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1 Introduction

In this chapter, we address three challenges relating to patient involvement in HTA. Firstly, we reflect on some of the terms often used by HTA researchers, patient organisations and HTA bodies to describe the patients who participate in HTAs including terms such as ‘patient’, ‘patient advocate’, ‘patient representative’, ‘patient partner’ and ‘consumer’. This challenge has previously been described in relation to healthcare in general. Dent and Pahor write: ‘The whole arena of patient involvement within healthcare is riven with problems of meaning, definition and purpose’ (Dent and Pahor 2015, p. 549). In addition, they argue that the topic is further complicated when one attempts to compare practices across countries (Dent and Pahor 2015). Secondly, we discuss how the choice of term and hence the choice of participants may influence the realisation of goals with patient involvement in HTA. We argue that a challenge lies in confusion about the goals for implementing patient involvement in HTA, particularly when these goals compete with a variety of other goals such as cost containment or decision-making based on strictly defined clinical effectiveness. Thirdly, we address the challenge relating to leadership and organisational change, because some of the goals for patient involvement in HTA will require new ways of organizing and leading HTAs. We briefly introduce three models for organising patient involvement in HTA. The aim of the chapter is to contribute to the current debate on the use of terms to describe patient involvement and the nature of the associated goals and organisation supporting this involvement.

Traditionally, in some countries, HTA has been seen as the provision of independent evidence-based assessment of the safety, clinical and cost-effectiveness of health technologies with patients’ experiences and preferences as useful but not essential components (Chap. 1). In other countries such as Denmark, patients’ perspectives and experiences have been an integrated part of a full HTA (Kristensen and Sigmund 2007). Part III of this book is a presentation of how different countries around the world have worked with patient involvement in relation to HTA. However, although EUnetHTA (Chap. 24) has integrated patient and social aspects in their HTA Core Model® and presents these aspects as an integrated part of an HTA process equally with other domains, EUnetHTA states that economic evaluation and clinical effectiveness until now have played a much more dominant role in HTAs.

In addition, some HTA researchers and decision-makers receiving HTA reports see the introduction of patient involvement in HTA as a potential hazard in achieving independent evidence-based decisions and question the ability for ‘nonexpert’ or ‘lay’ members of the public to be involved in highly technical assessments and decisions (Russell and Greenhalgh 2014; Lopes et al. 2015). On the contrary, others believe that we have only begun to incorporate patients’ perspectives and involve them in the HTAs to incorporate patient values and need to further develop patient involvement (see Sect. 3.3). To meet this challenge, it will be necessary for HTA practitioners to acquire new skills, new knowledge, new practices and new competencies. Similarly, shared understandings of the relationships between HTA researchers, practitioners and patients will need to be developed, critically reflected upon and implemented. Reflection about leadership and distribution of power in decision-making are therefore crucial to the HTA process.

Overall, there may be a tension between the evidence arising from clinical trials and economic models and the patient-based evidence arising from the perspectives and experiences of patients (Chap. 4). In particular, the value placed on a service or technology by patients may run counter to the values expounded in assessments. Patient values may be different to those held by other experts in the HTA process or by citizen at large (Street et al. 2008). In addition, patients’ views and preferences, similar to the views and preferences of health technology developers and clinicians, may be skewed by a vested interest in the outcome. These interests need to be acknowledged and managed appropriately. HTAs need to be patient centred (Bridges and Jones 2007) but must also recognise what is best for all patients across the board (see evaluation of equity in Chap. 2). It is possible that since patient groups often value a fair and transparent process, as long as these two conditions are met, they may be reconciled to the outcome. However, this notion itself deserves further scrutiny.

Understanding who will be asked to contribute, how and when, is important in sorting out this potential conflict of views in HTA. We argue that reflection on the terms used to describe the patients that are involved in HTA and the ideas underpinning their use is essential before we can discuss and consider how, when and on what basis patient involvement can be effectively implemented in HTAs. In particular, we argue that we need deep reflection on the overarching goals of patient involvement in HTA and the role of patients and their representatives in the HTA processes.

2 Terms Used to Describe ‘Patients’ in HTA

The terms used in HTAs to describe patient involvement are crucial in defining the roles and positions of those involved. A review of the role of patient and public participation in HTA, published by Menon and Stafinski (2011), provides an indication of the range of the terms used to describe such participants. We argue that the choice of term to some degree defines the type of participants who will be involved, their specific qualifications and competences and their interests. Work to define the goals for public and patient participation and hence the nature of the selected participants is already well underway in some organisations. For example, the European Medicines Agency (EMA) only invites patients to sit on scientific advice committees discussing a clinical trial, in order that the committee may hear the experience of someone living with the disease (EMA 2014). However, only European umbrella organisations may sit on committees that shape the processes and policies in order to incorporate the broader policy perspectives of the organisations. Nevertheless, in reading HTA reports and HTA articles, it is clear that, in many cases, the terms chosen are used with little reflection or consideration of these issues. In the following, we reflect on some of the terms often used to describe patient involvement in HTA.

