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Kohlhammer VW, author; Schildmann J, Buch C, Zerth J, editors. Defining the Value of Medical Interventions: Normative and Empirical Challenges [Internet]. Stuttgart (DE): W. Kohlhammer GmbH; 2021.
This chapter describes how patients and the social perspective can be included in health technology assessments (HTA). Utilising an HTA is a common procedure for defining and describing the value of a health technology for health policy decision-making. The HTA reports are produced on medical interventions to map the value of a health technology for various stakeholders and serve as a basis for information. The evidence from the HTA report should enable health policy decision makers to decide whether or not a health technology should be approved and/or reimbursed.
The integration of the patient and social perspective into the evaluation process of health technologies is highly relevant in the health policy context. It provides an important contribution to understanding what the value of an intervention is for users. At the European level, the HTA Core Model®, which was developed by the European Network for HTA (EUnetHTA), offers a framework for orientation on how to elaborate the domain.
The aim of integrating patients and social aspects into the HTA report is to understand the needs, values and preferences of the users of a medical intervention better. The domain provides information on important facets of the value of a health technology for users, including moral values and information needs. Relevant outcomes are particularly the motivation in favour or against the intervention under investigation, access to, experience with and expectations of the intervention or to better understand possible unmet needs.
Nevertheless, the current state of the research is that although the inclusion of the patient perspective is considered an important issue by HTA agencies worldwide, there is a lack of implementation. The integration of patients is currently still rare and insufficiently systematic. Moreover, in most cases, there is no evaluation of the additional benefit of including patients or the patient perspective.
There are various ways to include the patient and social perspective in the HTA process. They can be included by either secondary data analysis or collecting primary data. The evidence can be qualitative or quantitative. A systematic elaboration of the patients and social aspects domain requires more resources in the production, but there are good reasons to elaborate the patient perspective and social aspects comprehensively. As an example of an HTA report with a focus on patients and social aspects, we describe the HTA commissioned by a German HTA agency on integrative mistletoe therapy for patients with breast cancer in addition to standard therapy.
In this project, it was already foreseeable before the start of the HTA project that there might be only a few randomised controlled trials on the effectiveness of mistletoe therapy in patients with breast cancer. If the evidence on effectiveness as a basis is unclear, it is difficult to make a recommendation. Nevertheless, mistletoe extracts are in demand. In order to understand better why this is the case, the patients and social aspects domain has also been systematically elaborated in this HTA report.
Although it is not possible to estimate what influence the results on patients and social aspects will have on health policy decisions, it is evident that the elaboration has provided valuable indications as to the value of mistletoe therapy for users.
“What is the value of health interventions?” and “How to depict this value?” were the main questions that we discussed during the conference week. This chapter describes the empirical challenges that arise when patients and social aspects (SOC) are included in the evaluation of medical interventions in health technology assessments (HTAs). The topic is very relevant because it is important for decision makers to understand why patients, relatives and physicians want to have and use a medical intervention, sometimes even if the evidence base is insufficient to demonstrate clinical effectiveness or cost-effectiveness. Information on the experiences and expectations of health technology users in HTA reports can also be an important source of information for guideline authors to discuss these elusive aspects in guidelines.
After many years in which there was no internationally accepted definition of HTA, a joint task group, under the leadership of the International Network of Agencies for Health Technology Assessment (INAHTA) and Health Technology Assessment International (HTAi), has managed to create a definition of HTA for which there is a consensus on a global level. The new definition of HTA is as follows:
HTA is a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to inform decision-making in order to promote an equitable, efficient, and high-quality health system. (O'Rourke et al., 2020, p. 2)
Four notes were added to clarify the meaning and scope of the definition in order to find a consensus among all institutions involved. The first note clarifies the broad scope of the term ‘health technology’:
A health technology is an intervention developed to prevent, diagnose or treat medical conditions; promote health; provide rehabilitation; or organize healthcare delivery. The intervention can be a test, device, medicine, vaccine, procedure, program, or system. (O'Rourke et al., 2020, p. 2)
The second note emphasises that the process has to be “formal, systematic, and transparent, and uses state-of-the-art methods to consider the best available evidence” (O'Rourke et al., 2020, p. 2).
