Tag Archives: medical innovation

Department of Health Medical Innovation Bill events

The Department of Health are hosting public consultation events in Leeds, Cardiff and London.

The Department of Health say:

The Department of Health on behalf of HM Government is proposing to clarify the law in certain circumstances in order to encourage responsible medical innovation.

Medical innovation has been vital to the dramatic rise in life expectancy of the last century. This country has a proud heritage of medical innovation from Alexander Fleming and the discovery of penicillin to Sir Peter Mansfield’s enabling of magnetic resonance imaging.

However, where doctors are innovating outside of a research study, one of the barriers to improved treatment is the possibility of medical negligence claims and the pressure this can place on doctors to practice defensive medicine.

Consequently, instead of becoming the latest medical pioneers, doctors may feel obliged to follow standard treatments, even where those standard treatments have poor outcomes.

The Government is committed to doing whatever is needed to remove barriers that prevent innovation which can save and improve lives.

We must create a climate where clinical pioneers have the freedom to make breakthroughs in treatment.

It is important to be clear that we are not suggesting that doctors are concerned about the possibility of a negligence claim in relation to all the treatment decisions they take.

However, where there is insufficient or no evidence about the effects of a treatment, a doctor may hesitate to offer it.

We are keen to hear views, especially from doctors, on whether medical innovation is being unduly constrained by the possibility of litigation where there is insufficient or no evidence about the effects of a treatment that might otherwise be offered.

We also want to hear from patients whose experience suggests that the possibility of litigation has been a factor in their doctor’s attitude to possible innovation.

This event is an opportunity for those with an interest to join us to discuss the issues in order to inform the formal consultation process. Click here to view the online consultation.

If you are unable to attend this event, you may prefer to attend the following alternative events around the country:

The dates are as follows:

Cardiff
18th March 2014
13.30-17:00

Leeds
2nd April 2014
13.30 – 17:00

London
10th April 2014
13:30-17:00

Please note these events are organised by the Department of Health.

Supporting innovation – exposing the maverick

The Saatchi Bill will make it much easier for doctors to innovate safely in the interests of their patients.

At the same time, it will expose the doctor who wishes to exploit their patient, preying on them and their vulnerability in order to attempt a reckless experiment.

Doctors will not be protected by the Saatchi Bill unless they go through a rigorous and specific process to ensure that the attempted innovation is the right course of action for the patient.

As the draft Bill states in paragraph 2 (3a) a doctor wishing to try a new treatment – for example in the case where standard treatments aren’t working – must consult a body of senior and relevant medical experts and get their consent.

The doctor must also record their opinion, including and dissenting voices.

The decision of the panel of experts must then be presented to the patient – including any contrary opinions if there are any – and the patient must of course also agree to go through with the innovative treatment.

Finally, the note of the opinions must then be attached to the patient’s consent form as a permanent record.

Only then will the doctor be legally covered by the Bill.

The Bill imposes a much higher standard of consent than other health legislation.

For example in order to section a patient, the Mental Health Act requires the signatures of only two doctors.

Currently, it is easier now for a doctor to indulge in reckless experimentation and maverick medicine, than it will be if the Bill becomes law.

As it now stands – without the Saatchi Bill – a doctor can attempt to persuade a vulnerable patient to embark on a dangerous treatment path.

This is so because the doctor technically doesn’t need to refer to a panel of experts before trying the non-standard procedure. He or she can act alone.

So, the Medical Innovation Bill supports and encourages reasoned innovation – and exposes the maverick.

Lord Woolf: Former Master of the Roles and Lord Chief Justice:

[The Bill] could give confidence to medical practitioners engaged in the field of treatment of cancer that in appropriate circumstances they could safely recommend and implement a course of treatment, or non-treatment , which some, or indeed the majority of their professional colleagues, might regard as unorthodox.

I have come to this conclusion because it is, in my view, undoubtedly the case that there is insufficient certainty as to the course which courts will adopt in this country at the present time when faced with an allegation that a medical practitioner’s treatment of a patient was inappropriate because of its innovative nature.

[There is] a risk that the present state of the law could inhibit the proper development of treatment of particular cancers.

Any way of avoiding this by legislation, in my opinion, should be welcomed. I would therefore hope that in the Lords, at any rate, your Bill will be well received.

About Lord Woolf

In Memory of Chloë Drury

Chloe Drury by Mike Thomas

I gripped the wooden lectern tightly with both hands to try and prevent myself breaking down with emotion.

It was a year ago last Friday. The lectern was at the front of St Mark’s Church, Purley – and I was starting to speak at the Memorial Service of my best friend, Chloë.

In front of me were hundreds of faces, young and old alike. They were there to celebrate and remember an ultimately, short but extremely well-lived life.The life of Chloë Drury.

On Wednesday the 27th of February last year, I received a call from a friend, she sat me down, she looked me in the eye and told me that Chloë was going to die, that there was nothing anyone could do anymore and it was going to happen in the next few days.

I sat in shock, I tried to ask questions about when it would happen, and how did they know?

