Open Letter - Medical Innovation Bill

Today's Daily Telegraph includes an open letter in support of the Medical Innovation Bill (aka the Saatchi Bill). The letter was conceived and organised independently of the official Saatchi campaign. The letter, which carried 52 signatures, was edited for publication. The full text is reproduced below:

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The Letters Editor

Daily Telegraph

London

29/01/15

Dear Editor,

We note with considerable interest the successful third reading of the proposed Medical Innovation Bill, aka 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, pancreatic cancer and others have seen no clinically relevant improvements over those same decades. Refractory metastatic solid tumours remain a challenge and a significant cause of morbidity and mortality. Furthermore, for many less common diseases the landscape of clinical trials is barren.

While it is true that clinicians have lee-way to prescribe drugs ‘off-label’, we know from our direct experience with patients that viable clinical options are not being accessed in the vast majority of ‘terminal’ cases. When all standard therapies have failed, and there are no clinical trials available for the patient, the response is almost uniformly to move that patient into palliative care. Too often it appears that clinicians are reluctant to try treatment alternatives – be they metronomic chemotherapies, repurposed non-cancer drugs with evidence of efficacy or compassionate use/medical needs programs. Note that these are all options with often considerable levels of clinical and pre-clinical evidence; this is not junk science or quackery.

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 under the terms of the Bill. This ground-breaking registry will enable us to mine and analyse real world data so that we are not dealing with a set of anecdotes, but validated and clinically useful information and so providing greater patient protection than exists at present. Physicians treating patients with no other options would be empowered to evaluate off-label interventions with the highest evidence of efficacy.

The reluctance of physicians to explore alternative options may not be solely due to a fear of litigation, as Lord Saatchi contends. There are other social, cultural and institutional barriers at work – individual and institutional comfort zones – which often preclude off-label prescribing. However, if the passing of the Bill affects a change in thinking such that there is a greater willingness to explore potentially helpful treatments, then it will have provided benefit to patients. Passing the Bill sends a positive message that encourages responsible use of off-label options. Not passing the Bill sends a strong negative signal that off-label usage is neither encouraged nor supported.

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

Yours Sincerely,

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

Saatchi Bill and Medical Anecdotes

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.

The Medical Innovation Bill

What happens when you reach the end of the road with the standard cancer protocols? A patient starts with first-line treatments, usually a combination of chemotherapy, surgery and radiotherapy, depending on diagnosis and disease staging. If the response to that first-line isn’t positive, the disease continues to spread, then there’s a second-line and possibly a third-line treatment, depending on the type of cancer and how the patient responds to these often toxic treatments. And if the disease is still there at the end of this gruelling process? At this point the prognosis is pretty grim. What happens then?

At this point I should say that I’m speaking from direct personal experience with my son, George, who died almost three years ago from metastatic osteosarcoma that resisted every treatment thrown at it. And from the experience gained in the years following his death in trying to help other people in a similar situation, often also suffering from Li Fraumeni Syndrome as George did.

What happens? In the first instance the lead oncologist will often look for a clinical trial, hopefully a Phase II or Phase III trial. But often the patient will have already been on a trial and come out the other side with the disease still active. In practice if there are trials available, and often they are not, it will be a Phase I trial. The aim of a Phase I trial is to find the right dose to use in a Phase II trial, which is looking to see how well a drug works. Efficacy – how well the drug works – isn’t normally the primary outcome from a Phase I trial.

And if there’s no trial, or you don’t want to go on a Phase I trial with a new drug that might have toxic side effects and no effect on your disease? Then you are in the realm of palliative care. If there’s no standard treatment and there’s trial, then the emphasis is on trying to keep a lid on the symptoms and trying to preserve what quality of life you have given the advanced state of the disease.

But what if there’s something that doctors are using in another country? What if there’s a drug that has been shown to have some effect in your type of disease? What if there are other options that aren’t covered by the standard protocols?