F. Patient and Consumer Involvement in HTA

The field of HTA increasingly recognizes the importance of involving patients and other “non-professional” or “non-expert” individuals. Indeed, these individuals often have developed expertise about their disease, condition, and care. Their involvement can provide perspectives and experiences that are not available from others, but are integral to HTA. Their participation in HTA also helps to fulfill HTA good practices for openness, transparency, and accountability. The value placed on patient participation and input should be commensurate with that of other participants in HTA.

In this context, the terms “patients” and “patient representatives” may also be used to mean patient advocates, consumer representatives, family members, non-professional caregivers, citizens, and the public. These individuals and groups may have different interests and bring different perspectives. While recognizing these differences, this section generally uses the broadly inclusive term “patients” to refer to one or more of these groups. Also, the terms “involvement,” “engagement,” and “participation” of patients are sometimes used interchangeably, although some users consider the term “involvement” to be more inclusive or integral to the HTA process than the other terms. This section uses all of those terms, and “involvement” is used broadly to encompass various levels of inclusiveness in the HTA process.

In most countries, patient involvement in HTA is still emerging, with considerable variation in how patients are recruited and supported for involvement, the HTA processes in which they are involved, the role they have in determining HTA findings, and how potential conflicts of interest are addressed (Kreis 2013). Across HTA organizations, patients are participating in more aspects of HTA, including various combinations of the following (see, e.g., Facey 2010; HTAi Patient and Citizen Involvement Interest Sub-Group, Good Practice Examples 2012):

• Servings as members of HTA boards, committees, and workgroups
• Identifying potential topics for HTA
• Early identification of potential target groups for HTA reports
• Setting priorities among HTA topics
• Identifying health outcomes and other impacts (economic, social, etc.) to be assessed
• Reviewing proposals or bids by outside organizations/contractors to conduct HTAs
• Submitting evidence for HTAs
• Reviewing draft HTA reports and recommendations
• Helping to design and prepare patient-friendly HTA report summaries
• Dissemination of HTA findings to policymakers, patient groups, and other target groups
• Evaluating the use of HTA recommendations

Patients are also increasingly involved in helping to set research agendas and design clinical trials and other primary data collection (e.g., selecting health outcomes and other endpoints for study that are of particular relevance to patients).

A systematic review of 142 studies published through 2011 of patient engagement in health care research more broadly found that most patient engagement occurred in agenda setting and protocol development, with less occurring during the conduct and translation of research. The review reported that patient engagement improved clinical trial enrollment and retention, design of study protocols, selection of relevant outcomes, and securing research funding. This review found no studies comparing methods of patient engagement. The main challenges identified included the time and funding to implement patient engagement and concerns by some stakeholders that patient engagement is “tokenistic,” i.e., not genuine (Domecq 2014).

According to a 2013 report of an international web-based survey, a majority of HTA organizations have formal public involvement or engagement activities, most of which involve lay (i.e., non-professional) representatives of organized groups or, to a lesser extent, individual patients, consumers, and community members. Most public involvement or engagement activities have entailed communication or consultation with those members of the public, while some activities have entailed direct participation in the form of citizens’ juries, consensus councils, and similar groups (Whitty 2013).

Through direct involvement in the HTA process, patients can share experiences, perspectives, and evidence that complement or even contradict evidence from such traditional sources as clinical trials and clinicians’ observations. Certainly, direct patient involvement can improve the understanding and accuracy of patients’ needs and preferences. Further, patient experience, perspectives, and evidence might address the burden of a disease or condition on patients, family members, and caregivers, including dimensions of health, pain, quality of life, social and economic burdens. Such patient input might also address the impacts on patients and others of health technologies, such as benefits and harms compared to alternative therapies, ability to work, difficulties in access or adherence, impact on their life, and economic impact, including any out-of-pocket costs, associated travel costs, income loss, and caregiver costs. Formal patient evidence can be obtained via qualitative or quantitative means through primary data collection or secondary analyses (Bastian 2011; Danner 2011; Facey 2010; Health Equality Europe 2008).

An analysis published in 2009 of a random selection of HTA reports (published in English or Scandinavian languages) from INAHTA-member agencies found that about one-third addressed patient-related issues (i.e., concerning patient views on the use or impact of technologies). These mainly addressed psychological issues such as patient worry and discomfort, ethical issues such as patient acceptance of the technology, social issues such as impact on patient daily life, effect on quality of life, and patient information. There was great variation in the comprehensiveness and methodological transparency of assessment of patient-related matters; only a minority of reports had explicit questions about patient issues (Lee 2009).

