Tag Archives: medical innovation

Telegraph: The fear of being sued is ruining modern medicine by Dr Max Pemberton

Dr Max Pemberton writes about why he supports Maurice Saatchi’s Medical Innovation Bill in the Telegraph.

Telegraph

By doctor and Telegraph journalist Max Pemberton

Published 9th December 2012

Read in the Telegraph.

Doctors are too scared to deviate from evidence-based medicine and innovation is being stifled.

In recent years there has been a seismic shift in the way that doctors practise. The mantra that young, fledgling doctors repeat to themselves endlessly is “evidence-based practice”. On the face of it, this seems sensible. Of course doctors should only prescribe or recommend treatments for which there is a clear, empirical evidence base. Modern medicine is founded on the principles of scientific inquiry; a hypothesis is put forward, tested and proved or disproved. But, as any doctor will tell you, in real life things are a lot messier than that, and nowhere more so than in cancer management.

Here, evidence is not always as clear and decisive as we would wish it to be. When I worked on a paediatric cancer ward a few years ago, I remember being struck by how little evidence was available to support many of the treatments that were at the clinicians’ disposal. While evidence-based practice is noble in theory, in reality, it’s simply not always realistic, given the complex nature of cancer when its multiple variables, contributing factors and idiosyncrasies are taken into account. This is what makes medicine as much an art as a science.

But worryingly, while doctors know that evidence-based medicine is not always the best choice for treating their patients, they have, in recent years, become increasingly scared of deviating from the standard treatments available to them, even if these don’t appear to be working. One man is trying to change this. Maurice Saatchi, the advertising guru who sits in the House of Lords, last week launched his Medical Innovation Bill, with the specific aim of changing the current culture within medicine that makes doctors fearful of the new and untested. I give it my wholehearted support because I despair at the way medicine is heading.

It’s a tragic indictment of modern medicine that too often innovation is jettisoned in favour of the status quo – not because it’s in the patient’s best interest, but because of the fear of being sued. This defensive medicine is at the heart of so much clinical practice now. Several factors have coalesced to create an environment whereby evidence-based medicine is something to hide behind, rather than simply a gold standard to inform decision-making.

The seeds of this defensiveness were sown in the medical profession’s consciousness following the fall-out from the Harold Shipman case. One of the unintended consequences of the regulation that came in as a result was that, suddenly, doctors and what they did for their patients was under incredible scrutiny. It was no longer assumed that the doctor would, de facto, have the patient’s best interests at heart. The authorities became increasingly suspicious of doctors and what they got up to behind the closed doors of their surgeries. A culture of fear crept into the medical profession. This was against the backdrop of the insidious creep of the compensation culture and the rise of the no-win no-fee lawyer.

Also, as hospitals have become run increasingly like businesses, so a new ruling class has emerged from within health care – the managers. They tend to be wary of any innovation or deviation from protocol that might expose the hospital to litigation risk. With often little or no experience of health care at the coalface, they struggle to grasp the speed, daring and courage needed for medical innovation. So they hide behind protocols and policies and mete out punitive consequences for any clinician who deviates from them. Protocols have solidified into monolithic rules – not to help patients but so they could be waved across a courtroom to defend the hospital against complaints.

The current climate has resulted in a loss of professional autonomy and transmogrified doctors into tick-box automatons, no longer guided by guidelines but strangled and suffocated by them. Mavericks used to flourish in medicine, but they have now been stamped out – branded unacceptable variables of unpredictable risk.

All of this has coalesced to mean that the fear of being sued has ruined modern medicine. It’s not good for the doctors, who are constantly questioning what they do and don’t do – not on behalf of the patient, but because they fear having to justify what they are doing in front of cross-examination. And it’s not good for patients, who are denied the chance of cutting edge, untested and unlicensed treatments when they have little or nothing to lose. Things that might be in the patients’ best interests are not pursued, meaning that nothing moves forward for them, or indeed future generations.

It’s this that the Saatchi Bill is designed to address. Its origins lie in Lord Saatchi’s devastation as he watched his wife, the novelist Josephine Hart, die of ovarian cancer last year. He has managed to turn his grief and sense of loss into something that has the potential to bring untold benefit to future sufferers.