2.1 Patient

Etymologically the concept ‘patient’ stems from Latin patientem, meaning bearing, supporting, suffering, enduring and permitting (Harper 2016). Today the term ‘patient’ usually refers to an individual with a diagnosed disease or disorder who is using or has used the healthcare system due to their need for treatment and care. In HTA this term is often used in a general way to state that patients’ views, experiences, preferences, needs and involvements are of importance although frequently without reference to why that is the case. The homepage of the HTAi Interest Group for Patient and Citizen Involvement in HTA (the HTAi Interest Group) states, for example, ‘Our vision: Patient and citizen perspectives improve HTA’ (HTAi 2016a). Throughout the different links on the website, the term patient is taken for granted in that no explicit reflections on the nature of the term are presented in the text. The most detailed exploration of the term can be found in the stated aim of patient involvement: ‘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’ (HTAi 2016a).

Some authors explicitly describe the term patient. Bridges and Jones (2007, p. 32) do so by describing what a patient is not: ‘To be relevant, the patient’s perspective (rather than one of a citizen, tax payer, provider, or payers) must be addressed’. In contrast, Facey et al. (2010, p. 335) describe a patient as ‘any current or potential health service user or beneficiary of a health technology’ and ‘a person who has valuable experiential knowledge about a specific illness or condition…’. The inclusion of a broad spectrum of patients in HTAs is difficult. For example, it is likely that patients with no or low education, homeless patients, patients living alone, patients experiencing high levels of ‘social shame’ (such as with venereal diseases, alcohol dependence or substance misuse) or patients of lower social position may be more difficult to include in an HTA. Although many organisations are conscious of these omissions and seek to be inclusive, we would argue that patient representation in an HTA will always be incomplete and the notion of inclusion of ‘patient views’ inherently flawed. See Sect. 12.8 for the production of knowledge, where the authors state that knowledge is always both reductive and selective.

2.2 Patient Advocate and Patient Representative

The terms patient representative and patient advocate are often used interchangeably. This is, for instance, the case on the homepage of the HTAi Interest Group in the Frequently Asked Questions (HTAi 2016b). Here, a distinction is made between a patient and a patient representative or advocate in HTA, but not between an advocate and a representative. Hofmann and Saarni (2011) see advocacy as a potential aim for the patient representative in supporting ‘empowerment’ and ‘autonomy’ for patients and ‘improvement of the health systems’ Alternately the role may be seen as more circumscribed demanding ‘more and better services’ for their patient group (Hofmann and Saarni 2011). In a qualitative study from Australia, some participants ‘questioned the legitimacy of umbrella organisations and some patient (“consumer”) representatives in representing the wide range of patients and patient organisations with some interest in the process (Lopes et al. 2015). In this case “patient advocate” appears to be a more appropriate term since the individual will advocate on behalf of patients generally but does not necessarily “represent” all patient groups. Patient advocates or representatives are described as participating on behalf of patients or representing the views of a particular group of patients, survivors or carers: “Sometimes this will be for people who have a debilitating or rapidly progressing disease or limitations in their communication skills”’ (HTAi 2016b). The term patient advocate or representative may also be used to describe a professional who supports the patient and their caregivers (carers) through the illness journey and assists them in negotiating with the often complex health systems. In HTAs these terms are usually used to describe an individual or an organization promoting the interests of a broad group, for example, cancer survivors or patients with a rare disease or the views of a very specific group, such as patients with Alzheimer’s disease. Patient advocacy organisations are frequently non-profit. The modern concept of the patient advocate arose in the 1950s with concerns about the involvement of cancer patients in research trials (Keating and Cambrosio 2012).