Regarding the definition of the value of medical interventions, the third note emphasises the many facets of the concept of value, which is composed of various dimensions:
The dimensions of value for a health technology may be assessed by examining the intended and unintended consequences of using a health technology compared to existing alternatives. These dimensions often include clinical effectiveness, safety, costs and economic implications, ethical, social, cultural and legal issues, organizational and environmental aspects, as well as wider implications for the patient, relatives, caregivers, and the population. The overall value may vary depending on the perspective taken, the stakeholders involved, and the decision context. (O'Rourke et al., 2020, p. 2)
The fourth note refers to the point in the life cycle of the evaluation. Health technologies are assessed via HTAs prior to or during their market access or within the process of re-evaluating an intervention which is already implemented as far as the stage of disinvestment of a health technology, in order to summarise the evidence currently available as a basis for decision-making by health policy decision makers (O'Rourke et al., 2020).
Before we describe how including SOC into an HTA report enables extra value for decision makers and patients in the end, it is important to understand how HTA reports are compiled. The systematic and transparent way of conducting an HTA is described in guidelines and handbooks. These documents usually also define the basic structure of the report. In addition, the methodology is mainly predefined and standardized.
Best practice recommendations of the European Collaboration in HTA (ECHTA) Working Group 4 (Busse et al., 2002) suggest that an HTA should include a comprehensive background section describing the nature of the health problem with information on the burden of the disease, its epidemiology and who is the target population. Furthermore, the technology under assessment should be described and it should be stated at which point the technology stands in the product life cycle. In addition, alternative treatment options should be mentioned, including information on which treatment is currently the standard in practice.
There are five main domains that are relevant to consider (Busse et al., 2002):
Recommendations from 2002 on which aspects to include in HTAs already contained psychological, social and ethical aspects as the main outcomes and an important part of HTA. Busse et al. (2002) stated that the correct way of approaching these aspects depends on the knowledge that is already available and may also comprise qualitative data or knowledge from other disciplines. The hierarchy of study designs describing the levels of evidence is not applicable to this domain (Busse et al., 2002).
A more recent methodological framework is the HTA Core Model® (EUnetHTA, 2016a), which is a further development of the suggestions of the European Collaboration in HTA Working Group 4. The EUnetHTA HTA Core Model® (EUnetHTA, 2016a) was developed and refined during the >EUnetHTA Project (2006–2008) and the two follow-up projects, EUnetHTA Joint Action (2010–2012), EUnetHTA Joint Action 2 (2012–2015), and during Joint Action 3 (2015–2021) (EUnetHTA, 2018). The project aim was to achieve a common understanding within Europe of what is a good methodology to generate an HTA report to facilitate collaborations across countries and enable efficient sharing of results.
Domains of the HTA Core Model® are (EUnetHTA, 2016a):
Health problem and current use of technology (CUR)
Description and technical characteristics of technology (TEC)
Safety (SAF)
Clinical effectiveness (EFF)
Costs and economic evaluation (ECO)
Ethical analysis (ETH)
Organisational aspects (ORG)
Patients and social aspects (SOC)
Legal aspects (LEG)
It is noteworthy that the ethical analysis and the SOC part are divided into two different domains in the HTA Core Model®.
There are two types of HTA reports: rapid relative effectiveness assessments (REAs) and full HTA reports. An REA only includes the domains CUR, TEC, SAF and EFF, but full HTA reports comprise all nine domains when applicable to the health intervention assessed (EUnetHTA, 2020).
The HTA Core Model® (Version 3.0) (EUnetHTA, 2016a) is the current framework for European HTA collaboration. The production of joint REA reports should support a more efficient use of resources by avoiding duplication of work, because the same new health technologies are coming to the market in many European countries, which will usually be assessed by national HTA agencies. The joint REA reports are intended to be taken up in the national assessment.
European collaborations do not have the objective of making health policy decisions on a European level. The aim is for recommendations for reimbursement decisions to be made exclusively at a national level and that each country decides independently which services are to be integrated into the catalogue of healthcare services. The collaboration shall solely facilitate the exchange of evidence between HTA agencies that is applicable to different countries, such as evidence on effectiveness and safety. Domains, which can be different for different countries, will mainly be compiled for each country separately. It is suggested that a SOC domain is included on a national level.