Chloe Drury - Medical Innovation Bill
Chloe Drury – Medical Innovation Bill

All I remember thinking is that I felt desperately helpless. That night I stayed surrounded by friends, trying to find comfort in each other, anything not to be alone.

All night I relived the time I spent with Chloe, the first time we met, the days spent in her company, the last time I ever saw her. I thought about her dreams and aspirations, the things that she’d been so excited about, and which now would never be fulfilled.

When the news finally came through early the next morning that Chloë had passed, there were no more tears, no more uncontrollable emotion just silence, I was completely numb.

Mike Thomas and Liam Ryan at the Houses of Parliament 24th Feb 2014 speaking alongside Lord Saatchi as he launched the public consultation LIVE via Google Hangout.

Over the next few hours I had to have conversations that I wish never had to happen, to be bearer of the worst news possible, to look people in the eye, friends and family alike, and tell them that Chloë had died.

Watching them breakdown in front of me was unbearable.

The following weeks, were the most difficult. As a friendship group we spent nearly every day together, making sure that no one was alone for too long.

Being alone let you think too much, it allowed you to dwell and let reality set in, it let you realise that you would never see Chloë again, that you never got to say goodbye.

These painful thoughts would stay with me for months.

Eventually, we had to return to school, we had to return to everyday life.

But no matter how normal you try to make it, it’s impossible to pretend that nothing has changed.

For me personally, my school attendance was almost non existent, the last place I wanted to be was at school, because at that point in my life, lessons and exams didn’t matter, grades and university places weren’t important, I’d lost a friend and it had stripped me of all motivation. I had no drive.

There were two factors that help me deal with Chloë’s passing. Firstly the unwavering and resolute support that we all received from friends, dealing with this alone would have been undoubtedly impossible.

Secondly, the sheer bravery and optimism of her mum, Debbie Binner.

Despite what she has had to endure, she is always looking for a positive. Debbie has turned loosing Chloë into her driving force.

It was Debbie who urged me to speak in St Mark’s Church, who gave me the opportunity to say goodbye properly. When I stood at that lectern and spoke, I felt like I had finally let Chloë know how much she meant to me.

I hope that I have given you an insight into the pain that losing a friend causes, for young people like me.

And if you remember just one aspect of my words, I urge for it to be this one.

Chloe’s death has brought about many questions about cancer treatments.

And I urge you to ask yourself this question: Have you done everything you could have, were you bold enough, brave enough to demand more, more innovation and more accessibility to cancer treatments, so that more lives are saved where possible?

For Chloë and the hundreds of others who find themselves in her situation, Make sure the answer is yes.

This blog is based on a speech by Mike Thomas, who spoke at the House of Lords on Monday 24 Februrary about the death of his friend Chloë Drury and in support of the Medical Innovation Bill. Chloë died on 28 February 2013, of Ewings sarcoma aged 18. 

Telegraph: Lord Saatchi launches the consultation on his medical innovation bill – live from the House of Lords

Today Lord Saatchi launched his Medical Innovation Bill LIVE via Google Hangout from the House of Lords.

The politician was joined by a panel of experts including Telegraph doctor Max Pemberton in a live video stream from the House of Lords today where they will call on the public to support his Medical Innovation Bill.

Introduced by Lord Saatchi last year following the death of his novelist wife Josephine Hart to ovarian cancer in 2011, the Bill seeks to give medics greater freedom to test out cutting edge treatments on dying patients.

Currently doctors are forced by the threat of legal action to stick to standard procedures, even when they are proving ineffective.

Lord Saatchi’s bill has already received huge public support and health secretary Jeremy Hunt promised to support legislation on the issue after the public consultation which is launched today and ends in May.

Read in the Telegraph 

 

Harrison, Duchenne and I

Harrison and Alex Smith

When Harrison was just 4 years old, I took him to his pediatrician thinking he might have a mild physical delay and may need physical therapy.  Within 2 weeks, a blood test and a visit with a neurologist provided us with the most devastating diagnosis imaginable.  Duchenne muscular dystrophy.  The neurologist explained to us that Harrison’s muscles would rapidly deteriorate, he would lose the ability to walk, to use his arms, to bathe himself, to go to the bathroom on his own. Eventually Duchenne would attack his heart and lungs and the disease would take his life.  We have nothing to stop it he told us.  It’s 100 % fatal, I wish I could tell you it was cancer.

I swung in to action, I challenge myself daily…mentally and physically to do the best I can.  I’ve started a charity, Harrison’s Fund, to generate as much cash as I can to put into breakthrough Duchenne research to find a treatment for this generation of children and young adults. We have already invested hundreds of thousands of pounds. I’ve become a networker, at all times, this never really switches off, I’ve started a not for profit race team, Harrison’s Racing,  I’ve literally gone from  Average Man to Ironman, completing my first Ironman triathlon in October last year. But, becoming just an Ironman doesn’t seem quite enough for me now… next in the challenge list is to take Harrison on an Ironman with me. Drag him in a boat behind me for 3.8km, take him on the 180km bike leg and then push him the no small matter of a 42.2km marathon, all back to back in under 13hrs and 47mins, a world record time. Harrison will also be an Ironman… that’s just cool! I am in the process of assembling the team to get this done, from building a bespoke catamaran to drag, to building the bike and Harrison machine and the running chair needed to take a child that no longer really fits in any of the running chairs out there anymore.