If a health technology is contentious with entrenched views held in different patient advocacy organisations, there is potential for the debate to become polarized. In this case, the views of a vocal minority—not necessarily shared by the broader patient community—may skew the HTA processes. For example, the broader views of the deaf community may not have been heard in the debate as to whether prelingual deaf children should receive cochlear implants (see Batterbury 2008). However, there are few published examples of this phenomenon, and therefore, it is difficult to judge the real impact in HTA. Alternatively, the interests of high profile diseases, such as breast cancer, with numerous strong patient advocacy organisations, may dominate, such that those advocating for low-profile diseases struggle to be heard. In aiming for a balanced deliberation and community discussion about the adoption and/or public funding for a particular technology, patient advocates may be bound by the community they serve and may not be able to respond reflectively to evidence which runs counter to the community view. Similarly, some HTA practitioners have expressed concern about the influence of the pharmaceutical or medical device industries on the views propagated by patient advocacy organisations (Lopes et al. 2015). Caregivers may also act in the capacity of patient advocate or representative. This may be a challenge, since caregivers ‘may have their own experiences and views which can form part of the knowledge base’ (HTAi 2016b). However, it is important to stress that the needs of the caregivers are important and that they need to be considered as representatives in their own right.

2.3 Patients as Consumers

Some researchers have argued that the term consumer is broad enough to encompass patients, members of the general public, caregivers, etc. (Royle and Oliver 2004; Bastian 1998). Certainly, the term includes citizens who irregularly access systems of healthcare, for example, for screening programs or vaccines, but who may not be seen as ‘patients’. However, including patients within this umbrella term assumes an equal relationship between the ‘seller’ and the ‘consumer’. In the case of a patient, such a relationship would generally not be possible since patients frequently have few options in terms of their treatment and insufficient information on which to make choices and decisions. In addition, the patient may be seriously ill and burdened with the demands of his/her disease. Some diseases lend themselves more readily to the notion of patients as consumers than others, that is, the disease burden may not be as high, there may be a large number of long-term survivors or the disease may be better understood in the general population. In general, although patients are consumers of health technologies, the usual contractual arrangement between seller and buyer does not apply since it is clearly an unequal partnership. In most cases, the patient, particularly a patient in a publicly funded system, is unable to change their provider or treatment options or it may be difficult to do so. Tritter (2009, p. 285) states that it is important to be aware of the tensions between involvement and consumerism, since promoting patients as consumers may limit the evolution of patient and public involvement. The term might be considered particularly inappropriate in the case of shared decision-making which is based on mutual respect rather than a commercial agreement.

2.4 Patients as the Public

Because of their potential for partisan views (3.3), viewing the patient as representative of the broader public is usually inappropriate. On the other hand, the assumption that patients will not recognize the broader societal consequences of particular decisions may also be erroneous (Hodgetts et al. 2014). Clarity around the role of public or patient input will assist in teasing the two areas apart. That is, in HTAs, the public interest and the patient interest are usually different, and therefore, their input should be collected separately. Individuals who represent the interests of patients should not also be required to represent the interests of the broader public. In an article which conceptualised the term public involvement, Gauvin et al. (2010) highlighted some of the challenges: for example, their research indicated that there was little agreement in the literature as to who ‘the public’ is or ought to be, or the most appropriate terms to define ‘the public’ (Gauvin et al. 2010, p. 1522). They argued that until recently, the public was referred to as ‘consumers’ as a reflection of the market-oriented ideologies of the 1970s and 1980s.

2.5 Patients as Experts

Patients may be considered experts of their own experience of the disease condition and in terms of the applicability and importance of any technological application. This term is often connected to the argument of why to involve patients in HTA. For instance, Geissler from the European Patients’ Academy (EUPATI) wrote: ‘They [patients] are the experts on quality of life. They know how it is to live with a disease. They can probably assess one against the other…’ (EUPATI 2016). ‘Informed research, assessment and decision-making are not possible unless patients are involved…’. The term patients as experts is diametrically opposite to the idea of patients as ‘lay’ representatives. A patient as expert may be defined as a patient representative of a specific group of patients. They may also be involved as experts together with different experts among the healthcare professionals, because of their knowledge, networks and ability to contribute. Today different organisations such as EUPATI offer training courses for patients as experts. In an article by Hartzler and Pratt (2011), the authors discuss the different input that patient’s expertise and clinicians’ expertise may contribute. For example, they demonstrate that where patient expertise contained personal topics carried through narrative-style action strategies and perspectives, clinician expertise was medical, knowledge oriented and prescriptive (Hartzler and Pratt 2011).

The different terms we have reflected on here are all normative statements. Normative statements are usually understood as positive or negative, good or bad or right or wrong. In relation to research or HTAs, the terms such as patients as experts, patient representative and patient advocate or patients as partners (Chap. 8) are valued as positive and good. Positive statements are often taken for granted becoming factual statements attempting to describe reality. Although normative statements and norms and their meanings are an integral part of human life, it is important that HTA researchers reflect on them and, for instance, ask questions such as ‘What types and kinds of meaning are inherent in the terms we use’.

