Tag Archives: innovation

Film: The professor who ‘cured’ his cancer with a cocktail of everyday pills and 20 years on remains disease-free

In the Telegraph today Ruth Wood writes about new documentary Surviving Terminal Cancer

From the filmmakers:

“This film charts the remarkable story of Ben Williams, professor emeritus of experimental psychology at University of California, San Diego. Diagnosed in 1995 with a lethal cancer, a primary brain tumour called glioblastoma multiforme, he was given just a few months to live.

But a natural born maverick, and rigorous scientist, Ben decided he would not go down without a fight. Nineteen years later his story is an inspiration to patients the world over, whilst his case is dismissed by the medical community as just one of a handful of statistical outliers.”

Watch the trailer

From the Telegraph:

ONE day, some two decades ago, Ben Williams set out from his home in San Diego, California, to cross the border into Mexico in search of acne tablets. The 50-year-old psychology professor didn’t actually suffer from acne. What he had was the deadliest type of brain tumour, a glioblastoma multiforme that was the size of a large orange. A leading neuro-oncologist from Texas had suggested that a skin treatment, called Accutane, might help him.

Prof Ben Williams was diagnosed with the tumour in March 1995. “The entire right side of my brain was infested with a tumour,” he recalls. “Apparently brain tumours as large and ugly as mine are a notable event. Soon half the neurology department had shown up to look at the scans.” He underwent surgery the following afternoon. However, relations with his neurologist soon became strained due to his dogged insistence on researching his own treatments.

His doctor wanted him to stick to the standard regimen of surgery, radiotherapy and chemotherapy. But the rebellious Harvard alumnus insisted on adding to this a veritable cocktail of drugs – in addition to the acne pills, there were blood pressure and insomnia tablets. All were cheap and had little or no toxicity, and for all of them Williams had gathered some credible evidence from scientific trials that they might reduce his tumour, boost his immune system and make chemotherapy more effective. But none had been approved in the United States for use in the management of brain tumours, so his own specialist had dismissed them.

“He said I would drive myself crazy researching all these things and that I might hurt myself,” recalls Prof Williams, whose story is told in a new film, Surviving Terminal Cancer, released online today. “I almost laughed. Hurt myself? I had the most aggressive kind of brain tumour. I was expected to die in a year. What did I have to lose?”

Even today the average life expectancy for patients with glioblastoma multiforme is just 15 months, with survival rates highest among young people. Fewer than 10 per cent of people aged 50 and above survive for five years. So it is against all odds that Professor Williams has just celebrated his 70th birthday and 20 years of clean MRI scans.

“I’d been told that my chemotherapy wouldn’t get rid of the tumour completely or indefinitely, so I focused on finding agents that might make chemo work better for me,” he says. Sure enough, after his fourth round of chemotherapy in 1996, Prof Williams’s tumour had vanished. It has never returned and thousands of people, including oncologists, have sought his advice since on ”beating” a cancer known in medical circles as “The Terminator”.

In mainstream oncology Professor Williams is considered a freak case and his strategy of fighting cancer “using every potentially efficacious agent I could lay my hands on” attracts suspicion. Yet a growing number of specialists and researchers say there is evidence that some of the common pills taken daily by millions for other ailments, could be ‘‘repurposed’’ to help in the battle against cancer.

“We just need to look in our medicine cabinets,” says Pan Pantziarka, scientist and UK spokesman for the Anticancer Fund, a Belgian non-profit organisation run by researchers and doctors. “There is strong evidence that some of the medicines we use every day have anti-cancer properties.”

At present, the pharmaceutical industry is using advances in our understanding of genetics to create so-called ‘‘magic bullets’’, a new generation of ever smarter, ever more targeted therapies. These act like snipers, interfering with specific cell proteins or signalling pathways that have a role in cancer. Major successes with this approach include the chronic myeloid leukaemia drug imatinib (Glivec) which blocks a protein that makes cancer cells grow and divide.

But, according to Professor Angus Dalgleish, Foundation Chair in Oncology at St George’s Hospital, University of London, many targeted therapies are eventually doomed to failure.

“Cancer will do everything it can to survive and avoid being hit,” he says. “It’s like a traveller who wants to cross London on the Tube. Yes, you could block him by taking out a major station like Oxford Circus, but he’ll just switch to a different line. It’s the same with cancer. After getting hammered by one agent, the tumour quickly reinvents itself through evolution. I always delay giving these targeted therapies as long as possible because I know they’re not going to be working a few months down the line.”

