I’m impressed of with the direction of the Fabry Support & Information Group (FSIG) and the offerings they have.
In August 2016, they had a Fabry Family Get Together at the Hospital and Clinic of the University of Iowa with a noted expert in Fabry disease, Dr. Robert. J. Desnick, who also happens to be the director of the Center for Fabry disease. He gave a talk on the disease, sharing his perspectives as an expert, and also discussed enzyme replacement therapy and the direction of future treatments. Following the event, there was a Q&A session.
Also in attendance were representatives from Sanofi Genzyme, Protalix Biotherapeutics, and Amicus Therapeutics, who are some of the key leaders in biotechnology and served as cosponsors. Undoubtedly, these folks are committed to meeting the unmet needs of Fabry disease patients and their loved ones to develop effective therapies and support services.
I don’t know how the event turned out as I only recently learned of it, but I would be most curious to learn if it was a success and the upshot of the information that they provided. The fact that this event was even held is very impressive for a rare disease that affects fewer than 4000-5000 Americans—if females are also included in these stats.
It amazes me that these biotech companies are able and willing to support this rare disease community—especially when it costs approximately one billion dollars—yes, one billion!—to bring a drug to market. This figure includes the sometimes 20+ years of scientific research and development, various phases of clinical trials where the drugs are tested at various doses, using animals, then, testing humans, etc. It takes years to develop therapies and years to gather the data and submit to the FDA for approval. And with such a small disease population, their return on investment has to show profit in order for them to continue and bring the drug to market.
Some families are outraged over the cost of biologic drugs, some of them costing as much as $50,000 for two rounds of treatment. I’m just saying there’s a reason why the drugs are so expensive—I get that. And I also know that employees of these companies also have heart. They really do care. But I’m going to stick my neck out there in support of the right for EVERYONE to have access to effective treatments regardless of their income.
Do you believe in socialized medicine? Do you think it could ever work in America? And if so, do you think Americans would be willing to pay much higher taxes in order to pay for socialized medicine like we do Medicare?
Do you have an opinion about how the costs for medicines for rare diseases should be shouldered? Have you had any experiences getting help paying for your medicines? Tell us about it!