Harrison is now nearly 8, and we live each day with the knowledge that because he’s got a duplication of exon 51 of his dystrophin gene, his symptoms continue to progress and he continues on a steady and rapid physical decline.

Harrison’s doing incredibly well considering but he has reached a plateau all children with Duchenne  do around there seventh and eighth birthdays.

Harrison Smith

This is a stage of progression that is marked by an accelerated decline…  It is marked by …

Shock – the shock of his legs buckling without warning, sending him tumbling to the ground. It is marked by the shock of not being able to open the jar he used to be able to

Fatigue – he is plagued by the fatigue of exerting great effort just to do “normal” things – like keeping his balance and getting up from the floor.

Strategizing – thinking about how we organise and prioritize a day for the family that will not wear him out yet still let him experience all the world has to offer.

Disappointment – the searing realization that he can’t play organized sports, my lack of words or adequate comfort when he walks into the house fighting back tears because his brother or friends just rode off on their bikes and he can’t be with them.

Routine –  daily stretching sessions, and regularly physical therapy appointments and doctor appointments.

Hanging on – to what may be the last time he’ll complete a task or have the energy to help me coach his brother Williams under 6 rugby team.

This is the time when kids are moving faster and pushing limits and growing and looking forward to the future.  And I’m terrified that Harrison may not have one.  Annual events like birthdays and the end of a school year are marked by conflicting emotions – they’re marked by relief that we were given the gift of another year.  And by grieving one less year we have together.

While these symptoms are heartbreaking, I am also filled with the sobering reality that this is MILD compared to what we will face in the very near future.  There is a train racing toward my little boy and I’m running as fast as I can to scoop him up and save him, but I’m acutely aware that I may not make it in time.  We need to affect massive change to help stop the train.

Children at any age – whether they’re 3 or 5 or 7, like Harrison, should be afraid of the dark.  Of the bully on the playground.  Of the monsters under their bed.  But they should NOT be afraid of needles and biopsies and surgeries and falling and breaking bones.  They should NOT be afraid of no longer walking or of being unable to feed themselves or of losing so much strength in their arms that they can no longer hug their mum’s and dad’s.  Most importantly, they should NOT be afraid of dying.

I’m writing this blog today because this medical innovation bill has the very real potential to help doctors and clinicians slow down the train. In our case the risk of doing nothing is not nothing, the risk of doing nothing is fatal. Fatal every single time. You never survive this.

In any innovation in medicine there is an element of risk, and between clinicians and patients there has to be a level of permissible risk particularly when the population is small and the need is great. In terms of acceptance of risk – our community has already demonstrated our philosophy on this – we have one option right now – it causes cataracts and growth stoppage and osteoporosis, and weight gain and immune system suppression – and this drug is still being studied in the Duchenne population. There are debates over dosing and efficacy and this drug doesn’t even change the final outcome for Duchenne patients.  Yet, our children’s doctors prescribe it, and in fact encourage its use.  It’s part of the standard of care for Duchenne.   Through this use of steroids, we’ve already shown that we’re willing to assume risk.  We understand that the long-term risks of steroid therapy are known, and the long-term risks of a new therapy would be unknown.  But it’s also quite straightforward and simple logic that helps us understand that long-term risks can only become clear when something has been used for the long term – and the only way to get to the long term is to begin.  We have to begin somewhere. Sometime.  And the time has to be now.

What we are not willing to do is assume the risk of doing nothing. If a potential therapy shows promise of stabilization or improvement over what would be expected without any treatment, and it shows safety, then patients and parents should be given a choice to try it with long-term studies taking place concurrently.  Because, at the end of every discussion and assessment of a therapy, we must never lose sight of the reality that the risk of having Duchenne far outweighs the risk of most potential treatments.  And our children must be the beneficiaries of our best effort, of our most noble intentions, and of our greatest commitment to safety AND speed. Because at the end of the day, these children are not a statistic.  They are not a commodity. They are not someone’s science experiment.

They could be YOUR boys or grandchildren…..and they may not be, but the responsibility for saving them belongs to all of us.  I believe we are close to a treatment. We are so close that my son Harrison is part of a generation that will either be the last to die from Duchenne or the first to survive.  We must have a great sense of urgency and we must always remember that the children should NOT serve the science, but the science must always serve the children.

With this bill and the innovation it would encourage we have the potential to move forward and reduce Duchenne from a 100% fatal condition to that of a chronic condition. The time is now, we don’t have time to waste, #maketime.

 http://www.harrisonsfund.com

 

Telegraph: End in sight for cervical cancer?

Ian Hampson is finding it hard to contain his excitement. A molecular virologist with a passionate interest in understanding the relationship between viruses and cancer, he thinks that he and his wife, Lynne, may – he stresses the word may – have found a revolutionary self-help therapy for women with pre-cancerous cell changes in the cervix.