It costs more than $1billion (£650 million) to bring a new cancer drug to market and takes more than a decade. As drug companies face an increasingly uphill battle to invent new chemical entities that can be patented, they pass on the cost of a 90 per cent-plus failure rate, expensive trials and marketing to the NHS, insurance companies, healthcare providers and patients. Such is the spiralling cost that the Government now runs a separate £280 million-a-year Cancer Drugs Fund to shield NHS budgets – and even that is expected to overspend by £100 million this year. In January, the Government announced that 16 life-extending drugs would no longer be paid for by the Fund because of cost-cutting.

The good news is that early-stage laboratory experiments and clinical studies, as well as large scale epidemiological research point to the potential cancer-fighting properties of dozens of existing medicines that millions of people take safely every day for other ailments. Aspirin is the most high-profile example. Research funded in part by Cancer Research UK shows that it can significantly cut the risk of bowel, throat and stomach cancer if taken daily by people aged 50-65 (although the CRC warns on its website that aspirin can have side effects and should not be taken regularly without medical advice).

The Repurposing Drugs in Oncology (ReDo) Project, an international collaboration between the Anticancer Fund and US-based non-profit organisation Global Cures has identified 70 potential agents for which there is evidence of cancer-fighting properties. These include the diabetes tablet metformin, cholesterol-lowering statins, the antacid cimetidine, the de-worming tablet mebendazole, the anti-fungal itraconazole, and all the drugs Professor Williams took as he battled his brain tumour.

“Most modern cancer drugs are known as targeted therapies because they are aimed at very specific targets inside cancer cells,” says Dr Pantziarka. “But these older medicines are known as ‘dirty drugs’ because they have multiple targets, interfering with more than one protein or signalling pathway at a time. Used in combination, they could be very effective.

“If these medicines were coming out today, some would be blockbuster cancer drugs. But most are no longer covered by patents so the pharmaceutical industry has no financial incentive to investigate them.”

Even though these medicines are not officially labelled as cancer drugs, doctors are legally entitled to prescribe them “off-label” if there are solid grounds to believe they will be beneficial. Indeed, Lord Saatchi’s Medical Innovation Bill, which is supported by the Telegraph, aims to give oncologists (and specialists in other fields) more confidence to be experimental in treating terminally ill patients for whom all existing therapies have failed and who are prepared to take risks. But in practice, most oncologists are unwilling to prescribe drugs that have not passed final-stage (Phase III) clinical trials for a particular type of cancer, and even more wary of combinations that may interfere with conventional treatment or have unforeseen side effects.

Professor Dalgleish is one of the few UK doctors willing to think differently. “Say we have a patient who is fit and healthy in many ways but is going to be dead within months. If that patient asks me, ‘Is there anything else I can do?’ I will say, ‘Yes. The data suggests you could consider metformin which appears to selectively reduce glucose uptake by tumour cells as opposed to normal cells. I suggest aspirin to tackle your inflammation and let’s correct your vitamin D levels to boost your immune system.’

“I call it creative compassion because it’s not in the rule book and it’s what I would want for myself if I were in the same position. I wouldn’t want to just be told to go and see the palliative care person.”

Professor Justin Stebbing, of Imperial College London, the oncologist who treated actress Lynda Bellingham before her death from bowel cancer last year, agrees with the approach, though he tends not to prescribe off-label drugs himself.

“That’s not because I’m ethically opposed to the idea, but it’s not something I do every day so I’m not knowledgeable about dosages,” he says. “However, I don’t have a problem if patients get the drugs elsewhere and am open to referring them to trials that test these alternative approaches.

“As a profession, we can be cruel to cancer patients, giving them treatments that are horribly toxic with minimal benefits. I find it very frustrating when my colleagues are dismissive of patients who want to try other things that are non-toxic and may extend their lives.”

Professors Dalgleish and Stebbing are the co-authors of a study into “cocktail cancer therapy” currently taking place at the Care Oncology Clinic in London. Over the next five years, the private clinic run by biotech firm SEEK is aiming to treat more than 10,000 cancer patients with a combination of four ‘dirty drugs’ — statins, metformin, the de-worming drug mebendazole and doxycycline, a common antibiotic.

Gregory Stoloff, founder of SEEK, says it’s a non-profit trial funded by patients themselves, who pay £400 for the initial consultation, then £200 every three months to cover the cost of the drugs, consultations and the trial.

