FAQ for Patient Groups and Individual Patients

Below are some important terms from our Glossary to keep in mind when reviewing the FAQ.

  • Health technology: Any form of intervention to improve health, such as drugs, devices, medical equipment and procedures relating to health care and its services, including prevention, diagnosis and treatment of a condition.
  • Health technology assessment (HTA): The systematic evaluation of the clinical effectiveness and/or cost-effectiveness and/or the social and ethical impact of a health technology on the lives of patients and the health care system. Its main purpose is to inform health care policy makers. The process advises whether a health technology should be used, and if so, how it is best used and which patients will benefit most from it. Assessments vary, but most look at the health benefits and risks of using the technology. HTAs may also look at costs and any wider impacts that the technology might have on a population or on society.
  • Ethics: The study of values and customs to understand the principles according to which humans should live. Health ethics describes the principles of good medical practice that determine what is good for individual people and society as a whole. These principles include relationships between patients and doctors, and the use of technology.

The main aim of patient involvement is to ensure that HTA assessments and decisions are informed by the special knowledge/unique perspectives of those with the lived experience of a health condition and its management, or who are able to speak on behalf of patients as their informal carers.

Groups that represent patients and/or carers (patient/consumer organisations, charities etc) are involved, and this can be instead of, or in addition to, involvement of individuals.

An understanding of patients’ beliefs, values, expectations, needs and preferences can be essential to maximising the benefits of health care and and optimizing value delivered to patients.

Patients’ views may differ from those of health professionals or researchers and should not be assumed by other stakeholders.

HTA is a systematic analytical process at the interface of science and policy making, which is influenced by the values of the people who participate in the decision-making. Involving patients and their carers enables their values to be included in the deliberative processes. The result is that assessments take account of the knowledge, needs and preferences of those whose lives are shaped (functionally, economically, socially and psychosocially) by the health conditions, available treatments and the technologies being assessed. Typically, patient involvement is required to consider specific condition, diagnosis or treatment related issues and provides experiential evidence, including on the relevance of the outcomes reported and the needs of patients.

It is not always possible to identify patients with a direct experience of a specific health condition who are in a position to participate in particular HTA processes, or to be on committees. Patients with certain diseases or health conditions, such as rapidly progressive or debilitating diseases; with neurological or cognitive effects, or stigma attached; and patients in palliative care may not want or be able to directly contribute. In these instances, another person with an understanding of this patient group’s needs and preferences, such as someone with a condition that shares similarities to that being considered or a carer or patient representative group may be able to fulfil this role. In such situations, gathering patient experiences and sociological research about the needs and preferences of the specific patient group may be especially important for informing the HTA.

Additionally, some HTA committees that make decisions across multiple diseases or conditions wish to have public members, often termed ‘lay members’, who are not there to represent any one disease or condition. In this case, patients may participate so long as they are able to consider issues wider than their condition.

Where HTA processes are to permit initial market entry or public funding of a technology, few patients may have experience of the particular technologies. This experience is likely to be limited to patient access schemes, familiarisation studies, clinical trials or special programmes such as for compassionate use. Knowledge of the lived experience of a condition and identification of unmet needs with existing care pathways are more important to an assessment than knowledge of the technology or procedure under consideration. Patients who have experience of the technology, or the technology it is being compared against in the HTA and can share this experience would be particularly useful in informing the assessment.

An individual patient and a patient group may provide different but equally useful perspectives and contributions to an HTA assessment. A patient and/or their caregiver can bring rich knowledge formed from living with the health condition, its diagnosis and treatment. They may also be informed about the experience of other patients through their personal interactions.

A patient group may be comprised of patients or representatives of a patient community. If it is the latter, their knowledge will be less rich in specific experience but may be informed by a wider group of patients. A representative of a patient group may be able to consult their members to respond to the HTA processes. The capacity to provide representation and input into HTA processes can be a challenge for health consumer and patient support organisations.

General organisations representing broader patient and user interests may not share the same perspectives as disease-specific groups.

Increasingly representatives from patient groups are not patients (working in a voluntary capacity) but are trained, mostly highly educated volunteers and healthy employees who ‘have the knowledge and skills’ to influence decision making and the policy makers. However, they are without the lived experience of the condition.

Patient groups vary greatly in their size and remit (for example in providing patient education, support, services, research) and often their role and function is dependent on available resources (personnel, funding). Smaller groups may lack the resources to contribute in the same ways as large groups; they may be closely in touch with their patients meaning that they are in a good position to contribute to HTA processes.

When involving any individual or patient group, it is important to have transparency about any potential conflicts of interests that they may have. Awareness of the activities and funding sources (of groups) provides context about the information that is provided, and accountability to other stakeholders.

Participants can contribute to HTA processes in a number of ways, such as the following.

