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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.

Do you think FDA cares what patients think? They have an entire section of their website explaining how much they care.

This was my first time at one of their public comment meetings and it clearly wasn't intended as an event to engage in any thoughtful discussion.  It was a staged event carefully managed to record things in the record and to check a box on a checklist somewhere, but there was no engagement. No one was seeking information or understanding.

Imagine the scenario. You get 4 or 5 patients who get to speak for 3 or 4 minutes. No one ever asks them a question, it is just to record that the public spoke.  The first public speaker was a lobbyist from Ralph Nader's old operation Public Citizen. Not a patient, not a doctor, not someone who could speak to the patient journey.  His job was to read the FDA staff comments into the "public" record.  In a magic act, that "person from the audience who would like to help", is called a shill.

To be clear, I have no problem with any lobbyist group speaking up, but it is wrong to come on a panel discussing life and death drug therapy and imply that you are a medical doctor with knowledge rather than a professional PHD lobbyist reading someone else's script. We had real physicians there who know liver disease intimately who were mostly ignored.

This was theater, but one that offends me. There were a couple of people in the OCA trial who died of billiary disease.  I think a statistic they came up with in voting against the drug was that it could cause 1 or 2 excess deaths per 1,000 patient years. That is something to consider certainly.  The fact that we lose about 90 friends a day to liver disease was not considered. We know the drug works for some, we get their testimony in the peer groups, so my question is how many people should get a chance to take the risk knowing that 1 or 2 of them per 1,000 patient years may die?

Another argument presented by the FDA panel was that the doctors may not be able to manage patients with these extra risks. No one was allowed to comment on the odd fact that the drug is actually approved to treat bile duct disease and doctors manage those patients every day. Real physicians I've spoken with believe they are equipped to manage the side effects.

The FDA staff is opposed to approval of this drug.  I might agree if it was not the only option we currently have for a disease with no treatments at all. That is a risk benefit analysis patients should be allowed to make in consultation with their physicians. Instead we dance with the Red Queen who Louis Carroll described this way.

The Red Queen I pictured as a Fury, her passion must be cold and calm – she must be formal and strict, yet not unkindly; pedantic to the 10th degree, the concentrated essence of all governesses!

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 doctors know how to manage those risk factors.

For me, the most important thing about the drug was that by showing that for some people it could reduce fibrosis proved that for a larger group it would be able to slow or stop progression.  For someone who has not yet become a cirrhosis patient that is the most important thing.  Don't make me get really sick before you pay attention to my problems.

I see that thousands of you have not yet taken the survey, The State of NAFLD/NASH Care in America. Do you think organizations like FDA will pay attention if there is little evidence that you care?

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