I can tell you about one of the organizations & the disease.
PSC is a rare bile-duct disease that destroys the liver and puts the patient at risk for a half-dozen cancers. My daughter is one of approximately 30,000 people nationwide with the disease and has been living with it since at least 2003 (based on symptoms) and was diagnosed in 2009. There is no approved medical treatment, and the surgical treatment (liver transplant) is not a cure - I know people who have had as many as 5 liver transplants.
PSC Partners Seeking a Cure is - generally - a fantastic organization. It leverages small amounts of money (in research terms) into very significant research by funding (among other things) small proof of concept trials. For its size, it is extremely effective at raising money and using it well. It awarded nearly a half million in research grants in 2017, spread among 7 projects. It also curates a database of diagnosis, family history, quality of life, medications, surgeries, overlapping diseases, and patient demographics that is made available to researchers world-wide to address the problem inherent in rare diseases - there isn't a significant enough population in one geographic area to do traditional research.
That said - the organization is fairly medically conservative. There is a medication that has worked well, particularly in the pediatric population. There are patients who have been using it for decades - and have been in clinical remission for that long, there is not data to prove cause and effect. These are well documented in case studies and in smaller research projects that are too small to be routinely accepted as valid. Even though this medication is not of a nature that would be covered by this law, I firmly support the right to try for all PSC patients. The organization does not.
My concern is that the motivation for this letter may be the same kind of paternalism that (it feels to me like) is the driving force behind the medication that leads the organization to discourage people from making private arrangements with their physicians to try the medication that seems helpful in the pediatric population. The second paragraph in the letter is what makes me at least mildly suspicious.
So - yes - when organizations like PSC Partners Seeking a Cure speaks in connection with a bill that will impact PSC patients, it is good to pay attention. Just make sure to also keep your brain in gear and not merely accept the recommendation without understanding where it comes from.
= new reply since forum marked as read
