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 treatments over the past 20 years found that most approvals for cancer indications were designated as orphans. These included common cancer indications, not just those that were rare or ultra-rare. “Are we still getting what we thought we were paying for?” asks Joseph Ross (doi:10.1136/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 (doi:10.1136/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. The vaccine’s deployment is part of a wider campaign against RSV, including maternal vaccination during pregnancy, although experts are urging a slower pace on such a vaccine currently being fast tracked in the US (doi:10.1136/bmj.p1021).5

SARS-CoV-2 treatments were, of course, developed at warp speed. Looking to the future of covid treatments, the focus is not so much on the “new” as on continuing current research to improve access to drugs we already know work (doi:10.1136/bmj.p872).6 This is not to rule out the need for new drugs, especially antivirals. For patients with long covid, in particular, substantially more progress is needed. In the meantime, helping patients back to work is key (doi:10.1136/bmj.p800).7 S Thompson identifies health service colleagues who could return if supported with adaptations and flexibility, “but they are denied them.”

As the World Health Organization officially declares an end to covid as a global health emergency (doi:10.1136/bmj.p1041),8 one sign that covid is abating is that the post-covid wave of violence in UK primary care is beginning to recede (doi:10.1136/bmj.p995).9 But a legacy of this scarring time is a phalanx of protective measures such as panic buttons, screens, and CCTV in GP surgeries. Some doctors lament this shift, while others think a zero tolerance approach to violence against staff is the only response. Sadly, these conditions may feed into UK GPs’ rock bottom satisfaction with their job (doi:10.1136/bmj.p966).10 If Rishi Sunak’s bid to ease pressure on GPs fails to land this week, a rescue plan will be desperately needed (doi:10.1136/bmj.p1046).11