Submission to the ALS Caucus 2025

• In the opinion of ALS Action Canada (ALSAC), a fundamental problem at CDA has been the relatively limited influence of expert Neurologists in the reimbursement review of drugs for ALS, and when such advice has been sought, it has been rejected by CDEC. 

• CDA is a joint federal/PT agency and therefore Health Canada has a responsibility to ensure that CDA provides greater opportunity and responsibility to Clinical Experts in ALS for reimbursement decisions. 

1. Timeline of ALS Action Canada’s (ALSAC) interactions with Canada’s Drug Agency (CDA) and Health Canada (HC) from November 2023 – March 2025.

March 2023: Minister of Health announced the National Strategy for Drugs for Rare Diseases.

November 2023: Joint submission by ALSAC and the ALS Society to Suzanne McGurn, CEO of CDA, and Hon Mark Holland, Minister of Health, outlining scientific and clinical problems with CDA’s approach to reimbursement for ALS drugs, resulting barriers to drug access, and failure of the National Strategy to address these problems. (References: “Meeting request” email to the CEO of  CDA, November 30, 2023; “Urgent need” email to Minister of Health, November 30, 2023).

January 2024: Presentation (virtual) by ALSAC to the Drugs for Rare Diseases Directorate, Health Canada, detailing the problems for ALS patients that were caused by CDA’s decision processes, and explaining their origins and clinical invalidity (Reference: “Slide deck” January 17, 2024). 

September 2024: Meeting at the offices of Health Canada’s Drugs for Rare Diseases Directorate in Ottawa. Subsequent email discussion with Director General, Daniel MacDonald to clarify details.   

September 2024: ALSAC reception in Ottawa for MP’s, including the Minister of Health, Government officials, other ALS societies, and representatives of the NHL (supporting ALSAC’s fundraising for ALS Research. Opening remarks by MP Peter Fragiskatos, Chair of  the ALS Caucus of federal MP’s; speech by Matthew Brown, person living with ALS; speech by Andrew Darke on the invalidity and drug access problems caused by CDA’s reimbursement criteria.(Reference: Summit Recep TXT.doc, September 24, 2025). 

December 2024: Publication of two papers in the Canadian Journal of Neurological Sciences on the problems caused by CDA’s routine use of the strict numerical inclusion criteria used in clinical trials, as the basis of its reimbursement decisions,. (References: Darke AC, CJNS (2024), 51, 603–605, doi:10.1017/cjn.2023.307; Zinman L et al, CJNS  (2024), 51, 606–607, doi:10.1017/cjn.2023.320  

January 2025: CDA’s “Consultation on Additional Improvements to Drug Reimbursement Reviews”, including increased participation by Clinical Experts. Deficiencies submitted to CDA by ALSAC. (Reference: “Feedback on Proposed Improvements” email to Suzanne McGurn, January 15, 2025).  

February 2025: Through an introduction by Hon Peter Fragiskatos MP, email discussion with the Drugs for Rare Diseases Directorate regarding the need for Health Canada to exert influence over CDA, which the Directorate determined to be not possible. (Reference:”Connected through Peter Fragiskatos emails to Andrew Taylor, February 10-17, 2025). 

February 2025: ALSAC reply to CDA’s request for names of Clinical Experts to advise CDEC on an upcoming review of an ALS drug. (Reference: “Clinical experts” email to Suzanne McGurn, February 10, 2025). 

March 2025: ALSAC met with CDA’s “Engagement Group” to address concerns over the limitations placed on the role and responsibilities of Clinical Experts in the reimbursement decision process. (Reference: Drug reimbursement review.doc).

2. Conclusions based on ALS Action Canada’s communication with the Drugs for Rare Diseases Directorate, Health Canada, and Canada’s Drug Agency (December 2023 – February 2025):

A.The Minister’s announcement in March 2023 of the $1.4 billion National Strategy for Drugs for Rare Diseases stated: “This funding will help provinces and territories improve access to new and emerging drugs for Canadians with rare diseases, as well as support enhanced access to existing drugs, early diagnosis and screening for rare diseases”. As currently implemented, this Strategy will not meet the needs of patients living with ALS.  

QUESTIONS FOR THE MINISTRY OF HEALTH:

• How will the current implementation plan enhance access to existing drugs, specifically drugs for ALS, given that existing drugs (NOC 2019, or before) would have completed HTA reviews and  therefore, availability through provincial drug plans would be under restrictive reimbursement criteria, which will not be modified under the National Strategy? 

• New drugs for ALS (NOC after 2019) that are considered for eligibility under the National Strategy, will also have completed HTA reviews. CDA reviews of these drugs cannot be expected to include less restrictive reimbursement criteria than imposed on the last two ALS drugs reviewed by CDA. Will the plan to improve access to new and emerging drugs for ALS include re-evaluation of reimbursement criteria by independent clinical experts prior to – or concurrent with – inclusion of ALS drugs in the National Strategy? 

Unless CDA is required to place much greater emphasis on expert advice from ALS Neurologists, rather than their current .. almost exclusive .. reliance on the opinion of their Canadian Drug Expert Committee (CDEC), access to currently marketed and future new drugs for ALS will remain both seriously restricted and inequitable, because of CDA/CDEC’s current approach of simply restricting access to ALS drugs to patients who  meet the strict numerical inclusion criteria in pivotal clinical trials of the drug.  

Since HC has a representative on the board of CDA and has responsibility for its operation, and given the 2019 Report by HESA on Access to Treatment for Rare Diseases, which called for the auditor general to audit HC’s management of CDA: 

• Has HC implemented HESA’s recommendation for an audit of CDA?

• Will HC commission an expert panel of ALS Neurologists to assess CDA’S current HTA review practices for ALS drugs and propose a clinically valid framework for reimbursement reviews of ALS drugs that CDA will adhere to?

• Will HC require CDA to establish an Advisory Council for all drugs for rare diseases (ref: FDA model) that would have the primary responsibility for recommendations on provincial drug plan coverage, and whose recommendations would be public?   

• Will HC make all drugs for rare diseases immediately available under the National Rare Diseases Drug Funding Strategy, to all patients who meet the approved HC Product Monograph Indication, (as for other federally funded, healthcare delivery programs).

Will HC ensure that CDA follows Good Regulatory Practice and provide a more meaningful role, than is their current practice, for the lived experience of patients, in reimbursement decision making, particularly for drugs for rare diseases as exemplified by the FDA’s Rare Disease Innovation Hub – ref: Shore et al, 2024: https://doi.org/10.1001/jama.2024.20358.