My Time as a PBC Patient Representative for the FDA

In September of 2024, I served as the PBC Patient Representative for the United States Food and Drug Administration. The FDA was reviewing the medication Ocaliva, which was made by the pharmaceutical company Intercept. That was a life-changing day for me.

Reviewing whether a PBC treatment was safe

In April of 2024, I had met the Global Liver Institute's Director for Rare Disease, who had nominated me with the FDA for this role. After an interview and a lot of paperwork, I was chosen as the PBC patient representative. I did some online learning to try to prepare for my role being on the Gastrointestinal Drugs Advisory Committee, or GIDAC. But looking back on it now, I was not prepared at all for what I was walking in to.

The FDA was reviewing Ocaliva, which was a second-line therapy to treat PBC. Ocaliva was originally approved under Accelerated Approval. Meaning the findings of the clinical trials performed at the time were enough to approve the medication, but a confirmatory trial was still needed. So, the medication was approved for patients, and the final end-point trial would be performed in the meantime, so patients could have access to the medication.

Unfortunately, Intercept's trial ran into some major issues that led to regulators not having the data they needed to prove its safety. The committee meeting I was part of was helping to make the decision if the medication was proven to be safe. And unfortunately, the only thing to go off of was data received from an incomplete clinical trial.

An issue: patients were dropping out of the clinical trial

Many factors affected the trial. But the main one I really want to discuss is patients dropped out of the trial when they realized they were on a placebo.

With primary biliary cholangitis, a patient has to be on medication to control the disease and prevent liver damage. So, patients left the trial to try to gain access to medications instead of a placebo.

With long-term confirmatory trials, patients may have to be on placebos for 8 or more years. That's a huge risk to patients' lives, and it's an unethical ask in my opinion as a patient. Because of that, patient retention became a big issue regarding obtaining data for the clinical trial. Intercept wasn't able to obtain enough data proving the medication Ocaliva was safe for regulators to approve.

Voting on whether the medication should be allowed

At the GIDAC meeting, the panel and I listened to the FDA share their points against the medication. We also heard Intercept share their points on the data they were able to obtain, and the issues they ran in to. In the early afternoon, the public was allowed to speak both in person and remotely. I saw patients I knew and considered friends speaking. And that's what I wasn't prepared for.

I wasn't prepared for friends of mine to share their experiences on the medication and how it had helped save their life and managed their disease without complication. The emotional aspect is what broke me that day. Even though statistical data didn't show efficacy, the real-world data of patients' lived experience did - and to me, that is what mattered most.

Out of the entire list of panelists, I was the only person to vote that the medication should stay on the market because they were able to show at the right dosage and with the right patient, Ocaliva worked. But ultimately Ocaliva was removed from the market in the United States.

I was upset for patients

I was really grateful for my Uber driver that day who let me cry in his back seat all the way to the airport without trying to ask me any questions. I was upset for patients, and I was scared for patient's lives in the future.

Because not every medication works for every patient, and all patients deserve access to life-saving therapy.

Trying to change how clinical trials work

That day I really felt a calling for the role of real-world evidence in clinical trials. If patients dropped out of trials for one medication because of placebos, they likely would drop out of another trial for the same reason. And that can make obtaining the needed regulatory data difficult.

Over the last few years at the International PBC Summit hosted by the PBC Foundation in the United Kingdom, we have been working on bringing in real world evidence to clinical trial data. The end-goal is to end long-term placebo arm trials in PBC. I believe we will be able to make it a reality one day. Because I think it's unethical to ask a patient to not be on any treatment for an extended period of time and risk their life.

We just need the regulators to work with the patients living with PBC to develop a way to satisfy both parties and not risk patients' lives.