“Oncologists refer patients to us who have run out of treatment options and we put all of them on the treatment right away,” he says. Because nobody is given a placebo, this is not a controlled clinical trial. But SEEK hopes that treating thousands of people will create enough data to enable an effective comparison with cancer survival rates in the rest of the population. “Controlled clinical trials are all very well but when you have people who are expected to die within months they want treatment now. And these are very safe medicines that people have been taking for decades.”

Although Phase III clinical trials remain the gold standard for science, they don’t necessarily serve cancer patients well, says US campaignerDominic Hill, in whose film Prof Williams appears, along with oncologists researchers and other cancer survivors.

“Today’s lifesaving treatment for HIV [called antiretrovirals] is given to patients despite it never having passed a randomised Phase III clinical trial,” Mr Hill, whose brother-in-law Andreas died of a brain tumour in 2010, points out.

“How many cancer patients must die before the regulators recognise that this approach needs to be adapted to oncology?”

Dominic Hill’s film Surviving Terminal Cancer is free to watch onsurvivingterminalcancer.com

The Drugs in Question: the evidence for and against

Metformin: Several studies suggest that tumours grow more slowly in cancer patients who take this anti-diabetic drug. Early-stage clinical trials are investigating its potential to prevent various cancers including prostate, breast, colorectal and endometrial.

Statins: Preclinical studies suggest these cholesterol-lowering heart drugs may prevent various cancers and stop them spreading. One recent meta-analysis associated a daily statin with a significant risk reduction of liver cancer.

Mebendazole: There is evidence this drug – usually prescribed to treat parasitical worm infections — may inhibit cancer cell growth and secondary tumours, though no clinical trials have been completed.

Cimetidine: This over-the-counter antacid has direct anti-proliferative effects on cancer cells, inhibits cell adhesion, reduces tumour angiogenesis (growth of blood vessels essential to a developing tumour) and also boosts anti-cancer immunity in various cancers.

Itraconazole: The common anti-fungal treatment is also thought to be anti-angiogenic and has shown promise as an agent for prostate cancer, non-small cell lung cancer and basal cell carcinoma, the most common kind of skin cancer.

Isotretinoin: This acne drug, marketed as Accutane, is occasionally used to treat certain skin cancers and neurological cancers as well as to prevent the recurrence of some brain tumours, although some studies suggest it is ineffective.

What does Cancer Research UK think?

Professor Peter Johnson, chief clinician at Cancer Research UK, said: ‘It’s completely understandable that when faced with a terminal cancer, patients and their doctors want to explore every possible treatment option, but it’s important to balance the wish to do something against the problems such as side effects and false hopes.

“Cancer doctors in the UK are very innovative and often try new treatments if they and the patient feel this might be of benefit. The thing which sometimes prevents us from using different treatments is not our reluctance or a fear of being sued, but NHS funding. The NHS requires good evidence a treatment will work, which is why we carry out research trials, and more UK patients go on clinical trials than anywhere else in the world.

“Clinical trials are essential to find out whether new treatments are safe, if they are better than the standard care, and which patient groups will benefit. The only way to improve cancer medicine and show that a new treatment should be given to patients is by analysing trial results, which are scrutinised to make sure that they are ethically sound and scientifically valid. There are many different ways to do trials, but they all have the same goal: to find the best and safest forms of treatment. The people who take part in trials know that even if they themselves don’t benefit, they will help other people in the future.

“It doesn’t make sense for patients to withhold information from their doctor about other medicines they are taking. Many drugs, even those sold as natural remedies, can interfere with each other and the results can be very dangerous, or even fatal.”

Update on the Saatchi Bill

The Medical Innovation Bill, which would protect patients and doctors who want to try experimental treatments, is just days away from either becoming law — or becoming a footnote in the history of scientificadvance.The Private Member’s bill sponsored by Lord Saatchi has been debated four times in the House of Lords, tested and amended by Labour, Tory and Lib Dem peers and scrutinised and approved by doctors, lawyers and judges in the upper chamber. It is supported by both the Government and Opposition front benches. It is also backed by patients and their families – more than 40,000 have signed up as Medical Innovation Bill supporters.The Bill now includes a register that will see the results of all innovations recorded so they can be shared and used by other doctors and researchers. Having been amended and passed by the Lords, the Bill must now be approved by MPs in the Commons. But time is running out – Parliament has only weeks to complete its business before it is dissolved ahead of the May election. It is now up to the Government to find time for the Bill to be debated. If this does not happen the Bill will fall.

