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Research, Clinical Trials and You 

“Patients are the benefactors of medical research. Yet, in the past, patients were regarded solely as passive objects in research-based development of new treatments and medicines. Research happened ‘for’ or ‘to’ patients, who played no active part in the research process.  

In recent years, however, there has been a shift in research practices. Patient involvement in research, also known as Patient and Public Involvement (PPI), is increasingly recognised as a valuable part of healthcare research. Rather than ‘for’ or ‘to’, research is now ‘with’ or ‘by’ the patient.” 

Hørder & Nielsen (2020).  

Patients as partners in research – The challenges for researchers of patient involvement 

But before we explore involvement – what are clinical trials? 

To help make decisions about treatments and care, we need evidence about what works best.  

Clinical trials are often used to find out if new treatments and techniques are safer and more effective than the ones that already exist or to discover if existing treatments can be used in different ways. 

Traditionally, treatments were developed and tested by researchers/scientists.  

According to Hørder & Nielsen (2020) most scientific, medical and clinical research follows a well-established path”. This traditional research process – or ‘scientific method’ – usually includes 6 steps: 

  • asking a question about something, 
  • doing background research to learn what is already known about the topic,  
  • constructing a hypothesis, or deciding on an outcome to be measured, 
  • experimenting to test the hypothesis, or prove/disprove a predicted outcome 
  • analysing the data from the experiment and drawing conclusions from the results 
  • communicating the results to others. 

However, if different studies looking at treatments, for the same condition, use different measures or outcomes to test and/or assess results it can be difficult to compare or combine their results. 

For example – if Study A only measures length of stay in hospital for a procedure, and Study B only measures complication rates associated with that procedure – we cannot compare or combine their results because they have used different outcomes. 

Most current clinical trials (in cancer) use similar primary endpoints or outcomes, for example, overall survival (OS). They may also include secondary outcomes, such as Progression Free Survival (PFS) and/or Health-related Quality of Life (HRQoL) scoring. 

Clinical trials – What are they and what to expect?  is talked about in our explainer video here 

But for these outcomes to be relevant, and meaningful, to patients, carers, health professionals, policy makers and the wider general public – there is a need for them to be clearly explained and agreed, as to relevance and importance. HRQoL is more than lack of side-effects or tolerability of treatment: it is about promoting, maintaining, sustaining independence, cognitive ability and being able to live as well as possible – “to be able to look after myself in every respect – not treatment ‘at any cost’” 

Unfortunately, patients and carers have not always been involved in trial design and outcome setting, however, that is slowly changing – and more frequently they are now being included: recognising the importance of their lived experience of a condition. 

. . .the patient’s perspective can inform the research goals. When researchers are open to this, the view of the patient can steer the direction of the research, reveal overlooked issues, influence study design – and, crucially, ensure that the outcome is patient-centred.” 

Hørder & Nielsen (2020). 

What is patient and public involvement – when it comes to research and clinical trials? – and why is it so important? 

What is Patient and Public Involvement in healthcare? 

It means that patients or other people with relevant experience contribute to how research is designed, conducted, and disseminated. 

NHS HRA 

The experience of the NIHR (National Institute for Health and Care Research) is that patients and the public are interested in being part of and getting involved with health and social care research for a variety of reasons.  Most of all, not only are they hopeful of benefiting from more treatments but they also want to help others who are experiencing the same condition as them. 

“The general public are broadly positive about health and social care research that happens in the UK – two-thirds are confident it improves people’s health and wellbeing (67%). Additionally, three-quarters think that, if they took part, they would be treated with dignity and respect (75%). This is also reinforced by high levels of willingness to participate, with just over two-thirds of UK adults saying they would take part if asked (68%)”. 

NHS HRA 2024 

The NHS Health Research Authority have found that: “Research teams which involve patients and the public run better studies because: 

  • they are more relevant to participants 
  • they are designed in a way which is acceptable to participants 
  • they have participant information which is understandable to participants 
  • they provide a better experience of research 
  • they have better communication of results to participants at the end of the study.” 

They include 4 key principles for meaningful involvement of patients and the public in health and social care research: 

Principle 1 : Involve the right people 

Principle 2 : Involve enough people 

Principle 3: Involve those people enough 

Principle 4 : Describe how it helps 

More details about the NHS HRA is available here  

However, according to an article published by Oxford NHS Foundation Trust in 2023:  

“Nearly 80% of clinical trials fail to recruit patients within their agreed-upon timelines, with 19% terminated given unsuccessful participant enrolment.” 

INCA’s SCAN report, also published in 2023, co-authored by our own CEO Catherine Bouvier, found that  

“Only 17% (403/2359) of patients had participated in ‘NET’ trials,  

while … 67% wanted to participate in trials if eligible.”  

Awareness of trials was found to be relatively low in both healthcare professional and patient groups (28% and 16% respectively): and  

Patient understanding as to the purpose of clinical trials varied – 

from poor/fair (22%) to very good/excellent (39%) , with 15% reportedly uncertain” 

So, what can we do to address some of these challenges? 

Hear from Kate about this topic – click here to play 

To be able to make informed choices based on individual preferences, about treatment and/or trial involvement, people need to be adequately informed about their options and involved in setting their own potential outcomes: “what matters most to me”. 

