Durhane Wong-Rieger is the President & CEO of the Canadian Organization for Rare Disorders (CORD). In her first article for PharmaBoardroom, Durhane explains the changing landscape for access to rare disease drugs in Canada and the actors involved in getting there.

 

The irony of the government processes, which, on the one hand, could facilitate expedited access to necessary and promising DRDs and, on the other, allow prohibitive price controls that discourage entry of life-saving and life-enhancing therapies, including clinical trials and early submission.

 

For anyone not paying close attention, you may have missed Health Canada’s quiet release of an important webpage: “Canada’s regulatory approach for rare disease drugs: orphan drugs.1”  In 2017, when Health Canada unveiled its modernized Regulatory Review of Drugs and Devices (a.k.a. R2D2) and “pulled” the 2012 “draft” Canadian Orphan Drug Regulatory Framework, the rare disease community, including the Canadian Organization for Rare Disorders responded with initial disappointment and scepticism.  I think we called it the “kiss of death2.”  

 

So, why have we now become strong advocates for orphan drug review under R2D2?  First, experience to date with orphan drug submissions to Health Canada has been very positive, including early clinical advice to review clinical trials with small patient populations to expedited pathways.  Second, even though we lost the proposed option for aligned FDA/EMA/Health Canada “orphan designation”, there is, in fact, considerable alignment of Health Canada with the US Food and Drug Administration and the European Medicines Agency (and other regulators) in terms of data sharing and work sharing.  Third, Health Canada is offering aligned regulatory-health technology assessment reviews with the Canadian Agency for Drugs and Technologies in Health (CADTH) and l’Institut national d’excellence en santé et en services sociaux (INESSS) to expedite the process from regulatory approval to reimbursement recommendation.3

 

This brings us to a major new reason for optimism for Canadian orphan drug access, despite previous pushbacks from our HTA agencies and the public drug programs.  In October 2018, the provincial/territorial governments called for consultation on a newly released “Supplemental Process for Complex/Specialized Drugs (including Drugs for Rare Diseases)4.  To understand the significance of this announcement, one has to go back more than a dozen years, to 2006 when the Canadian governments created the first (and only) jointly funded program for one rare disease drug (actually two drugs for one disease, Fabry’s Disease).  At the same time, the governments committed to setting up an “expensive drugs for rare diseases” program, an initiative that vanished with a change in government.

 

Fast forward to September 2014, following continuous advocacy from the patient community, the provincial health ministers (finally) created the Expensive Drugs for Rare Diseases Working Group (EDRD WG) with a mandate to explore the management of rare disease drug therapies with evidence-based approaches.  That announcement was followed by four years of almost total “radio silence”, punctuated by numerous individual, family and patient group protests over denied or delayed access to life-saving and life-enhancing drugs.

 

So, why is the proposed supplemental process creating a buzz among patients and prescribers?  The “stated” primary objective is to implement a proactive, consistent, fair and transparent process … for the purpose of making responsive funding decisions.  As importantly, it recognizes the need for “modifications to the current national review process” for drugs based on criteria including disease severity, unmet needs, cost per patient, and disease prevalence.  Drugs may be submitted concurrently to Health Canada, CADTH, the Patented Medicine Prices Review Board (PMPRB), and the pan-Canadian Pharmaceutical Alliance (pCPA) to reduce overall submission review time. In recognizing the limitations of clinical trial data, the WG proposes the collection and assessment of real-world evidence (RWE) to address the evidence gap and to inform continued funding, including potential changes in funding criteria, price changes or renegotiations, or delisting.

 

Interestingly, the proposed Supplemental Process reinforces the recommendations for a “managed access” process to rare disease drugs outlined in Canada’s Rare Disease Strategy.  This comprehensive strategy was released by CORD in May 2015 following a year of multi-stakeholder deliberations.5

 

Sadly, the yin (optimism) offered by the proposed EDRD supplemental process is challenged by a serious yang (negativism), namely the proposed federal regulatory changes announced in June 2016.  These changes will allow the PMPRB to impose arbitrary (not evidence-based) and draconian (up to 60% to 90%) price reductions on certain new and current prescription medicines, especially those for rare diseases.6  CORD is particularly concerned that potential PMPRB reforms will create powerful disincentives for rare disease drugs to be available in Canada, or at least, not until they have been successfully stabilized in terms of pricing and use in other jurisdictions.  

Similarly, the government-funded pCPA, which negotiates the price of drugs paid by public drug plans, submitted a brief to the House of Commons’ Standing Committee, which was conducting special hearings on “Barriers to Access Treatment and Drugs for Canadians Affected by Rare Diseases and Disorders.” The pCPA, on behalf of the public drug plans, strongly endorsed the regulatory changes allowing for PMPRB strict price controls.  

 

The irony of the government processes, which, on the one hand, could facilitate expedited access to necessary and promising DRDs and, on the other, allow prohibitive price controls that discourage entry of life-saving and life-enhancing therapies, including clinical trials and early submission.

 

Nevertheless, CORD, on behalf of the rare disease patient community, chooses to be optimistic, focusing on the opportunities and advocating vociferously against the barriers.

 

References:

  1. https://www.canada.ca/en/health-canada/services/licences-authorizations-registrations-drug-health-products/regulatory-approach-drugs-rare-diseases.html
  2. Health Canada gives ‘kiss of death’ to planned policy for rare-disease drugs.  National Post, Oct 16 2017.  https://nationalpost.com/news/politics/health-canada-gives-kiss-of-death-to-planned-policy-for-rare-disease-drugs
  3. Health Canada Offers HTA-Aligned Reviews for Drugs and Biologics. Regulatory Focus, 25 June 2018. https://www.raps.org/news-and-articles/news-articles/2018/6/health-canada-offers-hta-aligned-reviews-for-drugs
  4. BIG NEWS! Provincial Working Group on Expensive Drugs for Rare Disease (EDRD) holding Consultations on a proposed “Supplemental Process” that could be a winner! https://www.raredisorders.ca/provincial-working-group/
  5. Now is the time: a strategy for rare diseases is a strategy for all Canadians.  http://www.raredisorders.ca//content/uploads/CORD_Canada_RD_Strategy_22May15.pdf
  6. Guidance document on changes to the Guidelines. Presentation to the Steering Committee – June 25, 2018.  http://www.pmprb-cepmb.gc.ca/view.asp?ccid=1376