All posts by Medical Innovation Bill

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.

 

My son Daniel

By David Thomas

The Medical Innovation Bill encourages doctors to take responsible risks, if this is what a fully-informed patient wants. Ensuring appropriate safeguards is, of course, vital.

The Bill should be seen as part of a wider picture. Palliation apart, the aim of medicine is to give those suffering from serious conditions the best chance of getting better. A statement of the obvious, perhaps, but in my experience it does not always happen.

My son Daniel, then aged 17, was diagnosed with a life-decimating bone cancer, Ewing’s sarcoma, in 2006. He had the standard treatment and was well looked after. He got into remission and on with his life. But prognosis was dismal.

So, naturally, we scoured the world for possible solutions. I found out about insulin growth factor inhibitors. There was a good deal of excitement about them in the Ewing’s world at the time. Unusually for cancer treatment, there seemed to be few side-effects. There were a number of clinical trials. A principal criterion for Roche’s was that a patient must have disease measurable by scan, standard in oncology so that efficacy can be measured. At that time, Daniel, in remission, had no measurable disease.

But a special diagnostic test, RT-PCR, indicated that he still had microscopic cancerous cells, as was expected. Now was the time to hit those cells, when they were at their weakest and had not mutated. We needed Roche to accept Daniel onto their trial, either by interpreting the criteria flexibly (I’m a lawyer and provided the interpretive ammunition) or by extending them to include RT-PCR–positive patients. If the inhibitor killed Daniel’s microscopic cells, Roche could chalk up a response. The company blanked me. Daniel’s doctor, involved with the UK trial, appeared unwilling to advocate for his patient and felt constrained by confidentiality not to release important information, an intolerable situation.

I turned to the MHRA, the UK clinical trials regulator. My argument was that, with rare conditions (especially involving children), the MHRA had a duty to ensure not only that trials are safe but that they produce as much useful data as possible. Allowing RT-PCR-positive patients onto the trial would increase the data pool. However, the MHRA responded that, safety apart, trial criteria were for the drug company.

I was willing to take the regulator to court. But Daniel then tested negatively in another RT-PCR, in Barcelona. This was great news but it effectively destroyed my legal arguments. We put the inhibitor on the back-burner.

Daniel eventually relapsed. After much failed treatment, he had a different IGF inhibitor but, although he showed some response, his cancer was by now far too powerful. He died in October 2011, just short of his 23rd birthday, a classics student at Oxford, full of dreams and promise and surrounded by love.

Would earlier access to an inhibitor have helped? It is fair to record that overall they have been a disappointment. But there have been some remarkable results. Sadly, the system was not flexible enough to allow Daniel access at the most opportune time. That might just have cost him his life and much suffering (and the NHS tens of thousands of pounds on wasted treatment).

Medical treatment should seek to squeeze every last drop out of current knowledge. There are many aspects to that, not least ensuring that clinical trials are regulated for the benefit of patients, not the convenience of pharmaceutical companies.

Lord Saatchi’s Bill has the laudable aim of putting the interests of seriously ill patients at the heart of decision-making.  Everyone, surely, must support that objective.

 

David Thomas is a consultant to Bindmans LLP and for several years was legal officer to Child Poverty Action Group. He is a former chair of the RSPCA, trustee of Compassion in World Farming and member of the advisory panel for Burma Campaign UK and is currently a trustee of a Casa Alianza UK, a street children charity.

He has written extensively about legal and related issues, especially in the campaigning context, and used to teach judicial review and human rights law, primarily in the context of welfare benefits.

He is a former member of the Law Society’s mental health and disability committee and of the Legal Services Commission panel advising on the grant of legal aid in public interest cases. He is also a part-time judge. 

Powerful plea from patients in support of Medical Innovation Bill

The Sunday Times have today published a letter in support of the Medical Innovation Bill from 100 patients and family who have lost loved ones.

The group, which includes Andrew Lloyd Webber, Melvyn Bragg and the publisher Gail Rebuck, widow of the Labour grandee Philip Gould, today issue a powerful plea for dying patients to be given access to experimental drugs and other treatments.

The Bill has moved recently into the House of Commons, having been passed unanimously by the House of Lords.

Last week senior oncologists wrote to Telegraph this week 100 patients & family write to Times.

The letter this week to the Sunday Times follows a supportive letter last week to the Telegraph from senior oncologists, researchers and patient groups.

→READ: Letter to the Telegraph from senior oncologists, researchers and patient groups.

READ: Letter to the Sunday Times – a powerful plea

We are a group united by grief.

