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The active involvement of Patients and Patient organisations in (the) clinical trial (development process)

Whilst patients have always been involved as subjects in research, traditionally, patient organisations are rarely seen as possible partners in clinical research. The active involvement of patients or patient organisations in the clinical trial development process is thus relatively new (9, 14).

During the last couple of decades patients and their advocates have been moving away from their “passive” involvement of participating as a research subject and information provider, towards becoming more actively involved in the clinical trial development process. One of best known examples of patient involvement in clinical research is that of HIV Aids activists who challenged researchers in the early 90’s on their research design (15). More and more, patient organisations and other stakeholders have started to realize that what patients talk about amongst themselves is in fact expert information on their diseases (16). It is believed by many (9, 13) that patients’ experiential knowledge is valuable in the process of Research and Development. 

Experiential knowledge is the knowledge patients have due to the daily experience of living with a certain disease. It is a more practical type of knowledge and could complement the researchers’ scientific knowledge. Experiential knowledge could be useful in developing therapies that are adapted to the real needs of patients. Therefore patients can provide an alternative more relevant view on matters of health and illness (17-19). Ian Chalmers (founder of the James Lind Alliance, UK) summarised the value of the patient’s perspective as “We need to recognise that patients have the experience and skills that complement the researchers. They know what it feels like to suffer a particular disease and to undergo the treatments with their various side effects. They will have a good idea of which research questions are worth asking, and when a question should be framed differently (20) validating the patients part in the research process”.

This idea might be difficult for researchers to adhere to, because science traditionally sees the scientific method as the only valid way of knowledge generation. Experiential knowledge, according to some, lacks the objectivity, verifiability, universality and rationality of scientific knowledge (17).

But a question that more and more people ask themselves is: is everything that is studied by researchers, desirable (21)?

A number of scholars agree; patients could contribute by making research more socially relevant with their experiential knowledge (18, 22) (11). Various authors have reported on the added value of experiential knowledge. 

These studies showed that both the research itself and the involved patients benefit by putting their experiential knowledge to use in the research process. These benefits are summed up below:

The benefits that have been described for patients themselves 

  • Gaining knowledge and research skills (23-25).
  • Greater self esteem and confidence (24) (26) (27) of the patient representative that is involved in the process.
  • Making use of their experience and knowledge on their condition (utilizing their “experiential knowledge”)(24).
  • Acceptance of patients as equal partners in the clinical trial process and the creation of a sense of ownership (28) (25) (29) (30) of the research.
  • Access to funding (24) (25) for bringing researchable topics to the research agenda that otherwise would not be taken into consideration.
  • Increase understanding of the nature and purpose of a clinical trial (31).
  • Create a bridge of understanding between patients and researchers (32).
  • Health care and therapies that are more representative of patient’s “real” needs (19, 33-35).

The benefits that have been described for the research

  • Changes in information material given to patients (26) (36) (24) (31) (29) (28).
  • Changes in the design of the study and aspects of research such as: ways of collecting data, analysis of qualitative data, research questions, -tools, -priorities and -outcomes  (37) (29) (38) (39) (40) (41) (42) (17) (43) (30) (44) (45).
  • Increased recruitment and better recruitment strategy  (31) (37) (29). Donovan et al. reported increased recruitment rates from 40% to 70% (31) (28) (42).
  • Increased response rates (42).
  • More patient relevant research findings and methods. (29) (9) (43) (27) (44).
  • Challenged the assumptions made by researchers (37) (24) (32) (30) (44).
  • Wider dissemination of findings.

Various problems with regards to clinical trials have been mentioned by patients

  • First of all the trials do not reflect real life because study participants do not represent typical patients.
  • Furthermore many clinical studies replicate work already conducted elsewhere, which means that patients undergo unnecessary experimentation.
  • Another often heard problem is the poor and often cryptic level of information given to the study participants.

Although there have been numerous benefits described  that result from the collaboration of patients and researchers in clinical trials and research active patient involvement in the clinical trials development process is far from being common practice.

