tolvaptan (Jinarc)

following a full submission under the orphan process

tolvaptan (Jinarc®) is accepted for use within NHS Scotland.

Indication under review: to slow the progression of cyst development and renal insufficiency of autosomal dominant polycystic kidney disease (ADPKD) in adults with chronic kidney disease stage 1 to 3 at initiation of treatment with evidence of rapidly progressing disease.

In a phase III placebo-controlled study tolvaptan, after 3 years, had significantly slowed the rate of disease progression as measured by impact on the rate of increase in total kidney volume (TKV) in ADPKD patients who were deemed to be at high risk of disease progression and had relatively preserved renal function. The study inclusion criteria included (list not exhaustive): age 18 to 50 years old, TKV ≥750ml and creatinine clearance ≥60ml/minute.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of tolvaptan. This advice is contingent upon the continuing availability of the patient access scheme or a list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.