Alagille Syndrome

Experts Discuss Breakthroughs in Addressing Patient Priorities for Primary Biliary Cholangitis

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In a recent episode of the European Association for the Study of the Liver (EASL) Studio, experts gathered to shed light on patient priorities in primary biliary cholangitis (PBC), including a growing recognition of the significant symptom burden faced by patients.

Robert Mitchell Thain, CEO of the PBC Foundation, emphasized the need to redefine ‘normal’ for patients living with PBC. He stressed that beyond biochemical improvements and liver structure, achieving a ‘normal’ quality of life is equally crucial.

“Yes, we’re looking for normality within the structure of the liver, so looking at the histology of the liver, but we’re also looking for normal as in quality of life and living what is genuinely a normal life with PBC,” he said.

A national audit in the United Kingdom found that only 60% of clinicians ask their patients about their symptoms and how their quality of life is affected by PBC. This prompted a call for action, urging healthcare providers to proactively engage with patients about their experiences.

“The best and first thing we can do when we serve PBC patients is to ask them about PBC and their PBC in particular,” Thain said. “I think we must do a bit of a better job as time goes by both asking these questions and having patients be able to share their voice because I think it’s quite noticeable in recent years that there’s been a lot more industry interest and focus on therapies for PBC.”

Alexander Artyomenko, MD, PhD from IPSEN, Stuart Kendrick, PhD from GSK, and Pam Vig, PhD from Mirum Pharmaceuticals highlighted pharmaceutical companies’ commitment to developing therapeutic agents targeting symptoms in PBC. They underscored the importance of understanding the multifaceted impact of symptoms on patients’ lives.

“I think companies are always looking for new opportunities to make a difference because that’s how they can fulfill their mission and deliver what they really want to do for patients,” Dr Kendrick said. “There has been a movement recently to identify things that really matter to patients because if we can address the important needs for patients, then there’s a good chance that we’ll make successful medicines.”

Vig noted a recent video made by Mirum that emphasized the importance of understanding what it means to have pruritus without normalizing severity because the patient lives with it day-to-day.

“I think that it’s very clear that there’s a significant unmet need in patients’ quality of life,” Dr Vig said.” Some of the work that we’ve done at Mirum is to try to understand what actually happens in the clinic and how we can educate and ask a different set of questions.”

The panel discussed the significance of patient-reported outcomes (PROs) in assessing symptom severity and impact. They emphasized the need for tailored tools to effectively measure symptoms and their influence on patients’ daily lives.

The conversation further delved into the complex interplay of symptoms like itch, fatigue, and cognitive issues. While progress has been made in treating itch, there remains a significant unmet need in addressing fatigue and cognitive symptoms.

Collaboration emerged as a key theme, with stakeholders emphasizing the importance of early dialogue and continuous communication between patients, clinicians, and the pharmaceutical industry. This collaborative approach is seen as essential to optimize care pathways and develop more effective treatments.

“I think the key to this, is each of us coming together as a progressive and -looking partner, acknowledging that we have one part of the puzzle, but we need you to fill in the gaps,” Thain said.

Just like hepatocytes fit into their designated spaces in the background, he said, each of us has a specific role in the bigger picture.