FDA, a dance with the Red Queen in liver disease Wonderland
As I reported earlier, this was the outcome of the FDA meeting on the first drug candidate to successfully meet the FDA's requirement of reducing fibrosis by at least one stage.
The FDA advisory panel voted to reject Intercept's application for OCA as a treatment for NASH
Despite meeting the goals set by FDA, of reducing fibrosis by at least one stage, the GIDAC panel today voted against approval of obeticholic acid as a treatment for NASH.Read more
FDA advisory panel votes against NAFLD/NASH therapy
The FDA advisory panel voted to reject Intercept's application for OCA as a treatment for NASH
Despite meeting the goals set by FDA, of reducing fibrosis by at least one stage, the GIDAC panel today voted against approval of obeticholic acid as a treatment for NASH.
As a patient, I'm very concerned by this decision. The concern expressed by the panel was that there was an increased risk of bile duct disease in a small number of patients in the clinical trial. The exact statistics will be published but we believe that the number is small compared to the number of people who will die from the disease if untreated Personally, I'd like to be able to have a chance to decide that risk for myself. I believe that most hepatologists know how to manage those risk factors.Read more
FDA sausage being made tomorrow, will they kill another NASH drug
The FDA is finally going to act on the first drug for treatment of NASH
The FDA staff does not support approval - you can watch the meeting live if you care
Click this link to join the meeting 9:00 AM EST Friday May 19th
A little background. The Intercept drug obeticholic acid or OCA is the first drug to meet the FDA requirement of reducing NASH fibrosis by at least one stage. There is no therapy for NASH and it is a serious unmet need. We all know that and the meeting tomorrow will end with a vote of whether to recommend approval.
It is a public meeting and I invite you to watch. If you have NASH this could be significant for you. They have a brief window for public comment and I get 4 minutes to speak on our behalf as patients.Read more
AI is coming!!! We are all going to die -- or maybe not -- clickbait anyone?
I am both amused and appalled by all of the stories about AI that are pummeling us lately. This is the latest fashion doing the walk down the breaking news parade route.
OK Wayne, just stop. You might know a little about liver disease but this is serious. I heard all the billionaires say its awful.
Maybe we could just take a breath. We need to remember that the world today runs on media exposure and audience. In the long ago we used to see cartoons about the guy in the public square with a sign saying "The World Is About To End". That was good for a chuckle. Today it's someone in a suit on TV with a new book or available for speaking tours. We have upgraded our doomsayers but now they are very successful at provoking our fear.Read more
The ICER man came, the ugly, the bad, and the good
IN BRIEF: ICER HATES obetacholic acid, and dislikes resmetirom
THE UGLY-- ICER voted 14 to 1 not to recommend obeticholic acid as a treatment for NASH
ICER voted 8 to 7 to reluctantly recommend resmetirom
A question that I found to be very distressing personally was defeated 9 to 6. The question: Would having a treatment for NASH have any effect on caregivers? UnbelievableRead more
The ICER man cometh, do you know how insurance denies you drugs?
New drugs are coming for liver disease, but will we be able to get them approved by insurance?
In a few weeks the FDA will have its public comment meeting for obetacholic acid for treatment of NASH. This is in response to an application by Intercept for approval. If FDA gives it a go ahead, the issue of the cost of the drug will be the next big thing.
Remember this, the insurance company is not actually in business for your health. They don't get bonuses because you were cured. Their job is to not spend all of the premium money so they carefully manage what they are willing to pay for.Read more
Here at last! Here at last! The CARE SURVEY is here at last
With drugs coming soon, I REALLY NEED YOUR HELP!!!!!!
In the 2022 survey only about 10% of the patients contacted bothered to help. We need to do better. Here are links.Read more
Important information for NAFLD/NASH patients
The FDA will consider two drugs for treatment of NAFLD/NASH this year. I know we have been saying that therapy is coming for several years only to be disappointed. It is easy to be a little cynical about the prospects. This is a government agency and it seems like delay is what all agencies do best, but I do believe we will finally have a chance to move forward.Read more
Dilemma, what to write about, butt crack deoderant or patient abuse
I've been seeing ads for butt crack deodorant and I've wondered what societal changes have made that a "thing". I've been soooooo tempted to write an April Fool blog about all the things that could go wrong if sniffing replaces fist or elbow bumps as the way we greet people in an age of pandemic fear. But, I'll leave those imaginings to you. I want to talk about doctors who are clueless about liver disease.Read more
There are some things that make me mad, but who should I blame?
If you are like I was, I knew that our life expectancy in this country had declined recently, but I had no idea. I was shocked by this report by NPR.