We are the bereaved – widows, widowers, brothers, sisters and parents who have lost loved ones to incurable diseases.

We are the parents fighting for the lives of our children who have cancers and degenerative diseases.

We are the patients dying for an answer to our own illnesses.

We have never met each other. But we share a bond of pain and fear.

And we are united in our support for The Medical Innovation Bill.

Not because we believe that it is the silver bullet.

Not because we think if it is passed that tomorrow there will suddenly be new cures for cancers, for Duchenne Muscular Dystrophy and other killer diseases.

We support the Medical Innovation Bill because it gives us hope – hope that doctors will feel more confident to try novel approaches to killer diseases for which current treatments are known not to work.

We support the Medical Innovation Bill because it offers hope to people yet to face what we have faced.

We support the Medical Innovation Bill because it will inspire doctors to innovate and to collect and share the results of their innovations so that medical science is advanced.

We know it will give doctors confidence and legal clarity to try more and to do more.

The patient’s voice has been drowned out. We have been patronised and told we must leave it to the experts.

But we have watched – and are watching – our families die. Some of us are watching our own bodies die.

Doctors have the medical experience.  But we have the human experience.  Nobody knows more about these fatal diseases than we do.

As the Bill proceeds to The Commons, our voice will be heard.

 Gail Rebuck

Sir Michael and Lady Pakenham

Lord and Lady Lloyd-Webber

Victoria Gray

Antonia Wellington

Lord Bragg

Sir Christopher Bland

Frieda Hughes

Vita Paladino

Lord Foster

Debbie Binner

Tom Parker-Bowles

Sara Parker Bowles

Richard Kitley

Mavis Nye

Ray Nye

Omaira Gill

Alex Smith

Rose Fletcher

Claire Cowley

Paul Cowley

Annette Gration

Philip Gration

Pat Hay

Julia Samuel

Sir Henry Keswick

Vivian Duffield

Neil Hay

Mary Toms

Nathan Toms

Barbara Whitehead

Julie Cooper

Christine Winters

Esther Driscol

James Driscol

Maurice Chambers

Beverley Chambers

Gemma Chambers

Clare Smith Daughter

Stauroulla Parker

Michele Parker

Dorothy Vaux

Diane Salisbury

Betty Salisbury

Pauline Debra

Debra Stuart

Stuart Faulser

Jan Weston

Cathy Dear

Elaine Bounds

Karyanne Todd

Amanda Reynolds

Steve Wride

Linda Wride

Richard Elson

Jackie Elson

Dr Irene Kappes

David Wilshire

Faye Wiltshire

Patricia Wiltshire

Dawn Fiddler

Barbara Scott

Robert Scott

Barbara Hampel

Billy Jenkins

Hannah Richards

Angela Davies

Michael Lasseter

Pan Pantziarka

Gail Mathe

Elaine Mitchell

Louis Brooks

Julia Travers-Wakeford

Lawrence Tansley

Sue North

William Pope

Diana Boyle

Robert Johnson

Karen Waldron

Ian White

Gayle McElhinney

Patricia Stubbs

David Williams

Jonathan Stubbs,

Julie Williams

Jane Weitzmann

Jen Selig

Sally Greene

Emily Crossley

Nick Crossley

Tony Levene

Paul Fitzpatrick

Lord Smith of Clifton

Alex Johnson

Andy Johnson

Lara Veitch

Natasha Bramble

Kerry Rosenfield

Doron Rosenfield

Emma Hallam

Andy Hallam

Steven Ho

Why Ebola treatment has parallels with the Medical Innovation Bill

Ebola Virus ©NIAID
Ebola Virus ©NIAID

A leading epidemiologist and governor of the Wellcome Trust has said there were “obviously parallels” between the Medical Innovation Bill and the sanctioning of untested therapies for Ebola patients.

Prof Peter Smith was on the World Health Organisation’s panel which permitted the use of untested therapies for the fatal Ebola virus.

Prof Smith said the WHO panel’s guidance “gave the go-ahead to use products for treatment that were not licensed for treating Ebola.”

Prof Smith said there were similarities between the Medical Innovation Bill and WHO Ebola Guidance.

“There are obviously parallels,” he said. If I had a cancer that say had a 70% mortality in six months and there was an experimental therapy and there’s no data on it, but which might actually improve that survival – and it looked at if it wasn’t going to kill me tomorrow – then I might well want the opportunity of taking that drug or whatever it was. And that’s the situation the Ebola patients are in.

