The Academy of Medical Sciences published a working group report examining the sources of evidence for assessing the safety, efficacy and effectiveness of medicines.
Sources of evidence for assessing the safety, efficacy and effectiveness of medicines
The Academy's report on the ‘Sources of evidence for assessing the safety, efficacy and effectiveness of medicines’ explores the strengths and limitations of different methods of assessing evidence and describes evolving approaches that are being, or have been, developed to address some of the major limitations of current methodologies.
The full report is available to download on the right hand side of this page.
This project is part of a wider workstream looking at enhancing the use of scientific evidence to judge the potential benefits and harms of medicines.
'Evaluating evidence in health' evidence-gathering workshop
As part of this sub-project, the Academy held an evidence-gathering workshop on ‘Evaluating evidence in health’ jointly with the Wellcome Trust on 21 October 2015. This one-day meeting brought together over 60 delegates from across the healthcare landscape to:
- Examine the strengths and limitations of evidence from different sources for determining risks and benefits of medicinal products.
- Explore how weaknesses in current approaches might be addressed, including discussion around evolving and novel trial designs, and methods of data collection, analysis and meta-analysis.
- Consider the future sources of data and how they might be used as evidence.
- Discuss how evidence can be effectively communicated to stakeholders, including patients, citizens, healthcare professionals and the media.
- Generate practical suggestions for enabling the better use of research evidence in healthcare decisions.
A report of this workshop is available to download on the right hand side of this page. This report does not represent a formal Academy of Medical Sciences position on the evaluation of evidence in health, but instead serves to reflect the wide-ranging discussions that took place at the workshop.
Financial contributions towards the project
Arthritis Research UK, the British Heart Foundation (through a Strategic Funding Award), the British Pharmacological Society, the British Society for Immunology, the Medical Research Council, the Naji Foundation and the National Institute of Health Research Health Technology Assessment Programme kindly provided financial contributions towards this work. Funding from a core grant from the Department for Business, Energy and Industrial Strategy to the Academy was also used to support this project. This was an independent project that had the support of the Chief Medical Officer. Funders did not approve the conclusions and recommendations and were not sent a draft of the report for approval.
For information on questions and queries that were asked at the beginning of this project, please click here.
As part of the workstream on ‘Enhancing the use of scientific evidence to judge the potential benefits and harms of medicines’, the sub-group:
- Explored the strengths and limitations of results and conclusions that originate from different study types or data sources to answer a range of research questions as well as to evaluate the risks and benefits of medicinal products.
- Started to consider the implications of its findings for the communication of evidence, including the availability of the evidence around the risks and benefits of medicinal products. Initial concepts were further explored within the other elements of the overall workstream.
This sub-group study did:
- Aim to develop a list of broadly applicable principles relating to the presentation, interpretation and weighting of evidence to enable a range of stakeholders (including patients, the public, healthcare professionals, and the media) to better consider the risks and benefits of medicinal products.
- Draw on case studies and examples to illustrate these principles; for instance, the use of the cholesterol-lowering drug statin, drugs to treat childhood leukaemia, hormone replacement therapy, the early treatment of psychosis, and vaccination.
It did not:
- Seek to address all areas of contention, nor to replicate the work performed by the Medicines and Healthcare products Regulatory Agency and the National Institute of Health and Care Excellence.
- Consider case studies that did not relate to medicinal products. Surgical interventions, medical devices, screening procedures, and so on, were beyond the scope of this study.
- Consider how evidence is used to inform the cost-effectiveness of medicinal products.
Professor Sir Michael Rutter CBE FRS FBA (Chair of the Working Group from May 2015 to November 2016) is Professor of Developmental Psychopathology at the Social Genetic and Developmental Psychiatry (SGDP) Research Centre at the Institute of Psychiatry, Psychology and Neuroscience, King’s College London. His research interests include the use of natural experiments and animal models to test hypotheses about causation; the use of epidemiological longitudinal studies for the same purpose; gene-environment interplay; and studies of psychosocial risk. He founded the SGDP in 1994 and was its first honorary director. He retired from his administrative posts in 1998 but remains active in research and teaching. His textbook on child and adolescent psychiatry remains distinctive in attention to both conceptual and statistical issues, as well as the integration of science and clinical work. He is Governor of the Coram foundation and Chair of the Scientific Advisory Group for the Canadian Institute for Advanced Research programme on Child and Brain Development.
