Karen Lowton, PhD
Senior Lecturer
Ageing & Health
School of Social Science and Public Policy
King’s College
London, UK

People with cystic fibrosis (PWCF) are often asked to take part in clinical research projects. These projects aim to understand and improve the experiences related to the condition, the treatments taken and the care received. This article considers the reasons why PWCF choose, or refuse, to take part in such research.

What sort of clinical research is carried out?

CF research studies include trials of new drugs or new ways of giving established treatments. They also focus on psychosocial research, which aims to understand more about how people experience different aspects of living with the condition. While some studies involve a questionnaire or trying a particular medicine in a blind study, some studies involve more than one method, and so researchers may ask participants to undertake one or more tasks. However, PWCF need to remember that they can change their minds and withdraw from the study even after agreeing to participate. It is also vital for individuals to remember that saying no to research will not affect their usual or planned future treatment and care. The range of tasks that people may be asked to do is wide, and may include:

A randomised controlled trial

This involves one drug, or mode of treatment, being tested against an established drug or treatment. Participants typically complete a variety of measures such as questionnaires, scales, and physical measures over a period of time. The trial is “blind,” meaning that participants and investigators do not know whether participants are in the ‘new treatment’ or ‘comparison’ group. This method provides the most objective results. Obviously this method is not possible for some trials, such as those investigating whether it is better to receive intravenous antibiotics at home or in hospital.

Observation

This method involves looking at what people do, either by a researcher observing, or using video recording. One recent example involved observing how families behave during the mealtimes of young children with CF (1).

A personal interview

Some projects aim to capture individual’s reasoning and explanation for their beliefs, attitudes and behaviours. Typically, personal interviews take place at a location suitable to the respondent, and are recorded so that the researcher can accurately note what is being said.

Review of ethical issues

All research conducted with individuals and/or their families requires ethical approval. This is to safeguard the rights, safety, and wellbeing of all participants, and to ensure that the researchers are not being unduly influenced by their funders or sponsors. There are a variety of groups that perform ethical review, depending on where the research is located, and the type of research undertaken. In the UK, the Central Office for Research Ethics Committees (COREC; www.corec.org.uk) oversees approval mechanisms for all research involving patients using National Health Services. Virtually all British universities have committees to oversee research undertaken by their students and members of staff. In North America, Institutional Review Boards perform ethical review.

All clinical research studies keep patient data confidential (for example when physical measures are made), or in an anonymised form (for example when parts of people’s interviews are quoted in reports). This ensures that anyone reading the research report is unable to connect individuals who took part in the project with specific responses. Researchers on every project will inform potential participants about how they plan to keep their data private as well as the length of time the data will be kept.

Why do people generally take part in research?

Much work has been done to discover why people with chronic illnesses choose to take part in some clinical research projects, but not others. For example, an opportunity to improve personal health through access to new treatments or experts has motivated people with vascular disease (2), acute cardiac conditions (3), and cancer (4). Finding out more about individuals' reasons for taking part in research enables researchers to design studies that will be of greater benefit to the patient community, and remove unnecessary barriers to participation that may exist whilst protecting those who may be vulnerable. Some reasons for participation appear the same regardless of the disease or condition people have. Many people believe that taking part in clinical research allows them access to new treatments or modes of care that they would not normally be able to have. In addition, it may enable them to be treated by experts or specialists in that disease, and be given extra time in their appointments. Reasons people give for refusing are commonly the burden of the research, and the financial or personal costs of travel, childcare, time away from work or other responsibilities.

Why do people with CF take part in research?

Like people with other chronic conditions, the reasons why people with CF choose or refuse to take part in research are varied. A group of 31 people between 18-30 years of age, attending a specialist CF centre in the UK took part in personal interviews and shared their motivations for, and experiences of, clinical research participation. Everyone confirmed that they had been asked to participate in at least one research project other than the current one. These projects included giving blood or undergoing scans for physiological research, allowing researchers to take nasal samples using a probe, taking new drugs, such as Pulmozyme ® , or completing health questionnaires for psychosocial projects. However, many patients were unclear about the precise methods and/or aim of the research that they had previously participated in. Although the majority of clinical research that patients spoke about did not involve taking new substances, there were other tasks (e.g. undergoing scans or blood tests) involved that were not part of their routine medical care.

Trust in the CF Centre

Men and women used a variety of sources to gain information about current and developing treatments, and they appeared satisfied with what they learned. These sources included the CF Trust, other patients, Centre healthcare staff, and the Internet. In thinking of their CF Centre on a general basis, patients reported prompt access to clinical staff and treatment, and believed that staff would never put them in a position where their health or wellbeing was at risk. Moreover, participants trusted that the staff would ensure their wellbeing in research participation, believing that staff knew the best current treatment and care for individuals and were committed towards working towards new therapies. However, some reported having lost trust in a particular healthcare professional. (Additionally, some commented that they were less likely to agree to participate if the research tasks that needed to be completed were not attractive.

Thinking about the risks and benefits of participation

PWCF felt the “risk” involved in participation depended on the type of research and the their state of health at the point the request was made. Individuals calculated risk more carefully when they were asked to participate in drug development or gene therapy research, than, for instance, when they were asked to have blood drawn. However, participation in drug development projects was viewed as ‘necessary’ or ‘unnecessary’ depending on the individual’s current health state and the efficacy of their routine treatment to maintain it, with people in relatively good health wishing to decline drug development research until their current health, or lack of treatment efficacy, suggested that a new drug may bring benefits.

