House of Commons - Clinical Trials - Science and Technology Committee

Clinical Trials - Science and Technology Committee Contents

1 Introduction

1. The UK has long been a world leader in the field of medical research. Since the birth of the pharmaceutical industry over a century ago, only the USA has discovered and developed more drugs, and British researchers have played a central role in many of the breakthroughs that have revolutionised both scientific understanding and standards of patient care in the past 100 years.[1] More recently, this strong heritage has underpinned the development of a life sciences industry described by the Government as "one of the most successful globally" today.[2] The UK's 4,500 pharmaceutical, medical technology and medical biotechnology firms together employ over 165,000 staff, generate annual turnover of over £50 billion and spend nearly £5 billion on research and development each year.[3] It is perhaps therefore unsurprising that the Government has stated its desire to make the UK "the location of choice" for the £700 billion global life sciences industry, ensuring that "life sciences will continue to be vibrant in the UK and a key contributor to sustained economic growth and health improvements".[4]

2. Clinical trials, which test the safety and efficacy[5] of medical treatments, provide the body of scientific evidence on which this industry is built. Human medicines cannot be sold without permission from a licensing authority and permission will not usually be granted unless a clinical trial has demonstrated the medicine's success in treating the condition for which it will be marketed.[6] Clinical trials, in addition to generating valuable scientific evidence, also provide patients with an important way of accessing products that have not yet reached the market, offering hope to those for whom existing treatments have failed.[7] The necessity for a clinical trial to be performed before a medicine can gain regulatory approval means that trials are also big business. The global market for outsourced trial services[8] alone is expected to be worth £19 billion a year by 2015 and the clinical trials market as a whole has been estimated at £29 billion.[9]

3. Historically, the UK captured a large slice of the market for clinical trials, enjoying in 2000 the third largest share of global trials, behind only the US and Germany.[10] Between 2000 and 2006, however, the UK's ranking dropped to ninth and Britain's global share of patients in pharmaceutical trials fell sharply as trials moved to other jurisdictions.[11] Between 2007 and 2011, the total number of UK trials also decreased, putting jobs at risk and making it increasingly difficult for British patients to benefit from participation in cutting-edge medical research.[12] In the last two years, several efforts have been made to halt this decline. The Government's creation of the Health Research Authority in December 2011 was intended to streamline the process through which researchers could obtain permission to conduct a UK trial, making it easier to get trials up and running.[13] In addition, the Government has stated its hope that the planned revision of the 2001 EU Clinical Trials Directive—the key piece of legislation regulating trials across the EU—would make it easier to conduct a trial in the UK.[14]

4. There are also long-standing concerns that, wherever in the world they take place, the methods and results of many trials currently remain hidden from public view. This lack of transparency, say campaigners, undermines public trust, breaks the ethical pact between scientists and those participating in trials and leads to clinical decisions being made on the basis of incomplete evidence, potentially leading to poorer outcomes for patients.[15] The topic of clinical trial transparency received renewed attention in late 2012 with the publication of Bad Pharma, a book by Dr Ben Goldacre that accused the pharmaceutical industry of deliberately suppressing unfavourable trial results from public view.[16]

5. In December 2012, we issued a call for written evidence addressing the following questions:

a) Do the European Commission's proposed revisions to the Clinical Trials Directive address the main barriers to conducting clinical trials in the UK and EU?

b) What is the role of the Health Research Authority (HRA) in relation to clinical trials and how effective has it been to date?

c) What evidence is there that pharmaceutical companies withhold clinical trial data and what impact does this have on public health?

d) How could the occurrence and results of clinical trials be made more open to scrutiny? Who should be responsible?

e) Can lessons about transparency and disclosure of clinical data be learned from other countries?[17]

6. During the inquiry, we received written submissions from 62 organisations and individuals and took oral evidence from 21 witnesses, including:

Researchers, clinicians and others involved in the running of clinical trials in the UK;
Representatives of commercial and non-commercial funders of UK trials, including the pharmaceutical companies GlaxoSmithKline (GSK) and Roche;
UK regulators and governing bodies, including the Medicines and Healthcare Products Regulatory Agency (MHRA), the Health Research Authority (HRA) and the Department of Health's National Institute of Health Research (NIHR); and
The Government, represented by Lord Howe, Parliamentary Under-Secretary of State for Quality, Department of Health, (hereafter "the Minister") and David Willetts MP, Minister of State for Universities and Science, Department for Business, Innovation and Skills (hereafter, "the Minister for Universities and Science").[18]

We would like to thank those who provided written and oral evidence.

7. During our inquiry, several developments affected the regulatory landscape for clinical trials and the ongoing debate about trial transparency. This Report therefore considers these developments alongside the questions specified in the terms of reference. Chapter 2 defines the term "clinical trial" and outlines the current regulatory and governance requirements for those based in the UK. Chapter 3 considers the reasons for the recent decline in the UK's share of clinical trials, and offers suggestions for how this might be put right. Chapter 4 examines the subject of clinical trial transparency, considering the different levels of data that can be generated by and disclosed for a clinical trial, and evaluating recent developments designed to make trials more transparent. Chapter 4 also considers how emerging evidence from clinical trials can be better incorporated into clinical practice in order to improve medical outcomes. Chapter 5 sets out some final conclusions and recommendations.

2 Department for Business, Innovation and Skills, Office for Life Sciences, Strategy for UK Life Sciences, December 2011, p 4 Back

3 Department for Business, Innovation and Skills, Office for Life Sciences, Strategy for UK Life Sciences, December 2011, p 4 Back

4 "What we do", Office for Life Sciences, Gov.uk, accessed September 2013; Deloitte, 2013 Global life sciences outlook, p 1 (USD values converted approximately to GBP based on Oanda exchange rate on 29 August 2013) Back

5 Efficacy tests whether a treatment works under ideal conditions, while effectiveness tests whether a treatment works under real-world conditions. Generally, a treatment tested under trial conditions tests efficacy rather than real-world effectiveness, since trial conditions are usually to some extent artificial. Back

6 MHRA, Medicines and medical devices regulation: what you need to know, 2008, p 5; Council Directive 2001/83/EC Back

7 Q 92 [Professor Sikora] Back

8 Outsourced clinical trial services, performed by third parties known as contract research organisations (CROs), include activities such as trial planning and design, patient recruitment, bioanalysis and data analysis Back

9 "'The Clinical Trials Market Will Exceed $30BN by 2015' says visiongain Report", PR Newswire, 5 July 2011, prnewswire.co.uk, accessed September 2013; "Clinical trial basics: global clinical trial market", James Lind Institute, jliedu.com, accessed September 2013 (USD values converted approximately to GBP based on Oanda exchange rate on 29 August 2013) Back

10 Kinapse, Commercial clinical research in the UK: A Report for the Ministerial Industry Strategy Group (MISG) Clinical Research Working Group, November 2008, p 19 Back

11 Kinapse, Commercial clinical research in the UK: A Report for the Ministerial Industry Strategy Group (MISG) Clinical Research Working Group, November 2008, p 19; Ev 79 Back

12 Ev 53, para 4 Back

13 Ev 54, paras 10-12 Back