Professor Baum: three simple principles of clinical practice

Professor Baum’s rapid response to Margaret McCartney’s BMJ column ‘Withdraw Saatchi’s quackery bill’ may be read in full here

I have also included it here in full, for in its skilled brevity it sums up everything that is wrong with the (still) proposed legislation.

Re: Withdraw Saatchi’s quackery bill
30 April 2014
Professor Michael Baum, Emeritus Professor of Surgery UCL, London NW11 6PT

There is one thing worse than controlled experimentation and that is uncontrolled experimentation.

There are many patients we can no longer help but there are none that we are incapable of hurting.

When patients are judged beyond cure they are never beyond compassionate care.

The supporters of the Bill seem to have overlooked these three simple principles of clinical practice.

In 45 years experience involved in clinical cancer research I have never once encountered the law as an impediment to innovation.

Blink and it’s back, again, twice

In a fascinating display of parliamentary quick change tango, Lord Saatchi’s creation has hotfooted not only back to the Lords to be read a second time in July (with the hopes of subverting usual procedure and fast-tracking straight to the lower house for its completion- with a time limit of one year from its reappearance) but, according to the Bill’s website, it is now replicated in the PMB proposed by MP Chris Heaton-Harris in the Commons.

Of course, if this is the case then it’s gone from a, relatively, restrained misrepresenting of benefit by using the word ‘innovation’ rather than ‘experimentation'; to a wholesale misrepresentation of content. The original never contained anything that would have enabled access to treatment,at all, despite the heavy lines spun to encourage loud, urgent, understandable, support.  One suspects Lord Saatchi’s new bill will be the same when it suits him to dupe supporters into believing it’s had the ok-go from the Lords; and will be an entirely new and fresh venture when the hideous problems and wall of opposition, from doctors’ representatives, patient charities, research groups and patient safety experts, to the original attempts are pointed out.    Nobody’s quite sure whether the Bill site has told the truth or not because the Bill probably hasn’t been written yet, despite having its first reading yesterday. Nor can anyone know how many willing helpers put their names in for the PMB ballot to run it for Saatchi; brownie points all round, right?
And what dazzling speed – Britain’s got talent for something, anyway, just apparently not appropriate, considered, transparent health policy or legislative drafting.
From the website:
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With a typically overt disregard for quality of life or respect for human worth, the mass guinea pig market was announced in the Telegraph as well, will they be this Bill’s media partner too?
On the plus side, Tredinnick’s cleared off temporarily for his gig at Glastonbury.

Anticoagulation, powdered owl & an outlier on the spectrum of reason

A GP with decades of experience in practice and medical education, Dr Wollaston became an MP in 2010 after an open primary in Totnes. Her invaluable experience and insight on national and local issues of importance as well as her specialist expertise made her the ideal choice to inform the Westminster vacuum.

She was soon appointed to the Health Select Committee, a cross-party committee to hold the government and health bodies to account and make policy recommendations. In 2014 she was elected Chair. She did a fantastic job in the role and it would be a terrible disservice to health policy, the committee and the government’s credibility if she were not Chair again. Very few are as qualified and appropriate for the role and,  felt keenly with the absence of such MPs as Julian Huppert on this occasion, we need to maximise the insightful few that we have. She’s also a rather great role model.

Her challenger for the role is David Tredinnick.

Why is that news? Well it’s perhaps not that newsworthy I guess, and that’s what’s scary. 

David Tredinnick’s appointment to the Health Select Committee itself was really quite shocking, but that was way back in 2010. Though inapposite, add in the company of other desperately unqualified persons such as Nadine Dorries and it’s no longer a surprise.   

Tredinnick, MP is so out there he’s ‘a hallucinogenic substance in his own right’.  He is a big supporter of his own variant of astrology, not only claiming for personal development courses, but once claiming expenses from taxpayers for £755.33 of ‘computer software and consultancy to investigate whether astrology can be linked to alternative medicine.’