Almost by chance, the Hampsons discovered in their lab at St Mary’s Hospital, Manchester, that the drug lopinavir, licensed for the treatment of HIV, attacks the strain of human papillomavirus (HPV) that causes virtually all cases of cervical cancer. An as-yet unpublished clinical trial has found that lopinavir capsules, when inserted into the vagina, appear to kill off abnormal cells in the cervix. There appear to be no side effects.

Cervical cancer is still the main killer of women in the developing world, causing more than 280,000 deaths worldwide annually, so the idea that women might use a simple vaginal pessary to protect themselves against it is an exciting prospect.

But the results of Dr Hampson’s trial are also promising for women in developed countries. Up to 40,000 women in England alone are found to have abnormalities of the cervix every year, following smear tests or biopsies. At present the only treatment for high grade, pre-cancerous changes is invasive – and far from risk-free – surgery.

Sensational news, then, but it has taken Dr Hampson 12 years and a battle against numerous obstacles – including UK red tape, lack of interest and funding – to reach this point. “Worldwide, one woman dies every two minutes of cervical cancer,” he says. “The results of the early trial are staggering, but I might have to wait another six months to get this study published, and another year to get a bigger study off the ground. I want to get this treatment on the road.”

The story begins in 2002, when Lynne, a lecturer in viral oncology at the University of Manchester, was studying HPV16 – the main cancer-causing strain of HPV – and analysing how human proteins interacted with virus proteins.

“It’s well known that viruses like HPV take over human cells by hijacking their ‘waste disposal systems’ to make cells throw away certain proteins inappropriately,” Dr Hampson says. “Human cells use enzymes called proteases to get rid of waste proteins in different ways. Lynne’s results suggested that HPV was enhancing the function of a specific type of protease.

“The findings made me speculate that there might be a drug that could selectively block this particular function of the virus. A class of drugs used to treat HIV, known as protease inhibitors, primarily targets the HIV protease enzyme; but previous work has shown they could also block the same type of human protease, which our results had shown was important for HPV. It was an epiphany.”

At his wife’s suggestion, they got hold of as many protease inhibitors as they could lay their hands on (using a national Aids repository in the US, which supplies small quantities of drugs for research). “We literally smashed up tablets and stuck them into cultures both of HPV and cervical cancer cells,” recalls Dr Hampson. Several seemed to have activity against HPV and cancer, but lopinavir was the most potent. “Lopinavir was remarkably toxic. We could see the cells dying in the lab.”

By 2011, he says, they had shown that lopinavir was “multifunctional”, meaning it inhibited both viral proteins and the proteins in cancer cells. Still nothing had been proven clinically, and as Dr Hampson soon discovered, setting up a patient trial proved difficult.

Lopinavir is made by the US company Abbott Laboratories as part of a combination drug for HIV called Kaletra. The good news was that, as one of the most widely prescribed drugs worldwide and already licensed for both children and adults with HIV, it would not need extensive safety testing. The bad news was that it was only licensed for oral delivery. This formulation would not, at the strength needed, get enough of the drug delivered to the cervix.

“Our work showed we needed a 10-fold higher concentration of the drug at the site of infection – the cervix – than you can get by taking the drug orally,” says Dr Hampson. “The obvious solution was to reformulate the drug, so it could be applied locally as a cream or pessary, and organise a clinical trial.”

But the cost of reformulating the drug meant there was little interest from drug companies, cancer charities or research agencies. Then one of Dr Hampson’s PhD students pointed out that one oral form of the drug available was a soft gelatin capsule, which might “melt very nicely” in the vagina, and could be trialled as a pessary.

There was another obstacle, though. In Europe the makers of the capsule had phased it out in favour of a hard tablet. A generic lopinavir capsule called Lopimune, made by an Indian company, was only licensed for Africa.

“I wanted to bring some Lopimune into the UK for a trial,” says Dr Hampson, “but it would have involved a lot of red tape – and would have cost a fortune. I felt as if I was banging my head against a brick wall.”

The same PhD student, Dr Orora Maranga, pointed out how useful such a self-help treatment – if it worked – would be to women in Africa, where there are few screening programmes to pick up pre-cancerous conditions, or surgical facilities to treat them.

So it was that, near the end of 2011, the Hampsons finally won approval from Kenyatta National Hospital in Nairobi (where Dr Maranga was then a senior registrar) for a preliminary trial of lopinavir in women with pre-cancerous cells in the cervix. In return they would set up a cervical-cancer screening programme – needed to identify women suitable for the trial – and facilities for surgery, if it was needed.

Funding such a trial was difficult, and Dr Hampson is enormously grateful to those who supplied money or equipment. Donors included St Mary’s, Central Lancashire Healthcare Trust, a company called Hologic, a UK philanthropist, Ken Cholerton, and a small charity on the Isle of Wight, the Caring Cancer Trust.

Finally, last year, in a trial overseen by Dr Maranga, more than 800 Kenyan women were screened for cervical cancer. Of those, 21 per cent were found to be HPV positive, of whom 40 had pre-cancerous cells in the cervix. Twenty-three of this group had high-grade disease – where there is a higher risk of cancer developing and for which the only treatment is surgery. A further 17 had low-grade or borderline disease.