  • As the ‘subjects’ of research such as through interviews, questionnaires, surveys and other forms of consultations and qualitative research, or in trials of intervention effectiveness and potential harms. In this way, they provide part of the evidence.
  • By providing experiential knowledge of living with the condition and the disease burden, its diagnosis and treatment, including pathways of care (for cost-effectiveness analyses), the disease burden, identifying gaps in health care, and identifying and addressing needs.
  • By providing perspectives on the social and societal implications of the health condition or disease and its treatment (or lack of treatment and support).
  • By providing feedback and submissions to HTA processes, whereby issues and outcomes of importance to the patient groups are identified, including unmet needs, and identification of sub-populations in need of effective treatment options.
  • By taking part in decision-making process to contribute the perspectives of patients, their needs and values.
  • By prioritising technologies (and diseases).

These contributions may be sought and collated in a range of approaches including formal qualitative research that may be conducted by a range of researchers including patient advocacy organisations themselves.

Patients and their caregivers are important for identifying the signs and symptoms of a disease that have the greatest impact on their functional and psychological aspects of living, and how great that impact is for themselves and their families. Additionally, they can indicate whether outcome measures used to assess the effectiveness of an intervention or technology are really providing the information that is important to the patients; or that they are a meaningful measure of disease state and what matters to patients. Patients (and their carers) are also the best people to identify what possible harms or adverse effects are being triggered by treatment, as they live 24 hours a day with the disease and know their bodies well.

Patients and their caregivers are able to actively participate in collection of information (data on patient-reported outcomes) that assists clinicians and researchers to assess the state of a disease, its treatment, and how effective a drug, device or procedure is in managing the condition. They know what is most worrisome to them and needs to be addressed to improve physical, mental and social performance and the ability to function and participate in daily living.

Patients and their carers may be called on, or have the opportunity, to participate in consultations. These may be in the form of interviews, focus groups, completing surveys or questionnaires, and making written submissions in response to calls for information.

Deliberative processes provide the opportunity to gain knowledge about a condition and its treatment. Deliberation offers a learning experience for patients and professionals, where each becomes aware of the other’s experiences and knowledge. It involves processes where patients can impact on group decision making and influence professionals’ opinions.

No, this is not the case. HTA agencies seeking input are likely to look at how the information is presented and organised, the content, and who submits the information. That is, the representativeness of the patient population that is covered by the group, how the group collects its information and from whom is considered. The content and how it fits into the evidence or knowledge base and decision making is what contributes to the quality of the information provided. Views of minority groups of patients, and differing views may also need to be considered.

Considerations, regardless of the size of the patient group, are:

  • the timeliness of feedback from patients;
  • the breadth and relevance of the group of patients who are consulted; and
  • who makes the decisions on the issues and topics that are included in the input.

Whether the points above could be influenced by confounding interests or the potential for profit from the stated position.

It is appropriate to request this information of any person or organisation making a submission to an HTA process. An important question for HTA agencies or bodies is ‘do the submissions present the views of patient organisations – and the patients themselves’? HTA bodies or agencies seeking input are in a position to look at how the information is presented and organised, the content, and who submits the information. Some HTA agencies ask for an overview of the patient organisation, its aims and membership; corporate members and joint working or sponsorships; and those playing a significant role in compiling the input or submission.

How a group collects its information and from whom, and therefore the credibility it can be given in terms of patient experiences, needs to be considered. Who makes the decisions on the issues and topics that are included in the input, and whether this can be influenced by confounding interests or the potential for profit from the stated position, are important aspects to be considered by both patients and the HTA agencies.

It is important to note that ‘declaring’ an interest may not be seen by the HTA agency as a ‘conflict’, depending on the nature of the interest declared, and so may not stop someone from participating in HTA processes or patient organisation evidence being considered. It is, however, important that people considering that information are aware of the declaration of conflicting or confounding interests.

Important considerations are the identification of patient needs that will make a difference to their wellbeing if addressed, gaps in treatments that are available, factors related to the use of the technology being assessed, relationships to availability of healthcare services and providers; and how the new technology fits in current clinical pathways. Measures of effectiveness, the relevance of outcomes measured in clinical evidence, and possible harms can all be assessed from well-presented and organised patient and patient organisation input that clearly identifies its source.

Measures of involvement of patients and their organisations provide information on the effectiveness of HTA agencies in engaging with patients and their caregivers. The numbers of patients and organisations providing input and the diversity of groups or patients may matter, so that a broad spectrum of views is obtained in terms of representativeness of the patient perspective.

Patients and patient groups need to have good knowledge about their own disease, its management and treatment and how these work for them. This is in a broad group of patients if they are actively participating in decision making committees as representatives. They may also need some education and training about specific issues relevant to their role, the expectations of them, and HTA processes relevant to their engagement.

Preparation may include education on the role they are to play and what they can expect, the role of the event or committee they are engaging in, background information on the HTA organisation, processes and where to find the information, support and knowledge they need to actively participate.

Patient representatives on advisory committees are required to have background knowledge in evidence based medicine and consumer matters, to be conversant with relevant technical matters for the committee, be able to effectively represent the views of those who they represent (unless they are public or general lay members of the committee) and actively and competently participate in meetings. It is important that they are clear about who they represent, how their knowledge of patient aspects comes about, who they are accountable to, how they present themselves, and to what end they ask questions. This helps them to have the capacity to respond on the spot and to utilise, and if need be create, their position in providing the patient perspective.