Where there are benefits to be had there are barriers to be overcome. These barriers have been expressed in concerns that both stakeholder groups have when it comes down to working together in the context of a clinical trial.

Concerns of Patients and Patient Groups

  • Concerns on taking on a new role (46).
  • Concerns that their views are not taken seriously (47).
  • Longer duration of trial (29) (48) (33).
  • Representativeness of the consumers who participate (29).
  • Conflict of interest between need of the trial and patients’ group (29) (43).
  • Professionals act according to a traditional biomedical model (48).
  • Power relations (44).
  • Non-structurally organized patient organisations (49).

Investigators’ concerns

  • Lack of knowledge and understanding of consumer involvement (50) (33).
  • Concerns whether consumers have necessary knowledge and skills (47) (49) (44).
  • Not seeing the added value of patient involvement (44).

For barriers to be overcome in establishing successful partnerships in clinical research, needs need to be fulfilled. The literature has been scanned to identify what these needs are

  • Need for strategies to educate public at large about the importance of research (9)
  • Need for dedicated organisational resources that facilitate patient involvement (e.g. money, coordinators, support) (39) (51) (52) (42) (53).
  • Need for training and support for patients to enable them to be involved in research (50) (39) (54) (55) (52) (56) (57) (42) (49) (53) (58).
  • Need for efforts to encourage a change in health professional attitude and greater understanding of patient involvement are required (51) (52) (55) (50) (42) (9).
  • Need for strategy to involve consumers effectively (55) (51).
  • Need for Interactive and long-term methods of patient involvement (39) (51) (53) (59).
  • Need for agreement and understanding between the two parties (54) (52).
  • Need for the identification of the right mix of consumers to be involved in clinical research especially for consumers groups  who do not come forward readily (e.g ethnic minorities) (54) (55).
  • Taking into account anxieties of patients of taking on a new role (46). If people are better informed about research (what it is and why it is important) they will be more willing to participate in as research as subjects, and also be more empowered to get actively involved (22).
  • Need to facilitate dialogue on the equal input of researchers and patients in research (60). One should give a voice first to stakeholder who is the least powerful (61) (2).

Overcoming barriers and fulfilling needs; methods from the literature

Even though not common practice, the literature does give some examples of how the active involvement of patients in clinical trials can be established. A number of authors have suggested and in some cases trialled methods to achieve working partnerships in clinical research. A formula that has been described in the literature is one that tackles the challenges toward patient partnership is Hewlett et all’s FIRST approach (46). This method describes how the barriers can be overcome and the needs fulfilled in order to have the right basis in place in which any working relationship between patients and researchers is possible.

  • Facilitate: inclusion and contribution. The principal investigator (PI) was found to be the key to facilitating the inclusion of partners in research. Partners should be included at early stages of protocol design.
  • Identify: projects, patients, roles. Identify research projects which can benefit from partnership. Patients need in depth experience of the health issue concerned and also need to feel confident that their experience is important. A job description clarifies the role of the partner and creates realistic expectations.
  • Respect: contribution and confidentiality. Respecting a partners contribution means acknowledging their value (for example by reimbursing expenses). The need to respect confidential information should be determined in an agreement.
  • Support: communication and working. Initial input from PI was considered essential. Other good practices were: early feedback from PI, peer support.
  • Train: research methods and processes. Because research is an unknown territory it was considered useful to provide training on quantitative and qualitative research methods, basic concepts of statistics and measuring outcomes. 

 Another example is the work performed by The James Lind Alliance (23) that divided the process of a working partnership on urinary incontinence into phases towards achieving an end result of the identification of research questions from the patient’s perspective. The process is described below:

  • Initiation phase in which potential partner organizations and representatives are identified.
  • Consultation phase in which questions and uncertainties are identified trough existing sources and consultation with patients, carers and clinicians.
  • Collation phase in which a search trough literature is done to see if any of these questions have already been answered. The issues that remain unanswered  will be made into clear questions.
  • Prioritisation phase in which unanswered questions are prioritised by parties through consensus.
  • Reporting phase in which findings will be reported to NHS Health technology assessment (NHS HTA) programme and the Medical research council (MRC) and published.

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