The domain “Patients and Social aspects (SOC)” in the HTA Core Model® provides guidance on methodological issues arising when compiling the SOC domain (EUnetHTA, 2016b).
There are two main ways of integrating information on patients’ aspects: (1) to summarise existing evidence in a secondary data analysis or (2) to gain primary data by conducting interviews or a survey. Both primary and secondary data analysis can be qualitative and/or quantitative. There are numerous different guidelines on how to conduct an HTA because each national HTA authority has its own handbook on how to conduct an HTA report properly.
In addition to different options of generating evidence on SOC, it is important to use a systematic and methodologically robust process for synthesizing and reporting the results. The SOC domain should be elaborated comparable to other domains to facilitate the integration of the domain into the HTA structure. However, neither a domain on social or ethical aspects nor qualitative evidence have been integrated into HTA reports in a standardized manner in the past, as described in section 03.1 (Merlin et al., 2014).
There was a recommendation published by EUnetHTA in 2019 on how to integrate patient input in REAs (EUnetHTA, 2019), and this document is updated regularly. There are also initiatives that are constantly working to improve patient and public involvement and publishing guidance, such as the book by Facey et al. (2017) or the framework by Abelson et al. (2016).
How the integration of SOC is to be carried out according to the EUnetHTA Core Model® is described in more detail in sections 3.2, 3.3 and 3.4.
Before we describe how SOC can be included in an HTA report and present an example of how we have integrated the domain into an HTA on the integrative mistletoe therapy in breast cancer patients, we explain why these aspects should be included.
Our HTA report is a practical example of how patient experiences influence the value of a technology.1 In this chapter, we will not go into more detail about whether and why the patients and social perspective should be included, but we refer to the clear statement of the HTAi interest group for patient and citizen involvement, which sees great potential in the integration of the SOC domain. The interest group for patient and citizen involvement at HTAi states its position clearly on their website: “Vision: Patient and citizen perspectives improve HTA” (HTAi, 2020a).
In general, SOC should be integrated into HTA to understand the perspective of people affected by the health technology. The domain and the term “people affected” usually refers to patients (if necessary, represented by their parents or other relatives), individuals and caregivers (IQWIG, 2017b). Caregivers come from the private environment of the patient, as family or friends, and are not healthcare professionals paid for the service (EUnetHTA, 2016b). The SOC domain provides information on what motivates patients to claim a treatment or intervention, respectively, what motivates individuals to use preventive interventions. Experiences regarding the impact of a health technology on everyday life and the perceived value of the quality and the benefit of the health technology can only be provided by those people affected (EUnetHTA, 2016b). Information on issues which promote or prevent a demand can also identify underlying unmet needs of the patients. Unmet needs can be diverse and may relate to information or social needs, uncertainty on the effectiveness and safety of a treatment, or limited access. The unmet needs are sometimes easy to meet, for example, if a patient wants to have the opportunity to discuss treatment options with a trusted doctor.
The SOC domain does not only include information on the motivation for using a certain health technology but also on how information is communicated to patients or where patients’ needs, such as the need for information, are not met within the healthcare system (EUnetHTA, 2016b). Good counselling on a health technology, for example, improves the health literacy of the patient so that they can be a responsible decision maker regarding their own treatment process.
In addition to informing the patient about individual risks regarding the treatment, each therapy planning needs a benefit-risk assessment on an individual level before suggesting a therapy to the patient (Ernst and Klein, 2017). In case unmet needs arise that are related to patient information, communication, access to a treatment or other topics, the SOC domain would be the appropriate place to provide information on these aspects if available and suggest how to react adequately.
Since decisions on the implementation and refunding of health technologies are increasingly complex and have to be made under scarce resources, some countries (e.g. Australia) try to expand public engagement in order to create more transparency (Wortley et al., 2017).
A mapping review at EU level summarises the HTA processes of HTA agencies in Europe and describes similarities and differences in the approach leading to the creation of an HTA product (Chamova, 2017).
The German HTA agency Institute for Quality and Efficiency in Healthcare (IQWIG) involves patients in the dossier assessment (IQWIG, 2017a), in the suggestion (called “IQWIG ThemenCheck Medizin”) and selection of topics for HTA, and offers the public the chance to react in written form on study plans and preliminary HTA reports (IQWIG, 2017b).