The drafting of the Bill, Lord Saatchi explains, is designed to safely advance the freedom of doctors to innovate and strive for advancement, rather than simply accept the status quo because it means that no one can sue them. This doesn’t mean that doctors will have free rein to experiment on a patient. They are still bound by professional guidance and their duty of care still remains to their patient. But what it does mean is that, in cases where the evidence is shaky or wanting, or is not yet clear, the Bill sets out a code by which doctors can try alternatives. In this way, it actually offers the patient more security than they have at present because it provides, for the first time, a robust legal framework to encourage responsible innovation in diagnosis and treatment.

One in three of us will get cancer. If the survival rates are going to improve, doctors must be free to innovate, and this is what Lord Saatchi’s Bill does. I hope our politicians can see this, too.

→READ in the Telegraph: Dr Max Pemberton writes about why he supports Maurice Saatchi’s Medical Innovation Bill in the Telegraph.

A balance between evidence-based medicine and responsible innovation

Charlie Chan Surgeon
Charlie Chan, breast cancer specialist surgeon

By Charlie Chan

The Medical Innovation Bill is in the process of re-drafting. The consultation is likely to take into account the many comments from patients and doctors. Many of the concerns raised in the consultation have centred on the issue of possible “quackery”.

The Bill’s critics have suggested that this new piece of legislation might encourage non-scientific “experimental” treatment, which might be viewed as bordering on charlatan behaviour. However, the Bill does have important safeguards to ensure protection for the patient, and scientific advancement.

Concerns by some that this Bill will lead to reduced scientific integrity and a lower emphasis on clinical trials, can be allayed. In fact, the Bill will only protect doctors, who are planning to treat patients, who have exhausted standard treatments and existing suitable clinical trials.

For some patients with common life-threatening diseases (e.g. breast cancer, bowel cancer, heart disease), there may be several standard treatments and a multitude of trials. However, for patients with rare diseases, there is often no treatment at all and few if any randomised trials.

If one considers the sum total of patients with “rare cancers”, their sum total exceeds the number of new cases of the top four cancers every year in the UK. Yet for this significant proportion of rare cancer sufferers, there are few treatments and a dearth of clinical trials.

For patients, both young and old, with rare diseases, the current scientific model of large randomised clinical trials means that they are predominantly excluded from scientific research and the development of new treatments. It is surely not the right thing to do to ignore this large number of patients, for whom the current status quo is failing.

An important concept being considered for this Bill is a Central Data Repository, which would record all patients having novel treatments. Ideally, this should be open access to both doctors and patients, so that people are able to learn rapidly about the efficacy, if any, of novel treatments for these difficult clinical problems.

Rather than inhibiting the development of clinical trials, this data repository can then provide a valuable resource for generating novel hypotheses for new studies. These clinical trials can then underpin any promising benefits shown by patients having innovative treatments.

Thus, the current status quo of clinical practice, based predominately on the randomised clinical trial might even be improved, and a sensible balance struck between evidence-based medicine and responsible innovation.

As Sir Austin Bradford Hill, one of the godfathers of modern clinical trials, said in 1966:  “Any belief that the controlled trial is the only way would mean not only the pendulum had swung too far but that it had come right off its hook.” Perhaps now this Bill might restore the pendulum into its rightful position.

Charlie Chan, Consultant General Surgeon

Charlie Chan is consultant surgeon, specialising in the treatment of people with breast cancer and malignant melanoma. He has been a national committee member at the Association of Breast Surgeons, and the British Association of Surgical Oncology. He has also been the senior author for UK surgical guidelines for the management of breast disease. He is on the Trial Management Group for 3 large UK breast cancer trials.

What is the Medical Innovation Bill ?

The Medical Innovation Bill is designed to help medical doctors innovate new treatments and cures safely and responsibly for cancer and other diseases.

Lord Woolf
Lord Woolf

“At the moment, the doctor’s hands are tied – by concerns about professional reputation and potential negligence claims,” says Lord Woolf.

“That needs to change.”

Lord Woolf, Former Lord Chief Justice, former Master of the Rolls.

He was the first Lord Chief Justice to be President of the Courts of England and Wales.

Sir Michael Rawlins
Sir Michael Rawlins

“The Saatchi Bill will allow responsible innovation.” says Sir Michael Rawlins.

“From trying out things in individual patients, that can lead onto research and benefit thousands of other patients.”

Sir Michael Rawlins is Chair of Medicines and Healthcare Products Regulatory Agency, former chairman for National Institute for Health and Clinical Excellence (NICE) and President of the Royal Society of Medicine

The Times: Our letter to the Editor

Published in The Times today our letter to the Editor in response to the letter published Monday 28th April regarding the Medical Innovation Bill.