Sir Alasdair Breckenridge CBE FRSE FMedSci was Chairman of the MHRA for 10 years. Previously, he was Chairman of the Committee on Safety of Medicines and Professor of Clinical Pharmacology at the University of Liverpool. He currently chairs the Advisory Scientific Committee of the Centre of Regulatory Excellence for the Government of Singapore and was a Non-Executive Director of University College London Hospitals from 2012 to 2015.
Professor Nancy Cartwright FBA is a methodologist and philosopher of the natural and social sciences specialising in issues of causal inference, objectivity and the nature of evidence in the natural and social sciences and for social policy. She currently holds joint appointments at Durham University and the University of California, San Diego and has worked previously at Stanford University and the London School of Economics and Political Science. She is a fellow of the British Academy, the American Academy of Arts and Sciences, the German National Academy of Science (Leopoldina) and the American Philosophical Society, and is a recipient of a MacArthur fellowship. She has also been President of the American Philosophical Association (Pacific Division) and of the Philosophy of Science Association. She has been awarded a number of grants for her work, including from: A. K. Knight; the Arts and Humanities Research Council; the British Academy; the European Research Council; the John Templeton Foundation; the Latsis Foundation; the London School of Economics and Political Sciences Seed Fund; the Durham University Seedcorn Award; the Spencer Foundation; and the US National Science Foundation.
Professor Dame Nicky Cullum DBE FMedSci is Professor of Nursing and Head of the School Division of Nursing, Midwifery and Social Work in the School of Health Sciences at the University of Manchester. Her research mainly focuses on the epidemiology and management of complex wounds such as leg, foot and pressure ulcers and non-healing surgical wounds. She was a founding member of the Cochrane Collaboration and has been Coordinating Editor of the Cochrane Wounds Group since 1995. A particular interest is how research evidence of relevance to clinical nursing decisions is produced and that evidence is translated into practice. She founded the Centre for Evidence-Based Nursing at the University of York in 1995. She is a member of the Royal College of Nursing and a Fellow of the American Academy of Nursing. She has a number of current grants, either as a principal or co-applicant, from the NIHR.
Mr Edward Green is a patient representative on the Great Ormond Street Hospital Members’ Council (Patient and Carer Constituency), where he strives to improve the overall patient experience by listening to patients and families and holding the executive to account. He has helped drive the implementation of new systems for patients and clinicians to communicate and work collaboratively throughout the period of care across the NHS. He is also a Business Consultant at Block Solutions, a technology focused consultancy.
Professor Deborah Lawlor FMedSci is Professor of Epidemiology and Deputy Director of the MRC Integrative Epidemiology Unit at the University of Bristol. Her research is concerned with women’s reproductive health, and how their pregnancy experiences and characteristics relate to their own, their offspring’s, and their grandchildren’s future cardiometabolic health. She has developed and applied a range of novel methods for obtaining best estimates of causal effects in population science. She is currently the Chair of the MRC Population Health Sciences Group and a member of the MRC Strategy Board, and has served on various boards and panels for the MRC, Wellcome Trust and British Heart Foundation. During the working group project, she also served on the Academy of Medical Sciences’ Council. She has received a number of grants, either as a principal or co-applicant, from: Alcohol Research UK; Australian Government National Health and Medical Research Council; British Heart Foundation; Economic and Social Research Council; European Research Council; Medical Research Council; Medtronic External Research Programme; NIHR; the UK Clinical Research Collaboration; the US NIH; the US National Institute on Aging; and Wellcome Trust. She has recently received industry support for her biomarker research from Medtronic and Roche Diagnostics.
Professor Mahesh Parmar is Professor of Medical Statistics and Epidemiology, and Director of both the MRC Clinical Trials Unit and the Institute of Clinical Trials and Methodology at University College London (UCL). Until 2014 he was an Associate Director of the National Cancer Research Network since its inception in 2001, an organisation which has more than tripled the number of f patients recruited to cancer studies in England. The Unit he directs at UCL aims to deliver swifter and more effective translation of scientific research into patient benefits, particularly in infectious diseases and cancer, by carrying out challenging and innovative studies, and by developing and implementing methodological advances in study design, conduct and analysis. The Unit receives educational grants and free or reduced priced drugs for many of the trials that it runs, including from: Abbott, Amgen, Astellas, AstraZeneca, Bayer, Baxter, Boehringer Ingelheim, Bristol-Myers Squibb, Cipla, Gilead Sciences, GlaxoSmithKline, Janssen, Janssen-Cilag, Lilly, Merck, Merck Serono, Novartis, Roche, Sanofi-Aventis, Sanofi Pasteur, Tibotec, Virco, World Health Organization (WHO)/Global drug facility (GDF). Professor Parmar has received a number of grants, either as a principal or co-applicant from: Cancer Research UK; Department of Health; European Science Foundation; MRC; and NIHR.