However, what is usually missing in HTA across different HTA agencies is the inclusion of the patients’ perspective in the HTA report in the form of a literature review, as is done in other domains. To summarise the relevant evidence on SOC helps to represent the patients’ perspective better.
Lehoux and Williams-Jones (2007) see an important responsibility of HTA agencies is to also constitute the diversity of social and ethical issues in addition to evidence on clinical and economic questions. Already addressing aspects as social justice and transparency in the HTA report helps decision makers to bring together public expectations, (moral) values, and evidence on effectiveness, safety and cost-effectiveness (Lehoux and Williams-Jones, 2007). A summary of existing evidence on the elusive topic of SOC makes it easier for decision makers to make sure they do not accidentally ignore important aspects.
The SOC domain in the HTA Core Model® contains eight assessment elements (see Table 1), which constitute the relevant outcomes for the domain. These assessment elements are assigned to three different thematic groups: patients’ perspectives (which also include caregivers), social groups’ aspects and communication aspects.
Assessment elements for the patients and social aspects domain according to the EUnetHTA HTA Core Model® (EUnetHTA, 2016b, p. 348).
Some aspects may be irrelevant for the intervention under assessment, but each aspect should be checked for relevance. If it is not possible to answer all research questions, it is important to state which questions could not be answered based on the existing literature (EUnetHTA, 2016a).
We will first consider the current situation since the extent to which SOC are included depends heavily on the commissioning HTA authority. Subsequently, we describe briefly how the creation of the SOC domain, as described in the HTA Core Model®, is carried out methodically as a systematic review.
Despite the recommendations above, social aspects have often not been treated as a separate domain in the HTA report in the past. Two surveys – one paper-based survey in 2010 and one web-based follow-up survey in 2013 – were conducted within the 53, respectively, 56 member organisations of INAHTA to find out more about the core dimensions that are regularly used by HTA agencies (Merlin et al., 2014). A total of 45 (approximately 80 per cent) agencies participated in at least one survey. The participants were asked about their practice of including the nine different core domains – similar to those stated in the HTA Core Model®. The question should be answered regarding how likely it is that the institution addresses the respective area in the HTA product. An HTA product is, for example, a full HTA report, a rapid review or a mini-HTA. Regarding social aspects, 50 to 74 per cent of institutions answered that they generally include social aspects in full HTA reports (number of answers: 31). The inclusion of social aspects for HTA products with a smaller extent, such as a rapid review or a mini-HTA, is less than 25 per cent and may also be zero (Merlin et al., 2014).
Many HTA agencies in developed countries have standardized processes for the involvement of patients and the public. Patient engagement is described as important by several HTA societies, for example, HTAi, INAHTA or EUnetHTA (EUnetHTA, 2016b; HTAi, 2020b; Menon and Stafinski, 2011). Although patient and public participation (PPI) has increased in recent years, the effects of these initiatives were hardly evaluated by HTA agencies for a long time.
Nowadays, the involvement of patients is more established. A recent discussion paper published by Single et al. (2019) reports the opinion of experts on patient involvement from HTA agencies on the development and impact of patient involvement in HTA. Patient involvement had a positive impact in the institutions participating in terms of clarifying uncertainties and complementing clinical and economic evidence. The conclusion of the experts is that, so far, patient involvement has made a difference, especially in the recommendations on when and how the technology can be used (Single et al., 2019).
A survey of the Patient and Citizen Involvement Group of HTAi with 15 responding HTA agencies (response rate of 27.8 per cent) tried to evaluate the potential usefulness of PPI initiatives. Fourteen respondents involved patients and ten involved the public in HTA. They recruited via the INAHTA and personal contacts. Approximately half of the institutions evaluated their PPI activities and used the insights to improve the activities and react on education and training needs (Weeks et al., 2017). These results could indicate a gap in evaluating PPI initiatives in HTA agencies in general or that PPI initiatives are still neglected.