Sir,

The evidence for the need for the Medical Innovation Bill is compelling. Around 18,000 doctors and patients replied to the Department of Health consultation supporting the Bill, many confirming that they have experienced the deterrent effect that an increasingly risk-averse culture is having on responsible medical innovation.

The objections in Mr Poole’s letter (Apr 24) relate to details of the Bill that were not in Lord Saatchi’s original text and will not be in the text that he intends to introduce early in the new session. The Bill team will soon publish a new draft which meets concerns expressed by legal and medical professionals.

Doctors must be given the freedom to innovate responsibly, with the confidence that the law will protect them if their decision is made with the support of a responsible body of medical opinion.

They must not be forced to wait until their decision is tested in expensive and traumatic litigation or disciplinary proceedings. Nobody wants that, except perhaps a small group of lawyers who make their living from the existing litigation-focused system.

The Bill will be an opportunity for all those who are concerned that the legal system is not properly serving patients with rare diseases, whose hope rests entirely on innovation.

Patients want to know that every responsible avenue is being explored in order to help them, and that doing nothing is no longer the easy and safe answer.

Good doctors must be given the protection and encouragement of the law to innovate; and bad doctors must be deterred from innovating without support of their colleagues.

The Bill achieves both aims, and is to be welcomed by lawyers, doctors and patients.

Daniel Greenberg
Parliamentary Counsel to the Medical Innovation Bill team

Telegraph: ‘This isn’t quackery, it’s good practice’

The Bill Team’s Dominic Nutt talks to Prof Walker, University of Nottingham

Paediatric cancer specialist Prof David Walker’s work is a bittersweet affair. At times he is able to deliver the happiest of news to parents: that he and his team have saved the life of their child. Equally, he is the one who has to tell a fearful mother and father that there’s nothing more he can do, that their son or daughter’s cancer has spread beyond the reach of his drugs and the surgeon’s knife, and that their child will die.

Based at Nottingham University for the past 22 years, working at Nottingham Children’s Hospital and Nottingham Medical School, Prof Walker combines a passionate commitment to the welfare of his patients with a deep respect for scientific discipline.

He wants to see more and better therapies added to the armoury of paediatric cancer treatments, to save the lives of more children, and is frustrated by the obstacles that block the path to new cures for cancers.

Medical science decrees that no new treatment should be used until it has been thoroughly tested in randomised clinical trials. Trials are the gold standard, Prof Walker stresses, and where possible patients should be in a trial if one is available. “Research does require regulation to introduce new drugs and treatment safely,” he says.

But trials alone cannot meet the desperate need for new treatments, in particular for rare cancers. “There will never be enough trials, they take years, and in any event there are rarely trials for less common diseases. There just aren’t enough patients.”

Around one in 600 children under 16 are diagnosed with cancers. The most common, such as leukaemia, account for a third of cases, and treatment success rates are high. But some are hard to treat, other than with surgery, such as the highly malignant rhabdoid tumours, which start in the kidney. There are no standard therapies for these cancers and less than one in five children diagnosed with a stage III or IV rhabdoid tumour will survive beyond four years.

With such rare childhood cancers, it is hard to gather enough patients to form a trial in one country alone, and these have to be organised internationally.

“The problem is that there are many different tumour types and sub-types in childhood cancers, each having an unique biological signature justifying a unique scientific rationale for their treatment with modern drugs in development,” says Prof Walker. “They are all rare in the population and there is a huge element of luck as to whether in your child’s case there is a trial available.”

So, while potential new drugs may exist, they cannot be used. “A drug may be available, the scientific rationale for its use may exist, but patients cannot receive it if no trial has been organised to assess its effectiveness and toxicity,” says Prof Walker.

He also points out that the “big four” cancers – breast, bowel, lung and prostate – dominate media coverage as well as fundraising, research and trials. Researchers want to work on the common cancers because their research will be better funded, while drugs companies are more interested in this area as there is a bigger market, and a greater potential profit.

If you have a less common cancer – and there are hundreds – your chances of getting on a trial are limited and the chances of there being innovative treatments are, in many cases, zero. Yet taken together, less common cancers – defined as a cancer that affects five people or fewer in 10,000 – account for more than half of all British cancer deaths. One in six of us will develop and die from a rare cancer.

The lack of trials in this area means doctors who want to cure their patients, rather than just manage their deaths, are caught in a scientific circular argument. There is no evidence that a new treatment will work, so it cannot be used to find out if it does work.