3 Goals for Patient Involvement in HTA

The terms the HTA researchers choose to use and hence the choice of participants may influence the realization of goals with patient involvement in HTA including capacity building, patient empowerment and the knowledge gained. Although it is not always apparent why HTA organisations involve patients in HTA, there are a range of potential goals for patient involvement (Abelson et al. 2007). The choice of goals sets in motion a ‘different set of instruments and actors’ (Abelson et al. 2007, p. 40). Defining the goals for patient involvement will help define the type of participants needed to meet these goals. In HTA these goals may be:

  • Democratic goals seek to achieve more informed, transparent, accountable and legitimate assessment to improve the decision-making processes. As such the processes must be seen as fair and transparent drawing broad-based support from patients and the broader community. Transparent representation of stakeholder views safeguards against perceived bias in HTA and in particular bias due to political pressure in decision-making (Busse et al. 2002; Liberati et al. 1997; Gallego et al. 2011; Van der Weyden and Armstrong 2004). Furthermore, these goals draw on the notion that citizens have rights and responsibilities which extend to patients as citizens. Including citizens in decisions which will directly affect them is a fundamental tenet of participatory or deliberative democracy. Patients in HTAs are the citizens who will be most impacted by an assessment report and the ensuing decision, and therefore, it could be argued and have a special place in the HTA process

  • Scientific goals seek to promote a more robust and comprehensive scientific basis for HTA that incorporates social and ethical values (Chap. 2), as well as patients’ issues, lived experiences, outcomes and preferences

  • Instrumental goals are based on the belief that involving patients in HTA will improve the HTA assessment and make better quality decisions across all stages of an HTA. Understanding the lived experience with a disease may be crucial to the way in which an assessment is framed and conducted. For example, Street et al. (2008) and Facey et al. (2001) drawing on reports of patient experience demonstrate how the lived experience with diabetes may impact on the acceptability and uptake of technologies to detect and treat diabetic retinopathy

  • Developmental goals are tied to the move towards increased patient empowerment with patients as partners in their own care. Not involving patients in HTAs restricts the ability of patients to define the treatment choices available to them, a fundamentally disempowering and disenfranchising move. Increasing public understanding of health technologies and HTA strengthens the public’s and patients’ competence and capacity to contribute to health technology policy issues (Gauvin et al. 2015)

On occasion, depending on how and when patients are involved in the HTA process, these goals may conflict or give rise to bias or skewed assessment particularly since patients and caregivers involved in an HTA may have a vested interest in the outcome. This may be particularly the case where an expensive medicine only extends life by a few weeks or months or does not show long-term benefit. For this reason, patients, patient advocates or patient representatives might in some cases be viewed as ‘partisans’ in that they may have strongly held beliefs about a technology and therefore less amenable to engagement in ‘evidence-based’ discussion. In particular, a group of patients may be influenced by a persuasive individual or through the input of stakeholders such as clinicians or health technology developers. This does not necessarily mean that their views are unreasonable or that they should not be included in debate but rather that those patient groups may be unwilling to change their views in the light of clinical evidence. In mitigation, we should also consider that evidence-based medicine itself represents a particular world view and in some cases may neglect the needs and priorities of patients (Jensen 2004; Hansen 2004) and that many systems have processes for management of such conflicts of interest. Yet in some cases, the impact of partisan views may result in coverage for procedures or technologies which are not evidence based. For example, in the 1990s, the threat of litigation, often supported by state legislative mandates, forced many health management organisations in the USA to fund high-dose chemotherapy with autologous bone marrow transplants to patients with breast cancer. It was not until 1999 that it became apparent that the procedure provided no benefit compared to standard dose treatment and that in many cases, had caused unnecessary suffering (Deyo 2009). It is therefore important that HTA researchers make a well-argued analysis of the technology under assessment addressing the range of stakeholder perspectives. Partisan views may of course not only come from patients but also from other stakeholders such as clinicians, health technology developers and/or researchers. These kinds of stakeholders may also skew or bias the process.

4 Organising Patient Involvement

It is clear that the goals of patient involvement and the choice of participants (patients, patient advocates, caregivers, etc.) will have an impact on the organisation of patient involvement in HTA and the mechanisms for that involvement (Chap. 5). However, the implementation of patient involvement cannot only be concerned with goal setting and choosing the right term. It is also necessary to examine the role of leadership and organisational aspects, particularly the relationship between the participants and the researchers in the HTA or the members of an appraisal committee. Two Danish researchers have suggested a framework of three models of patient involvement, which we briefly describe here. The models are based on research literature about user involvement in healthcare (Holm-Petersen and Navne 2015). Each of the models reflects a different point of departure in relation to the person who is setting the agenda, that is, the doctor, the patient or both of these. We would argue that these three models may be highly relevant in relation to HTA and patient involvement to understand the relationship between the ‘patient’ and the HTA researcher (see also Chap. 5 for other models).