In the House of Commons he has stated emphatically that blood does not clot on a full moon, and surgeons won’t operate on full moons. Indeed his fear of the moon has been raised on a number of occasions.   He launched a tirade of EDMs at the indignance of people who knew what they were talking about to talk about stuff, like homeopathy; and relied upon so-completely-unambiguously-discredited studies to support his claims, even those which claimed to cure cancer and where authors asked to be removed from papers. 

Like Australia have concluded, our Science and Technology Committee found homeopathy useless. David Tredinnick then joined the Committee.  

He still believes astrology should be used more often in healthcare in the UK.  Recently in 2015 he reiterated the virtues of astrology as “a useful diagnostic tool enabling us to see strengths and weaknesses via the birth chart” and proclaimed that astrology and complementary medicine “would help take the huge pressure off doctors”. So much for Francis and safe staffing. Perhaps NICE have already a-okayed this as it’s clearly cheaper to replace legitimate health policy with the nuttier side of woomongery’. 

‘How to handle the ongoing issue of having this bizarre, deeply wrongheaded man in a position of influence over matters that demand evidence-based decision making?’ asked Adam Rutherford, 5 years ago.   The indulgence of his personal interest in astrology to the detriment and exclusion of real matters for his constituents and for the health of the nation has not gone unnoticed in parliament either. 

But now this man, who Professor Brian Cox politely termed ‘an outlier on the spectrum of reason’  not only wants to be on it, he has been nominated to chair the Health Select Committee. 

Beating cancer with astrology

Beating cancer with astrology

Almost impossible to parody it’s not actually funny.

These are not the affable, eccentricities of your distant uncle or your old headmaster. This is not harmless.

Championing discredited studies as evidence in parliament, including for cancer treatment, is deeply contemptible. Steering health policy in this way is like crashing it into the dover cliffs. It’s not “the awesome power of the moon” flinging it there – it’s Westminster sinking itself, and the rest of us in the process. 

More from Professor Cox here. 

 

Novel use of procedures to avoid scrutiny

Saatchi’s Medical Innovation Bill (more properly titled the unfounded novel use, irresponsible human experimentation and removal of redress Bill) was not, in the end, rushed through in the last parliament, despite the best attempts of Lord Saatchi to use his weight and talents to create extra time and special rules. In his shock that this, sanctioned by the conservatives, was not going to work out, he proclaimed that Nick Clegg was a nasty murderous sort with blood on his hands, and had handed down a death sentence to patients.

All Nick et al had done was say, hang on a minute – medical organisations, patient charities and medical defence orgs don’t want the Bill, patient safety experts and legal experts are up in arms abut the Bill, and Wales unanimously condemned it, and were quite horrified by it. Perhaps we shouldn’t rush this dangerous and widely condemned draft legislation in these circumstances and instead it warrants detailed scrutiny, at best, and certainly not to be pushed through in this cavalier fashion because of who Saatchi is how much power he wields or how much he donates.

Who he is does not make it right to risk (limitless) patients’ lives, safety, dignity and their quality of life.

Saatchi’s crew then went flat out trying to pressure the Lib Dems to cave in to the Conservative deal to allow this monster of a danger to be passed without any scrutiny by deploying the tried and tested setting up of petitions and shouting in the press at a crucial pinch point before the election. Thankfully the Lib Dems had more integrity, and some very sensible MPs who understood science. (Though Sarah Wollaston is a stalwart of sense, she has not been listened to by her own party – and has found herself surrounded by some extraordinarily inept characters.)

At the HealthWatch public debate on the Bill held at King’s College London in March, Nigel Poole QC and Nick Ross spelled out the problems with irrefutable clarity and sense.

Alas clarity, sense, science and integrity are being challenged once more by the undead Bill, raised from the ground and re-entering the House of Lords on the 8th June.

