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There is no way to know when our observations about complex events in nature are complete. Our knowledge is finite, Karl Popper emphasised, but our ignorance is infinite. In medicine, we can never be certain about the consequences of our interventions, we can only narrow the area of uncertainty. This admission is not as pessimistic as it sounds: claims that resist repeated energetic challenges often turn out to be quite reliable. Such ‘‘working truths’’ are the building blocks for the reasonably solid structures that support our everyday actions at the bedside.

William A. Silverman. Where’s the evidence? 1998

We have been colleagues for many years, sharing not only professional camaraderie but also a deep-seated conviction that medical treatments, whether new or old, should be based on sound evidence. Our collective experience of healthcare and healthcare research suggests this is often not the case. And that is what encouraged us to write this book.

IE’s curiosity about the evidence underpinning the treatments she prescribed to patients was heightened during her career in medical research. When she became a medical journalist at The Lancet she encountered flagrant attempts by some pharmaceutical companies and researchers to economise with the truth by distorting or embellishing their research results. HT’s unexpected invitation to participate in a clinical trial of doubtful quality made her realise that she ought to be an active participant in the quest for progress concerning her treatment, not a more or less passive recipient of care. She went on to campaign vigorously for collaboration between health professionals and patients to ensure worthwhile research with good quality patient information. IC’s obsession with rigorous assessment of the effects of the things that doctors do to their patients began when he was working in a Palestinian refugee camp: some of his patients seemed to be dying because he was practising in ways that he had
been taught at medical school. Since then he has strenuously promoted the view that decisions in healthcare should be informed by unbiased evidence from relevant research, particularly the results of systematic reviews of controlled trials.

Every year, studies into the effects of treatments generate a mountain of results. Sadly, much of this research fails to address the needs of patients, and even when it does, the evidence is often unreliable. We hope our book will point the way to wider understanding of how treatments can and should be tested fairly. This is not a best treatments guide to the effects of individual therapies. Rather, we highlight issues that are fundamental to ensuring that research is soundly based and designed to answer questions that matter to patients and the health professionals to whom they turn for help.

In Chapter 1 we describe how some new treatments have had harmful effects that were unexpected; the hoped-for effects of others failed to materialise; and some predictions that treatments would not work were proved wrong. Furthermore, some useful results of research have not been applied in practice. In Chapter 2 we highlight the fact that many commonly used treatments and screening tests have not been adequately evaluated. Chapter 3 gives some ‘technical details’ – here we outline the basis for fair testing of treatments, emphasising the importance of paying attention to reducing potential biases and taking
account of the play of chance; this chapter also introduces concepts such as randomised clinical trials and placebos, and the need to review systematically all the relevant evidence. In Chapter 4 we describe some of the numerous uncertainties that pervade almost every aspect of healthcare, and how to tackle them. Chapter 5 contrasts the key differences between good, bad, and unnecessary research into the effects of treatments. In Chapter 6 we point out how much of the research that is done is distorted by commercial and academic priorities and fails to address issues that are likely to make a real difference to the well-being of patients. Chapter 7 maps what patients could do to ensure better testing of treatments. And in Chapter 8 we present our blueprint for a revolution in testing treatments – practical measures that could be started now to bring this about.

Each chapter is referenced with a key selection of source material; other sources of information are included in the Additional Resources section at the end of the book. For those who wish to explore issues in more detail, a good starting point is the James Lind Library at www.jameslindlibrary. org.

This site also hosts an e-mail contact point for readers of Testing Treatments – do send us your comments to

Although we describe the harm that some inadequately tested treatments have caused, it is certainly not our intention to undermine patients’ trust in their health professionals. Our aim is to improve communication and boost confidence. But this will only happen if patients can help their doctors critically assess treatment options. We hope that you, the reader, will emerge from this book sharing some of our passion for the subject and go on to ask awkward questions about treatments, identify gaps in medical knowledge, and get involved in research to find answers for the benefit of everybody.

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