by June Kinoshita, Director of Research and Patient Engagement
Making sure that every person with FSH muscular dystrophy gets the best possible medical care is a high priority for us. Far too many people remain undiagnosed. Too many struggle to find useful guidance on managing their symptoms. It’s a daunting task to update the medical profession, considering that most physicians see only a handful of FSHD patients in their career. The information from that muscular dystrophy lecture in medical school is likely outdated by now. Much has changed just in the past five years.
How can we bring this new information to doctors? One way is to create a continuing medical education (CME) course, such as the FSHD Masterclass that the Society produced this year. Another is to publish an updated standard of care article in a medical journal. That’s why the FSHD Society, in collaboration with the European Reference Network, has recruited a stellar team of international authorities on FSHD to write a new, unified international standard of care. The team includes patients who will make sure that the concerns of those affected by FSHD are addressed at each step. We will be leading a complex project to review the medical literature systematically and objectively across 11 key topics, develop consensus recommendations, write the article, and get it published in a high-impact medical journal to ensure that it gets international visibility and adoption in many countries.
There’s a second reason why this publication is urgently needed. As was pointed out in the white paper from our 2019 Industry Collaborative Workshop, an up-to-date standard of care is vitally important for clinical trials:
“Site-to-site differences in standard of care represent a potential barrier for the conduct of clinical trials, as efficacy assessments may be influenced by baseline level of care to an extent that trial results are confounded. FSHD care guidelines have been developed in 2008 and 2015, but are likely out of date. Continuing efforts to improve, update, and disseminate care standards are essential in optimizing patient management and feasibility of interventional clinical trials.”
The new international standard of care, which we expect to complete by the end of 2022, is an extraordinary measure we are taking to ensure that people living with FSHD, no matter where they live, have access to high-quality care based on the best available evidence and consensus recommendations by the most experienced FSHD clinicians in the world. With several FSHD clinical trials anticipated over the next 2-3 years, the updated standard of care will help to ensure that discrepancies in patient care will not undermine these trials.
I would like to receive a printed copy of the FSHD Advocate by mail rataher than online. Is that still available?
Yes it is! All you need to do is join our contact registry to ensure that we have your address and indicate that you wish to receive USPS mailings from us.
https://www.fshdsociety.org/for-patients-families/join-fshd-research-contact-registry/
Reading between the lines, its a scary proposition that big Pharma and the Industrialized and profit driven health care corporations will want to decide, based upon some cost/benefit algorythmn who is deserving of being given the magic pill, and who isn’t. Being in my 60’s and semi-retired, I’m a goner for trials and treatment. Obviously, my earning and tax paying impact cannot compare with someone with begining progression who is 25.
It’s not the companies so much as the payors, both private insurers and government, that act as gatekeepers. They evaluate whether and how much to reimburse for medical treatments based on data on their efficacy and impact. That’s why it’s so important for the FSHD community to be involved in the conversation about the effects on health, medical costs, quality of life, and non-medical costs of living with FSHD.