Twenty-three international patient organisations – including Swiss HePa – have addressed an open letter to the relevant regulatory authorities. Together, we are calling for modern regulatory pathways towards the full approval of effective second-line treatments for Primary Biliary Cholangitis (PBC).

Long-term placebo-controlled confirmatory trials may expose people living with a progressive rare disease to considerable risks. Participants in a placebo arm may remain without effective treatment for an extended period, even though elevated ALP and bilirubin levels are associated with disease progression.

Modern solutions are therefore needed: alternative study designs, real-world data and the meaningful involvement of people living with PBC and their patient organisations.

People living with PBC must not only be part of the conversation – they must be part of the solution.

For Swiss HePa, responsibility does not end with regulatory approval:

• Swissmedic is responsible for modern approval pathways and regulatory requirements.
• The Federal Office of Public Health (FOPH/BAG) plays a central role in reimbursement and effective access to treatments.
• Policymakers and healthcare stakeholders must create the legal, financial and structural conditions that allow medical innovation to reach people.
• Patients and patient organisations must be involved early, meaningfully and as equal partners in these decisions.

Approval, reimbursement, access to care and patient safety must not be considered in isolation.

Innovation must reach the people who need it.