On 27th January 2016 the National Institute of Health and Care Excellence (NICE) released guidance recommending the use of panobinostat, in combination with bortezomib and dexamethasone, for the treatment of adults with relapsed or relapsed and refractory [R/R] Multiple Myeloma [MM] who have received at least two previous regimens (including bortezomib and an immunomodulatory agent).
The main reason for this recommendation was cost efficiency as a result of the patient access scheme granted by Novartis for the use of panobinostat.
Key clinical evidence came from the PANORAMA-1 randomised controlled clinical trial (n=768), which compared panobinostat with a placebo on a background of a bortezomib and dexamethasone combination regime. An indirect comparison of panobinostat in combination with bortezomib and dexamethasone, with lenalidomide plus dexamethasone was also assessed, as well as a semi-Markov model comparing the cost-effectiveness of panobinostat with dexamethasone with lenalidomide plus dexamethasone.
The committee discussed concerns regarding the uncertainty of the evidence, such as the relevant age group and the fact that a large number of patients had received a stem-cell transplant, and had been receiving bortezomib for a long period. It was also noted that only a sub-group of patients fell into the category for which panobinostat has marketing authorisation (i.e. RRMM having had two or more previous treatments). Despite this, the results of the PANORAMA 1 trial were deemed relevant to NHS patients, and panobinostat in combination with bortezomib and dexamethasone was thought to be clinically effective.
Taking into account all the evidence, the incremental cost-effectiveness ratio for panobinostat, in combination with bortezomib and dexamethasone, was deemed to be no higher than £25,000 per QALY gained.
In summary, due consideration was given to both the relevant clinical evidence, plus the calculated cost-effectiveness ratio for panobinostat in combination with bortezomib and dexamethasone, and outweighed the benefits of the current regime of lenalidomide plus dexamethasone, to treat RRMM patients that have received two or more previous treatments. This is in line with the approval of panobinostat granted by the EMA in August 2015.