“I think the situation here [with Ebola] was sufficiently dire that there was encouragement to actually shortcut the normal processes. I mean, to use therapies for which there may not be as strong an evidence base with respect to safety as you would normally require – but these were special circumstances.”

Another parallel with novel Ebola treatments and the medical Innovation Bill is the idea that in cases where a full trial is impossible – for lack of patient numbers, or because of an imminent likelihood of the death of the patient, innovative treatments could form the foundation of a full trial at a later date.

He said while it was impossible to draw sound data from a small number of cases, such innovations would “at least produce the evidence needed to justify doing a proper trial.”

“Treating a small number of patients with a new therapy, can’t make that judgment with any confidence in a small number of patients but at least it will produce the evidence needed to justify doing a proper trial. One of the strategies that’s being used for the new Ebola treatment is to really just do that.”

Acknowledging there was a risk in prescribing treatments that had not been through full clinical trials, he added: “You don’t want to give [patients] treatments that would hasten their mortality but you’re more inclined to take that chance because they’ve got this severely life threatening condition anyway.

“The mortality rate is still very high and there’s really nothing on the shelf that is licensed to treat these conditions and that, I think, is the incentive to use things which have been either been slow to being developed or really are experimental.

“These [treatments] might have worked against other viruses but one doesn’t know if they’re going to work against this virus. Obviously you’re prepared to take more risks with respect to something that is going to be given to someone who has quite a high chance of dying anyway than you would in someone who is perfectly healthy.”

However, Prof Smith said novel treatments should only be used in the best interests of the patient – and not as mini-experiments.

“If you are giving a certain therapy to a patient then you have to believe it’s going to do them some good. But you may do that with considerable doubt as to whether it will actually will do them good – but you have to believe it will do them more good than harm.”

As with the Medical Innovation Bill, which will require a mandatory reporting of the results of medical innovations – something that does not happen at the moment – Prof Smith said doctors using novel Ebola therapies should also collect data. “You should maximise the information you get out of that and measure as much as you can even though it’s not a formal trial.

Letter to the Telegraph – Pass the Medical Innovation Bill say senior oncologists, researchers and patient groups

Letter to the editor of the Telegraph from senior oncologists, researchers and patient groups.
Letter to the editor of the Telegraph from senior oncologists, researchers and patient groups.

 

Letter to the Telegraph published today in support of the Medical Innovation Bill (Saatchi Bill) from senior oncologists, professors across numerous disciplines, cancer and rare disease CEOs, research groups, early access to medicine campaigners and patient advocates.

They ask, “Ultimately the question that must be addressed is: what can we responsibly offer to those patients for whom there are no suitable clinical trials?”

Letter to the Telegraph on Medical Innovation Bill (Saatchi Bill)

SIR – We note the successful third reading of the proposed Medical Innovation Bill (the Saatchi Bill).

While there have been significant advances in cancer treatments in recent decades, there remain areas where there has been no meaningful advance. Diseases such as glioblastoma, sarcoma or pancreatic cancer have seen no clinically relevant improvements over those decades.

While clinicians have leeway to prescribe drugs “off-label”, we know from our direct experience with patients that viable clinical options are not being used in the vast majority of “terminal” cases. When all standard therapies have failed, and there are no clinical trials available, the response is almost uniformly to move that patient into palliative care.

We do not dispute that the clinical trial is necessary in order to identify those advances that work and those that do not. However, the evidence base for medicine can come from many different sources. Data collection is a necessary corollary of increased off-label usage and the new registry included in the Bill will record information (including side-effects and outcome data) in every instance of an innovative treatment. This ground-breaking registry will enable us to analyse real-world data, thereby providing greater patient protection than exists at present.

Ultimately the question that must be addressed is: what can we responsibly offer to those patients for whom there are no suitable clinical trials?

