As Medicaid, Private Insurers Begin To Lift Curbs On Pricey Hepatitis C Drugs more patients are gaining access to medications with a cure rate of over 90%. The increased access however has not been without significant legal and lobbying efforts as questions over lifting accessibility restrictions and covering the hefty price tags of these drugs are grappled with by states, government agencies, drug manufactures and commercial insurers. While an average 12-week course of treatment can currently range between $54k and $95k, this price is expected to decrease as more drug treatments come on the market and competition increases. Executive director of the National Association of Medicaid Directors conveys that the desire to make these drugs available to everyone in need is there however, "with the prices they're commanding, something has to give."
Community education and dialogue play an important role in shaping how the bleeding disorders community processes, understands and applies scientific research. This is certainly true when it comes to the recently published results of the SIPPET (Survey of Inhibitors in Plasma-Products-Exposed Toddlers) study and its implications for treatment options. Following circulation of BloodStream host, Patrick James Lynch's article, The SIPPET Study & MASAC’s Recommendation: What Does It Means For Me?, we learned of additional writings by community members aimed at helping to deconstruct the specifics of the research and translate the scientific findings into important considerations for individuals and families.
Trained immunologist and community member Murali Pazhayannur has created a site, The SIPPET Studied which helps to simplify the study and its findings, while also posing thoughts and questions about MASAC's recommendations. Additionally, community member Michael Birmingham's What the heck is a SIPPET, and why should I care? post lends his voice to break down what exactly was examined in the SIPPET study and how its findings might inform decisions about treatment.
I am not a doctor or a medical professional. I am, among other things, a writer with severe hemophilia A and a tolerized inhibitor. Following the article I wrote in January summarizing the SIPPET abstract, the National Hemophilia Foundation (NHF) engaged me to write a patient-facing article summarizing NHF’s Medical and Scientific Advisory Council’s (MASAC) recommendation in response to SIPPET’s publication.
In agreeing to do so, my goal was to help patients and families understand MASAC’s recommendations related to the SIPPET study on inhibitor formation, and to empower their decision making in choices about care.
To improve my understanding of the issues at hand, I was invited to be a guest listener on MASAC’s conference calls discussing SIPPET and was copied on the ensuing emails with draft after draft of what is now a completed recommendation. I hope this article helps provide some clarity on what is admittedly a complicated set of study results. To that end, the article is written in my language, not in dense medical-ese.
Once again, I am not a medical professional, and any decision about you or your child’s medical care should be made by you, your family, and your child in conversation with your usual healthcare team.
One of the many challenges that come with a hemophilia diagnosis is that you sorta kinda have to become something of a medical expert. And an insurance expert. And a packing expert (factor can fill a suitcase in no time!).Read More
Promising results have been reported in early clinical trials of Emicizumab, otherwise known as ACE910. This subcutaneous therapy is being developed for individuals with hemophilia A, with and without inhibitors. ACE910 has been on a fast-track approval process since September 2015 when it was given breakthrough therapy status by the U.S. Food and Drug Administration. Subsequent study is currently being conducted by the makers of the drug, Tokyo-based Chugai Pharmaceutical Co. Ltd. Its hope is that ACE910 will soon offer a new prophylactic treatment option to individuals with hemophilia A.
On June 3, 2016, Shire announced its combination with Baxalta in a $32 million merger that will notably expand its global reach. The combination positions Shire as the, “global leader in rare diseases.” In addition to the significant anticipated revenue, the announcement also signals substantial development and innovation of next generation treatment therapies. Read more about the combination here.
Long-lasting recombinant treatment for hemophilia A, AFSTYLA, has received FDA approval. The new drug, which its manufacturer CSL Behring announced as available today, “offers patients an opportunity for excellent efficacy with a strong safety profile and twice-weekly dosing--potentially helping patients to fit treatment into their active lives,” says Lisa Boggio, Assistant Professor of Internal Medicine, Hematology and Oncology, Clinical Director of the Rush Hemophilia and Thrombophilia Center, and AFFINITY clinical development program study investigator. Read more about AFSTYLA and its approval here.
After decades of study, gene therapy is showing exciting possibility for the treatment of hemophilia B patients. Researchers at Spark Therapeutics have reported promising results in a study of four hemophilia B patients who were injected with viruses containing the gene that codes for factor IX. Factor IX protein which clots blood is otherwise absent in the bodies of hemophilia B patients. While the small study size is keeping scientists from declaring an overall cure, the consistently positive results observed in each of these four patients is being received by scientists as quite encouraging for future one-time treatment possibilities for hemophilia B. In addition to the optimism regarding a potential cure for segments of the hemophilia population, these findings also have encouraging implications for the treatment of several other rare diseases.
Last week Precision BioLogic announced its donation of coagulation diagnostic products through the World Federation of Hemophilia (WFH) Humanitarian Aid Program. The donation delivered products to the University Teaching Hospital in Yanoudé, Cameroon to aid in the diagnosis and treatment of individuals with bleeding disorders. Executive Vice President of Precision BioLogic, Paul Empey says, “We’re excited to make this donation and directly support WFH’s efforts to close the gap in care between those with bleeding disorders who receive proper diagnosis and treatment and those who do not.” The WFH Humanitarian Aid Program seeks to ensure treatment for all patients with bleeding disorders despite current limitations in access to care in developing countries throughout the world. Learn more about the WFH Humanitarian Aid Program here.
Queens University researcher, Dr. Paula James, has developed a self-administered bleeding assessment tool (self-BAT) to aid women in determining whether they may have a bleeding disorder. Bleeding disorders in women frequently go undiagnosed as individuals are often unfamiliar with the symptoms and/or mistake them for normal. The website LetsTalkPeriod.ca which houses self-BAT, “aims to increase awareness of the signs and symptoms of bleeding disorders.” Through increased understanding of what constitutes abnormal bleeding, women who suffer from bleeding disorders can better access treatment to limit unnecessary health complications.