Patient-centered care implies that patients, their values, preferences, and individual life and health goals are at the heart of care processes and that patients are involved in care decisions  

(Person-Centered Care: A Definition and Essential Elements. J Am Geriatr Soc. 2016; 64:15–8.) 

To facilitate this goal, studies must examine the effects of care, based on parameters that matter to patients and thereby enable them to make an informed decision. 

(Kersting et al. (2022) Patients’ perspective on supposedly patient-relevant process and outcome parameters:  

a cross-sectional survey within the ‘PRO patients’ study’. BMC Health Serv Res 22, 72) 

European Patients’ Academy on Therapeutic Innovation (EUPATI) is a great example of promoting PPI. Their vision “is to improve health outcomes through the contribution of patients and patient representatives as valued stakeholders.” 

The EUPATI Patient Involvement in Medicines R&D Roadmap (11 minute) Video can be watched here 

Hørder and Nielsen’s review found that, “according to many researchers, the potential of PPI varies according to the type of study. While it may be more easily incorporated into clinical trials, it may be seen as less relevant to basic research (which seeks to expand knowledge, rather than lead directly to a new treatment, for example).” 

Interestingly, they also found that most researchers expressed a view that they are themselves changed by the process. For example, working closely with patients can force researchers to discuss their work in more accessible language, avoiding scientific jargon. As a result, researchers improve their ability to share their work with a wider audience. 

“It is clear that there is no ‘one-size-fits all’ approach to incorporating PPI in a project.  

However, there are a few common factors that are important in successful PPI – and institutions should ideally have the proper support in place to allow researchers to address these points.” 

As the studies referenced to above have concluded, and Kate has so eloquently said, there is a need for greater involvement – and for aims and plans to come off the page and be put into action. 

All agree that: 

Patients should be involved at an early stage in the research process.  

This allows patients to understand and influence the aims of the project at the planning stage.  

It also encourages them to participate in discussions and enables them to have a sense of ownership over the work. . . and for researchers . . .the patient’s perspective can inform the research goals. 

The ‘lived experience’ is a vital and rich source of information: researchers should ensure that patients understand how and why their contribution is valuable. They have life skills and knowledge that don’t disappear on diagnosis – and real experience of that diagnosis and its impact.  

If addressed properly, these factors will strengthen both the research and the collaboration between patient and researcher. 

Training and education around research and PPI – beneficial to both researchers and patients:  

For the patient, this could cover the fundamental ideas of the research process: that research builds up knowledge that is based on data, how it takes place is based on certain principles and theoretical assumptions, for example.  

For the researcher, training in communication – which could cover everything from listening to the lived experience and ‘what matters most’ – to discussing methods and results, in a way that is meaningful to all involved.  

Researchers and patients could even participate in training together, which would help to build their relationship – their partnership. It can also help clarify roles and responsibilities. 

Institutions that ‘run’ research should offer practical support for PPI.  

Apart from training, this could involve extra financial support – in meeting patients’ transport costs, for example. Providing facilities to host meetings and ensure there are mechanisms for ongoing feedback and collaboration throughout the research process.  

Institutions could also take responsibility for ensuring diversity in PPI: in the ethnic, cultural, and educational backgrounds of participants. 

Put simply: 

“The researcher does not know what she does not know until she has asked the patient.”  

Kristina Staley 

Quoted in Hørder & Nielsen (2020). 

Further resources and information: 

Ancora AI : serves as a bridge connecting cancer patients with cutting-edge clinical trials, empowering them to make informed decisions about their treatment options. Our podcast series (mentioned below – features Ancora AI’s founder talking about ‘humanising’ the clinical trial process, ensuring patients can access personalised trial options that cater to their unique needs. Season 2 Episode 3 

COMET stands for the ‘Core Outcome Measures in Effectiveness Trials’ Initiative. It recognises the expertise and crucial contribution of patients and carers in developing core outcome sets and research more generally – and is focused on developing resources to encourage involvement. It is funded by the UK Medical Research Council and the European Union and has links with researchers, and patient representatives, across the world: www.comet-initiative.org  

EUPATI The European Patients’ Academy on Therapeutic Innovation  

Institute of Cancer Research : Clinical trials in cancer Barriers in access to clinical trials, especially in light of the Covid-19 pandemic (2021) PDF   

National Institute of Healthcare Research (2024) Barriers and best practices to improving clinical trials transparency at UK public research institutions: A qualitative interview study 

National Institute of Healthcare Research: Be Part of Research – https://bepartofresearch.nihr.ac.uk/  

National Library of Medicine (US) Clinical trials database: https://clinicaltrials.gov/  

Neuroendocrine Cancer UK Advancing Neuroendocrine Cancer Research Campaign 

Not Just NE Cancer Takeover Series: Season 2: This takeover series dives deep into the world of research, exploring its significance, methodologies, and impact. Through interviews with researchers and esteemed organisations, each podcast episode offers valuable insights into the realm of neuroendocrine cancer research. 

RareCan – an organisation in the UK that specialises in finding clinical trials for patients with rare and less common forms of cancer. Also features in our podcast series – see link above – Season 2 Episode 2.