Our Position Paper

In June 2020 we started researching and writing a Position Paper which surveyed the ALS landscape in Canada and then developed draft recommendations to tangibly improve the lives of Canadians diagnosed with ALS. We believe this was the first time a review like this has ever been undertaken in Canada. Next we circulated the paper to stakeholders and experts to obtain their feedback. We then finalized our paper which can be found here. The Position Paper laid the foundation for Our Plan below.

A Time of Unprecedented Hope

In recent years, the ALS story has changed. It is no longer just about wheelchairs and medically-assisted dying. It now includes unprecedented hope given that:

  • there are over 160 drug companies worldwide working in the ALS space.
  • there are ~80 ALS clinical trials going on worldwide including several in their final stages.
  • in September 2020, Amylyx Pharmaceuticals announced that its drug, AMX0035, slowed the progression of ALS in a clinically meaningful and statistically significant way in a Phase 2/3 trial.
  • in November 2020, Brainstorm Cell Therapeutics announced that its stem cell therapy, NurOwn, produced positive results in a sub-group of ALS patients in a Phase 3 trial and also resulted in positive bio-marker data. One member of our group, who participated in the trial, believed it slowed down and even temporarily reversed his ALS progression.
  • there has been a breakthrough therapy in the fight against genetic ALS. Chris Snow, the Assistant General Manager for the Calgary Flames, publicaly announced in December 2019 that he had been diagnosed with ALS in June 2019. Chris has a type of genetic ALS that affects 2 percent of ALS patients and that had already killed his father, two uncles and his 28 year-old cousin. The average life expectancy for this type of ALS is 6 to 18 months. Almost immediately after his diagnosis Chris enrolled in a Phase 3 clinical trial at Sunnybrook Health Sciences Centre in Toronto for those with his type of genetic ALS. Chris has since been able to participate in this same clinical trial out of Calgary. The therapy, tofersen by Biogen, is working in that Chris’ ALS progression has essentially stopped since he has been in the trial.
  • there are new, innovative clinical trial formats being initiated, e.g., the HEALEY ALS Platform Trial (see below).
  • there are new, bold, patient-led advocacy organizations demanding action, not just awareness, e.g. I AM ALS.
  • there has been unprecedented legislative action in the US focused on ALS treatments and research.

The Canadian Reality

The reality, however, is that this activity has been largely happening outside of Canada and the current Canadian drug approval process can take two or more years which is longer than the expected lifespan of an ALS patient.

In other words, if a scientifically validated cure is made available in the US tomorrow, and a Canadian is diagnosed with ALS the following week, that Canadian may not survive to access the therapy. They will most certainly have to pay for it out of pocket or through private / employer insurance and if these options are not available, they will, in all probability, die before provincial coverage is made available.

Our Plan

As such, we have developed the following plan to allow Canadians with ALS a chance to live:

  1.  Secure immediate access to new therapies that get approved in other countries. In order to achieve this we are focused on:

A. Project Orbis

      • Canada is a signatory to several international reciprocal agreements, treaties, and conventions that serve as precedents for an ALS focused-agreement that would fast track the approval process in Canada for new ALS therapies.
      • The best example we found is Project Orbis, which was initiated in September 2019 and involves Health Canada, the USFDA Oncology Center of Excellence (OCE), the Australian Therapeutic Goods Administration, Singapore’s Health Sciences Authority (HSA) and Swissmedic. It provides a framework for the concurrent submission, review and approval of oncology products among these countries.
      • The first therapy approved under Project Orbis occurred in April 2020 allowing the immediate use of the treatment in all signatory countries.
      • Applying this type of framework to ALS therapies would eliminate the standard 180-day review period under Health Canada’s Priority Review, during which 500 Canadians, including several members of ALS/SLA Action Canada, will likely die. Canadians with ALS do not have the 180 days to wait for a review period.
      • We are calling on Health Canada to enter into a Project Orbis type agreement with the US and other countries for ALS therapies.

