Advocacy

Equitable, timely and affordable access to therapies

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.

The ALS community measures time not by months or years, but by loss – loss of function and loss of life. During a 180-day expedited timeline for Health Canada to review priority drugs, for example, 500 Canadians will die of ALS. Equitable, timely and affordable access to tomorrow’s therapies shouldn’t be a luxury – it should be a given.

How we advocate

There are more than 60 ALS drugs currently in the development pipeline and only two Health Canada-approved ALS therapies that have an impact on disease progression. 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 reality of ALS.

We urge the Government of Canada and the Province of BC 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 provinces, territories and federal government should collaborate to address the health care and funding inconsistencies across Canada that currently result in inequitable access to new therapies across the country.
  • The provincial government should implement a real plan that streamlines the process for newly-approved treatments moving through the BC Public Drug Program.
  • 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.

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.

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.

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 PMPRB compares drug prices to from seven to twelve countries.

The proposed changes to the regulations could affect the ability of Canadians living with ALS to access innovative and complex therapies in a timely and equitable manner, as well as create an even larger discrepancy between what therapies and clinical trials are available in Canada versus other countries.

Radicava (edaravone)

In October 2018, Health Canada approved edaravone for the treatment of ALS. Edaravone is the first drug to be approved by Health Canada for the treatment of ALS since the approval of riluzole in 2000. Approval from Health Canada means that edaravone can be marketed and sold in Canada.

What happens next

Other considerations, like the price of the drug and whether it will be covered through provincial drug plans, are separate steps in how drugs become approved and available to Canadians. The Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d’excellence en santé et en services sociaux (INESSS) in Quebec provide recommendations and advice to federal, provincial, and territorial drug plans regarding reimbursement decisions. Each publicly-funded drug program conducts an independent review of the therapy before ultimately deciding whether to cover the drug at no cost. The CADTH, Ontario Public Drug Program and B.C. PharmaCare processes include an opportunity for patient input.

How we advocate

Throughout the drug access process for edaravone, we have worked with the ALS Societies across Canada to bring forward the experiences and perspectives of Canadians living with ALS.