A specific example of the lack of including SOC in HTA, even when these aspects would be relevant for the assessment of the value of the intervention, is a knowledge synthesis by Potter et al. (2009) on how ethical, social and legal aspects (ELSI) are included in HTA for prenatal/preconceptional and newborn screening. The literature review and an associated workshop with different HTA agencies in which the findings were discussed showed that ELSI and public health ethics are highly relevant in the context of applying the screening intervention when they were taken into account. A second result was that ELSI have to be evaluated on a national level. The diversity between countries concerning cultural values, stakeholder communities and contextual factors need a flexible approach regarding how to address ELSI. Approaching ELSI should be adapted to each target population of the HTA (Potter et al., 2009).
The authors of the publication criticise omitting elaborations on ELSI in HTA reports because it does not solve the problem that these aspects have to be considered when making the decision on reimbursing an intervention or permitting market access. It simply shifts the assessment of ELSI to a later stage in policy decision-making (Potter et al., 2009). The more transparent the process is from excerpting study results on ELSI to the conclusions within an HTA report, the better health policy decision-making committees can justify their decision towards the public.
According to the SOC domain of the EUnetHTA HTA Core Model®, the methodology of how to elaborate the domain in a good practice depends on the existing knowledge on relevant outcomes. The relevant outcomes related to the health technology under assessment are stated in Table 1 (EUnetHTA, 2016b).
The process of defining the appropriate method starts with a literature search. If a current systematic review of patients’ and social aspects has not yet been published, one has to be conducted. If the existing literature does not cover relevant aspects, an additional primary study can be conducted to solve the problem of missing information. Asking patient groups or patient organisations, for example, in focus groups is also an option (EUnetHTA, 2016b).
In practice, HTA agencies have to deal with limited budgets and the extent of patient involvement and how the domain shall be elaborated will be defined by the HTA agency that commissions the HTA.
The quality of the studies and reporting of the results within the publications for all studies included in the systematic reviews as part of an HTA has to be appraised to judge the strength of the evidence and enable transparency. One challenge is the huge amount of appraisal tools available for the critical appraisal of studies – as well as for qualitative and quantitative studies. A review conducted by Crowe and Sheppard in 2011 included 44 critical appraisal tools, of which five (11 per cent) included a comprehensive guideline on how to use the critical appraisal tool and how it was developed (Crowe and Sheppard, 2011). There was no test for reliability for 77 per cent of the appraisal tools and no information available about a validation of the tool for 25 per cent. Hence, the authors advise was to be careful when selecting and using an appraisal tool (Crowe and Sheppard, 2011). A mapping review on critical appraisal tools for qualitative studies shows a similar situation. Munthe-Kaas et al. (2018) conducted the mapping review with the aim of finding a critical appraisal tool that would be suggested for use in the GRADE-CERQual (Grading of Recommendations, Assessment, Development and Evaluations – Confidence in the Evidence from Reviews of Qualitative Research) assessment (Lewin et al., 2018). A total of 107 tools were identified, of which 40 have been published since 2010 (Munthe-Kaas et al., 2018). New tools do not usually refer to older tools or justify why a new tool was needed. The authors of the review conclude:
the plethora of tools, old and new, indicates a lack of consensus regarding the best tool to use, and an absence of empirical evidence about the most important criteria for assessing the methodological limitations of qualitative research. (Munthe-Kaas et al., 2018, p. 1)
The Cochrane Qualitative and Implementation Methods Group (Noyes et al., 2018b) suggests using the checklists of the Critical Appraisal Skills Program (CASP) (CASP, 2018a, 2018b, 2018c, 2018d, 2018e).
There is no appraisal tool available in the CASP program for cross-sectional studies. Alternatively, two other checklists could be used: one tool is developed by Downes et al., the AXIS critical appraisal tool (2016), and the other is developed by the National Heart, Lung and Blood Institute (2020).
There are several methodological options to analyse the data, but we only present one option, which is commonly accepted in the literature (Higgins and Green, 2011; Noyes et al., 2018a). Both the qualitative and the quantitative research paradigm provide relevant information on SOC. Both types of data can be analysed separately and presented in the same chapter of the HTA report. If this option is chosen, the analysis of quantitative data is similar to evidence in other domains. Evidence is summarised, including relevant inferential and descriptive statistics, and presented in tables and narrative summaries.
