FAQ for HTA Agencies and Policy Makers

The main aim of patient involvement is to ensure that HTA assessments and decisions are informed by the special knowledge or 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 or 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 maximizing 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.

Meaningful patient involvement is where patients take an active role in activities or decisions that will have consequences for the patient community, because of their specific knowledge and relevant experience as patients.

The involvement must be planned, appropriately resourced, carried out and evaluated according to the values and purposes of:

  • the participating patients or patient organisations,
  • other participating organisations and funding bodies, and
  • the quality of their experiences during the involvement activity.

Citizen involvement aims to give people a greater say about the delivery of healthcare in their community and to increase the transparency of decision-making processes. Involving citizens can reveal beliefs, values, needs and preferences of the community. Citizens and the public (that is those not affected by a health condition), may bring a different perspective than patients and their informal carers. Citizen or public involvement is important when considering generic issues such as community values about how funds are allocated, availability of treatments, healthcare priorities and other issues to do with an ethical health system and societal values.

When we talk about involving patients in HTA processes, we are referring to two distinct processes.

  1. Patients involved as subjects in sociological and qualitative research to inform an assessment
  2. Patients involved as participants in the assessment and decision-making process

In some countries, it is legislated that patients or the public are involved. The most common way is by participation in HTA committees; however, they can be involved throughout the assessment process in:

  • (a) topic proposal and selection – prioritising HTA topics
  • (b) giving feedback on proposed HTA protocols
  • (c) scoping – informing what is ‘in’ and what is ‘out’ of an assessment
  • (d) submitting evidence
  • (e) formal consultations
  • (f) evidence assessment and interpretation, sitting on HTA committees as part of the decision-making process; in some systems they can vote, in others they do not
  • (g) dissemination of HTA findings

Patient involvement can come from individual patients, their informal carers, patient representatives, advocacy groups, and patient and consumer organisations. Different types of perspectives may be sought at different points in an assessment, and agencies differ in how they seek these perspectives.

Who is involved, and how, when, and where they are involved, depends on the HTA organisation’s specific reasons for involving them. These reasons must be clearly articulated and agreed upon with those being involved, so that their role and what is expected of them is clear. HTA agencies should set this out in formal processes and documents, for transparency and consistency.

A patient is someone who is actively receiving healthcare treatment for a health condition. The condition may be acute, recurring or a long-term condition that persists or may worsen over time. It may be a risk factor for disease, such as high blood cholesterol levels. Women using maternity services and people using mental health services (often termed ‘service users’) may be considered ‘patients’ in HTAs.

Patient representatives or advocates may participate on behalf of patients. Sometimes this is for people with a debilitating or rapidly progressing disease, or those with limitations in their communication skills. Sometimes informal carers act in this capacity, but they may have their own experiences and views which can form part of the knowledge base.

Some organizations have patients/consumers with special knowledge and their own networks and choose to speak on behalf of a broader community of patients as advocates. They can provide strategic input to broader health system issues by speaking from a population perspective rather than ‘their own story’.

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; those 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 fulfill 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.

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 (e.g., in providing patient education, support, services and research), and often their role and function is dependent on available resources (personnel, funding, etc.). 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.

The involvement of patients and carers with a strong voice is seen to benefit policy-making by

  • adding support to the assessment of value and ensuring aspects of value that are important to patients and carers are adequately considered and understood;
  • making HTA more relevant to the everyday care of patients;
  • identifying areas of need for patients and their informal carers;
  • supporting participative democracy (giving people a say beyond the ballot box or election of government);
  • increasing the transparency and accountability of HTA processes;
  • increasing the relevance and credibility of the recommendations;
  • establishing a culture of knowledge exchange and mutual respect (e.g., between HTA researchers, health professionals, patients and community);
  • helping patients and carers to contribute to identifying risks and harms, to improve safety and quality of care;
  • managing expectations of patients and their carers;
  • raising awareness of the HTA processes and how healthcare interventions are made available to them;
  • increasing understanding and buy-in for contentious issues; and
  • increasing public confidence and trust in the healthcare system.

It is important that the patient input reflects the interests of patients themselves. It needs to be made clear where the input may reflect the views of a specific group of patients (and not the general population). Patients are not a homogeneous group and so looking for the ‘normal or average’ patient or an overall group perspective could be misleading and unhelpful. Patient involvement may be undertaken to learn about the wide variety of views held by patients rather than necessarily finding a majority point of view.

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 itself 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 sponsorship; 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, therefore, 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.

Pragmatic and qualitative clinical studies can provide useful data to support follow up of the clinical data (from randomised controlled trials with well defined, non-subjective outcomes where appropriate) presented to meet regulatory requirements. In particular, it can be used to identify potential harms, the use of the interventions within the health system and social structure, and the relationship of the interventions to social values and beliefs.

Consultation with users of health care, and involving them in deliberative processes (where all the information on an intervention is presented for consideration), can effectively provide the patient and healthcare user perspective. That is, actively involve the patients and not just have them as the subjects of clinical and qualitative research.

Patient data is one type of evidence that forms the evidence base for a HTA and ideally it is used throughout the HTA, alongside clinical evidence and economic considerations.

Qualitative research (such as surveys, questionnaires, interviews and focus groups) and studies that look at patient reported outcomes, social values, psychosocial (e.g., sense of well-being), socioeconomic and functional outcomes can add patient experience to the knowledge base on which decisions and recommendations in the HTA process are made.

HTA agencies can ask for submissions from patients and their organisations. These may define likely clinical pathways of a person with a particular health condition, provide the experience of patients and their caregivers, and identify shortfalls and gaps in their diagnosis and treatment. An important aspect is unmet needs that have an impact on their well-being and ability to function and participate in life. These submissions can influence decision making and contribute to analyses of the cost effectiveness of a technology.

Overall, patients and their informal carers can be engaged in

  • prioritisation;
  • consultation, patient surveys, interviews, focus groups, etc.;
  • responding to invitations to provide patient and patient group submissions, to protocols, decision making meeting agendas;
  • decision making committees (as patient representatives);
  • formulating recommendations, wording and important aspects to address; and
  • dissemination of information about new health technologies.

Important considerations are the identification of patient needs that will make a difference to their well-being 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 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).

The weight given to all input and evidence in an HTA may vary according to the quality of the information and the question or aspect being considered. Typically, different types of evidence are used to answer different types of questions in an HTA. Usually evidence or input from different sources, including patients, gives an HTA a broader, richer picture of the way a technology may be used. However, sometimes different sources of input and evidence provide conflicting information. When this happens, the HTA needs to weigh up the quality of the information and may need to undertake further questioning, discussion or research to resolve the conflict.

Patient perspectives can influence the priorities and considerations factored into cost-effectiveness analyses, recommendations, and how the information is disseminated. Patient input is valuable because it is the user perspective and is different, without professional protectionism and business implications. As a result patient groups need not try to disguise this value by attempting to make their inputs more quantitative in order to be taken notice of and to influence the decision makers. The patient perspective is influenced strongly by the lived experience, and knowledge of the healthcare and other resources available to them. Relational resources include their social networks, sense of identity, disposition. Cultural resources include education, which impacts on communication and organisational skills; economic, socioeconomic, and occupational assets that influence their self-esteem, competencies, and sense of self-efficacy. Cognitive resources, the knowledge patients possess and acquire, ideological frameworks, values, norms, political or religious ideologies can all influence that perspective.

This makes it very hard to identify the ‘normal’ patient and a single answer represented by numerical values.

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.