Worse still, the attempt to limit scrutiny and avert the gaze of noble Lords who may know what they are talking about has gone to new lengths. Lord Saatchi has given notice to the House to agree a motion that Standing Order 46 (no two stages of a Bill to be taken on one day) be dispensed with, allowing the Bill, which would need to be identical to that which left the Lords, to career straight through to the Commons without any stages of examination by learned peers, and be pushed through on the nod. If there’s one thing we need, it is proper scrutiny, particularly in light of the overwhelming opposition to the Bill from those who know what they are talking about, including those in parliament and experts in patient safety such as Sir Francis QC and Sir Ian Kennedy QC.

More info: Stop the Saatchi Bill

politics.co.uk and openDemocracy

A welcome decision

Stop the Saatchi Bill Alliance statement

The Stop the Saatchi Bill Alliance welcomes the decision not to move the Medical Innovation Bill at its second reading.

While we firmly support innovation, we were joined by countless charities, experts, professional and patients’ organisations in our concerns that the Bill, which was set to apply to all patients, all doctors, all conditions and all treatments, was both unnecessary and unacceptably dangerous.

The law of negligence does not impede responsible innovation and the Bill was poorly targeted on this baseless premise.

Some further concerns included that the Bill would undermine clinical trials and introduce contradictory and dangerous amendments to the law, removing a patient’s ability to access redress without providing any additional rights of access to treatment, helping neither doctors nor patients.

Importantly, it was widely agreed to be a serious threat to patient safety.

However well-meaning the originator and motivations of this private member’s Bill, it is a disgrace that the Government and Department of Health supported and encouraged dangerous primary legislation based on such lack of evidence and with such ill-conceived expectations.

The Stop the Saatchi Bill Alliance will firmly reject and repudiate any future moves to bring such legislation back. There is no legal impediment to medical innovation to be solved.

Read: Media articles about the Bill

The Saatchi Bill public debate – HealthWatch & KCL

The HealthWatch debate at King’s College London

The debate took place 4 March at KCL with Professor Sir Michael Rawlins and Daniel Greenberg proposing the motion ‘this house supports The Medical Innovation Bill’  with Nick Ross and Nigel Poole QC against.

Here’s the full debate:   HealthWatch Debate – Medical Innovation Bill

and a rather spiffing live-blog account of it at nhsshakeup.co.uk

 

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Crash call and a close call

Crash call and a close call

Scientific progress is not
linear. Newer does not automatically equate to better.

Rapid uptake of treatments with decreased critical evaluation  is driven by a number of factors including patient demand, industry and ‘gizmo idolatry’– the new, shiny drug or other technology is considered by many to be a better bet than the alternatives.  These new gizmos are more enticing – and not just on a profit basis-  the latest, the greatest, they are laden with assumptions of advancement and laced with hope for the most desperate.  Imagine turning a drug down, a new drug, with the hype and the sparkle and the plausible science, where accepting it means the newest, the best, surely?  accepting it means hope?

gizmo

Gizmo – Gremlins (1984)

Drugs that sound like they make sense and work in animal models may have catastrophic effects in healthy people – TGN1412 is just one example.  Even in drugs which have completed Stage 1, 2 and 3 trials, true adverse effects and true outcomes may remain unknown – still relatively few people will have taken the drug overall.  The results are often unpublished, undisclosed even to regulators, advisory or commissioning authorities and outcomes are tampered with to spin a drug company’s data for patenting and marketing purposes. In the population however, of unwell people with comorbidities, polypharmacy and physiological and pharmacogenetic differences, a drug that appears to be a logical, new, shiny upgrade on the old one may be anything but.   Large scale accurate trials of a drug and the open publication of all results are essential to understand a drug’s real spectrum of effects in vivo. The sooner that the data is available and may be seen transparently the sooner a drug’s therapeutic and all other effects will be determined.

The consequences of not having this data are clear. The drug (or any medical technology/device/procedure) will be in use in the population for longer before the effects become apparent.  In a new drug likely to be used in a significant number of people this is, obviously, a problem – the sooner a drug is introduced and taken up on a larger or ad-hoc scale, the longer that drug is in use without adequate assessment and data, the more people there will be who take the drug and over a longer period of time and yes, these people may be quite happy (for a while). They might have campaigned vociferously for access to the newest drug to treat their condition.