Professor Simon Thompson FMedSci is Director of Research in Biostatistics in the Department of Public Health and Primary Care, University of Cambridge. From 2000–2011 he was Director of the MRC Biostatistics Unit in Cambridge, an internationally acclaimed research institute in medical statistics. He held previous academic appointments at the London School of Hygiene and Tropical Medicine, and as the first Professor of Medical Statistics and Epidemiology at Imperial College London. His research interests are in meta-analysis and evidence synthesis, clinical trial methodology, health economic evaluation, and cardiovascular epidemiology; he has published widely in these areas. He has collaborated on a number of major clinical trials, including all the major recent UK national trials of screening and treatment for abdominal aortic aneurysms. He has been a strong advocate for biostatistics through his research papers, didactic articles, and contributions to courses and workshops both in the UK and abroad. He has also taken on a number of responsibilities for the MRC, the Royal Statistical Society, and other international professional societies. Professor Thompson has received a number of grants, either as a principal or co-applicant, from the MRC, British Heart Foundation, European Research Council, and NIHR.
Dr Julian Treadwell is a GP based in Wiltshire and an NIHR In-Practice Fellow at the Nuffield Department of Primary Care Health Sciences. He is Vice-Chair of the Royal College of General Practitioners (RCGP) Standing Group on Overdiagnosis and a member of the editorial board of the Drug and Therapeutics Bulletin. He has an interest in evidence informed prescribing and shared decision-making. He is a member of the RCGP and of the British Medical Association.
Professor David Webb FRSE FMedSci is the Christison Professor of Therapeutics and Clinical Pharmacology at the University of Edinburgh and leads a European Society of Hypertension (ESH) Centre of Excellence in Edinburgh. His research focuses on renal and vascular aspects of hypertension, and he runs a Scottish translational medicine and therapeutics clinical PhD programme funded by the Wellcome Trust. He is a Non-Executive Director of the MHRA and Chair of the Scientific Advisory Committee for the National Institute for Biological Standards and Control (NIBSC). He is President of the British Pharmacological Society (BPS), taking over Presidency in 2016. He is also Honorary President of the European Association for Clinical Pharmacology & Therapeutics (EACPT) and Clinical Vice-President of the International Union of Basic and Clinical Pharmacology (IUPHAR). His research is supported by grants from the British Heart Foundation, Kidney Research UK, the MRC and the Wellcome Trust.
Dr Stephen Webster is Director of the Science Communication Unit at Imperial College, which is responsible for training science graduates wishing to become media professionals, policy advisors and public engagement specialists. His academic background is in zoology and the philosophy of biology. Dr Webster was for many years a school science teacher in London. For six years he was Chair of the Wellcome Trust’s public engagement award scheme, the Society Awards. He has broadcast widely for BBC radio, including dramas and several documentaries on the nature of science. Dr Webster’s books include a biography of Charles Darwin and an edited collection of essays on the role of silence in science communication.
This Working Group was initiated in part as a response to the public debate over the risks and benefits of statins. Because of Amgen’s involvement in the development of cholesterol-lowering treatments, it was decided that the participation of Christine Fletcher (Executive Director of Biostatistics and a Regional Head in Global Biostatistical Science at Amgen) in the Working Group could be viewed as a conflict of interest. Christine Fletcher therefore stepped down from the Working Group. She did not participate in the project after 21 December 2015 and was not involved in discussions about emerging conclusions and recommendations. The Academy is grateful for her contributions to the first two Working Group meetings.
Representatives from the Medicines and Healthcare products Regulatory Agency (MHRA) and the National Institute of Health and Care Excellence (NICE) were invited to join the discussions as observers to clarify factual points. They did not input or have sight of the study’s conclusions and recommendations. The observers were:
Dr Sarah Branch, Deputy Director - Vigilance and Risk Management of Medicines Division, MHRA
Dr Ian Hudson, Chief Executive, MHRA
Professor Andrew Stevens, Chairman of a NICE Appraisal Committee, Professor of Public Health and former Head of Department and Division of Primary Care, Public and Occupational Health at the University of Birmingham
Head of Policy (currently on maternity leave)
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