B. PMPRB’s Proposed Pricing Regulations

      • We are extremely concerned that the federal Patented Medicine Prices Review Board’s (PMPRB) proposed new pricing regulations will lower the likelihood of ALS therapies coming to Canada, rather than increase them.
      • Based on the regulations initially proposed, for approximately 80% of new therapies, the price set arbitrarily by the PMPRB would be far below the drug sponsors’ cost of product research and development. As a result, many drug companies have indicated they will not bring new therapies to Canada on a priority basis.
      • The PMPRB’s new pricing regulations were scheduled to come into effect on July 1, 2020. However, the PMPRB recently announced that the new pricing regulations will be delayed until January 1, 2021.
      • On June 19, 2020, a revised set of draft guidelines were issued (referred to as the “June 2020 Guidelines”), followed by a consultation period.
      • ALS/SLA Action Canada’s submission to the PMPRB can be found here.
  1. Bring the HEALEY ALS Platform Trial and therapies to Canada
    • For the first time, an innovative clinical trial – the HEALEY ALS Platform Trial – has commenced in the US. Borrowing from successes in cancer drug development, the Sean M. Healey & AMG Center for ALS at the Massachusetts General Hospital, in partnership with the Northeast ALS Consortium (NEALS) and TackleALS, are leading the first Platform Trial initiative for ALS. Dr. Merit Cudkowicz is spearheading the trial.
    • Traditionally, each trial evaluates one drug at a time, and requires lengthy start-up and execution times. Platform trials, conversely, test multiple drugs at the same time, using specialized statistical algorithms and tools. New regimens (drugs) can be added as they become available thereby decreasing or eliminating the gap in time from identification of a rationale therapy to testing. Thus, the focus is on the disease, rather than any individual experimental agent, and the platform remains open long-term until successful cures are found.
    • The Platform trial will be hosted at 54 sites across the US.
    • The Platform trial organizers and therapy sponsors have expressed an interest in expanding this trial into Canada subject to regulatory approval and funding (~$10 million).
    • We are working to bring this trial to Canada as soon as possible.
  2. Ensure timely Provincial health plan coverage for ALS therapies
    • Radicava (edaravone) was approved in October 2018 by Health Canada.
    • However, it took until April 2020 for the pan-Canadian Pharmaceutical Alliance negotiation process for this drug to conclude. (The pan-Canadian Pharmaceutical Alliance is an alliance of the provincial, territorial and federal governments that collaborates on a range of public drug plan initiatives),
    • Following the pan-Canadian Pharmaceutical Alliance negotiation process each provincial government still then had to approve Radicava before it was covered through their respective provincial health care programs and available to all Canadians.
    • To date, eight provinces have approved edaravone (Radicava): Quebec (April 2020), Ontario (May), Alberta (June), Saskatchewan (June), Manitoba (June), New Brunswick (June), Nova Scotia (July) Manitoba (July) and BC (August). As such, it took ~18 months before Quebec approved this drug and ~22 months before BC approved it. These delays cannot be justified in relation to a disease like ALS.
    • We are calling on the Provinces to adopt an accelerated approval process for new ALS therapies.
  3. Increase ALS research / treatment funding in Canada
    • The current level of public and private funding of ALS research only enabled the ALS Society of Canada to allocate $1.4 million to research in its 2019 funding program; $1.4 million is grossly inadequate to fund a research program that affects tens of thousands of Canadians with neurodegenerative disease. We are currently considering the most effective research funding target(s).
    • We are calling on the federal and provincial governments to provide meaningful and ongoing funding for ALS research.

Actions Undertaken To Date

In the short time since we formed our group, we have accomplished the following:

  • we developed a national consensus among our members on objectives, gaps, priorities and stakeholders as it relates to ALS
  • we formed a 7-person Executive Committee with a cross section of experience
  • we put together an external advisory team that has significant experience in health policy, neurology/pharmacology, strategic communications, etc.
  • we have initiated and continue to foster relationships with key stakeholders and influencers including members of the federal ALS Caucus, Ministers of Parliament, Health Canada, the ALS Society of Canada, and provincial ALS societies
  • we researched and wrote our Position Paper and obtained feedback from stakeholders and experts
  • we made a submission to the federal government for the 2021 budget requesting funding to support our ALS Plan that can be found here
  • we have engaged in discussions with the Massachusetts General Hospital  and McGill University / Montreal General Hospital about how we can help bring the HEALEY ALS  Platform Trial to Canada
  • we identified the multi-country Project Orbis approval framework for new cancer  drugs as a model for approving new ALS therapies and tabled this idea with Health Canada.  This idea is now being advanced with worldwide patient led organizations to gain global support and momentum
  • we made a submission to the Patented Medicine Prices Review Board (PMPRB) concerning the proposed new drug pricing rules that can be found here
  • we made a submission to the Canadian Agency for Drugs and Technologies in Health (CADTH) that can be found here
  • we have developed a presence and following on Twitter and FaceBook