Apellis Pharmaceuticals Inc., a biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through the inhibition of the complement system, has initiated its PEGASUS Phase 3 clinical trial assessing the safety and efficacy of APL-2, a novel inhibitor of complement factor C3, in patients with PNH compared to eculizumab.
The Guide to Living Well with PNH has been updated to include information on the latest news in PNH research, navigating the complexities of accessing treatment through clinical trials, traveling with PNH, and more! Please click here to read more and to download or order your copy today.
Alexion Pharmaceuticals, a clinical stage biopharmaceutical company focusing on the development of life-transforming therapies for patients with devastating and rare diseases, recently announced positive results from a phase 3 study of their second generation of Soliris® (ALXN1210), for the treatment of PNH. The press release announcing these results can be read here.
Ra Pharmaceuticals, a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for complement-mediated diseases, recently announced interim results from their ongoing, global Phase 2 study evaluating RA101495 for the treatment of PNH. The press release announcing these results can be read here.
This post is for all patients living in Ontario who might be affected by the new OHIP+: Children and Youth Pharmacare Program. Beginning January 1, 2018, all children and youth 24 years of age or younger in Ontario will be able to get their prescription medications for free by simply showing their Ontario health card number and a prescription. Coverage will be automatic, with no upfront costs.
The Canadian Association of PNH Patients (PNHCA) is thrilled to announce the creation of a new page on the PNHCA website called “What’s New in Research.” For the last 20 years, there has only been one Health Canada-approved treatment for PNH patients, but that may change in the coming years. There is a significant amount of activity underway to discover and develop new treatments for PNH, and right now, there are 10 organizations currently conducting clinical trials around the world.
Rare Blood Disorders Supplement Barry Katsof, president of the Canadian Association of PNH Patients, recently contributed to a rare blood disorders supplement that ran in print in Maclean’s Magazine, as well as online at PersonalHealthNews.ca. The article discusses Barry’s journey with PNH, the challenges he faced at diagnosis and how advancements in treatment options are helping patients with rare blood disorders live their lives to the fullest. To read the full article, click here.
Drug Approval and Funding Process in Canada If you think that deciphering how prescription medicines are approved and funded in Canada is akin to learning a new language, you’re right. It incorporates a combination of multiple processes and variations on those processes, and is complicated and complex. Just figuring out all the acronyms is enough to boggle the mind. If you’re looking for a simple snapshot of the process, we’ve got the solution for you. The following infographic will help you navigate a drug’s journey – from “bench to bedside. To download an expanded version of the infographic, click here.
Every year, hundreds of new clinical trials are initiated to test new “experimental” drugs, or to test new uses of already approved drugs on humans, in a wide range of disease areas. These trials are conducted by researchers in countries all around the world, including Canada, in locations like hospitals, universities, doctors' offices, and community clinics. Through voluntary participation in clinical trials, patients get a chance to take part in research that could improve their health and help them access a drug, prior to its approval. Like all drugs, the ones used in clinical trials have potential benefits as well as risks, and since they are still being studied, there is usually limited information about safety and efficacy. Before deciding to take part in a clinical trial, discuss the potential risks and benefits with your health care provider, so that you can make an informed decision about your health.
In the fall of 2016, the Patented Medicine Prices Review Board (PMPRB) requested input from stakeholders and interested members of the public on the PMPRB Guidelines, to ensure they remain a relevant and effective tool for protecting consumers from high drug prices in a dynamic and evolving pharmaceutical market. In response, the Canadian Association of PNH Patients (PNHCA) provided a submission and it was included in the PMPRB Guidelines Modernization Discussion Paper, which is now available on the PMRPB website. PMPRB received 66 submissions representing more than 500 different organizations, including patient groups, provincial health authorities, academics, private insurers, patentees, health professionals, employers, unions, and advocacy groups.