Novartis' JAK inhibitor Jakavi patient populations to come under NICE microscope
This article was originally published in Scrip
NICE, the health technology appraisal institute for England and Wales, is to review Novartis/Incyte's orphan myelofibrosis treatment, Jakavi (ruxolitinib). The institute's draft appraisal scope, now out for consultation, indicates that the institute will be paying close attention to whether it can narrow the potential patient population.
The oral JAK1 and JAK2 inhibitor was the first treatment to win US approval for myelofibrosis, however, it is yet to be cleared by the EMA. Myelofibrosis is a potentially life-threatening blood cancer that is marked by an enlarged spleen, anaemia and decreased white blood cells and platelets. The only curative treatment currently available is haematopoietic stem cell transplant, but this is only suitable for patients fit enough to undergo treatment. Other treatment options include androgens, splenectomy, chemotherapy, splenic embolisation and radiation therapy and aim to relieve symptoms and improve quality of life.
In its scope, NICE suggests that drug is compared with standard management that does not include ruxolitinib. The institute will be examining the following outcomes: symptom relief (including pain and fatigue); overall survival; progression-free survival; response rate; adverse effects of treatment; and health-related quality of life.
The institute is seeking comments on what place the drug might take in the treatment pathway and what proportion of myelofibrosis patients might benefit.
The institute also seems to be considering whether to restrict the patient population to those who are unable to undergo haematopoietic stem cell transplant and is asking for comments on whether this would be advisable. It also wants to know if there are any other patient subgroups in whom ruxolitinib may be more clinically or cost- effective.
YM BioSciences is developing its own JAK1/JAK2 inhibitor, CYT387 as a potential treatment for myelofibrosis. Meanwhile, Promedior is developing PRM-151, a recombinant human pentraxin-2 protein.
Other manufacturers in the JAK inhibitor space will also be interested to see how the drug fares. Jakavi was the first drug of this class to win US approval.