Prof Walker believes that doctors should be able to try new treatments with such patients on an individual basis. Yet, he argues, the law, and the culture of defensive medicine which surrounds it, stands in the way of innovation. Doctors are protected if they stick to the well-worn path of “standard procedure” even if it leads to the death of the patient. But they may be vulnerable to legal action if they try something new and it fails.

Which is why Prof Walker supports Maurice Saatchi’s Medical Innovation Bill, currently going through a public consultation process by the Department of Health, which ends later this month. It seeks to provide legal protection for doctors who innovate in the interests of their patients. He argues that when patients are terminally ill and there is no trial for which they are eligible, a doctor should be free to innovate.

“We need to allow them to try new drugs outside of a formal trial and collect the data from those innovations to inform the next generation of trials,” he says. “The Saatchi Bill would protect individual doctors who try new, untrialled treatments, where there is a scientific rationale for their use, in patients who consent.

“This isn’t a licence for the maverick doctor acting alone – the Bill obliges the doctor to seek agreement from peers.”

The Saatchi Bill, he believes “will give the patients and their families additional choice and allow doctors to try new medicines in people who have nowhere else to go, and do it in such a way as we could all learn from it”.

Prof Walker also believes that even when an individual patient cannot be cured, this kind of innovation will advance medical science for future patients.

“When people are dying,” he says, “they all would like their passage through their illness to have some meaning and to learn from the loss of their life.”

He also explains that many cancer drug trials rightly focus hard on a tightly defined group of patients with the same tumour type, which has within it a particular cancer molecule, that the drug being tested is designed to block.

Using molecularly targeted drugs in patients with the same tumour drives robust results. But, says Prof Walker, “there is a weakness in the process, because it doesn’t tell you where else the new drug may work. It only tells you that the drug works on a very specific patient type with a very specific tumour which has within it that particular molecular target.”

The new drug may work in another rarer cancer with the same molecular target, he says. “But if I have a patient with a very rare cancer with the same molecular target, I can’t use it. Current rules require us to set up another trial in that tumour, and that’s expensive and requires collaboration with the drug industry, which may not wish to supply the drug for such a purpose if they don’t think it is commercially advantageous. So, in the meantime, the hospital won’t release that licensed drug for an unlicensed purpose because there’s no evidence that it works,” he says.

This is where the Lord Saatchi’s Medical Innovation Bill would help, he argues.

“If we could test drugs in a patient and record our results in an open-access database, this would contribute to our understanding of the application of this drug and perhaps help with selecting new drugs for clinical trial by identifying those with most promise; although it is important that the drug has already been tested fully on patients with a different cancer but with the same molecular signature.”

Opponents of the Bill argue that a law that supports doctors who want to try new treatments outside the trial process is a “quack’s charter”.

Prof Walker disagrees: “To offer an untried treatment and not learn from the process would be quackery.” “But to use untried treatment and to try to learn, responsibly, about the effect on the individual, and share that learning with others would seem to be not quackery, but actually responsible professional practice.”

Sign the petition for the Medical Innovation Bill: http://chn.ge/1pqY6lS

 

The word ‘lumpectomy’ was a surgeon’s insult

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Pictured: Geoffrey Keynes

The word ‘lumpectomy’ was a surgeon’s insult.

The surgeon Geoffrey Keynes could arguably be considered the patron saint of innovation. In 1922, Keynes, based at Barts Hospital, in London, developed the lumpectomy for breast cancer.

He did so in the face of global ridicule and hostility from fellow surgeons. He was swimming against the tide of orthodoxy, which he described as a religion that could not be easily challenged.

The accepted practice for dealing with breast cancer, developed by the all-powerful American surgeon William Halsted, was the ‘radical mastectomy.

The ‘Halsted Procedure’ was revered. This physically deforming operation involved removal of the breast tissue, skin, nipple, axillary lymph nodes and the underlying chest wall muscles.

It caused a great deal of psychological upset in many who underwent the procedure. It was a horrendous – but no one challenged it. Keynes – the brother of economist John Maynard – began using local excision and radiation therapy to treat breast cancer.

More than 70 percent of his patients survived five years – his survival rates were the same as those patients who underwent the Halsted operation, yet without the horrendous side-effects massive, debilitating surgery. For his pains he was ridiculed.

The Halsted brethren nicknamed the procedure the ‘lumpectomy’ – a low-minded joke.