It’s shiny. It’s new. It’s better than the others. Hope and hype are all powerful and entirely understandable. But that drug might harm. It might have many effects which were not apparent in the data presented to NICE or the EMA or MHRA that was likely funded and chosen by the pharmaceutical company manufacturing it, it won’t feature in ghost written and false-meta-meta analyses of a selected group or even the genuine articles, for it’s simply not available; and it certainly may harm those patients who are taking it.  If a drug has an effect, side effects are normal and idiosyncratic reactions always possible. A wider responsibility exists  to know what any risks are at least most likely to be before dishing the drugs out to great fanfare to big groups, and a responsibility to report adverse reactions appropriately thereafter through reporting schemes that are in place.  

A responsibility to not create unnecessarily or disproportionately heightened risks of harm – to treat the individual in their own best interests while maximising patient safety as much as possible. To first, do no (entirely avoidable, unjustifiable and reckless) harm.  Surely that’s what patients would be campaigning vociferously for, too?

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It is an unfortunate fact that many millions of people have taken drugs that were harmful and they did not find out until long after the harm was done. Others may have had an inkling before their last breath but it’s quite unlikely they realised they were being killed by their medicine.  We’ve all heard of thalidomide yet there are so many more examples of drugs that, when taken up too quickly, have had devastating effects. Many others still have prolonged unnecessary expense or increased relative deterioration by being no more or considerably less effective than existing options.

Take Stevie Wonder. He was given oxygen as a neonate. Makes sense right? Sure it made sense to give high-dose oxygen to premature neonates. Unfortunately, this was in place for over 12 years  before a causal link was made to ROP- Retinopathy of Prematurity (aka RLF).  Due to this delay and lack of a link, the therapy harmed many infants, causing permanent blindness in more than 10,000 – including Stevie.  

Women with breast cancer petitioned, in their tens of thousands, for bone marrow transplant to accompany high dose chemotherapy. It made sense – it would allow better toleration of a higher dose of treatment, right? The pressure was enormous from patient advocates, lawyers and even their doctors. Some launched legal actions to gain access to this new treatment.  Unfortunately despite the hype and intense demand, it was much more expensive yet conferred no benefit at all. Worse, it was far more toxic- leading to greatly impaired life quality and hastened deaths.  More than 42,000 women received the treatment before it was stopped – at a cost of $3.4bn to the US.

Baycol (cerivastatin) was a hugely profitable snazzy new statin taken by more than 6 million people. Unfortunately, although a new upgrade, it conferred no benefit compared to any existing treatment. Actually, it transpired that it had a serious adverse risk profile, including things like rhabdomyolisis. Including things like death. It was directly implicated in many  deaths – in reality thought accountable for hundreds, thousands of deaths and withdrawn by Bayer amidst legal disaster.  Other disasters include  Diethystilbestrol (DES), given in pregnancy to prevent miscarriage and widely used for other purposes, becoming routine. Unfortunately as there was no reliable data it took decades to link DES as the cause of cancer and other illness in the children of pregnant women, in which time thousands of women and children were harmed and/or died.  The anti-arrythmics Encainide and Flecainide caused excess mortality – with 50,000 deaths that were entirely unexpected and unexplained – 50,000 people who died when the drug companies had failed to undertake trials to observe any potential benefit, and surrogate markers and assumptions had been relied upon.  The ALLHAT trial finally revealed that, while a drug may confer little benefit over another for one variable, in this case BP, it can be very different in its effects on another- here, its effects on preventing MI.   Huge numbers of patients died unnecessarily through the delay in realising that they were being prescribed a drug that did not work as well as the other on the outcome they should have been interested in, rather than a surrogate.  While they were dying, the drug company were cashing in as the newer much snazzier drug was much more expensive.

