Anticipating approval this summer of the first drug for PFIC, Albireo hopes to add Alagille syndrome and biliary atresia to odevixibat labeling. Firm has modeled its commercial plans on the success of other rare disease firms.

Bristol Myers Squibb says it is common for cell and gene therapies to revert to the standard timetable as ide-cel follows liso-cel in losing its accelerated assessment status at the European Medicines Agency. Separately, the company has discontinued development of orva-cel, a CAR-T cell therapy that was earlier in the development pipeline.

Dermatology, chronic kidney disease and liver disease therapies stand out in a crowd of user fee goals that is actually bigger than at the same point in 2020.

Executives share their expectations and hopes around the multi-faceted topic of patient access to medicines. From health inequities to value assessment and reimbursement trends, expectations for change abound.