Qualitative evidence is analysed by qualitative thematic synthesis, including a coding process, which is conducted by two independent researchers. “Coding” is the first step in a text analysis. The researcher reads the text line by line and summarises the meaning of a thematic unit in one word or phrase. This phrase is called “code”. Both researchers must check the consistency of the interpretation of codes to make sure that the code depicts the meaning of what is said in the text. This check of consistency of the codes and the meaning of the text is repeated until both researchers agree. The second step is to deduce descriptive themes from the codes. This means that codes that are related to the same topic are merged into one descriptive theme. In a third step, the researchers must interpret the descriptive themes and make judgments on how the content of the descriptive themes can answer the research questions (Thomas and Harden, 2008).
A focus on the SOC domain is still a special feature in HTA reports. One example for the elaboration of the SOC domain is the HTA report conducted recently on “Safety and efficacy of additional treatment with mistletoe extracts for patients with breast cancer compared to conventional cancer therapy alone”, commissioned by the German Institute of Medical Documentation and Information (DIMDI) (Schnell-Inderst et al., 2020, under review). This report will presumably be public by the end of 2020. Previous assessments of mistletoe therapy in cancer patients hypothesized that there will be few studies available to assess safety, effectiveness and costs of a mistletoe therapy (Horneber et al., 2008; Kienle et al., 2009; Lange-Lindberg et al., 2006). However, mistletoe therapy has been used for many years. The section on SOC was elaborated in detail to understand why patients and doctors use additional mistletoe therapy and what they expect from it.
The HTA reports commissioned by DIMDI are often based on secondary data and systematic literature reviews or, in some cases, decision-analytic models. The contracting authority limits the sources of evidence to published literature, which has to be synthesized in a systematic review. We compiled the information regarding the SOC domain through a secondary data analysis of qualitative and quantitative studies.
A systematic literature research was performed to collect data for the secondary data analysis. A comprehensive search in medical databases (MEDLINE, EMBASE, Cochrane Library, the databases of the Centre of Reviews and Dissemination [DARE and HTA database], and CINAHL) was complemented by a search in databases from other disciplines, such as psychology, economics and social science, because medical databases probably do not contain all the relevant literature (Wessels et al., 2016). The database search was supplemented by a comprehensive search on the internet. The “Grey Matters” tool, published by CADTH Information Services, has been used to identify grey literature (CADTH, 2015). Search terms included various different aspects related to the SOC domain and aspects related to complementary and alternative medicine (CAM) (Franzel et al., 2013).
The inclusion criteria for the systematic review were:
Population: patients with mammary carcinoma, their family caregivers, dependents, physicians and nurses
Intervention: therapy with mistletoe extracts (subcutaneous, intravenous, in tumour tissue, in the gap between the lungs and pleura) additional to usual care (this has been extended to complementary and alternative medicine since there was scarce evidence on mistletoe therapy, but mistletoe therapy had to be reported separately)
Outcomes: attitude and expectations towards, acceptance of, experiences and satisfaction with, knowledge about, access to and use of adjuvant mistletoe therapy; type, extent and evaluation of patient information and evaluation of the doctor-patient communication concerning adjuvant mistletoe therapy
Study type: all study types were considered.
The check for eligibility of the papers was conducted by two independent authors. If the inclusion or exclusion of references was unclear, the authors solved discrepancies by discussion.
The study quality for the cross-sectional and qualitative studies has been assessed using the checklist for qualitative studies developed by the CASP Program (CASP, 2018c), as suggested by the Cochrane Qualitative and Implementation Methods Group Guidance (Noyes et al., 2018b). Since there was no checklist available for cross-sectional studies when the systematic review was conducted, the checklist for cohort studies has been adapted by the research team of the HTA report (CASP, 2018b).
We analysed and reported the studies as described in section 3.4. The two qualitative studies were analysed in a thematic synthesis (Thomas and Harden, 2008) and quantitative evidence was summarised in tables. Both qualitative and quantitative results were narratively described, and the themes derived from the coding process were put together with the results from quantitative studies.