In his autobiography, Keynes wrote: “A built-in dogma of thirty years standing dies hard, and I was regarded with grave disapproval and shaking of heads by the older surgeons of my own hospital.” Keynes then went on to develop new blood transfusion techniques which saved lives in WWII.

The Saatchi Bill is designed to support doctors who want to change things for the better but fear doing so because culture and law is against them.

What has the Saatchi Bill got to do with culture? How would it help a modern-day Keynes?

Innovation is change and change is uncomfortable. The law is one barrier to change, and it is a barrier that engenders and encourages a wider culture of inertia.

Changing the law, will encourage more doctors to innovate, and to pass on the sense of enterprise to others around them.

They in turn will pass on that enthusiasm and sense of hope that incurable diseases can be treated.

The Bill will be a fire starter.

Sign the petition for the Medical Innovation Bill: http://chn.ge/1pqY6lS

This Bill is very important, as the scientific world has changed

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By Charlie Chan, Consultant General Surgeon, Nuffield Health

In spite of medical advances over the last 50 years, many people across the world still die prematurely from cancer, heart disease, lung disease, and terrible degenerative diseases like muscular dystrophy and motor neurone disease.

For many of these people, the options for treatment and run out quickly, with no prospect of improvement in quality of life or life expectancy. For some, but not all, exploring new treatment in a responsible manner is something that the individuals may wish to do, if only to benefit future generations.

This Bill is very important, as the scientific world has changed. The last generation has seen an enormous change in our fundamental understanding of diseases and their processes.

Sign the petition for the Medical Innovation Bill: http://chn.ge/1pqY6lS

This has been underpinned by the massive changes in laboratory research, genetics, 3-D printing, and regenerative medicine. This knowledge could not have been foreseen over 50 years ago, when the Bolam case was first heard.

This new knowledge has been underpinned by the vast improvement in computer technology, which has benefited all parts of society. When treating patients and diseases, doctors and scientists are waging a conflict on 2 fronts.

The frontline remains the one of the individual patient, with the doctor at the bedside or in the operating theatre. Behind-the-scenes, there is a continuing intelligence campaign underpinned by scientific research in the laboratory.

100 years ago, communication between the front and Intelligence Corps might well have been done by carrier pigeon. Now, information exchange occurs rapidly in real-time between GCHQ and troops in Afghanistan. In medicine however, this communication or translation of new scientific knowledge to the bedside remains slow.

We are fast approaching and era when scientific research may outstrip our ability to deliver this to the patients. Hence, we need to address a new way to deliver innovative treatments.

That is not to say that the standard clinical trial model is dead. There is still much merit in the randomised trial as a paradigm. It is vitally important that the development of new standard treatments for large numbers of people is underpinned by solid statistical analysis and estimation of perceived benefits.

However, a new process for innovative treatments may provide many new hypotheses for new trials, which can then cement new techniques and drugs.

There are some colleagues who rightly have concerns that a change in the law may constitute a charlatan’s charter. However, the Bill contains safeguards to ensure that there is no quackery. All standard and trial treatments need to be exhausted, there needs to be logic behind the proposed treatment, and this treatment needs to be agreed within the peer group, prior to discussion with the patient.

The agreement within the peer group will need to be done in a timely manner. There is a significant challenge for the profession to establish a framework for such peer group discussions, which may occur on the local level or through a National Specialty Association.

However, it is well recognised that this must be something that can be done quickly for patients treated in a district general hospital by local consultants, as well as those managed in large teaching hospitals.

Some of my colleagues would strongly support some form of central data collection.

A central data repository to be analysed on a regular basis, in order to establish whether any putative innovative treatments have any merit for further investigation in large clinical trials.

This might be located in an academic university department, so that this might be independent of central government control.

The future is extremely exciting. Basic scientific research has enhanced greatly our fundamental understanding of many diseases, such as cancer.

This understanding of the basic diseases means that some new biological drugs may have multiple applications across different cancer types. It makes logical sense to exploit this basic science knowledge, particularly to benefit those patients with rare diseases, for whom a standard clinical trial may be impractical or financially non-viable.

Advances in regenerative medicine, particularly in the USA, now mean that organs can be printed in a matter of hours or weeks.

This will herald a completely different way in which we might manage people with cardiac, kidney, or liver disease.

We must grasp this opportunity to change things for future generations, otherwise these scientific advances may be for naught.