All 40 women were asked to insert a lopinavir capsule as a pessary, twice a day, for two weeks. At three months, each participant underwent a biopsy of the cervix, together with a smear test. In 19 of the 23 women with high-grade disease, the abnormal cells had disappeared. Two women now had low-grade changes, while two still had high-grade disease, for which they needed surgery.

Dr Hampson emphasises that this was an early trial, the type normally used to look at how well a new drug is tolerated. And he points out that there were no side effects. “We had a 90 per cent treatment response in the high-grade group – we couldn’t believe it,” he says. Although high-grade disease can sometimes disappear spontaneously after a year or two, this is rare after three months. There was also, he reports, “very high clearance” among women with low-grade and borderline cell changes.

Dr Hampson is now planning a larger controlled trial involving 500-1,000 women based in three sites in Africa – Nairobi, Kampala and Johannesburg. If he can raise the funds, that is. If the results can be replicated, the advantages for women everywhere are obvious. This potential treatment, less invasive than surgery, would also avoid the risk of premature labour in pregnancy that is associated with the operation.

“Research has found that one surgical excision of abnormal cervical cells increases the risk of premature delivery by 14 per cent,” says Prof Pierre Martin-Hirsch, a gynaecology oncologist at Lancashire Teaching Hospitals, who finds the results of this first trial “impressive”.

By contrast, Dr Hampson points out that in cases where the disease is persistent, “treatment with lopinavir can be repeated as often as needed, because it appears to be safe.”

Lopinavir could also be an option for women with low-grade pre-cancers who are currently offered a “watch and wait” approach – “a horrible time of anxiety”.

“While a vaccine is now being offered against HPV in some parts of the world,” he points out, “it will take 20 to 30 years before we have immunity.”

Cancer Research UK says it cannot comment on a trial that has not yet been peer-reviewed or published. “This is raising the hopes of patients,” warns a spokesman. “A peer review might find flaws in the trial. It may never get published.”

What frustrates Dr Hampson is the fact that, even if further trials were successful, it could be years before this pessary becomes available. There is now talk of of a “window trial” – one in which lopinavir could be offered to women with high-grade disease while they are waiting for surgery. “The drug has been shown to be safe, and the women have nothing to lose and everything to gain. If it works they will not need an operation.

“We are looking at this possibility, but clinicians in the UK are understandably cautious about using treatments off-label. And who can blame them? In our litigation culture, it doesn’t pay to take risks.”

Which is why Dr Hampson supports Lord Saatchi’s Medical Innovation Bill, designed to encourage doctors to innovate without fear of prosecution, and which is the subject of a government-backed consultation.

“At present there is a great reluctance to deviate from the accepted best clinical practice. Had we been able to obtain the particular drug formulation in the UK, it would have been far easier to convince a clinician to try the treatment – if the fear of litigation was reduced.”

The University of Manchester has filed a patent for treatment with lopinavir for HPV-related disease

Saatchi’s Medical Innovation Bill

Lord Saatchi’s Bill, now open to public consultation, would allow doctors to prescribe ‘’off label’’ drugs. These are drugs that have been tried and tested for one specific use (in this case lopinavir used to combat HIV). lopinavir is known to be safe, but doctors cannot easily prescribe it for other, non-standard uses. The Bill aims to speed up the development of new and innovative treatments for cancer and other diseases.

The Caring Cancer Trust has agreed to accept donations towards funding a phase 3 trial of lopinavir for pre-cancerous cell changes in the cervix. More details are available from their Virgin Money Giving page which can be accessed from their website http://www.caringcancertrust.com/

Guest blog: Fearful doctors ‘in a war against inertia’

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By Dr Lefever

The Medical Innovation Bill, to be introduced by Lord Saatchi, is intended to help patients by allowing their doctors to be less constrained by clinical orthodoxy.

Single issue fanatics, whose fervent beliefs have no scientific merit, will still be reined in. But doctors who can make a reasonable case for a non-standard approach will be able to do so. Clearly they have to have the support of the patient. Also they must convince a scrutinising panel

The Medical Innovation Bill intends to make it easier to define what is sensible and permissible innovation and, by contrast, what is reckless experimentation.

It would do this by setting out a clear protocol to follow before offering the patient an innovative or experimental procedure.

Under the bill, the lead clinician must consult with, and gain approval from, a multi-disciplinary team. Without approval, the innovation cannot be legally offered.

This approach is eminently sensible. Which of us would not want to brush aside ‘the way things have always been done’ if doing so might bring additional years of health and happiness to a loved one?

Doctors in the front line, right alongside their patients in the battle with disease and decay, want to do the best they can. So who is the enemy? Who has a vested interest in the status quo? Whose head would roll if something did not work out as hoped when something new is tried?

The answer at present is that we are witnessing a civil war. The doctor’s own career would be sacrificed if he or she steps out of line.

The National Institute for Health and Clinical Excellence (NICE) was intended to weed out mavericks. It has become the keeper of the purse for the NHS.

