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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. "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. 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.
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This week in The BMJ there is much reflection on the “new normal” of NHS services—that is, the reality of life after the first brutal years of the global covid pandemic.
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Even as the NHS scrabbles to fund pay rises, staff are faced with a pay cut in real terms, and unions are talking tough (doi:10.1136/bmj.o2166).1 Burnt-out junior staff are at the sharp end, with the lowest pay deal of all (doi:10.1136/bmj.o2118).3 At the other end of the career spectrum, Truss has pledged to “sort out” the pension problems driving senior doctors from the NHS (doi:10.1136/bmj.o2166).1 For Partha Kar (doi:10.1136/bmj.o2143) this is the crux of the problem: “Without consultants doing the procedures the waiting lists won’t get shorter.” Clinically vulnerable people are already being admitted to hospital after having their energy supplies disconnected (doi:10.1136/bmj.o2156).6 And the effect on children’s health of living in fuel poverty is both immediate and long term. Adults living in cold homes put their respiratory and cardiovascular health at risk, but for children it can lead to a lifetime of health inequalities (doi:10.1136/bmj.o2129).7 Michael Marmot and colleagues’ bleak assessment is an urgent call for action. 1 Wise J. New prime minister must prioritise NHS, say doctors’ leaders.
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[...]at least 24 of England’s 137 NHS trusts have declared critical incidents, but there is good reason to believe the effect on care goes far beyond this, and many hospitals are struggling under the radar (doi:10.1136/bmj.o60).1 The BMA warns that a dramatic slowdown in the provision of non-urgent care is causing “untold suffering” to the record nearly six million patients on waiting lists (doi:10.1136/bmj.o45).2 The army has been called in to help London hospitals (doi:10.1136/bmj.o47),3 and people are being asked to find lifts to emergency departments because ambulances are taking so long to arrive. Staff absences in hospitals are up by around 40%, and almost half of staff report illness from work related stress (doi:10.1136/bmj.o51).4 There is no reason to think that staff shortages in general practice are any different from acute services, says our GP columnist Helen Salisbury, whose practice has four staff members absent (doi:10.1136/bmj.o43).5 “It’s difficult not to be angry,” she says, “as this current surge of cases and burden of suffering was predicted and preventable: we had a chance to flatten this wave, and our government chose instead to do nothing.” Even without a plan to build a future workforce, there are sensible calls to mobilise more staff, such as by asking medical students to volunteer and by shortening self-isolation periods for infected staff, but these calls are not backed by the government (doi:10.1136/bmj.o38).6 “We should not be in this position two years into a pandemic,” says the NHS Confederation’s Matthew Taylor.