No one expressed that they ought to participate in research to ensure their good care in the future, or to be seen as a ‘good patient’. However, one man, aged 40, who at the time of his interview was considering assessment for lung transplant, stated that he believed that his taking part in research at the centre was mandatory; this never being the case.

Social Constraints

Individuals thought that consideration of the potential effects of research on their social life was essential when being asked to participate in clinical research. The social concerns most commonly identified were obligations to families and employers. Although some research tasks were of a short duration, for example those carried out within an outpatient visit, other projects took months for volunteers to complete and often involved frequent visits to the hospital. The constraint imposed by employment meant that people did not feel able to take time away from their job, especially if they were ‘saving’ that time for anticipated future periods of acute illness. Therefore, many working individuals felt that, although their employment stopped them from being involved in longer-term research projects, those that could be fitted in to a day already earmarked for an outpatient appointment were acceptable.

The need to involve partners and/or the wider family in decisions to participate in clinical research was a point made by many patients, especially those who had or were contemplating having children. Those who were trying for a child or considering undergoing fertility treatment frequently decided against participation in research at that time due to the unknown risks it could present to the foetus; in this instance social and health risks were considered together.

Health Constraints

The knowledge of already having outlived the predicted survival age for CF patients as a group and the likelihood of a declining health state with increasing age were significant constraints for participants in their late twenties or thirties. This group tended to say that now they were older they were ‘more careful’, only participating in research that they thought would not put their current health at risk, or would give them an immediate health gain. They were less likely, therefore, to participate in drug trials, preferring to participate in psychosocial research. The thought that something might ‘go wrong’ through their participation in research projects and lead to damaging currently good, carefully controlled health was enough to stop many from participating.

PWCF who had been asked to participate in trials that were testing new drugs such as Pulmozyme®, licensed for use in CF in 1994, appeared to use a carefully calculated approach to decision-making and risk management, as did those who had been approached to take part in gene therapy research. Here the risks to health were calculated much more frequently than social risks, although it was acknowledged that social benefits, for example being able to play sports with children or being able to work, would arise from any health benefits that arose.

Perhaps peculiarly to this patient group, some individuals, whose health could not be maintained by their current routine treatment, reported initiating contact with those at the Centre who were conducting the trial to ask if they could participate, sometimes asking more than once. This again was particularly true of the Pulmozyme® trial. One respondent stated that he managed to be recruited onto the trial just months before he had his heart-lung transplant.

A wish to ‘give back’ to others

Seventeen individuals explicitly stated that their motivation for participation in some research projects was to ‘help others’. More specifically, an additional seven stated that they wished to ‘give back’ to others as a form of repayment for the treatment and care that they themselves had received.

Although a willingness to ‘give back’ was evident, risks to the self were carefully considered and, especially in the context of gene therapy, often outweighed thoughts of benefit to others. During their calculation of personal risk, participants stated that they were conscious of the possible benefits to others that would arise from their participation in such trials. However, in this decision-making, the potential benefits to others were perceived as secondary to the risks to the individual, and a bonus if they did decide to participate. In some cases, the potential cost to the self outweighed any possible benefit to others, making the denying of possible benefit to fellow PWCFa difficult decision to reach. Two individuals who had refused to take part in drug development expressed feelings of selfishness and guilt.

People with CF closely defined the ‘others’ that might benefit from their participation in hospital research. The specialist centre was a recipient named by the majority of patients, and in these instances the patient was ‘giving back’ to the hospital and staff for the care already received. Other beneficiaries of ‘giving back’ were named as fellow patients, usually perceived as being in worse health or ‘unluckier’ than themselves, who may benefit from the research results, and past patients (those now dead) who had taken part in trials for drugs from which the patient was currently benefiting, for which participants paid their dues in recognition of what these people had effectively done for them. Future patients (children and those not yet born with CF) were also identified as those who would benefit from the treatments of the future, which the patients of today were helping to develop through their participation.

In summary, there are many motivations for choosing or refusing research participation. It is important for individuals to remember that no project is mandatory, further information about the study will be available from CF staff or researchers, and that withdrawal is possible throughout the project tasks.

1. Powers SW, Mitchell MJ, Patton SR, Byars KC, Jelalian E, Mulvihill MM, Hovell, MF, & Stark LJ. Mealtime behaviors in families of infants and toddlers with cystic fibrosis. Journal of Cystic Fibrosis, 2005, 4(3), 175-182.

2. Wilcox M & Schroer S. The perspective of patients with vascular disease on participation in clinical trials. Journal of Vascular Nursing, 1994, 12, 112-116.

3 Gammelgaard A, Rossel P, Mortensen OS, in collaboration with the DANAMI-2 investigators. Patients’ perceptions of informed consent in acute myocardial infarction research: a Danish study. Social Science and Medicine, 2004, 58, 2313-2324.

4. Madsen SM, Mirza MR, Holm S, Hilsted KL, Kampmann K, & Riis P. Attitudes towards clinical research amongst participants and nonparticipants. Journal of Internal Medicine, 2002, 251, 156-168.

This article is taken from Dr Lowton’s paper “Trials and tribulations: Understanding motivations for clinical research participation amongst adults with cystic fibrosis,” published in Social Science and Medicine, 2005, 61, 1854-1865. Social Science & Medicine provides an international and interdisciplinary forum for the dissemination of research findings, reviews and theory in all areas of common interest to social scientists, health practitioners and policy makers.