The General Medical Council has responsibility for putting doctors names onto the medical register, through monitoring medical education, as well as for striking off miscreants. Yet how much attention do they pay to considering whether doctors are properly trained for the job they actually do? For example, are GPs trained to counsel? Hardly at all. Yet that is half the work. Are specialists trained to take risks? No. But they have to.

Lawyers and Coroners judge the actions of doctors on what their colleagues would customarily do.

A profound inertia results from the limited vision of these guardians of public safety – with the consequence, as Lord Saatchi understands only too well, that doctors now fear more for themselves and their families than they do for their patients.

About the author

Dr Robert Lefever is regarded as the pioneer of addiction treatment methods and rehab centres in the UK. He established the very first rehabilitation centre that treated patients with eating disorders alongside those with drug and alcohol problems. He was also the first to treat compulsive gambling, and workaholism.

READ: Dr Lefever blogs for the Daily Mail

READ: Dr Lefever’s own blog

READ: http://www.doctor-robert.com/

There will never be enough trials – additional innovation is needed

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By Professor David Walker

Professor of Paediatric Oncology Faculty of Medicine Health Sciences, University of Nottingham

There will never be enough trials – additional innovation is needed I was at a meeting recently with clinical academics, charity chief execs, health regulators, representatives of the pharmaceutical industry – and one lone voice that cut to the heart of the matter.

That voice was Debbie Binner, whose teenage daughter had died of a rare bone cancer, Ewing’s Sarcoma. Debbie was the reason we were there. When her daughter, Chloe, died she had just turned 18. A few months short of her 18th birthday, the chance of a drug trial was offered.

But Chloe needed to be 18 to be eligible and was refused entry. Our meeting, held in the House of Commons with Paul Burstow MP in the Chair, was to discuss how we, the experts, could ensure that younger people had access to more trials of new therapies for their life-threatening conditions.

Many speakers spoke about new ways to reform the regulatory system so that it could work better for young patients. We heard about changes in the EU Clinical Trials Directive that is being enacted in Brussels, that may help.

Listening to my colleagues, I was struck by one thought: that if you put people in a room who are expert on a regulatory process they want to make better, but inevitably more rules.

What is needed is a feeling that we can work for the patients’ benefit using our established professional values and standards rather than a raft of ever more complex rules. Research does require regulation to introduce new drugs and treatments safely. That is true. But trials cannot be the only answer.

There will never be enough trials, they take time and money, there are rarely trials for the less common diseases. So when patients are in a situation with “nowhere else to go” and if there is no trial for which they are eligible, they need to be able to try treatments that might work, based upon the best judgment of their medical advisors. We need to allow them to try such new drugs that may be applicable and collect that experience to inform the next generation of trials.

The Saatchi Bill would do this, by protecting individual doctors who try new, licensed but untrialled treatments, on patients who have consented to such treatment outside of a formal trial. This cannot be – and isn’t – a license for the maverick doctor acting alone.

The doctor would also need to share the decision with peers as part of the multi-disciplinary teams that already exist in modern health systems. The Saatchi Bill would meet the needs of pharma and academia – but most importantly the patients and their families who need to be given additional choice – and experience the associated hope.

If organised with light touch this could offer valuable information to researchers whilst sustaining hope for families that “no stone is being left unturned”.

The Saatchi Bill offers a framework where practitioners would be supported in exploring innovative therapies, whilst building evidence of their applicability and effectiveness as part of the process of acquiring the evidence necessary to launch the next generation of trials. This is why I commend the Saatchi initiative.

→READ Professor Walker’s biog

→READ the full story in the Telegraph

→WATCH Lord Saatchi explain the Bill

Telegraph: Lord Saatchi Bill: We must liberate doctors to innovate

Lord Saatchi urges the public to seize a ‘once in a lifetime’ opportunity to change how medicine is practised in British hospitals.

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I don’t remember feeling anger when my wife, the novelist Josephine Hart, died in 2011 of ovarian cancer. I certainly didn’t want to thump anyone. It was a calamity, but grief is not a disqualification for rational thought.

I knew that no one had done anything wrong, or behaved badly, that the doctors who had treated Josephine were neither incompetent nor inefficient, that they did what they were supposed to do with drugs and chemotherapy. They followed “standard procedure”, even if the treatment was degrading, medieval and ineffective, and they knew it would lead to her death.

What puzzled me was how there could be such a ready acceptance in the medical profession of “standard procedure” in the light of the hundreds of thousands who die of cancer. In the case of advanced gynaecological cancers, such as my wife’s, “standard procedure” is 40 years old, offers poor quality of life, and the mortality rate is 100 per cent, with the survival rate correspondingly zero.

They did it, I came to understand, because “standard procedure” is the only safe route they can take legally. The patient, their family, their partners and their lovers may all be desperate for something else to be tried, something new, something innovatory, but the law prevents any innovation, however reasonably chosen and carefully managed. Why? Because it defines innovation as deviation from “standard procedure”, and deviation makes it medical negligence.