About the author:

Charlie Chan is a Consultant General Surgeon with a special interest in breast disease, skin cancer, and soft tissue tumours. He also has a varied practice in general surgery. He has extensive research interests, and is currently involved on the Trial Management Group for 4 large UK breast cancer trials.

On average, he performs 30 to 40 major breast reconstructions a year, as well as numerous cosmetic breast procedures. Amongst his breast cancer patients, he is normally able to conserve the breast in 70-75% of his patients. AIong with his colleagues in Cheltenham, James Bristol and Fiona Court, he is one of only a few surgeons in the UK who are trained in the new Breform™ cosmetic breast operation. He has contributed to numerous articles in the national press (Sunday Telegraph, Daily Telegraph, Daily Mail) as well as the local press and BBC Radio Gloucestershire.

He has written breast cancer guidelines for the Association of Breast Surgeons, organised national cancer surgery meetings for the British Association Of Surgical Oncology, and reviews oncology education across Europe for the Accreditation Council of Oncology in Europe.

Sign the petition for the Medical Innovation Bill: http://chn.ge/1pqY6lS

Launching the Early Access for Innovative Medicines scheme

By George Freeman, MP for Mid Northfolk

First published in the Spectator

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Imagine this: you take a routine trip to the doctors. Except it doesn’t turn out to be routine at all. Instead, the doctor tells you that you only have months to live. Worse still, there is no certified cure. There is a potential drug that could save your life, but it’s stuck in a regulatory tangle, waiting for approval which takes years. It might come on the market in a decade. But by then, of course, it will be too late for you.

33 days to change medical history: sign our petition to enable medical innovation: http://chn.ge/1pqY6lS

Ludicrous, surely? Yet that has been the dilemma facing too many over recent years, unable to get access to the drugs that could save their lives. Decades more of enjoyment, time with the grandchildren, a whole chunk of life wiped out when a cure could be sitting there unused.

As was announced on Friday 15th 2014, this is going to change thanks to the Early Access to Innovative Medicines scheme I have helped design. It has the potential to revolutionise drug development and completes the vision set out in the UK Life Sciences Strategy launched by the Prime Minister in 2011 and the NHS Innovation Health and Wealth reforms. The core vision was that the UK would be a crucible of medical innovations that could help all of us live better for longer. It is the same philosophy that lies behind the Saatchi Bill, putting medical innovation front and centre of healthcare and helping find new cures that work for patients.

 Medicine is currently undergoing a seismic shift, from a 20th century model (something done to us by Government) to a 21st century model (something we increasingly take a responsibility for ourselves). Revolutions in genomics and data are driving a new generation of targeted and personalised medicines, and reshaping the landscape of drug design. The old traditional ‘blockbuster’ Big Pharma model of the post-war years is ceding to the world of ‘translational’ or ‘experimental’ medicine in which drugs are designed with, and around, patients, their data and tissues, in clinical research facilities and hospitals.

 This is part of a wider revolution of patient empowerment. Instead of sitting passively waiting for new medicines, today’s patients are increasingly impatient and want access to innovative treatments now. Patient charities and advocacy groups are becoming powerful players in the new landscape: funding research, lobbying for access and forming new companies. The Early Access Scheme fast-tracks the best new life-saving medicines to patients earlier while maintaining robust safety standards. It can only happen if the new drug is targeted at an area of unmet clinical need, there is powerful data and evidence to show the drug is safe and the patient taking the drug has provided informed consent.

 With the right safeguards in place, it soon becomes clear that the benefits of this new scheme are profound. Not only will this help save lives by providing patients with life-saving drugs quicker, it can help the UK be at the forefront of this new world of twenty-first century healthcare. We have the chance to cement a competitive advantage (over the USA and Europe especially) as the global location of choice for designing, testing and proving the new generation of personalised medicines. Under this Early Access scheme, UK patients will be able to trial the most innovative medicines before any others. With the NHS seal of approval, we can then help sell these drugs to the Qataris, Saudis and emerging-world rich all over the planet, investing the revenues back into the NHS to the benefit of us all.

Nothing in healthcare is without controversy. But with innovative medicines, as with the recent controversy over patient data, the arguments on both sides boil down to one single point: who wants to be better for longer? One thing’s for sure – I do. I think you do too. If any of us got that diagnosis, we’d want to know we had access to the latest drugs. Last week’s announcement – and the pioneering campaign of the Saatchi Bill – helps make sure we do.