Of the 302 publications screened in title and abstract, 84 were checked for eligibility in full text. A total of 13 publications were finally included in the analysis on SOC. Ten of the studies are from a patients’ perspective, including two qualitative (Brandenberger et al., 2012; Gschwendtner et al., 2016) and eight quantitative (Drozdoff et al., 2018; Fasching et al., 2007; Fremd et al., 2017; Huebner et al., 2014; Moschen et al., 2001; Schönekaes et al.; 2003, Tautz et al.; 2012, Templeton et al., 2013) studies. Three studies focus on the physicians’ perspective (Kalder et al., 2001; Klein et al., 2017; Muenstedt et al., 2000).
All of the relevant outcomes that were stated in the inclusion criteria in section 4.1 have been addressed in the analysis within the HTA report. The main difference between our outcomes and those suggested in the SOC domain in the HTA Core Model® is a fourth main topic. In addition to patients’, social groups’ and communication aspects, the new topic is physicians’ aspects. The analysis showed that physicians also had a broad range of experiences with, beliefs about and motivational causes in favour of or against mistletoe treatment.
In this book chapter, we can only address the results and challenges of the HTA report very briefly. For more information, please see the full HTA report by Schnell-Inderst et al. “Safety and efficacy of additional treatment with mistletoe extracts for patients with breast cancer compared to conventional cancer therapy alone”, which is currently under review and going to be published by the DIMDI at the end of 2020. A summary in English will be available (Schnell-Inderst et al., 2020, under review).
The results of the SOC domain deliver valuable insights regarding the attitude towards and motivation in favour of or against supplementary mistletoe treatment. The results can enable decision makers to track medical decision-making on an individual level between physician and patient and enable responsible institutions to react to unmet information needs.
We faced some challenges related to the secondary data analysis on SOC related to the mistletoe therapy in breast cancer patients. Firstly, we had a problem due to the fact that mistletoe therapy was only addressed as one complementary treatment option of other complementary and alternative medicine in all quantitative studies from a patients’ perspective. Therefore, we had to widen the scope of the intervention to integrative therapies in breast cancer patients. We included complementary and alternative medicine studies as long as mistletoe therapy was mentioned separately. This led to the next challenge, because the term “integrative medicine” has not yet been used in English publications but only “complementary medicine” or “complementary and alternative medicine”. Unfortunately, up to now, the concept of complementary and alternative medicine is not clearly defined (Ernst and Klein, 2017) and comprises diverse supplementary treatments. Each study team defined for themselves which complementary and alternative medicine were to be included. This complicated the comparability and transferability of the results to the mistletoe therapy.
At this stage, it is not yet possible to estimate the impact of additional outcomes from the SOC domain. However, it has been confirmed that the evidence on efficacy, safety and cost-effectiveness available does not allow for a clear recommendation. The reasons for and against the use of integrative mistletoe therapy reflect the uncertainty among those treating and patients. At the same time, the results on SOC make it comprehensible why mistletoe extracts are, nevertheless, used and with what aim. Doctors might also find results from the SOC domain helpful in advising patients when they see that colleagues are equally unsure what to recommend and perhaps even that treatments are sometimes being performed with the sole purpose of motivating the patient. We are not saying that this cannot be a legitimate reason for using a form of therapy, but only that it is important to be aware of it.
In summary, in the authors’ opinion, it made absolute sense to evaluate the SOC domain in detail in the HTA on integrative mistletoe therapy in breast cancer patients. The motivation of the patient or physician for the application of the mistletoe therapy is based on a broad spectrum of reasons, which represent the value for patients in various ways. If the evaluation had been based solely on the clinical and cost-effectiveness of mistletoe therapy, the value of mistletoe therapy for patients would probably not have been sufficiently understood.
A justification why patients’ experiences should be taken into account when defining the value of a medical intervention is provided in another chapter of this book written by Ubels. He describes the value of additional information on patients’ experiences on a theoretical level, in the context of the capability approach and how this could be integrated in practice. Including the patient perspective in decision-making is also the subject in Napiwodzka's ethical discussion of potentials and challenges for the Polish healthcare system in this volume.
This is an open access article licensed under a Creative Commons Attribution 4.0 International License, which permits unrestricted use, distribution, reproduction and adaptation in any medium and for any purpose provided that it is properly attributed.
Monographs, or book chapters, which are outputs of Wellcome Trust funding have been made freely available as part of the Wellcome Trust's open access policy
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