In 2012, the NHS paid out £1.2 billion as a result of medical negligence claims, that figure had doubled in four years. And Treasury figures show that we have a pot of £24 billion set aside to cover future liabilities. That is a staggering sum, roughly half of the defence budget. The result is that, if you are a doctor in a hospital, you are all too conscious of the tide of litigation, and you become risk averse. And innovation averse.

There may not have been anything to stop Josephine dying, but the terrible thought that haunts me is that her death was a wasted death. Indeed, all 165,000 cancer deaths in this country every year are wasted deaths because science advances not one centimetre as a result of them. Nothing new is tried and so nothing can be learnt that might spare others. Scientific progress is being halted by the law and fear of negligence bills.

This culture has to change. Last year I introduced a Private Member’s Bill into the House of Lords that set out a legal framework “to encourage responsible innovation in medical treatment and to deter reckless departure from standard practice”.

It was drawn up with the help of the best legal and medical minds, and stipulated that, to innovate, doctors must have patient consent and the agreement of other senior medical experts and practitioners. They cannot go it alone, but they can go beyond standard procedure without fear of ending up in court.

Such Bills usually stand little chance of success, but two things were in my favour. The first was the overwhelming tide of support I received from doctors, lawyers and, most of all, from those who wives, husbands, sons, daughters, brothers and sisters have died wasted deaths from cancer.

“I truly hope with all my heart,” one correspondent told me, “that your Bill is a success and it changes for the better the treatment offered to cancer sufferers in the UK. It drastically needs to change. My husband was belatedly diagnosed with pancreatic cancer in February 2007. We were told (mistakenly) it was inoperable and that chemo was the only option, not as a cure, just to buy him more time, before finally a different surgeon at a different hospital agreed to operate to remove the tumour, saying he could’ve done so at time of diagnosis. The op was too late and the cancer had spread to his lungs. My husband died.”

Or another, who wrote that “when discussing the benefits/disbenefits of certain treatments with my GP, he pointed out that if he did not follow the “guidelines” and something went wrong, he could be open to a legal suit”.

And the second was that, last November, Jeremy Hunt, the Health Secretary, with the backing of the Prime Minister, announced his “wholehearted support” for my proposals, and promised government support to legislate to make them happen, after a public consultation.

But this had to be, he stipulated, “a full and open consultation, a consultation that gets the views of patients on the right balance between innovation and safeguards, a consultation that hears from clinicians on the problems they face in innovating and how to overcome them”. He has even agreed that responses to the consultation – which must be received by May – can be sent to the Department of Health via social media.

Mr Hunt has laid down the challenge. I’m appealing to Daily Telegraph readers to join with me, and the tens of thousands who have already given me their support, to make this the biggest government consultation response ever. We need to say loudly and clearly we want to try new treatments for cancer where the old ones are known to lead only to death. We want to escape being doomed to repeat an endless cycle of failure.

What’s wrong, you may ask, with the way we explore new treatments for cancer now? Clinical trials, random clinical trials, take a long, long time to produce results. It can take 15 years and £1 billion to come up with just one drug. I believe passionately that we will get no closer to a cure for cancer until doctors can test new treatments, in a controlled way, not on laboratory animals but on real patients, with real illnesses in real hospitals.

I believe that we are on the brink of a great medical moment, a once-in-a-lifetime opportunity for a change of culture, away from being risk averse, and back to the spirit of medical innovation that once led Alexander Fleming to the discovery of penicillin or Sir Peter Mansfield to enable magnetic resonance imaging.

In this new culture, we will be able go to our doctors and say, “have you tried everything? I understand there is a treatment out there that might help. Can you try it on me? I have nothing to lose.” And the doctor, for the first time, will be able to say yes.

I can’t promise you that, by itself, this change will cure cancer, but it could encourage the person who is out there right now, who may still be a child, and who one day may free us from this blight on my life, and yours.

Curing leukaemia

IN THE 1940s, the survival rate for childhood blood cancers was pretty much zero. At the time, the scientific literature argued that anyone trying to cure childhood leukaemia was cruel, because the result was always the same: death.

Prolonging the agony with needless, unproven medical interventions was wrong, it was argued; the condemned child should be made as comfortable as possible and allowed to waste away.

A few determined doctors in the United States and Europe challenged this defeatist sentiment. They tried treating the disease with folate, a B vitamin, and discovered that it got worse.

As a result, they tried drugs which reduced folate levels instead. This worked and led to the introduction of the still-used drug methotrexate.

Andy Hall, Professor of Experimental Haematology at the University of Newcastle upon Tyne, says: “What those doctors did then couldn’t be done so quickly now.

“Those doctors were close to the patients dying on the ward and not prepared to accept the status quo. Survival rates for children with leukaemia today are around 90 per cent.”

‘Off-label’ drugs

DEVIATING from the standard medical procedure can offer hope to those with the most dire prognoses — which is why Prof Angus Dalgleish, Professor of Oncology at the University of London and the Principal of the Cancer Vaccine Institute, is a supporter of the Saatchi Bill. He feels doctors are too often afraid to try new ideas, by prescribing drugs “off label” — for diseases for which they have not been licensed. “I have recommended logical, non-standard treatments to cancer patients who have run out of standard options,” he says. “I have seen on many occasions patients who have benefited dramatically.”

