Regulatory approval of new medicines has increased in recent years. In 2018, the U.S. Food and Drug Administration (FDA) approved a record 59 “novel” medicines (their active ingredients had never before been approved). In many therapeutic areas, it is now common to have several novel products approved contemporaneously that compete for similar patient populations. Examples include patisiran and inotersen for a hereditary form of amyloidosis and galcanezumab and fremanezumab for prevention of migraine. Randomized controlled trials (RCTs) that directly compare these types of agents are exceedingly rare.
In the absence of head-to-head RCTs, indirect comparisons are required to evaluate the comparative benefits and harms of multiple treatment alternatives. Methods for evaluating treatments that have never been directly compared have evolved significantly and are now widely used. Inclusion of new evidence in network meta-analysis (NMA), which allows for indirect comparisons of treatments linked through common comparators, has been found to identify clinical signals sooner than more traditional meta-analysis approaches. Such advancements can benefit health technology assessment (HTA)