For Canadians with Rare Diseases, Waiting for Treatment is not an Option
Research and Innovations Treatment for Mucopolysaccharidoses (MPS) has not been found yet. Ensuring proper detection and treatment is given in order to slow down and halt the progression of the disease is vital.
As Canadians, we pride ourselves on a health care system that is supposed to provide equal care for all. However, for the 1 in 12 Canadians — mostly children — who live with rare diseases, accessing the treatments they need can involve a litany of barriers and frustration.
Andrew McFadyen is the founder and Executive Director of the Isaac Foundation, a non-profit organization dedicated to helping those with mucopolysaccharidoses (MPS) and other rare diseases. The foundation is named for Andrew’s son Isaac, who lives with a severe and progressive form of MPS. “When you fall on the wrong side of the genetic lottery like our kids who are battling rare diseases,” McFadyen says, “every step along the path to gaining access to the treatments they need becomes a challenge.”
With progressive diseases, we must treat as early as we can
Though there is no cure for these diseases, there are treatments for many of them that can slow or halt progression of the disease. Unfortunately, even when these treatments have been approved by Health Canada, they often remain unavailable or very difficult to access while reviews and negotiations happen at the provincial level. These delays can devastate families.
“Waiting is not really an option,” says Dr. Aneal Khan of the University of Calgary and the Alberta Children’s Hospital. “The disease continues with its relentless course, and we are left helpless because we can’t treat it. If a patient must wait years to get their first dose, they could either die while they are waiting or see their disease become so advanced that treatment can no longer help.”
Fixing the system can save lives
As Canadians, it is our responsibility to work towards a better system. When the system fails to build a bridge between industry and government, it is too easy for both parties to see solving the problem as the responsibility of the other. But when this happens, it is the patients who lose out. “Collaboration is the key to paving the way forward for patients,” says McFadyen. “I know because it always works when we make that collaboration take place. Patients come out ahead when that happens, and it’s our best hope to save all of our patients in need.”
We must raise our voices and ensure that a truly collaborative conversation takes place. It is the responsibility of all Canadians to make it clear that we believe a better solution is possible, and that we want to work towards making it a reality. If our process is leaving vulnerable patients behind, we have the power and the obligation to change that. This can be fixed, if we work together to build the political will to do so.