Would you like to tell the FDA how you feel about having a NASH therapy available?
FDA has scheduled an adcom (advisory committee meeting) for Intercept's candidate drug to be the first approved treatment for NASH.
This is the public's opportunity to provide input to FDA as part of its deliberations about approving a drug. If you would like to have a chance to add your views to the discussion, this is how you do it.
Being unwell sucks, but we need your help
I know what it is to be unwell, but I need you to think about this result from our State of Care Survey. Only 16% of you reported feeling like you got enough information from your doctors.
The State of Care for NAFLD/NASH patients, So many things need to be better
I invite you to read the report of the 2022 State of NAFLD/NASH Care in America Survey. This survey looks at the experiences of patients from the perspective of their day to day lived experience. Many of the results are disturbing as they highlight how poorly liver disease is managed in the broader community. There are certainly pockets of excellence and patients fortunate enough to live in their footprint are well served. For many, the path is more difficult than it should be. We hope this work leads to progress in the care of the patient community at large. To read the report click on the image below.Read more
It's Charlie Brown time again with FDA, will they or won't they approve a NAFLD/NASH drug
People who are lost can walk in a circle and get back to where they started. It is a bit like that for the patient community. In 2020 we were getting ready for the FDA to hold an adcom meeting on the Intercept drug Ocaliva. Adcom is where they take public testimony and as patients we wanted to speak. This was to be the first drug that had met the targets set by FDA for a NAFLD/NASH therapy. We all knew it wasn't a perfect solution but when there is no therapy at all it was a place to start and we were very hopeful.Read more
Where have all the heroes gone? Gone to graveyards every one
My grandson asked about our family tree for a school project and I found myself thinking about the changing character of our society and wondering how we have become so dysfunctional.
I have been fortunate to have lived in a region that may be one of the last parts of the country to have retained aspects of the society that settled the West in the 1800's. Both sides of my family were part of the wagon train movement that came west and settled in Idaho.Read more
New guidance for NAFLD/NASH is here, what does it mean for patients
The new guidance for managing NAFLD/NASH has been officially published. This is the official guidance by the American Association for the Study of Liver Disease (AASLD) and it is intended to provide primary care and other providers with the information they need to provide care for their patients. A key element for us is that it supports early intervention for high risk patients and provides clear guidance for how to risk stratify and to determine who needs referral to a specialist.Read more
Would you risk your life to save your child? Should other people protect your child?
Very few parents would say that they wouldn't take risks to save their child. In fact many people would try to protect unrelated children from harm. These impulses are just part of our nature. But what are the limits to that? At what point do we stop being concerned about harm to our own or other children?
OK, this is a really strange question for a patient health blog but stay with me for a little while.Read more
For Patients, One Battle is Ending, But a Bigger One is Next
When I was a newly diagnosed patient with cirrhosis, one of the things that offended me was that my doctors had followed practice guidelines that said not to screen for asymptomatic disease. I had to be hospitalized before I learned anything about my liver getting sicker for at least a decade. Many of you can relate to that, as you have told your stories in the groups.
It hasn't been officially published yet, but the new guidance by the American Association for the Study of Liver Disease (AASLD) will soon release new practice guidance. Here is a summary slide of the key points.Read more
NAFLD Stigma Survey Project - Interested in Participating in a Patient Survey?
We have been asked by a colleague to help distribute this study on stigma to the patient community. Please find below information about this opportunity to share your thoughts and contribute to NAFLD research by completing an anonymous survey:Read more
Intercept announces resubmittal of Obeticholic Acid to FDA as a treatment for NASH fibrosis
INTERCEPT RESUBMITS NEW DRUG APPLICATION TO FDA FOR OBETICHOLIC ACID IN PATIENTS WITH LIVER FIBROSIS DUE TO NASH
A SURPRISE CHRISTMAS PRESENT FOR PATIENTS WITH FIBROSIS. IF YOU WOULD LIKE TO READ THE PRESS RELEASE CLICK HERE.
A bit of history may be helpful to many. Obeticholic Acid (OCA) was filed with FDA in 2019 but FDA didn't go through with a planned patient meeting called an adcom and instead issued what they called a complete response letter in June of 2020 and raised questions about safety. They did not reject the application they just punted. The department was going through some reorganization at the time and from a patient perspective we did not believe the sequence was proper but we were stuck with it. Most companies would have abandoned the effort at that point. To their great credit Intercept believed in the drug and continued their studies to answer the safety questions that had been raised. Now they have much more data and good evidence of safety which we believe should resolve that issue.Read more