Patient and public involvement in clinical trials

Abstract

Is established worldwide, but encouragement is needed to promote institutional collaboration and avoid duplication of effort

Patient and public involvement in clinical trials has been defined as “experimenting with” as opposed to “experimenting on” patients.¹ It is founded on the belief that a collaborative approach to testing treatments is vital if the uncertainties that matter most to patients are to be reduced.² In 1994, the ethicist Raanon Gillon proposed that not only morality but also scientific interest should combine to urge a “brave new partnership between clinical trialists and patients.”³

It is difficult to be precise about the origin of patient and public involvement but several early examples exist. Rose Kushner—a pioneer of patient involvement in the United States in the 1970s—was a freelance writer who also had breast cancer. She wrote a book, which was based on a thorough review of evidence of the effects of radical mastectomy. Her influence and attitude was such that she eventually reviewed new research proposals for the US National Cancer Institute.⁴ Her achievements helped inspire the work of the US National Breast Cancer Coalition.

Another early example of well organised and influential involvement occurred in the 1980s in the United Kingdom. The Association for Maternity Services convened a meeting of interested voluntary organisations and patient groups to encourage them to support the Medical Research Council’s proposals for a randomised controlled trial of chorionic villus sampling in pregnancy. Representatives of these groups were involved in conducting and promoting this important trial.⁵ Another example is provided by well organised groups of people with AIDS—first in the US and then in the UK—who challenged researchers’ approaches to conducting trials, which had overlooked patients’ preferred outcomes.^{2 6}

In 1997, the first international conference on breast cancer advocacy, attended by people from 44 countries and six continents, took place in Brussels. It was led by the US National Breast Cancer Coalition and supported by organisations from Panama, Belgium, the UK, and Israel. This meeting helped shift the balance towards consumer participation, which was becoming a reality at that time.⁷ The Cochrane Collaboration (www.cochrane.org/), an international organisation that puts strong emphasis on consumer participation, also took part in the meeting.

After a UK government enquiry in March 1995, in which patient involvement in the whole research process had been advocated,⁸ the Health Select Committee report on breast cancer services devoted a section to “Involving patients in research.” On the basis of written and oral evidence, ministers recommended that patient involvement at all stages of a trial, including the initial design, is essential and that initiatives such as the Consumers Advisory Group for Clinical Trials should be welcomed.^{6 9} Ministers believed that their recommendations would help to improve the standard of care for women with breast cancer in the UK. They also hoped that “as other specialties follow the lead, they may help to raise the standard of care for all cancer patients.” Subsequently, the Standing Advisory Group on Consumer Involvement in the NHS R&D Programme was formed in April 1996 to advise the Central Research Development Committee on how to boost patient involvement in the UK NHS research and development programme. The group included representatives of consumer bodies, health professionals, managers, and information specialists.

Patient and public involvement now goes beyond clinical trials. For example, an appointed group of 30 well motivated and informed lay members, the Citizens Council of the National Institute for Health and Clinical Excellence, (www.nice.org.uk/page.aspx?o=citizenscouncil) contributes to decisions about the prioritisation of healthcare resources on the basis of evidence from clinical trials. They help to decide whether interventions should be approved or rejected, or whether treatments should be available only in randomised controlled trials and not in clinical practice. Within the James Lind Alliance, lay people form part of a support and information group that works with health professionals to prioritise research questions.¹⁰ In both of these examples, professional and lay members focus on improving research processes and seeking fair systems that consider the needs of patients.

Since the recognition and acceptance of patient and public involvement, and the rapid accumulation of evidence regarding its worth,¹¹ patient and public involvement has been implemented here in the UK and in Europe, the United States, Canada, and Australia. In the UK, the National Institute for Health Research (NIHR) is now established as part of the government’s strategy, “Best research for best health.” The NIHR wants patients and the public to be involved in all stages of research, and, together with its partners—the UK Clinical Research Collaboration and Involve—has put structures in place to achieve and facilitate this.

Healthy development of the partnership between patients and the medical profession will depend on firm policy directives that encourage institutional collaboration to avoid wastage of resources and duplication of effort. It will be important to record and understand the social and cultural history of patient and public involvement, compile comprehensive databases, and undertake ongoing reviews of the effect of public involvement if we are to make progress and maintain balance and equality within this new partnership.