Rescuing orphan drugs

Rare diseases affect many millions of people; by one count Europe alone has 30 million such patients with more than 6000 conditions. So, developing the so called orphan drugs for these diseases really matters. The problem is that no one really knows if they are better than existing treatments, because the evidence requirements for regulatory approval are low, and many studies rely on unproved surrogate endpoints (among other reasons). Despite this, orphan drugs are usually highly expensive, and doctors are likely to be drawn to the newer drug without realising that its benefits are uncertain.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 (doi:10.1136/bmj-2022-072796).1 The US offers new evidence that not all drugs benefiting from orphan status are actually for rare diseases (doi:10.1136/bmj-2022-073242).2 A study looking at FDA approved cancer…
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