Why does medicine continue to work poorly? In the case of children with gender-related distress, the Cass Review, published two weeks ago, makes clear that for years doctors have been prescribing powerful hormones to these children despite a lack of evidence that this would help them.
“The reality is that we do not have convincing evidence about the long-term outcomes of interventions to manage gender-related distress,” wrote Dr Hilary Cass, a pediatrician charged with reviewing NHS gender services.
Dr Cass described how doctors had been largely influenced by a single Dutch study, which excluded children who had dropped out of the research because they developed problems with the treatment.
However, doctors applied the findings to many more children and, as she wrote, “abandoned normal approaches to holistic assessment.” So, for example, children did not have the impact of depression evaluated.
As a result, young people with gender dysphoria (where they feel their biological sex does not match their gender), who have often been suffering greatly, have received poor care from the health service.
High-quality trials of heart rhythm pills prescribed to people who had had heart attacks found they increased the risk of death
I have known many who turned to the NHS for help and ended up with surgery and medications whose side effects they later deeply regretted.
But sadly, this is just one part of a long-term pattern of health services acting without good quality evidence.
The only reason medicine should be authoritative is because it is based on science, where assumptions and hypotheses are tested and questions are asked about harms and unintended consequences. If it’s not based on science, frankly, it might as well be homeopathy.
It’s easy to think you’re helping when, in reality, you’re hurting people.
In the 1980s, it was recognized that some people who suffered heart attacks developed fatal heart rhythm disorders; These patients often had abnormal heartbeats and the logical assumption was that reducing them would reduce mortality rates.
And yes, trials showed that taking drugs called class Ic antiarrhythmics reduced those abnormal heartbeats.
However, when high-quality trials were finally conducted, it became clear that they achieved the opposite in terms of saving lives; in fact, the drugs led to an increased risk of death. What seemed “logical” was actively harmful.
And it is not enough to say that you have “evidence”: it has to be of good quality. Take arthritic knees, for example. For decades, surgeons performed “washout” arthroscopy, in which patients had incisions made in the joint that were then washed with saline; The idea was that cartilage fragments would be removed, making the joint work more efficiently.
Certainly, patients tended to report that it made them feel better. But in 2002, a trial compared knee arthroscopy with a placebo: Patients either had the real operation or a “placebo” procedure in which they also had small incisions made in their skin and surgeons “sprayed” saline to to make it seem like the actual operation had been real. happening, but he didn’t actually put the saline solution into the joint.
For years, pregnant and breastfeeding women with a family history of allergies were advised to avoid eating peanuts, in case they increased their child’s allergy risk. But peanut allergy rates in the population skyrocketed
Both the placebo and actual surgery groups improved, with patients describing less pain and better function over the following months.
Medicines that cannot help you can only harm you, either through side effects or costs to the patient and the health service. That’s why, unless in very specific circumstances, the National Institute for Health and Care Excellence (NICE) is now clear that “arthroscopic knee lavage alone should not be used as a treatment for osteoarthritis.”
There are other examples. For years, pregnant and lactating women with a family history of allergies were advised to avoid eating peanuts in case they increased their child’s risk of allergies; Parents were also warned not to give peanuts to children under three years of age.
But peanut allergy rates in the population skyrocketed; The problem was that the studies that supported this avoidance were mainly retrospective studies, relying on people’s memories of what they ate.
Then, in 2015, a “forward-looking” randomized controlled trial (the gold standard for testing a new approach, where a control group does not receive the intervention) found that babies with severe eczema or egg allergy actually developed fewer peanut allergies. if they were introduced as part of their normal transition to a solid diet, compared to children whose parents were told to avoid them (the control group).
In the case of gender dysphoria, what was missing from the research (that one Dutch study) was information about how young people’s feelings would change over time, without hormones or surgery.
In fact, we now have data showing that for most young people, “gender dissatisfaction” (in other words, feeling discomfort in the physical body) tends to improve with age, something that should give great hope amid of what could happen. be a huge desperation.
For decades, surgeons performed “washout” arthroscopy, in which patients had incisions made in their joints that were then washed with saline solution. But a 2002 trial found the procedure for knee arthritis wasn’t worth it.
The Dutch research also had no control groups, meaning we couldn’t compare what would have happened without medical intervention.
It is tempting to conclude that any improvement is due to the treatment, when in reality, the young people would have fared just as well (or better) without it.
How should we fix this and make sure we act on really good quality evidence?
That is what NICE was set up to do when it was founded in 1999. It was a move away from what was called “eminence-based medicine” (doing things because esteemed professors said they worked) and towards “evidence-based medicine”.
Dr. Cass makes it clear in her report that the doctors she met had the “best of intentions,” and I’m sure that’s the case.
However, the best intentions do not work well in medicine unless you also have good quality evidence on your side.
Pediatrician and best-selling author Dr. Spock, for example, was, by all accounts, a compassionate and caring man. He recommended, from 1956 until the late 1970s, that babies be placed on their stomachs.
After all, isn’t it obvious that vomiting would be less dangerous in that position?
But, as we now know, it was wrong; Dr. Spock had based it on theory, not high-quality evidence. Researchers later estimated that 50,000 infant deaths in Europe could have been avoided if high-quality evidence from the 1970s onwards suggesting it was better to place babies on their backs had been acted upon sooner.
I think part of the problem has been the way other doctors’ concerns about treatments have been addressed, often ending up having to go through whistleblowing procedures and employment tribunals.
That happened to Sonia Appleby, child protection lead, after she raised concerns at the Tavistock gender clinic at the Cass Review centre.
We have become accustomed to the pharmaceutical industry pressuring doctors to prescribe the latest drug, even if there are concerns about side effects, and although we are not yet good enough to resist this, at least (usually) there is an acknowledgment. that we should try to resist biased information.
However, in the case of gender dysphoria among young people, pressure groups seemed to have been welcomed in the NHS.
Training modules were often sponsored by activist groups, who have every right to express their views, but do not necessarily have experience in critically evaluating evidence. This also meant that the voices of those harmed by medical intervention were not heard enough.
Unfortunately, asking questions was often met with hostility (or worse) rather than a discussion of the evidence base.
A recent conference in London to discuss uncertainties about how to best treat children with gender dysphoria was attacked by protesters with smoke bombs.
Dr. Cass has made it clear that this should not be an “exceptional” area of ​​practice; My hope is that now we can really focus on the distressed young people who need help, and do it as best we can, using truly evidence-based, non-ideological care.
Dr Margaret McCartney is a GP in Glasgow. Her book, The Patient Paradox, is published by Pinter & Martin.