What is patient involvement and what are the benefits?
Patient involvement in research means the research is done with patients or by patients, rather than simply being done to them as passive subjects. Patient involvement generally means that patients, carers and relatives can all be involved. Patients and patient organisations can make a valuable and important contribution to research, and researchers are increasingly committed to involving them in their work.
The benefits of patient involvement – for both patients and researchers – are well documented. Patients have an opportunity to influence research that can be of benefit to them and involvement can help patients develop new skills and knowledge. Involving patients can improve the quality and relevance of research. Patients bring to research their knowledge and personal experiences of living with their condition on a daily basis.
How can patients get involved in research?
There are various ‘levels’ of involvement. For example:
- Driving Force: starting or keeping the research process going. This might involve financing the project, or campaigning for the research to be undertaken.
- Co-researcher: working as a partner alongside other ‘professional’ researchers. This might involve supporting the data collection by conducting a focus group or an interview.
- Reviewer: giving opinions on written documents. This might involve reviewing a participant information sheet or study protocol.
- Advisor: providing advice on different aspects of the research process such as how best to recruit participants, or how best to gain informed consent. This might involve being part of an advisory group or committee.
- Information Provider: providing information to researchers. This information is likely to relate to a patient’s experience of living with a particular condition, or the characteristics of patient organisation members.
- Research Subject: taking part in the research as a participant. In a clinical trial, this might involve providing a blood sample or testing a new treatment.
When can patients get involved in research?
Patients can get involved at any point of the research process – but patients do not have to be involved in all of the stages of any one project.
- Identifying and prioritising: patients can influence the areas in which research is funded.
- Designing and managing: patients can be involved in the design of research and its management, either employed as co-researchers or sitting as members on a steering committee.
- Undertaking: patients may get involved in data collection or analysis and interpretation of the results.
- Disseminating: patients may be involved in co-authoring and disseminating the outcomes of the research.
- Implementing: patients can influence how research findings are acted upon in the real world.
An organisation called INVOLVE provides very good detailed guidance for patient (and public) involvement with research.
Patient involvement in medicines development
The medicines development ‘cycle’ involves research and development (including clinical trials), but also the subsequent regulatory steps that are necessary before a medicine can be used by patients. The variety of ways that patients can become involved throughout this cycle is summarised below.
Patient involvement with industry-led research
Most medicines are developed as a result of industry-led research and development, and there has long been an appetite among patients and patient organisations to influence and be involved in medicines development (other than as volunteer subjects in clinical trials). The influence of patients tends to rely on individuals or patient organisations and pharmaceutical companies taking the initiative in making contact with each other. However, a new guideline to support the systematic involvement of patients in industry-led research and development will be released in late 2016 / early 2017 by the EUPATI consortium.
Patient involvement with regulators
European Medicines Agency (EMA)
The European Medicines Agency (EMA) is responsible for evaluating and authorising medicines in the European Union. Patients are involved in several activities at the EMA, such as reviewing information about medicines, being involved in the preparation of guidelines, and taking part in scientific advisory groups and being members of the EMA’s scientific committees. Patients are also represented on the EMA Management Board.
The group that advises the EMA on questions concerning medicines for human use (the Committee for Medicinal Products for Human Use, or CHMP) has a patient observer. In 2016, four of the EMA scientific committees include patients. Patients who are members of EMA scientific committees act in the same way as all other members. Patients bring their real-life experience of a disease and its treatments to the scientific committees. The committees are:
Committee for Orphan Medicinal Products (COMP)
The COMP includes three members representing patient organisations, for renewable terms of three years.
Paediatric Committee (PDCO)
The PDCO includes three members and three alternates representing patient organisations, for renewable terms of three years.
Committee for Advanced Therapies (CAT)
The CAT includes two members and two alternates representing patient organisations, for renewable terms of three years.
Pharmacovigilance and Risk Assessment Committee (PRAC)
The PRAC includes one member and one alternate representing patient organisations, for renewable terms of three years.
Medicines and Healthcare products Regulatory Agency (MHRA)
The Medicines and Healthcare products Regulatory Agency (MHRA) is the UK regulator responsible for medicines and medical devices. The MHRA is an executive agency of the Department of Health. The Board at the MHRA, which advises on strategy and oversees progress, does not include representation from any specific stakeholder group. Non-executive members of the Board are appointed by the Secretary of State after open competition.
Commission on Human Medicines (CHM)
The Commission on Human Medicines (CHM) advises ministers on the safety, efficacy and quality of medical products. The CHM includes one lay member. The CHM is supported by eleven expert advisory groups (EAGs), one of which is the Patient and Public Engagement EAG. Lay members sit on other EAGs.
Human Tissue Authority (HTA)
The HTA is sponsored by the Department of Health and regulates organisations in the UK that remove, store and use human tissue for research, medical treatment, post-mortem examination and education. The HTA also gives approval for organ and bone marrow donations from living people. The Chair and nine members of the HTA are lay. A Stakeholder Group, which advises the HTA on regulation, includes public members and other stakeholders.
Human Fertilisation and Embryology Authority (HFEA)
The HFEA is the UK’s independent regulator of treatment using eggs and sperm, and of treatment and research involving human embryos. This includes regulation of Preimplantation Genetic Diagnosis (PGD) and Preimplantation Tissue Typing (PTT). The Chair, Deputy Chair and at least half of the HFEA Members are lay, that is they are not doctors or scientists involved in human embryo research or fertility treatment.
Patient involvement in health technology assessment
The National Institute for Health and Care Excellence (NICE) provides national guidance and advice including health technology assessments of medicines and medical devices. Health technology assessment for a medicine or medical device can include a review of clinical evidence, cost effectiveness, and likely impact on the health care system and the lives of patients.
There is a variety of ways that patients can become involved with the work of NICE, from contributing to guidelines about specific treatments, to attending meetings held in public, to joining as a committee member.
Genetic Alliance UK examined the value of inclusion of patients in NICE’s Highly Specialised Technology programme, and subsequently published the Patient charter: patient perspectives and priorities on NICE’s evaluation of highly specialised technologies.
The NICE website outlines how patients, carers and the public can get involved in developing guidance for specific treatments.
Several NICE committees include ‘lay members’ i.e. people who are not trained or specialised in medicine or health technology appraisal (but who are not necessarily patients).
Patients organisations can also become involved through Patients Involved in NICE (PIN), which is a coalition of over 80 patient organisations. It is independent from NICE and the pharmaceutical industry, and aims to ‘use their combined knowledge, experience and direct contact with patients from a wide range of conditions, to ensure NICE puts patients, carers, and patient groups at the centre of all of its work’.
In Scotland, health technology assessments of treatments are carried out by the Scottish Medicines Consortium (SMC). The SMC runs a Patient Group Partner system. Patient groups register with the SMC in order to contribute to reviews of treatments, and are responsible for gathering contributions from individual patients and carers. SMC also runs a Public Involvement Network (PIN) to advise the SMC on its involvement of patients, carers and the public.
Genetic Alliance UK examined patient views on the process of access to medicines for rare conditions in Scotland, and published the Patient charter: Patient perspectives and priorities on access to medicines for rare conditions in Scotland.
The All Wales Medicines Strategy Group (AWMSG) advises the Welsh Government’s Minister for Health and Social Services on new medicines and prescribing. Patients can give their input when the AWMSG is assessing specific new medicines or prescribing approaches for existing medicines. Lay members are also included in the groups that perform the assessments of new medicines. The AWMSG has established a Patient and Public Interest Group to advise on patient and public engagement at the AWMSG.