SG, Boothe K. Universal prescription drug coverage in Canada: Long-promised yet undelivered. Healthcare Management Forum. 2016;29(6):247-254. doi:10.1177/0840470416658907.

2. Canada’s Orphan Drug Policy. Canadian Organization for Rare Disorders. https://www.raredisorders.ca/content/uploads/CanadaOrphanDPFinal-copy.pdf. Published July 6, 2005.

3. Forrest M. Health Canada gives ‘kiss of death’ to planned policy for rare-disease drugs. National Post. http://nationalpost.com/news/politics/health-canada-gives-kiss-of-death-to-planned-policy-for-rare-disease-drugs. Published October 16, 2017.

4. Lynd L, Klein P. Why Canada needs a clear policy on orphan drugs. The Globe and Mail. https://theglobeandmail.com/opinion/why-canada-needs-a-clear-policy-on-orphan-drugs/article28935317/. Published February 29, 2016.

5. Canadian drug price gouging for generics called ‘hard to celebrate’. CBC News. http://www.cbc.ca/news/health/drug-prices-generic-1.3441080. Published February 9, 2016.

6. Spurr B, Woods A. Canadian families pushing for a rare diseases national strategy. Toronto Star. https://www.thestar.com/news/canada/2016/01/20/canadian-families-pushing-for-a-rare-diseases-national-strategy.html. Published January 20, 2016.

7. Herder M, Krahn T. Some Numbers behind Canada’s Decision to Adopt an Orphan Drug Policy: US Orphan Drug Approvals in Canada, 1997–2012. Healthcare Policy | Politiques de Santé. 2016;11(4):70-81. doi:10.12927/hcpol.2016.24626.

8. The pan-Canadian Pharmaceutical Alliance (pCPA): Negotiations Status Update. PDCI Market Access. http://www.pdci.ca/the-pan-canadian-pharmaceutical-alliance-pcpa-negotiations-status-update-12/. Published October 11, 2017.

9. IRDiRC Goals 2017-2027: New rare disease research goals for the next decade. The International Rare Diseases Research Consortium. August 2017. http://www.irdirc.org/wp-content/uploads/2017/08/IRDiRC_Goals-2017-2027-Press-Release_20170809.pdf.


Cite This Article:

Rong M., Chan G., Palczewski K., Lewis K., Ho J. Pulling the Plug on Orphan Drug Policy. Illustrated by C. Nguyen. Rare Disease Review. July 2018. DOI:10.13140/RG.2.2.14330.18880.

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