Pan Pantziarka
The George Pantziarka TP53 Trust
Dominic Hill
Film maker & patient advocate
Professor Marc-Eric Halatsch
Professor of Neurosurgery, University of Ulm
Lydie Meheus
Managing Director, Anticancer Fund, Brussels
Dr Gauthier Bouche
Medical Director, Anticancer Fund, Brussels
Richard Gerber
Glioblastoma survivor and patient advocate
Professor Angus Dalgleish
St George’s Hospital, University of London
Professor Ahmed Ashour Ahmed
Professor of Gynaecological Oncology, University of Oxford
James Hargrave
Empower Access to Medicine
Dr John Symons
Director, Cancer of Unknown Primary Foundation
Flóra Raffai
Findacure
Professor Stephen Kennedy
Professor of Reproductive Medicine, University of Oxford
Dr Ian N Hampson
Reader in Viral Oncology, University of Manchester
Professor Andy Hall
Associate Dean of Translational Research, Newcastle University
Professor Emeritus Ben A Williams
Psychology, long-term glioblastoma survivor, patient advocate, Moore’s Cancer Center, University of California, San Diego
Dr Al Musella
President, Musella Foundation, founder The Grey Ribbon crusade: umbrella organisation for over 100 brain cancer charities
Professor John Boockvar
Director, Brain Tumor Center Lenox Hill Hospital NYC
Professor Emil J Freireich
Ruth Harriet Ainsworth Chair, Developmental Therapeutics, The University of Texas, MD Anderson Cancer Center, Houston, Texas
Brett Shockley
Patient advocate
Professor David Walker
Professor Pediatric Oncology, University of Nottingham
Laura Mancini
Clinical Scientist, National Hospital for Neurology and Neurosurgery, UCLH NHS Foundation Trust, London
John Morrissey
Adviser to the Children’s Cancer Research Fund
Stephen Western
Patient advocate, Astrocytomaoptions.com
Richard E Kast
MD, IIAIGC Study Center
Charlie Chan FRCS
Consultant Breast Surgeon
Professor Chas Bountra
Professor of Translational Medicine, University of Oxford
Dr Henrietta Morton-King
North Cumbria University Hospitals Trust
Dr Andrew Brunskill
Clinical Assistant Professor of Epidemiology and Health Services, University of Washington Seattle)
Vincent Galbiati
President & CEO of Tomorrow’s Cures Today, Washington DC
Neil Hutchison
Founder, Magic Water Pediatric Cancer Foundation, San Diego
Fiona Court
Consultant Oncoplastic Breast Surgeon, Cheltenham
Professor Alastair Buchan
Head of the Medical Science Division and the Dean of the Medical School at the University of Oxford
Dr Georgios Evangelopoulos
Patient advocate, lawyer & political scientist
Professor John Yarnold
Professor of Clinical Oncology at The Royal Marsden and Institute of Cancer Research
Professor Jerome H Pereira
Consultant General & Oncoplastic Breast Surgeon, Norwich Medical School University of East Anglia
Dr Lynne Hampson
Non Clinical Lecturer in Oncology, Institute of Cancer Sciences, Manchester
Dr Robert Kirby
Senior Lecturer, Hospital Dean, University Hospitals of North Midlands
Professor Gareth Evans
Professor of Medical Genetics and Cancer Epidemiology, University of Manchester
Dr Rupert McShane
Coordinating Editor Cochrane Dementia and Cognitive Improvement Group, Oxford University
Michael Shackcloth
Consultant Thoracic Surgeon, Liverpool Heart and Chest Hospital
Professor Vikas P Sukhatme
Professor of Medicine, Harvard Medical School, Co-founder Global Cures
Vidula Sukhatme
Co-founder Global Cures
Sarah Lindsell
CEO, The Brain Tumour Charity
Neil Dickson
Chairman, The Brain Tumour Charity
Alex Smith
Founder, Harrison’s Fund
Giles Cunnick
Consultant General & Breast Surgeon, Bucks Healthcare NHS Trust
Dr Piers Mahon
Biotech Consultant
Paul Fitzpatrick
Chairman, Duchenne Now
Dr David Faurrugia
Consultant Oncologist, Cheltenham General Hospital
Dr Chris Govender
Medical Officer in Addictions
Sue Farrington Smith
Chief Executive, Brain Tumour Research
Professor Steven Gill
Professor in Neurosurgery, University of Bristol

 

When relying on the Bill must medical experts who are offering their views to the doctor who is treating the patient actually see the patient?

It is often most efficient and effective for doctors to discuss the principles of treatment without examining each others’ patients.

This is not new and already happens currently.

Doctors will decide for themselves what information they need in order to offer views on treatment, and whether they need to see the patient – as is the case now.

GMC guidance or hospital protocols will also be relevant.

Why is there is no requirement that the other doctor must agree to or support the proposed treatment. The doctors may disagree.

The views must be recorded in the patients’ notes – no doctor who receives only negative responses will be safe in relying on the Bill.

To intentionally use known, supportive doctors and to exclude intentionally dissenting experts would be unreasonable and would expose the doctor to a claim of negligence.

No doctor who receives only negative responses will be safe in relying on the Bill.

However, the Bill does not allow one, single, dissenting doctor to effectively veto a novel treatment for which there is otherwise a clear consensus among other experts.

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.