Patient representatives need to be informed and able to make other decision makers understand the consequences for patients of the positioning of the clinical evidence base and the decisions being made, from a patient, carer and family perspective and perhaps from a societal perspective. The patient’s perspective is given as an autonomous subject and not as the object of experts’ discourses.

Being clear about who they are as patients and patient organisations, and why they are there, makes their participation relevant and legitimate, so they are not outsiders by design. The viewpoints of different patient representatives may be substantially similar or different, so become more aware of how and why these differences occur. It is always best to involve at least two patient representatives, not just one.

Representing and presenting the patient perspective requires time and deliberate, sustained effort. It also requires awareness that the patients and patient representatives and their views are open to varying degrees of alteration by those who will ultimately make the decisions in HTA processes. Patient representatives benefit from having community consciousness and being there to help others like themselves.

Patients are often engaged to provide political legitimacy to the HTA process. Their representativeness is a political term that is generally asked of patient representatives but not of healthcare professionals, researchers and other stakeholders. As lay people they need to know enough to participate in active debate and to ask pertinent questions. They do not need the same knowledge base as the ‘experts’ in clinical and research matters.

Such patient representatives are often assumed or expected to retain ‘ordinary norms and values’, yet what these are supposed to be is not clear. Their putative value lies in the differences between their perspectives and those of health professionals, researchers, economists, policy makers and the other stakeholders in the HTA process. Expectations are that they are devoted to the task, are knowledgeable about health care – and can work with experts who have formal authority within a structure (such as a professional society, university, or HTA agency). The capacity to be a participant from a patient perspective is shaped by that person’s interests and values, and by the contributions they seek to realise. They should be clear about why they want to engage and participate. Who they are relates to what makes them potentially relevant and legitimate as spokespeople.

Patients and their organisations should be offered education and training in order to:

  • Make involving patients (and lay people) in the often complex collective healthcare decisions of HTA more feasible, as some may not understand the available scientific literature or be aware of clinical and resource implications of the decisions being made. This can lead to uninformed opinions and unrealistic expectations by the patients and their representatives.
  • Improve their ability and competence in participating in specific HTA processes such that they can impact on decision making in a meaningful way.
  • Patients who are actively involved in HTA processes benefit from:
    • Education on the role they are to play
    • Understanding what is expected of them
    • Understanding what they can expect
    • Background information on the role of the committee or process, the organisation and overall HTA processes
    • Knowing where to find the information, knowledge and support to assist them in actively participating.

Patients or their representatives on committees may receive additional training on being part of a committee and on governance, so that they are aware of their responsibilities.

Patients are generally involved to present the collective patient experience, focusing on the patients’ issues, needs, experiences and preferences in relation to a given (type of) technology; or to ensure that the best interests of patients are considered.

Patient representatives benefit from being competent in presenting ‘what works’ for patients and the gaps in their health care; the need to understand issues related to the illness or condition, the need for intervention; factors influencing the implementation of particular health technologies, their appropriateness and acceptability; clinical pathways and a broad societal perspective. The representatives are able to present these perspectives in a way that is acceptable to other stakeholders (clinicians, researchers, health economists, policy makers as well as other patients, carers, consumer groups and decision makers).

Patient input is only one type of evidence that is considered in a HTA and its influence on a funding decision is affected by the quality and strength of the other evidence as well as the quality and strength of the patient input.

Patient experiences as knowledge must be presented as organised information. The information provided is likely to include: who uses/would use the technology; what benefits are to be experienced (and by whom); any (potential) harms; which of the patients’ needs are addressed, and the impact of this on their lives and that of their carers; potential limitations to use of the technology; and costs to patients, their caregivers and family.

The concept of what counts as knowledge by the HTA agencies needs to be considered, how information is sought from patients (and their carers), how many provide input, the representativeness of the sample, how the information gained is sorted and presented so that it is likely to be taken note of and scrutinised critically (in the scientific assessment, cost effectiveness analysis and by decision making committees). It needs to be clear what is meant by patient needs, what has been looked for, and what has been looked past.

Knowledge is relational – in the sense that it ‘attaches’ itself to relations among the people who form part of the dialogue. Patient information that provides input must, therefore, be clear and accurate about any assumptions made, categories and concepts used, and the choice of words.

Difficulties with involving patients are often attributed to lack of training, time and resources that patients and their organisations have, but the fundamental problem is related to uncertainties about these lay people’s role or purpose, their method of selection (representativeness), accountability and what they are expected to contribute and how.

People who put themselves forward to participate in HTA processes tend to come from high level occupations and social groups. When patients gain knowledge through their engagement, and become proficient in arguing for the interests and values of their peers, they may no longer be seen as representative. Excluding patients and lay people from scientific or technical committees can also be used as a way of limiting their involvement.

Patients are very human, with demonstrated emotions. Overall, their contributions can be devalued and dismissed or ignored by other members who may be more comfortable with quantitative data and employed health and research professionals who are there for the purpose of health technology assessment.

Healthcare professionals alone cannot presume patient issues and that they know what is most important for patients. The assumption among those involved in health care is often that ‘everyone’ is working for the ‘best interests of the patient’. This can hide genuine differences in the interests, needs and expectations of patients and their caregivers.