Equitable, timely and affordable access to therapies

Access to innovative therapies is an urgent issue for people living with ALS, yet the current drug approval and reimbursement processes in Canada do not function in a way that reflects the realities of what it means to live with this disease – a swift progressing, fatal disease with no cure. 

Because of how fast someone can lose their life to ALS, it’s critical that all people living with ALS are able to benefit from the therapies to come, as quickly as possible following Health Canada approval. But, if we consider the current drug access pathway and the significant time it takes for a new therapy to move from regulatory approval through to the reimbursement decisions that result in patient access, thousands of Canadians with ALS will die waiting for these much-needed treatments. 

We believe the entire drug access pathway must be streamlined and made more transparent and inclusive to address the needs of people living with ALS. This includes research and development, clinical trials, the process for new drug submission and approval, and public funding decisions.  

How we advocate

The ALS community measures time not by months or years, but by loss – loss of function and loss of life. There is no time to waste: for people living with ALS, the timelines associated with how drugs are evaluated and made available in Canada should not be a barrier to accessing future proven treatments. 

Now is the time for government to streamline the drug access pathway and improve its transparency while creating meaningful involvement for those who know the realities of the disease best. Government at all levels must work together to implement solutions that will result in faster access to proven ALS therapies to help those affected now, before it’s too late 

We urge the federal, provincial, and territorial governments to improve access to therapies, based on the following recommendations:  

  • Create an environment that makes Canada a country of choice for industry to bring new therapies, from research and development, to clinical trials through to new drug submissions.  
  • Coordinate, streamline, and increase transparency of the regulatory processes, timelines, and transparency associated with bringing a drug to Canada in order to enable patients to better access drugs and inform decision-making processes. 
  • The federal, provincial, and territorial governments should collaborate to address the health care and funding inconsistencies across Canada that currently result in inequitable access to new therapies across the country. 

Roadmap to expedited and equitable access to ALS therapies: The Time is Now

ALS Canada – in consultation with the broader Canadian ALS community – has released The Time is Now  position paper, which offers two concrete solutions to get Health Canada approved therapies to Canadians living with ALS in a timeframe that more accurately reflects the urgent needs of the community. The position paper proposes that: 

  • All ALS treatments, upon approval by Health Canada and the development of applicable clinical criteria/prescribing guidelines, be immediately publicly funded on an interim basis. 
  • All ALS treatments be approved, reviewed for public reimbursement, and publicly funded through a single condensed timeframe applicable to all jurisdictions in Canada within an 18-month timeframe. This pathway recommends Health Canada priority review for all ALS therapies.   

ALS Canada calls upon the federal, provincial, and territorial governments to work together to build a plan that would see these two solutions implemented across Canada.  

ALS Canada has demonstrated leadership in drug access reform through the following:

The development of a national pharmacare program has significant implications for all Canadians as it relates to access to therapies. There are also important considerations for the ALS community as we think about the treatment pipeline and the potential for new therapies to come to market in the coming years.  

As a member of the Health Charities Coalition of Canada (HCCC), ALS Canada and members of the ALS community participated in the summer 2018 consultation process led by the federal government’s Advisory Council on the Implementation of National Pharmacare, chaired by Dr. Eric Hoskins. We also submitted a written response in the form of a letter as part of the consultation process. The letter was in support of the recommendations put forward by the HCCC, with additional considerations unique to the ALS community.  

On June 12, 2019 the Advisory Council on the Implementation of National Pharmacare released its  final report. Included in the report are recommendations on a strategy for expensive drugs for rare diseases and that patients should be involved in aspects of the decision-making process. These recommendations are a step in the right direction for equitable, timely, and affordable access to the proven ALS therapies of tomorrow and speak to how collectively our voices can have an impact. 

Read ALS Canada’s letter. 

National Strategy for Drugs for Rare Diseases | Government Consultation

ALS Canada took part in Health Canada’s consultation on the development of a National Strategy for Drugs for Rare Diseases in March of 2021. 

To accurately reflect the experiences of the ALS community, we launched our own consultation survey in February 2021 and invited Canadians affected by ALS to respond by sharing their perspectives on how the government should move forward with a National Strategy for Drugs for Rare Diseases. Following our survey, we summarized the nearly 250 responses and put together a submission for the National Strategy for Drugs for Rare Diseases consultation.  

In summer 2022, Tammy Moore, CEO, ALS Canada, took part in a roundtable as part of Health Canada’s consultation on creating a National Strategy for Drugs for Rare Diseases, where she provided comments on the What We Heard from Canadians Report. The roundtable was an opportunity to reiterate the urgent need to change how drugs are made available in Canada and for Canadians living with ALS to access approved therapies in a timely way.  

