April 15, 2025
It is hard to believe the first quarter of 2025 is in the history books. In the space of hypertrophic cardiomyopathy and thick heart muscle disorders, the distance between the promise and the delivery of a reality freed from burden of disease is closer than it has ever been, not only in the United States but throughout the world. Unfortunately, we are navigating through some challenging health policy times, which will impact a large percentage of those with the diseases we seek to serve. While we navigate these challenging waters together, we remain committed to ensuring safe, accessible, affordable healthcare while ensuring the rights of those with disabilities are maintained. Last month, I attended two large conferences - one held in Stockholm, Sweden the other Chicago, Illinois. The research communities worldwide are holding their breath, waiting to see how we will move forward, even as we continue to develop new therapies, treatments and, even potentially, cures. It has never been more important to keep research moving, as we are so close to so many amazing improvements in our ability to care for those with thick heart muscle disorders, including HCM in all its forms, Amyloidosis, Fabry’s disease, Danon disease, and RASopathies. There was amazing science presented at the American College of Cardiology, where we warmly welcomed new president, Dr. Christopher Kramer, the original director of the hypertrophic cardiomyopathy center at University of Virginia. We look forward to his leadership and wish him well in his challenging tasks ahead. Later this month we will be in Orlando, Florida - please register and join us for this wonderful Bighearted warrior tour in person with our friends at AdventHealth and the incomparable Dr. Marcos Hazday. I even understand that there's some carpooling being organized from the Tampa area. If you're interested, reach out to the office and we will connect you. Maybe, the most impactful thing that will happen in the month of April is that we will conduct our second visit to Capitol Hill. Our lead topic this year is something you have heard us talk about at many prior meetings of the HCMA and podcasts; the generic drug quality issue. We are proud to be partnering with David Light, Co-founder and President of Valisure, and retired Colonel Vic Suarez, to ask House and Senate members to support the inspection of all generic drugs purchased by the Department of Defense and the Veterans Administration and make results of these inspections open for public use. Secondly, we are seeking rational oversight of health insurance companies’ abuse of prior authorizations and step therapy requirements. These issues cost an estimated 1.3 billion dollars a year and provide nothing to keep a patient safer or a physician's office running more efficiently. Common sense tells us to not waste money where there is no return. Additionally, prior authorizations and step therapies can keep patients sicker longer, ultimately costing the healthcare system more money. Our briefing will educate Representatives to act in an informed manner when moving policies that impact us all. You can watch the video of our briefing from Wednesday, April 9th on Vimeo: https://vimeo.com/event/5043266 If you wish to get involved or more informed on any of the issues above, I encourage you to visit the website, 4hcm.org , or reach out to the office and the staff will be happy to assist you. On the day before I head out to Washington DC, I will leave you with this one thought - we have come a really long way in our understanding of HCM over the past 60 years. We have increased the lifespan of those with HCM, through collaborative research and implementation of best practices in an organized fashion throughout this country. We have worked so hard to end suffering for so many, and we are succeeding in our shared goal to outsmart hypertrophic cardiomyopathy and other thick heart muscle disorders. It is important to continue the research into all aspects of these diseases, including the biological, the clinical, and burden of disease measurements. System improvements, positive impact of timely diagnosis and treatment, and the value to society of all of these big hearts being here, will help keep their families whole. So we're off to DC to try to educate others about what it really means to live in our ecosystem. Best wishes, Lisa
