Accelerating Canadian Regulatory Approvals

Budget

US$720,000 (FSHD Canada Foundation share) over Three Years

Lay Abstract

The Objective

Several promising treatments for FSHD are going into clinical trials very soon. Hopefully, these therapies will be approved for use by FSHD patients in the next couple of years. The objective of this project is to ensure that Canadians don’t fall behind the US and the rest of the world in getting access to these therapies. We also want to make sure that FSHD patients in Canada are reimbursed for the use of these therapies.

The Challenge

There are some major challenges to getting approval and reimbursement for a rare disease like FSHD:

• • Lack of ample clinical trial infrastructure to support FSHD trials.
  • Lack of clinical-trial-ready patients to meet the growing demand in FSHD research. 
  • Lack of organized Key Opinion Leaders (KOL’s) to guide biopharma and regulators.

These challenges are common globally. But Canada has an additional, unique challenge that must also be addressed.  According to a report from Innovative Medicines Canada:

“Medicines take longer to be covered in Canada compared to other countries due to its sequential and multi-layered drug review and public reimbursement process. Generally speaking, once Health Canada (federal regulatory agency, similar to the FDA in the U.S.) has approved the product, individual agency reviews to establish pricing and availability at the Provincial level are conducted consecutively instead of concurrently or before market authorization. Because public drug plan decisions are not made until after all sequential steps have been completed, patient access is considerably delayed and compromised. This can create wait times that can add years to the timeline for patients to access an FDA approved drug.”

We simply cannot let this happen. 

The Project

To get the job done, we will work jointly with the FSHD Society to leverage their resources and expertise in driving the regulatory approval and reimbursement process for FSHD patients in the US. We will work together on the following four primary goals:

1. To create the necessary data elements, analytics, and reporting in the FSHD Society’s patient registry needed by drug developers and Canadian regulatory authorities.
2. Expand the current clinical research infrastructure from three Clinical Trial Research Network sites to eight (8) sites by adding five (5) additional sites capable of supporting clinical trials in FSHD.  This will enable engagement with FSHD clinicians, Key Opinion Leaders (KOLs) and assist in garnering patient/data into the registry. 
3. To expand the existing community engagement programs from Ontario to geographic areas surrounding all eight clinical locations.  This will enable a robust, targeted approach to getting FSHD patients into the registry, as well as establish self-sustaining revenue streams to support the ongoing activities.
4. To establish key strategic partnerships and collaborations with Canadian organizations and consultants that can help us achieve the first three goals.  We will seek to establish a ‘Working Group’ to facilitate effective engagement.