The full film has been released today online – Watch it here 

Prof Chas Bountra talks about crowdsourcing science & medical innovation

Chas Bountra is a visionary. A human dynamo who seems to generate his own energy, who doesn’t know the meaning of ‘impossible’.

More formally, he is professor of translational medicine and head of the Structural Genomics Consortium at Oxford University.

Like many scientists working to develop new molecular entities – drugs to you and me – he realises the system is broken littered with barriers and blocks. It takes too long and is too expensive to develop new drugs for killer diseases.

He has said: “The cost of developing new drugs is spiralling. Analysis by Forbes [in 2012] showed that it cost as much as $12 billion to produce one new successful drug. “Most of the cost pays for failed projects that never see the light of day. From 1996 to 2009, research costs have almost tripled, while the number of drugs approved for use has more than halved. “Drug companies do similar research in parallel and in secret, and usually do not share their failures – and 90 per cent of potential therapeutic compounds fail.

It’s madness. “If companies shared their failures they would prevent each other going down blind alleys – and it would mean patients wouldn’t be used as guinea pigs for drugs that another company already knows won’t work.”

And like Lord Saatchi, he knows there is not one single answer to the problem.

His focus is on encouraging big pharma to share the results of their research so that both development time and costs decrease.

Like Lord Saatchi, he is pushing for greater innovation and recognises that the Medical Innovation Bill can play a part in speeding up the drugs discovery pipeline.

No one on the Bill team says innovation doesn’t happen now. It does. But, that is not an argument against more innovation.

As long as there are incurable diseases we must dedicate ourselves to greater, effective, innovation.

Prof Chas Bountra has been in the news this week (16th Feb 2015)  as part of a group scientists pooling their resources to work together on a treatment for dementia.

We visited him at Oxford University last year. In this video he talks about his support for the Medical Innovation Bill, highlights a few of the challenges within the industry and talks about the innovative work of the Structural Genomics Consortium at Oxford University.


If a doctor makes out the Saatchi Defence, will this stop a judge applying either of the Bolam or Bolitho tests to find that doctor to have been negligent?

If a doctor has satisfied the reasonableness and responsibility requirements of the Bill, the Bolam and Bolitho tests will have been applied and satisfied at the point of treatment, so there will be no opportunity for lawyers to hire doctors to go into court and re-argue the issue in the hope of forcing a settlement from a risk-averse NHS budget-manager.

Is the Medical Innovation Bill different from the current legal requirement that a decision to treat is one which would be supported by a responsible and rational body of medical opinion?

It is a practical application of the existing hypothetical test which asks doctors to imagine what colleagues’ views would be, but not to ask them unless and until the matter comes to trial.

Under the Medical Innovation Bill the doctor will actually ask colleagues for their opinion at the point of treatment.

The Medical Innovation Bill will support evidence-based medicine

Prof Walker - Medical Innovation Bill
Prof Walker – Medical Innovation Bill

One of the key tenets of the Medical Innovation Bill is that it will oblige doctors to record and share the results of incidental medical innovations in the Medical Innovation Register.

This applies to both positive and negative results.

→READ: About the Medical Innovation Bill Register

As such, the Bill will begin to build data around innovations in a way never before seen in this country.

We know that medical innovation already happens – though this bill will encourage more innovation by setting a clear process for doctors to follow that will ensure patient safety and offer the doctor legal clarity.

But currently, there is no register for innovation, no database, no record of success or failure.

Medical Innovation Bill - Oxford University
We are working with Oxford University to produce an open access database of treatments and outcomes under the Bill.

So no one can replicate those successes and avoid the failures.

Recording such data would also allow doctors and researchers to follow up on promising new therapies.

Dr David Walker, professor of paediatric oncology at the University of Nottingham says:

“If evidence started to amass under the Medical Innovation Bill, that a treatment works then it would support the establishment of new trials.

“Historically medicine has a large number of situations where new treatments have been developed by repurposing drugs for a new indication.

“Currently this step is hard to make as people say that although the drug exists, is licensed, it cannot be used off license if there is no evidence as it would be  wasting money that could be spent on licensed drugs for licensed indications.

“This is particularly damaging for people with rare conditions where research is rare – this applies to cancers in particular.