One example was a 63-year-old man with metastatic prostate cancer for whom the usual treatments were not working. “We agreed that he try a drug licensed at a high dose for another condition. Even though his other doctors thought his case was terminal, he had a marked clinical response and survived for another three years, dying not from his disease but due to the indirect chronic effects of his previous therapies.”

How war promoted modern surgery

War is a crucible for medical innovation. Medics are faced with men and women who are dying, often in large numbers and are driven to try new techniques, sometimes developed in the heat of battle. They have little to lose and all to gain – saving otherwise doomed soldiers from death.

In the Falklands war of 1982, Surgeon-Captain Rick Jolly OBE – a man decorated both by the British and the Argentinians for saving lives on both sides – operated in a field hospital with an undetonated bomb lodged near his operating table. He discovered that casualties left out in the cold because it was impossible to collect them from the battlefield fared well, in many cases, leading to the development of theory of therapeutic hypothermia, whereby patients can benefit from deliberate cooling.

Penicillin was first used in earnest in the Second World War. Doctors were aware of its benefits, but not necessarily how to use it and in what doses. However, knowing that personnel would likely die without it, doctors administered it, learning as they went. Military doctors facing injury and suffering on a massive scale during the Second World War also pioneered advances in antibiotics, anaesthesia and blood transfusions – advances that would usher in the age of modern surgery.

The innovating breast cancer surgeon

GEOFFREY Keynes could arguably be considered the patron saint of innovation. In 1922, the surgeon, based at Barts Hospital in London, developed the lumpectomy for breast cancer, flying in the face of orthodoxy. Back then, 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 a physically deforming operation involving removal of the breast tissue, skin, nipple, axillary lymph nodes and the underlying chest wall muscles.

Keynes, the brother of economist John Maynard, began using removal of the tumour and radiation therapy to treat breast cancer. More than 70 per cent of his patients survived five years, a rate that was similar to that in patients who underwent the Halsted operation, yet without the massive, debilitating surgery.

For his pains, Keynes was ridiculed — yet lumpectomy was gradually accepted as a standard treatment, with the Halsted operation rarely performed today.

The future?

How might the Saatchi Bill work in practice: a hypothetical case study.

Doctor Glenda Smith is treating a patient, Alison Jones, for a rare and life-threatening condition. She asks Dr Smith about a new kind of non-surgical treatment she has read about. Dr Smith discovers the new treatment has not been tested for Alison’s condition, although it has been used for other illnesses.

Under the present legal conditions, Dr Smith will feel safest to say that in the absence of published research, she cannot advise anything departing from the standard surgical procedure. If she innovates and Alison dies earlier than would be expected statistically with standard treatment, she will be vulnerable to disciplinary or legal proceedings.

Under the Bill, if Dr Smith was impressed by the arguments in favour of the new treatment, she could follow the process outlined in the Bill, which includes talking to other experts and to Alison and her relatives about the innovative treatment, and obtaining a consensus as to its use.

If the case came to court, Dr Smith could be confident she had followed the Bill’s processes and any court decision would be made in that light. There would be no opposing ranks of “experts” commissioned by the two opposing legal sides, after the event.

→Watch Lord Saatchi explain his Bill visit telegraph.co.uk/video

→Read: Telegraph stories and updates on the Bill:telegraph.co.uk/health/saatchi-bill/

 

 

Daily Mail: ‘It could lead to a cure': Jeremy Hunt praises Saatchi’s bid to overhaul ‘medieval’ cancer care

Lord Saatchi and Josephine Hart

→READ: article in the Daily Mail

By Stephen Adams for The Mail on Sunday

Advertising guru Maurice Saatchi, whose wife died of ovarian cancer, has won Government backing for his private member’s Bill calling for more innovation in treatment of the disease.

When novelist Josephine Hart died two years ago, the Tory peer described her treatment  as ‘medieval’.

Lord Saatchi has since fought to enable doctors to use procedures other than surgery, radiotherapy and chemotherapy.

He argued they are too often hamstrung by legal rules which mean they can be sued if they ‘deviate’ from standard treatments, even if they know it will do little good.

 The peer, founder of advertising giants Saatchi  & Saatchi, sponsored the Medical Innovations Bill to reduce the threat of litigation and ‘encourage responsible innovation’.

And on Friday, Health Secretary Jeremy Hunt praised the Bill, saying it ‘could lead to major breakthroughs, such as a cure for cancer’

Mr Hunt announced a ‘full and open consultation’ in the New Year to understand ‘the problems [doctors] face in innovating’ and strike ‘the right balance between innovation and safeguards’.

Last night, Lord Saatchi said: ‘I think the  Prime Minister and the Secretary of State want Britain to regain its place as the world leader  in medical innovation.’

He stressed patient safety was at the heart of his proposals. ‘We do not want reckless experimentation that puts patients at risk, and we don’t want patients to be treated like mice,’ he said.

Obtaining informed consent to take part in experimental treatments would remain essential, the peer stressed,  and doctors would retain full legal responsibility for  their decisions.

→READ: article in the Daily Mail