Rescuing orphan drugs

ABSTRACT

Philip Kranz and colleagues argue that drug regulators should not automatically assume orphan drugs are clinically "superior” for patients, in the absence of robust evidence of their clinical benefits (doi10.1136/bmj-2022-072796).1 The US offers new evidence that not all drugs benefiting from orphan status are actually for rare diseases (doi10.1136/bmj-2022-073242).2 A study looking at FDA approved cancer treatments over the past 20 years found that most approvals for cancer indications were designated as orphans. "Are we still getting what we thought we were paying for?” asks Joseph Ross (doi10.1136/bmj.p928).3 Evidence matters and can take many decades of endeavour to gather, as is the case for a new vaccine to prevent respiratory syncytial virus bronchiolitis in infants (doi10.1136/bmj.p1023).4 The RSV virus kills very young children, mostly in low to middle income countries, and a pandemic related surge in incidence resulted in many hospital admissions. FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications cross sectional analysis.