VIDEO: Better real-world data needed for FDA PBC approvals

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Really exciting and difficult time for people living with PBC. The community of doctors and patients knows where they want to get to. Normal tests, normal quality of life. The regulators have a duty of care to license therapies across all diseases according to regulatory framework. Patients have expectations but regulators require evidence. Evidence is very hard to generate in rare diseases which are slowly progressive, where hard endpoints, death and transplant, are infrequent, and where surrogate endpoints are already used in clinical practice.

Therefore, we are very mindful of what's going on at the EMA and the FDA as they look at the conditional approval of therapies for PBC. And we can only hope that the patient is kept in the center of those decisions because as prescribing clinicians, we feel that we have identified how to use second-line therapies safely and effectively. But we'll continue to work with patients and regulators to get better real-world data because we don't believe that long-term placebo-controlled endpoint studies are possible. We don't believe they're ethical.

We don't believe our patients will accept to stay on placebo for 10 years and even if they did, with early disease, we don't believe that there'd be enough events to demonstrate efficacy anyway. So we'll continue as a group of interested and academic clinicians with our patient partners to get better at providing the real-world data to the whole audience that we believe we already see in our own practices.