33 days to change medical history: sign our petition to enable medical innovation: http://chn.ge/1pqY6lS

 

Cardiff consultation: Answers to questions that came via Twitter

During the recent Department of Health public consultation meeting in Cardiff (18 March 2014) attended by doctors, academics and patients, we got some questions on Twitter we discussed.

Sean Kielthy – @LMKPartnership – in particular asked three questions which sum up what many others are asking.

Sean asked:

1. ‘Defining experts [in the Bill]. A homeopath may be an expert in their field. How do we stop them getting in?’

Answer: The Bill can only be used by qualified and registered doctors. The term ‘doctor’ is a legal one and homeopaths (for example) would not be protected by the Bill, if it became law.

Basically, you have to be a legally-recognised doctor. If not, the Bill doesn’t cover you.

2. ‘How do we keep the quacks out? [I want] science-based medicine only’.

Answer: Apart from the answer above – that you must be a doctor to be covered by the Medical Innovation Bill – you must also get the agreement of a panel of medical experts and other doctors before you offer a new treatment to the patient.

It is inconceivable that a quack doctor, treating a terminally ill patient, who comes up with a crazy ‘snake-oil’ treatment would get the support of a panel of other senior doctors.

To get agreement, the doctor will have to produce evidence or coherent reasons and a theory as to why the new treatment is worth considering.

The supporting doctors will be named as part of the sign-off process. At the moment, it is possible for a doctor to act alone without the agreement of senior and qualified peers.

So the Bill makes it harder for a quack to prey on a patient. After the Bill is passed, they will not be able to act alone.

3. ‘People in these situations [patients who are very ill] can be desperate. [I] don’t want to see quackery used and funding taken from science.’

Answer: Quacks will not be able to hide behind the Bill. In fact the Bill will expose quacks.

There is another question here, though – the cost of innovation. Will the Bill cost money? There is no logical reason to say that it will. An innovation may cost, it may cost less or it may cost nothing.

For example, it may conceivably be an innovation to do nothing. There are examples where it is felt that adjuvant chemotherapy for certain cancers may be at best only marginally beneficial, and that the associated potential side-affects of the treatment may outweigh the standard chemo treatment.

In this case, doing nothing would be an innovation. (This is not to advocate in any way that not having chemo is a good thing – it is simply a generic example).

However, if people argue that innovation costs money and that the Bill will inspire innovation and new and better treatments, then that is not an argument against the Bill – it’s an argument against all medical innovation and medical progress.

The Bill won’t divert resources from science. The Bill will help individual doctors help individual patients on a one-off basis.

By trying new ideas and techniques in this way, doctors will learn and the data they collect can be used by other doctors and scientists to inspire full medical trials and scientific discovery.

The Medical Innovation Bill works hand-in-hand with science to deliver new treatments for hard-to-cure diseases.

 

‘Protect the patient: nurture the innovator’ writes Norman Williams, President, Royal College of Surgeons

‘Protect the patient: nurture the innovator’ writes Norman Williams, President of the Royal College of Surgeons (RCS) in the March 2014 RCS bulletin.

→READ: the full article in the bulletin

The bulletin text is reproduced below:

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A few weeks ago I spent the weekend in Innsbruck, participating with several European colleagues in a surgical workshop that concentrated on a new minimally invasive technique of sphincter preservation in rectal cancer. This innovation has the potential not only to reduce the incidence of permanent stomas significantly but to do so with the minimum of trauma.

It occurred to me and my fellow participants that it might also, if developed appropriately, be possible to carry out the whole procedure via one orifice – no prizes for guessing which one! I also thought that if robotics develop at the estimated pace then the technique could be performed with even more precision, thus reducing the margin of error inherent in such an operation.

Now, I know many of you will be thinking that the President is in ‘cloud cuckoo- land’ but I would like you to reflect on the considerable progress we have made in surgery over the past 50 years. When I was a medical student there was no such thing as organ transplantation, cardiac surgery was in its infancy, joint replacement had just been introduced (but only for hips), and laparoscopic and microsurgery were both at a very rudimentary stage.

The pace of innovation has been truly remarkable and yet we in the UK could be criticised for dragging our heels when it comes to the dissemination of some developments.

Four years ago, when I was Chairman of the RCS Research Board, we published a report entitled From theory to theatre, in which we explored the barriers to translational research that threatened to stifle surgical innovation and identified a series of actions to deliver high-quality surgical research now and in the future.