“In childhood 60% of all drugs are off licenses many of our strongest treatments are a product of off license development or repurposing of drugs.”

The Medical Innovation Bill would could speed this process of re-purposing drugs up thereby enabling treatments to get to patients quicker.

Prof Walker added that this would create a system where the recording of pre-trial results of therapeutic innovation would become “a recognised and appreciated step in the drug selection process, particularly in rare conditions.”

“It could help to reduce the risks of failed trials”

→READ: About the Medical Innovation Bill Register

Dr David Walker is a Professor Paediatric Oncology
Children’s Brain Tumour Research Centre
Faculty of Medicine and Health Sciences
University of Nottingham

Guest blog: Saatchi Bill and Medical Anecdotes

By scientist Pan Pantziarka, a scientist in the UK employed by the Anticancer Fund of Belgium. Pan works on drug research as part of the Repurposing Drugs in Oncology project

Re-blogged with permission from  anticancer.org.uk


Opponents of the Medical Innovation Bill (aka as the Saatchi Bill), such as Sarah Wollaston MP, have been very vocal in attacking the Bill by making a number of false claims about what the Bill will do.

One such argument is that the Bill will undermine medical progress by doing away with clinical trials, and that instead we will just have to rely on individual anecdotes that arise from doctors using innovative off-label treatments on patients. In fact Sarah Wollaston even referred to the Bill as the ‘Medical Anecdotes Bill’ in her recent speech in the House of Commons.

There are a number of points that to raise in response to this false assertion.

First, there is no intention to replace clinical trials. The Bill is about treating patients with no place left to turn – these are people who have exhausted standard therapies and for whom there are few options left to explore. If a clinical trial is open and the patient is eligible then that is the place to go if it is in the patient’s best interest. There may be cases where it is the right thing to do, just as there are cases when it will not benefit the patient who is offered the additional choice of an non-standard treatment (for example an off-label drug with evidence of clinical activity in the patient’s illness). This will be decided on a case by case basis, what it will not do is force doctors to ignore clinical trials or undermine the trials process.

We also have to keep in mind that for many rarer cancers, genetic conditions and chronic illnesses there are few clinical trials available. Just as important, even when there are clinical trials open, many patients may not be eligible because of prior treatments, confounding conditions, age or physical status restrictions and so on. What are patients supposed to do when they cannot gain access to clinical trials?

Many of the opponents of the Bill act as if there are no unmet patient needs, as if every patient has access to trials and that there is no need to consider alternatives.

Secondly, rather than leaving us with a collection of anecdotes, the Bill offers the prospect of making a huge step forward in medicine through the creation of a central database to record patient data.

This database will be hosted at Oxford University, and work is progressing with the Department of Health to work out the details. A central database which will record each patient’s details, the treatment they receive and their outcomes is actually a hugely advance in medicine.

With all of the patient data recorded we do not have a collection of anecdotes but a database that we can mine and explore and use to design clinical trials based on the signals we can glean from real patient data. It also means that non-standard treatments that work can be identified more quickly, and just as importantly those that do not work can also be identified and discouraged.

This isn’t a replacement for the clinical trial, it’s an advance on the system we now have.

The central database is really the essential piece that makes the Bill so compelling. It offers doctors looking for innovative treatments a chance to explore and learn from the experience of their colleagues.

Many of the opponents of the Bill suggest that it is not needed – they claim that doctors are already free to innovate and explore non-standard therapies. But what is not in place now is a standard process for this to occur, nor a method by which doctors can learn from each other and help to evolve the breakthroughs we so badly need.

And, finally, a central database means that patient safety will be increased. As all data has to be recorded, those unscrupulous individuals prescribing crank treatments will have to reveal their data, they will no longer be able to keep their results away from scrutiny as they now can.

About the author:

Pan Pantziarka is a scientist in the UK employed by the Anticancer Fund of Belgium. He works on drug research as part of the Repurposing Drugs in Oncology project. He is also the co-founder and chairman of the George Pantziarka TP53 Trust, the only charity in the UK dedicated to supporting families with an inherited cancer predisposition condition called Li Fraumeni Syndrome. He lost his first wife to ovarian cancer when she was 29, and his son George died in 2011 at the age of 17 from osetosarcoma.

