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.
Click here to read the response from Health Canada.
Click here to read our reply to the Health Canada response letter.

#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.
Click here to review the presentation delivered to Health Canada.
Click here to read our letter to Health Canada on building a national strategy for high-cost drugs for rare diseases.

#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.

#4 Consulting on the incorporation of the patient perspective into the drug development process.

Click here to read our consultation letter on inclusion of patient perspective in drug development to Health Canada.

#5: Partnering with our multidisciplinary health care teams across Canada to address the Canadian adaptation of the international Duchenne Standards of Care.

Click here to learn more about our Defeat Duchenne Canadian Conference addressing the current standards of care for Duchenne in Canada.

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

Health Canada announced that the coming into force of the Patented Medicines Regulations has been delayed for another six months to July 1, 2022. As a result, implementation of the New PMPRB Guidelines (which in part rely on the amended Regulations) are also delayed until July 2022. Further information can be found on the PMPRB website:

2021 Federal Election

Learn more about our 2021 Federal Election initiatives in partnership with the Canadian Organization for Rare Disorders (CORD):

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

Nicola Worsfold

Director of Advocacy and Research

[email protected]