One of the more comprehensive efforts to gain patient input is that used for technology appraisals of the UK National Institute for Health and Care Excellence (NICE). Patients and caregivers provide input on: the personal impact of their condition; outcomes patients want the technology to improve; the impact of a technology on health outcomes, symptoms, physical and social functioning, quality of life; impact on family, friends and employers; ease of use, side effects and costs of the technology; patient preferences; subgroups who might benefit more or less from the technology; challenges to professional or researcher views; and areas needing further research (HTAi Patient and Citizen Involvement Interest Sub-Group, Good Practice Examples 2012; NICE Guide to the Methods 2013). Among other forms of patient and citizen input, NICE has a Citizens Council of 30 members that is demographically representative of the UK. The Citizens Council provides NICE with public perspectives on moral and ethical issues of which NICE takes account when producing its guidances (NICE Citizens Council 2014).

The Consumer Network of the Cochrane Collaboration involves health care consumers in the production of Cochrane systematic reviews. The most common role is consumer referee, which involves, e.g., commenting on protocols (plans) for new Cochrane systematic reviews, commenting on Cochrane reviews prior to publication, and helping to prepare or provide consumer perspectives on the plain language summaries of Cochrane reviews. Consumers may also help to search journals, disseminate information about particular Cochrane reviews, co-author reviews, and translate reviews and plain language summaries (Cochrane Consumer Network 2014).

Based on recent survey findings from HTA organizations, some of the main challenges to patient engagement in HTA include: lack of organizational capacity to involve patients, extensive time requirements, lack of agreed-upon methods for involving patients in HTA, lack of clarity about when in the HTA process to involve patients, lack of funding, and concerns regarding the scientific credibility of patient evidence (European Patients’ Forum; undated).

Greater experience with patient involvement in HTA and information sharing about these are leading to good practices for involvement of patients as members of boards, committees, and working groups as well as involvement and communications with patient groups and the broader public. These emerging good practices address such areas as those shown in Box X-1. While it may be impractical for most HTA programs to adopt all or even most of these immediately, those programs seeking to benefit from greater patient involvement can adopt more of them as time and resources permit. Also, greater involvement of patients and other target populations is gradually extending to development of evidence-based clinical practice guidelines, including to incorporate information about PROs, values, and preferences (see, e.g., Basch 2011; Brouwers 2010).

The HTAi Interest Sub-Group on Patient and Citizen Involvement in HTA has developed a set of resource materials, including examples of good practices for patient and citizen involvement from various HTA agencies, video and slide presentations introducing HTA and patient roles in it, a glossary, answers to frequently-asked questions, and others (see: http://www.htai.org/index.php?id=730#c2785).

Box X-1. Emerging Good Practices for Patient Involvement in HTA

• Ensure that all HTA processes are appropriately accessible, transparent, fairly applied, documented, and accountable
• Gain commitment of HTA organization to patient involvement
• Establish, review, and update policies for patient involvement in HTA
• Establish a dedicated unit or staff function for patient involvement, with explicit channels of communication and personal points of contact in the HTA organization
• Plan and provide for adequate budgets and other resources for patient involvement
• Conduct broadly accessible, transparent, and equitable (including relative to other types of participants) recruitment of patients to HTA boards, committees, and working groups
• Provide training and education for patient involvement in HTA, including appropriate materials, handbooks, reviewer/evaluation forms, etc.
• Provide training for HTA staff and other participants on working with patients and groups that represent them
• Ensure appropriately equitable rights (e.g., in voting) and responsibilities for patient members of HTA boards, committees, and working groups
• Provide adequate notice of meetings, due dates for input, and related scheduling information to patient participants to enable their sufficient preparation and timely participation
• Inform the variety of patient groups and other potentially interested groups about planned and ongoing HTA reports and ways to provide input to these reports
• Communicate in plain, patient-appropriate language, including in background materials and other documentation to ensure that patients are adequately informed for participation in HTA
• Provide patient participants with adequate financial support, including fees commensurate with those for other participants and reimbursement for travel and other out-of-pocket expenses (e.g., for child and other dependent care)
• Ensure that the context for patient involvement is welcoming and is not intimidating or coercive
• Make reasonable accommodations to ensure accessibility to HTA processes for patients and other participants who may have activity limitations (or are otherwise differently-abled)
• Provide HTA reports (and/or summaries of these) and other outputs in plain, patient-appropriate language and format, including for users with sensory (e.g., visual or hearing) limitations
• Provide for active, targeted dissemination of HTA reports and related products to patient groups
• Provide links from HTA organization web sites to those of relevant patient groups
• Organize special/targeted events, communications, or other activities for engaging individual patients and patient groups
• Seek to involve patient groups that are traditionally difficult to reach (e.g., ethnic minorities, underserved populations, stigmatized populations)
• Document/report patient involvement in HTA, including, e.g., levels of participation, impact on HTA deliberations, findings, and recommendations
• Provide recognition and feedback to patients regarding their participation

Sources: European Patients’ Forum (undated); Health Equality Europe 2008; HTAi Patient and Citizen Involvement Interest Sub-Group PIE Good Practice Principles 2012, Good Practice Examples 2013.