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Something  else- something that really made sense- was giving corticosteroids to patients who had acute head injuries. People did this, but there hadn’t been a trial. It just made sense – let’s reduce that swelling- and it had been the case for more than 30 years.  A review came about in 1997 and suggested perhaps this treatment conferred a 1-2% reduction in the risk of death.   Many doctors deployed this treatment. Others were less convinced and did not, but nobody knew.  A trial was finally launched.  The CRASH1 trial recruited more than 10,000 patients. It was hard to get approval, randomising unconscious people (who would have been randomised anyway in real life).   The results showed an excess mortality in patients treated with IV methylprednisolone compared with placebo – what was  immediately clear was that the treatment conferred no survival benefit at all, no advantage for these patients. Indeed, the steroids given to so many had been positively harmful. While a quarter of patients died from their injuries no matter what treatment they received, there were 2.5 extra deaths per 100 for those treated with steroids. ‘The risk of death was higher in the corticosteroid group than in the placebo group (1248 [25.7%] vs 1075 [22.3%] deaths; relative risk 1.15, 95% CI 1.07-1.24; p=0.0001), as was the risk of death or severe disability (1828 [38.1%] vs 1728 [36.3%] dead or severely disabled; 1.05, 0.99-1.10; p=0.079).’  In real world numbers of patients, that’s a lot of extra death. So unequivocal was the result, the trial was stopped early.  ‘The lethal effects we have shown might have been found decades ago’ wrote the study’s authors, lamenting a failure to ‘provide robust evidence’ to prove that ‘ prescriptions are likely to do more good than harm’. 

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Lord Saatchi’s Bill as he proposed would not benefit patients who want access to a treatment – it provides no right or means of access, and would not benefit doctors who are able to innovate responsibly under the current law. Its selling of the register of uncontrolled experimentation (that didn’t exist)  and promises of trying something out would prolong the time taken for reliable evidence to become available, reducing the evidence quantity and quality, knowingly promoting an archaic method which lengthens time and heightens risks sustained from any technologies that are unsafe.

Treating innovatively in a patient’s best interests, even where a doctor doesn’t know which is the better method, or has no data, is really not going to be found negligent under the current law, unless it’s so totally and ridiculously illogical and unsupported by any strand of reality.  But any treatment currently so so bad that it would be considered negligent  would be defensible under the Saatchi Bill’s proposals  -and what of cases of product liability and serious adverse drug effects?   No redress. No ethics committee equals no REC insurance cover. No implications at all for a pharmaceutical company with a harmful product?  

Interventions should be widely introduced in a critical, carefully targeted manner and yes, reliable evidence is required to ensure there is no unnecessary, excessive risk of harm to a patient or a larger group of patients.  Without accurate and systematic monitoring and reporting there will be diminished evidence regarding a drug’s safety or efficacy, widening the gap in knowledge required also for properly informing and gaining consent from a patient.   Rapid adoption of treatment with decreased critical evaluation has led to countless adverse events in recent history.  While many advances offer real advantages, others can be aggressively marketed and championed yet offer little, if any benefit; cause alarming side effects, serious harm, suffering and even death. 

There are problems with aspects of research and regulatory frameworks, there are problems with data access and publications, there are problems with bureaucracy and huge problems with funding.  A broken system perhaps. But you cannot mend a broken plate by smashing up your entire kitchen, destroying an aga and removing the sink.  While it’s possible to advocate for a number of methods to assess an intervention, it is impossible to rationally, or innovatively, advocate that anecdote, uncontrolled and irresponsible experimentation is anything other than a timehop straight back to an era we’d rather not repeat.  This imprecise, outdated approach is markedly out of step with current practice, as well as out of step with increasing precision approaches, targeting a drug to genomic features which are increasingly in use and can be incorporated into trial methodologies worldwide for some patients.  The Bill would have been an inappropriate step backward that places current and future patients at considerable risk of harm, delaying beneficial data as well.   It’s the opposite of innovative, the opposite of an advancement and patients deserve better. Patients deserve their lives to be valued. There is no excuse for this today.

The Medical Innovation Bill,  full of hope and packaged with hype-  would have harmed, not helped.

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