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? Unbelievable
There were a lot of issues that were discussed so I can't address most of them here. At the root of the negative votes is the belief within the ICER panel that NASH is not a progressive disease. As a patient who went from F2 to F4 in 5 years I objected to that view but I'm confident I was ignored.
The ICER meeting was very disappointing for patients. I can only skim the surface with this blog. If it is of interest, here is a link to my testimony
THE BAD: The ICER economic analysis makes assumptions that we don't support
The goal of the ICER exercise is to provide a presumably unbiased cost/benefit analysis of new drugs. This is a worthy goal. We question some key assumptions. I suppose they are trying make a general case, but the first rule of their analysis is that the drug cannot increase the total cost of healthcare. OK that means you have to replace some other cost. Since they don't believe that NASH is progressive, it is hard to calculate societal costs.
We weren't pleased that they used out of date pricing in their analysis. Just one example, they charged $350,000 for a liver transplant. Maybe in 1999 but try getting that today.
It was distressing to realize that there was only one liver doctor on the panel. It seemed clear that most had little knowledge of the disease. They certainly didn't appreciate the consequences to the caregivers and the families. They also did the cost analysis assuming a patient would take these drugs for the rest of their life. That just isn't going to happen.
They complained that they didn't have enough phase 3 data even though they knew that resmetirom was just finishing their trial so that data wasn't available. It was clear that the goal for holding this meeting had little to do with the real data. It was timed to be just ahead of the FDA deliberations about obeticholic acid. I'm disappointed because we need to understand cost/benefit but how do you trust an opinion that is so clearly an effort to manipulate the narrative.
If you are interested, here is a link to the early draft report from ICER a final report will be issued.
It is unlikely that the FDA will pay any attention to the ICER recommendation. The FDA is charged with making a medically relevant determination and is not supposed to be influenced by this kind of material. Hopefully they will be faithful to that responsibility.
This does however, point to a very important issue for patients. Insurance companies do use these reports as they decide who will get coverage and under what conditions. At the meeting some insurers talked about how they controlled access and even acknowledged that they might approve a drug because of price instead of what was best for the patient. At the foundation we hear stories all the time of doctors prescribing drugs that the insurer won't cover and patient simply can't afford. There is a moral question in here somewhere. How is it fair to provide Cadillac care to some and deny care to others because they are not well connected or well off?
Some of you have taken our 2023 survey, The State of NAFLD/NASH Care in America and we thank you. Most of you have ignored it
I do understand. I get many surveys that I just delete. Who has time? But if NAFLD/NASH affects you, you need to stand with us. I can huff and puff but without the support of the patient community I'm just a sad old guy making a fool of himself in the parking lot. Nobody cares until we band together so add your voice.
Do your part and take 20 minutes to help us along.