Further reading:

→READ: Oxford University offer to host the database

→READ:Prof Walker writes – there will never be enough clinical trials



Medical Innovation Bill – adjournment debate

George Freeman during Bill Adjournment debate
George Freeman, MInister for Life Sciences, speaking during the adjournment debate

Yesterday December 10, 2014 Sarah Wollaston MP was granted leave to hold an adjournment debate on the Medical Innovation Bill.

The Bill team were also invited to meet Dr Wollaston earlier in the week to discuss the Bill. We thank her for that meeting and for requesting the debate.

During the debate Dr Wollaston raised several important points about the Bill, which could formally come to the Commons in early January.

The points revolve around patient safety and scientific rigour – issues which have been raised by senior doctors and lawyers in the House of Lords and which have been discussed with peers in committee and in bilateral meetings with Lord Saatchi, health ministers Earl Howe, George Freeman and Department of Health lawyers and officials.

On the back of these meetings, amendments have been laid which address the issues raised, and they will be further debated on Friday (December 12) in the Lords.

Edit: This debate has now taken place. Read here.

Chief Medical Officer Dame Sally Davies and NHS chief Sir Bruce Keogh, who has inputted into the Bill himself, remain satisfied that the Bill is robust, safe and will enhance, not hinder scientific research.

Summing up in favour of the Bill, Minister George Freeman said:

‘I want to close with some supportive quotes the Bill has received from a number of important people, lest the House should form the view that it is unanimously opposed, which is not the case.


Dame Sally Davies, the chief medical officer, has said:

“I am confident that, with the amendments made in Committee stage, the Bill is safe for patients and has the potential to encourage responsible innovation.”


Sir Bruce Keogh, Medical Director of the NHS, said:

“Encouraging innovation in medicine and protecting patients are both of vital importance. That is why I am pleased that amendments have been devised to address concerns about patient safety.”


Sir Michael Rawlins, president of the Royal Society of Medicine, said that the Bill will allow responsible innovation and treatment:

“I believe the use of the provisions in the draft Medical Innovation Bill will benefit patients, especially those with rarer diseases, and the furtherance of medical science.”


A letter to The Telegraph from 40 leading medical professionals, including David Walker, professor of paediatric oncology at Nottingham university and Riccardo Audisio, the president of the Association of Cancer Surgery, said the Bill

“legally protects doctors who try out innovative new techniques or drugs on patients when all else has failed. This Bill will protect the patient and nurture the innovator. It will encourage safe medical advancement, while at the same time deterring the maverick, thereby recalibrating the culture of defensive medicine. Finally, it will work with evidence-based medicine and provide new data that will inspire and support new research.”

I hope very much that that is the case and that when the Bill leaves the House of Lords, the vast majority of qualified senior opinion in this field is able to agree with it. It is absolutely our intention to support the Bill’s noble intent to promote medical innovation, but equally our intention is to not undermine in any way the Government’s commitment to patient safety or the duty of care that all clinicians share and owe to their patients.’

→Read: The full text in Hansard 



BMJ: The “Saatchi bill” will allow responsible innovation in treatment says Sir Michael D Rawlins President, Royal Society of Medicine


By Michael D Rawlins President, Royal Society of Medicine

Published in the BMJ (British Medical Journal) 15th April 2014

You need to either be a member of the BMJ or to sign up for their 14 day trial.

Many doctors, myself included, will have occasionally tried to treat individual patients – where all else has failed – with novel interventions. This may be where there is no recognised form of effective treatment; or when existing one(s) have not produced the desired effect. The legal basis for doing so, at least for pharmaceuticals, has been the so-called “named patient” provisions of Section 9 of the Medicines Act 19682 which permits any doctor to be able “to sell, procure or supply a medicinal product to a patient under his or her care”.

Although my own experience has been disappointing there have been occasions, when intervening in such a manner, have subsequently led to significant advances. Indeed, such observations are arguably a form of n-of-1 trial.

Confidence in using the “named patient” provisions of the Medicines Act has though become eroded. A number of legal authorities have pointed out that departing from what is regarded as “established practice” or “the standard of care” leaves a doctor open to an action for negligence. The definition of “the standard of care” traditionally follows the Bolam principle2 as amended by the Bolitho decision3 but was forcefully criticised by Lady Butler-Schloss4 in her capacity as President of the Family Division of the High Court:

“The Bolam test ought not to be allowed to inhibit medical

progress. And it is clear that if one waited for the Bolam

test to be complied with to its fullest extent, no innovative

work such as the use of penicillin or performing heart

transplant surgery would ever be attempted”.