I am pleased to say the College rose to the challenge and, in addition to raising funds and influencing research councils and charities, has managed to develop an infrastructure that should markedly change for the better the way in which we carry out clinical surgical research. All in the research and development departments should be congratulated on their achievements.

I should also compliment the many surgical trainees who have contributed their time and efforts to developing trainee research collaboratives. There are more than 20 of these across the country, each carrying out some large clinical trials and audits as they try to answer important questions. All these efforts are to be applauded and celebrated but the challenges do not stop there.

The fruits of research are of little value if they are poorly implemented. Discovery only matters if it reaches all those patients who might benefit. The diffusion of surgical innovation has at times been painfully slow. The reasons are many, ranging from lack of evidence to training and capacity issues. We hope we are beginning to address some of these barriers with the actions mentioned above but others will require different solutions.

If we are to tackle the slow diffusion of innovation then we must learn from experience and not keep making the same mistakes.

That is why we commissioned a piece of work to investigate how we might improve. The report of this investigation, entitled From innovation to adoption: Successfully spreading surgical innovation, will be published soon. It sets out for the first time what makes adoption of surgical innovation different and why we need a new approach.

It is based on a review of five mainstream surgical procedures, across a number of specialties, in which patterns of uptake are analysed and the factors that helped and hindered surgical adoption in England are explored. As a result, a pathway of surgical innovation has been developed, which is made up of six critical factors:

1. Early identification of the promise of an innovation

2. Leadership to champion and advocate its adoption

3. Establishing the infrastructure to enable its use

4. Defining what should be implemented and how its impact will be measured

5. Developing levers and incentives to encourage appropriate adoption

6. Providing information to support clinical adoption and patient choice

These are vital factors if we are to achieve a more streamlined adoption mechanism but in addition I must stress that there is a responsible and ethical approach.

This applies particularly at the earliest stages but of course must be applied throughout the whole process. There is no room for ‘cowboys’ or the ‘have-a-go’ merchant.

My memory goes back to the early 1990s when a news conference was held at the College about the death of a patient that occurred at the hands of a surgeon performing an early laparoscopic procedure without appropriate governance arrangements in place.

The patient’s husband was a QC and the subsequent fallout was disastrous for the profession and set back laparoscopic abdominal surgery in the UK for a decade. Although governance has improved, a clear path showing how to support the innovator, while at the same time protecting the patient, is still lacking.

We need a transparent and supportive mechanism to assist the innovator in honing a truly new concept without the constant fear of litigation (or worse). Yet at the same time safeguards need to be in place so that patients are fully informed and protected from the unscrupulous practitioner.

This will not be easy but it is an issue that can no longer be avoided, otherwise it is only a matter of time before one of us is incarcerated for trying to advance his or her specialty.

I understand that several consecutive patients died when Professor Sir Roy Calne carried out the first liver transplants; I wonder what would happen to him nowadays were there to be a similar outcome. Complications are inevitable with new procedures and only by facing up to them and understanding them can they be overcome.

This requires courage on behalf of the innovator (and, indeed, the patient) and support from colleagues. Research ethics committees have their place but they are invariably composed of non-surgeons who do not have an appreciation of the problems. I would like to see our profession taking on this responsibility.

One way to do this might be for each specialty association to set up an Innovation Oversight Panel composed of individuals with experience of the trials and tribulations involved in surgical innovation. Such a panel could receive proposals for innovative procedures to assess whether or not they were feasible, appropriate and designed to improve the status quo.

The panel could then give approval for a small pilot study but request regular reports, so that when complications occurred they could help the innovator to resolve the particular issue. The process would be iterative and hopefully the new procedure would evolve so the trial could be widened to involve other groups. Ideally this would take place within the infrastructure discussed above to ensure more rapid dissemination.

Such a structure would enable the realisation of this piece’s title and is in keeping with the new Medical Innovation Bill promoted by Lord Saatchi, which is designed to improve responsible innovation in medicine and remove the fear of litigation.

I would exhort the officers of the specialist societies to think seriously about setting up an appropriate mechanism. It does not need to be the model I have outlined; they may well feel they could do better and I would be the first to applaud them.

However, unless a mechanism of some form is set up I fear that the progress of surgical innovation may be impeded by the imposition of stringent external regulation. I also fear that patients will be damaged unnecessarily and surgeons will be impugned or, at worst, indicted. Unless potential developments like the one I learnt about in Innsbruck can be developed speedily yet ethically, then disseminated appropriately, we are all the losers.