4.1 Service Minded Involvement

Service-minded involvement positions the patient as a consumer and/or the patient as the public. This model is in line with new public management ideas which emphasise a business approach to service provision including a focus on customer satisfaction. This may be a relevant model if HTA researchers, for instance, wish to investigate patients’ preferences, needs and expectations with respect to a treatment, a new device or a hospital service. We often see this kind of involvement in relation to patient satisfaction surveys, and it is the easiest way of organising patient involvement in HTA, because it is based on ‘professional management’ (Holm-Petersen and Navne 2015, p. 120). It is the professional—here the HTA researcher—who defines the relationship. However, as we discussed earlier in the chapter, it is not without problems particularly in positioning the patient as ‘consumer’ (see Sect. 3.2.3).

4.2 Supportive Involvement

The supportive involvement position may be understood in relation to patients as experts, patients as advocates and/or patients as patient representatives. This kind of involvement supports the patient as an active agent taking part in addressing the needs of a specific group for treatments, investigations and the use of devices (Holm-Petersen and Navne 2015, p. 121) and employing this knowledge in the assessment process. This way of understanding and organising patient involvement in an HTA prioritises patient autonomy, self-care and empowerment. This involvement approach places most of the responsibility on the patients assuming that they know best in relation to their own care. The role of the HTA researcher is then to support, inform and motivate the patient. This form of involvement is based on patient management and requires relational work between the HTA researcher(s), the patients and any relatives (Entwistle et al. 2012). However, as we discussed earlier in the chapter (see Sects. 3.2.1, 3.2.2, and 3.2.5), this way of understanding involvement also poses challenges related to which groups of patients will actually be able to be involved. Furthermore in relation to leadership roles and implementation possibilities, many challenges need to be considered, for instance:

  • HTA researchers may be reluctant to relinquish control and responsibility

  • Patients may come to the HTA process with limited skills to engage in problem definition and discussions due to training, disease burden, physical, psychological and or social-economic reasons (Entwistle et al. 2012, p. 122)

  • There may be ethical considerations in relation to leaving a decision to the patient/patient representative (Entwistle et al. 2012)

  • There may be an economic challenge related to face-to-face meetings instead of teleconferences (Holm-Petersen and Navne 2015)

4.3 Equal Partnership

The idea of equal partnership is especially drawn from the literature examining shared decision-making, where both the researcher and the patient contribute with relevant knowledge from their specific perspectives. The agenda is set by the patient and the HTA researcher together, and the relationship between them is based on equality. In relation to patient involvement in HTA, this can be connected to all the terms and to the three goals with patient involvement in HTA we have presented. However, as we especially discussed in relation to the notions of patients as partisans, an equal partnership approach also brings many challenges.

5 Conclusion

In this chapter, we have reflected on the intricate interrelationship between the terms used to describe patient involvement in HTAs and the goals and framing devices underpinning the involvement processes. We argue that whichever term HTA researchers use and whatever understanding and kind of patient involvement is chosen in relation to a specific HTA, it is essential to engage in critical reflection about terms, goals and the structural organisation of patient involvement in HTA. Part III of this book demonstrates clearly that patient organisations, HTA agencies and HTA researchers are increasingly sensitive to this issue and the needs to provide clear definitions and processes for patient involvement in HTA. This is important in order not to give rise to confusion and potentially dissatisfaction and mistrust between patients and HTA researchers and assessors. Involvement processes developed without due attention to these aspects risk being seen as ‘tick-box’ processes instituted to give the appearance but not the actuality of patient involvement. Patient involvement in HTA in the future must be more than well-meaning expressions and unreflective use of terms and goals. In particular, HTA researchers have a great responsibility and ethical challenge in being able to reach out to marginalised patients and patient groups who are hard to reach or involve, including patients with acute diseases, homeless people, people with low levels of education or communication difficulties, people with mental disorders and people without social networks. Qualitative and ethnographic research are especially well suited for such research (Chap. 12). Furthermore, there will often (if not always) be a power imbalance among the stakeholders in an HTA with the patients involved sometimes relatively poorly prepared and poorly funded for the debate compared with clinicians and health technology developers. However, as part III of this book demonstrates, training, mentorship and patient education have received increasing attention in an attempt to address the power imbalance or ‘asymmetry of knowledge’. We argue that there is a need for more systematic and rigorous research about the issues discussed in this chapter in order to effectively support the participation of patients and patient representatives in future HTAs.