The Medical Innovation Bill attempts to rectify this situation. It proposes legislation stating that it would not be negligent for a doctor to depart from the existing range of accepted medical treatments, for a condition, in carefully defined circumstances. These circumstances – allowing for responsible innovation – are laid out in later Sections of the Bill. They include a plausible basis for the use proposed treatment, and an assessment of the risks that could be reasonably expected to be associated with it. The Bill also proposes that, before embarking on such a treatment, the doctor should have discussed it with the patient, the multi-disciplinary team responsible for the patient’s care, and with the Institutions Responsible Officer. The Bill emphasises that its provisions are solely concerned with the patient’s best interests.

The Bill as originally introduced into the House of Lords in May 2013, as a Private Members Bill by Lord (Maurice) Saatchi, and has become known colloquially as “The Saatchi Bill”. The Department of Health is currently consulting on a draft version of it5. I was originally sceptical about the need for the Saatchi Bill but I have been persuaded otherwise. First, it is clear from the comments of Lady Butler-Schloss4, as well as other legal authorities Lord Saatchi has consulted, that there are serious legal impediments in civil law to using therapeutic interventions that do not represent the current standard of care. Second, although Lord Saatchi’s original Bill was confined to patients with malignant disease this restriction has, rightly in my view, been removed in the current draft. There are, after all, many other miserable conditions for which we have no, or very limited, remedies. Third, I did not believe that his suggestion for approval by a multi-disciplinary team, alone, provided sufficient safeguards: the inclusion, now, of agreement by a doctor’s Responsible Officer provides me with re-assurance.

Despite my strong support for the Medical Innovation Bill there are important consequences for the professions when (as I hope) it becomes law:

1) Just because a particular intervention appears to have been effective in an individual patient it cannot be assumed that the results are generalisable. Further research in the form of one or more randomised controlled trials, or case series, will be necessary to establish its effectiveness. To take a recent example, a case report6 describing the apparently successful treatment of generalised juvenile pustular psoriasis with etanercept, requires confirmation before it can be regarded as the current standard of care.

2) There may be occasions when Responsible Officers wish to seek other advice before approving the proposed use of an intervention in accordance with the provisions of the Medical Innovation Bill. This will often need to be provided very rapidly especially when a patient has a life-threatening illness. The Academy of Medical Sciences, or some of the specialist associations such as the British Pharmacological Society, could have an important role here in offering a speedy advice service.

3) If the intentions behind the Medical Innovation Bill are to be fulfilled, NHS hospital Trusts, and their Responsible Officers, will need to look on proposals sympathetically. Anecdotal evidence suggests that too many Trusts are fearful of departing from the prevailing standard of care because of the possibility of litigation. The Bill should provide them with adequate reassurance.

4) It is essential that the results of using the Bill’s provisions, in individual patients, are placed in the public domain whether or not they have been successful. This would allow others not only to learn from such experiences but – especially for interventions that appear to have been successful – to undertake formal research.

I believe that the use of the provisions in the draft Medical Innovation Bill offer benefits to patients – especially those with rarer diseases – as well as to the furtherance of medical science. Subject to the responses to consultation it is the government’s intention to have it on the statute book at the earliest opportunity5.


1. Medicines Act (1968). www.legislation.gov.uk/ukpga/1968/67 (accessed 12.04.14)

2. McNair J. Bolam v Friern Hospital Management Committee (1957) 1 WLR 582. www.e-lawresources.co.uk/Bolam-v—Friern-Hospital-Management-Committee.php (accessed 12.04.14)

3. House of Lords. Bolitho v City and Hackney Health Authority (1998) AC 232. www.bailii.org/uk/cases/UKHL/1997/46.html (accessed 12.04.14)

4. Butler-Schloss LJ. Simms v Simms (2002) FAM.83 para 48. www.bailii.org/ew/cases/EWHC/Fam/2002/2734.html (accessed 12.04.14)

5. Department of Health. Legislation to encourage medical innovation. www.gov.uk/government/uploads/system/uploads/attachment_data/file/285272/9959-TSO-2901828-Legislation_to_Encourage_Medical_Innovation.pdf (accessed 12.04.14)

6. Fialová J1, Vojáčková N, Vaňousová D, Hercogová J. Juvenile generalized pustular psoriasis treated with etanercept. 2014;2:105-8. doi:10.1111/dth.12065.

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


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.