The Canadian health care evaluation bodies (CADTH and INESSS) have both recommended not to reimburse for the use of caplacizumab in the treatment of adults with TTP. Answering TTP Foundation has written letters on behalf of Canadians who have survived an episode of TTP to express the community's shock and disapointment with this recommendation, in hopes of having the recommendation reversed.
The letter emphasizes the enormous challenge of rescuing an aTTP patient without caplacizumab. Existing TTP treatments are designed to control the underlying mechanism of disease, but require time to take effect, time that aTTP patients do not have. In aTTP, blood clots rapidly form in small blood vessels throughout the body and block the flow of oxygen-rich blood to the body’s organs, including the brain, kidneys and heart. Caplacizumab prevents the formation of clots that cause organ damage, allowing patients to survive long enough for existing treatments to take effect.Caplacizumab saves lives and prevents disability. Caplacizumab is the first new treatment developed for aTTP in the last 25 years. Peer nations have recognized its evidence-based utility by updating their international treatment guidelines to include the use of caplacizumab. For a country that pioneered TTP treatment by establishing the effectiveness of plasmapheresis (Rock 1991), Canada’s inability to adopt caplacizumab is causing undo suffering and death.
Answering TTP Foundation is seeking motivated and self-directed volunteers to develop a strategy and plan of action to address the use of caplacizumab in Canada. No experience is necessary. There exists other charities, and coalitions of rare-disease patient groups that will provide guidance and mentorship.
Sydney started the Foundation to fund TTP research and provide education and patient support. Her Toronto based network of friends and family have successfully implemented this model. We are now calling out for more champions. A group (patients/ supporters) who can step up and build out a much needed framework to engage with decision makers to ensure that patients in Canada can access new effective treatments for TTP.
"Our community requires self-motivated individuals to take on this vital new role. Current volunteers are already overburdened with running existing programs and meeting charity requirements." --- Sydney Kodatsky, Chair, Answering TTP Foundation.
If you are ready to seize this opportunity to give back, and to truly partner in helping the TTP Community, please email firstname.lastname@example.org.