Jesse's Journey Foundation

Defeat Duchenne. Change Lives.

Advocacy

As the leading Duchenne organization in Canada, we are here to serve you – Duchenne families – by representing your voice and diverse needs, as we advocate for the best access to healthcare and therapies with government, industry and the medical community.

Our priority: timely access to affordable treatments in Canada.

Jesse’s Journey in partnership with rare disease organizations across Canada including Muscular Dystrophy Canada (MDC), the Neuromuscular Disease Network for Canada (NMD4C), and the Canadian Organization for Rare Disorders (CORD), have undergone consultation with Health Canada on the development of a rare disease strategy. We are advocating for a pathway in Canada that supports early access to life-changing medicines, that are affordable, and accessible to everyone.

We are achieving this by:

#1: Working with Health Canada, our clinicians, and manufacturers to gain access to therapies not yet approved in Canada.

Click here to read our letter to Health Canada’s Special Access Program (SAP) regarding deflazacort supply interruption.

#2: Advocating for fast decision making in order to speed up access to medicines in Canada.

Click here to read our letter to Health Canada and a summary of our 2019 patient survey results.

#3:  Advocating against price restrictions imposed by the PMPRB (Patented Medicine Prices Review Board) to ensure that when therapies do become available, they’re affordable for all families.

Click here to read our consultation to the PMPRB.

2021 Canadian Federal Election

Make your voice heard! Join Jesse’s Journey in our urgent advocacy efforts for accessible and affordable treatments for those living with Duchenne muscular dystrophy in Canada.

How you can get involved:

The Canadian Organization for Rare Disorders (CORD) has developed a toolkit to make it easy to engage with candidates and campaign teams in your riding:

STEP 1: Find the candidates in your riding

Visit Elections Canada and type in your postal code.

STEP 2: Develop your key messages

Now that you know who your candidates are, what do you want to say to them? Very important: when interacting with candidates, make it personal – share your experiences with rare diseases and how they have affected you or someone you know. And don’t forget to communicate your ask(s)! Three suggestions are below:

  •  Duchenne muscular dystrophy: Share your story along with the following message: Duchenne muscular dystrophy is a rare, genetic, muscle wasting and life-limiting condition diagnosed in early childhood.  There are currently six treatments approved outside of Canada that could delay disease progression and help our children stay stronger for longer. Help us gain access to these treatments for our children in Canada.
  • National Rare Disease Strategy: We urge all candidates to jump-start the implementation of a comprehensive Canadian Rare Disease Drug Strategy building on the committed $500 million in annual federal funding starting in 2022. 
  • Federal Drug Pricing Reforms: Withdraw the proposed reforms of the Patented Medicine Prices Review Board (PMPRB) because they will limit access to treatments, clinical trials, and health research.

STEP 3: Start engaging!

Election candidates will already be very active in your community, hosting town halls and taking part in meetings invited by or alongside community leaders. The best way to follow candidates is through their Facebook pages and Twitter accounts, emailing or visiting their campaign office, or even when they knock on your door.

Click the button below to see CORD’s full Election Toolkit.

PMPRB

The PMPRB stands for Patented Medicine Prices Review Board, an independent quasi-judicial government body to inform and protect Canadians by regulating the price of patented medicines sold in Canada.

The PMPRB has proposed new changes that would lower drug prices to levels that would discourage or delay pharmaceutical companies from bringing new rare disease medicines into Canada. That means severely limiting and delaying access to treatments for children and young adults living with Duchenne.

We believe Canadians deserve life-saving and life-altering treatments, but restrictive pricing is making it impossible to get new therapies in Canada. In response, Jesse’s Journey has partnered with a variety of leading healthcare groups to advocate for the Duchenne community:

  1. Jesse’s Journey has partnered with Muscular Dystrophy Canada (MDC) and the Neuromuscular Disease Network for Canada (NMD4C) to provide a collective response to the PMPRB draft guidelines – read the full release here.
  2. Jesse’s Journey sits on the Board of Directors for the Canadian Organization for Rare Diseases (CORD) and fully supports their submission to PMPRB on the revised draft guidelines in collaboration with the Canadian Agency for Drugs and Technologies in Health (CADTH) – read the full release here.
  3. Jesse’s Journey participated and supports the Fight for Our Lives campaign, advocating for solutions to lower drug prices without harming patients – learn more here.

PMPRB Next Steps

On October 23, 2020, the PMPRB published the final version of the PMPRB Guidelines that operationalize the amended Patented Medicines Regulations, which come into force on January 1st, 2020. Our team is taking the time to carefully review the document and will provide an update for the Duchenne community soon.

What to learn more about our advocacy work or get involved? Contact:

Nicola Worsfold

Director of Advocacy and Research

[email protected]