What’s New in the PSC Research World?
Byers “Bud” Shaw, MD is a world renowned transplant surgeon who has saved the lives of countless patients.
The video from the March 2016 PSC meeting held at the FDA White Oak Campus is up!
FDA, AASLD, ACG, AGA, and NASPGHAN co-sponsored a meeting March 3-4 on Surrogate Endpoints and Clinical Trial Design for PSC. Watch the video from every session on AASLD's Featured Content page.
How do we measure whether new therapies are making a difference? What's a surrogate endpoint?
In part one of this series, we talked about the challenges associated with understanding a rare disease and developing new therapies, and we touched briefly on a meeting recently held at FDA on clinical research in Primary Sclerosing Cholangitis (PSC). In part two we discussed the importance of doing more clinical trials to understand what types of therapies might make a difference in PSC, and how SJI is working to support those trials with a nationwide Clinical Research Network. But when we're testing a new therapy, how do we measure whether that therapy is actually working and making a difference for patients with PSC? That's a more complicated question than it appears at face value. In today's post, part three of the series, we'll discuss surrogate endpoints in the context of therapeutic development for PSC.
What's the strategy for curing a rare disease?
In part one of this series, we talked about the challenges associated with understanding a rare disease and developing new therapies. As many of you are well aware, either in your experience as a patient or a medical researcher, it can be very difficult for specific rare diseases to capture the interest of pharmaceutical companies, especially when the cause of that disease is unknown like in PSC. There are many rare diseases (greater than 7000), and they are all competing for funding and attention; because rare disease patients are few and often spread out across the country, it is hard to enroll them in clinical trials in large numbers. That said, enrolling patients in clinical trials is the way forward for understanding PSC and what types of therapies will effectively treat it - we have to do it.
Why is it so hard to cure a rare disease?
In 2010 the Institute of Medicine released a report entitled Rare Diseases and Orphan Products: Accelerating Research and Development, in which they outlined the challenges associated with curing a rare disease. Namely, there are many rare diseases (greater than 7000), and they are all competing for funding and attention; because each rare disease affects a small number of patients, it can be hard to attract interest in pharmaceutical development for that indication; and because those rare disease patients are few and often spread out across the country, it is hard to enroll them in clinical trials in large numbers.
In addition to being Valentine’s Day, February 14th is also National Donor Day. Those that have received a life-saving organ, tissue, or blood donation know just how important that is. Whether you donate blood every few months or you are a registered organ donor, you are helping to save lives.
To repeat, that’s 42% of people in the US think that they can survive without a liver. This shocking statistic, which was derived from a survey of 511 people representative of US households, highlights a deficit in awareness of the vital, multifunctional role the liver has in the body. A deficit that could be costly to many, considering that it is not just alcohol that can cause liver damage - many common and controllable diseases such as obesity, high cholesterol and type 2 diabetes can put liver health at risk.