Read our  full submission here. 

Expensive Drugs for Rare Diseases

In 2018, ALS Canada was also invited to participate in a webinar consultation led by the Expensive Drugs for Rare Diseases Working Group (EDRD WG) on a proposed supplemental process for the national and jurisdictional drug review process for complex/specialized drugs, including drugs for rare diseases. The discussion focused on a variety of topics including how to ensure the proposed supplemental process is transparent, streamlined, and designed in consultation with patients and stakeholders. 

Following the webinar, we provided a written submission response highlighting the perspectives of the ALS community as it relates to ensuring a streamlined and transparent drug access process. 

Read the response. 

In June 2020, the Canadian Agency for Drugs and Technologies in Health (CADTH) launched a public consultation on its proposed reforms to its drug reimbursement review processes. Because CADTH’s drug reimbursement reports affect how people living with ALS can access the treatments they need, ALS Canada participated in this consultation to bring forward the experiences of the ALS community. In our survey response, we highlighted the need to shorten lengthy timelines associated with CADTH drug reviews and for the importance of patient voices as a meaningful source of input during the recommendation process. 

Read ALS Canada’s response.

Proposed framework for a potential pan-Canadian formulary

As a member of the Health Charities Coalition of Canada (HCCC, we responded to the CADTH’s consultation on a proposed framework for a potential pan-Canadian formulary. Our response brought forward the perspective of health charities across Canada and highlighted the importance of making innovative medicines accessible to Canadians living with ALS in an affordable, timely, and equitable manner.   

Read .   

In December 2017 the federal government proposed amendments to the Patented Medicines Regulations, the guidelines that govern how the Patented Medicine Prices Review Board (PMPRB) sets the price at which companies sell their drug to distributors across the country. The amendments are designed to create additional price determination factors for PMPRB, such as increasing the number of countries from which PMPRB compares drug prices from seven to 12. 

While intended to result in lower drug prices, the proposed changes to the regulations could also affect the ability of Canadians living with ALS to have timely, equitable access to future proven therapies, including the therapies and clinical trials that are available in Canada compared to other countries. To draw attention to the potential implications for patients, as a member of the Health Charities Coalition of Canada (HCCC, ALS Canada shared the potential for these unintended consequences for the ALS community by participating in several consultation opportunities. In 2018, ALS Canada submitted a response to the proposed amendments in the Canada Gazette Part I, and in 2020 ALS Canada submitted a response to two public consultations on the proposed amendments. 

UPDATE: On April 14, 2022, Hon. Jean-Yves Duclos, the Minister of Health, announced that the government will not proceed with the amendments related to the new price regulatory factors – meaning the reforms will only move forward with changes to the comparator countries.  

The government’s decision not to proceed with the amendments that could create barriers for companies to bring innovative medicines to Canada indicates that by working together to approach decision-makers, we can make our voices heard.

We thank the ALS community for their advocacy efforts in creating this change. Read more about the change HERE.  

Read ALS Canada’s response to the December 2022 PMPRB public consultation HERE.

Read ALS Canada’s letter to the Canada Gazette Part 1 HERE. 

Read ALS Canada’s response to the February 2020 PMPRB public consultations HERE. 

Read ALS Canada’s response to the June 2020 PMPRB public consultations HERE. 

Read ALS Canada’s response to the July 2021 PMPRB public consultations HERE. 

Read ALS Canada’s response to the December 2022 PMPRB public consultations HERE.

With issues like national pharmacare, healthcare funding, and drug access and affordability at the forefront of the 2019 federal election, it’s critical that ALS be part of this conversation. 

During ALS awareness month in June 2019, and the federal election campaign period that followed, ALS Canada provided a fact sheet for the ALS community to share with local candidates, political parties, and canvassers to help them better understand the role the federal government can play in helping to create a future with access to proven ALS therapies. 

Download the fact sheet.

In September 2018, we were invited to appear as a witness before the House of Commons Standing Committee on Health (HESA) related to access to therapies for rare diseases. Tammy Moore, ALS Canada CEO, spoke to the challenges faced by Canadians living with ALS related to accessing innovative and promising therapies before and after market approval.  

In March 2019 HESA released its report Canadians Affected by Rare Diseases and Disorders: Improving Access to Treatment. The report acknowledged the experiences of people living with ALS and provided 19 recommendations to improve access to therapies.  

Read the remarks here.