On 27th April 2017, Takeda's Multiple Myeloma (MM) drug Ixazomib (Ninlaro®) received a draft 'no' from the National Institute for Health and Care Excellence (NICE) for use in combination with lenalidomide and dexamethasone, for the treatment of MM patients that have received at least one prior therapy. Currently, ixazomib is licensed by both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) for this indication, but in practice is often used to treat relapsed and refractory (RR) MM patients who have exhausted other available therapies.
This is disappointing news for England and Wales, as it is unlikely that ixazomib (a highly convenient orally administered drug) will be routinely available on the NHS, limiting treatment options for RRMM patients. NICE have justified this decision on the basis of limited data to indicate that ixazomib is superior to other currently available therapies, that are also more cost effective. NICE argue that the pivotal clinical trial for ixazomib (Phase III TOURMALINE-MM1 trial) is ongoing, and therefore the data is not fully evaluable, particularly in terms of patient subsets that have received two or more treatments. Takeda is keen to work with NICE in order to revoke this decision and to establish more watertight cost-effectiveness calculations.