Recognizing and Re-balancing Risk and Benefit for All HTA Participants

Many patient involvement activities increasingly resemble research, and expose patient participants to similar potential harms. For example, we have observed a growing pressure to conduct more numerous and more detailed interviews with patients. Patient interviewees are typically limited to describing their experiences in response to prepared questions, and may not have the opportunity to direct the conversation to topics they deem relevant to the policy decision, such as the trade-off between cost and improvement in quality of life. Patient-contributed information is analyzed cumulatively and presented in a written report, mediated through the perspective and priorities of the analysts and report authors. Dissenting views may or may not be included. As the report is discussed, patient participants have little opportunity to respond, react and offer an alternative vision.

This form of patient involvement is qualitatively different from the way other experts are engaged, and very closely resembles research participation. Research has a rich history of ethical theory guiding the recognition and re-balancing of cost and benefit. These ethical principles can guide patient involvement in HTA, under the principle that we should work to “do good” throughout the entire process of the HTA and not only at its conclusion (Reference Bogolub4). This means that we must strive to maximize benefit and minimize harm to patient participants throughout their involvement. “Doing good” is more than just “not doing bad.” It means not remaining complacent that, if the HTA is done well, the resulting policy decisions will benefit patients and society, but striving to create benefit to the participants of the HTA regardless of the outcome of the assessment.

This could be facilitated through the adoption of the principle of patient empowerment as an aim of patient involvement (2;5), which requires consideration of the principles of self-determination, autonomy, responsiveness, and ensuring the “voice” of participants is at the forefront (Reference Pittaway, Bartolomei and Hugman6). It means using the principle of respect for autonomy to create strong structures to facilitate the collection of continuous informed consent to participate (7). As we strive not to do harm, we should consider the concept of therapeutic misconception, that is, whether a patient has chosen to participate because she assumes that the results of participating will directly benefit her. This is especially important when engaging patients who use the drug or device being assessed. At a minimum, we should strive to ensure that participation in patient engagement activities does not cause physical, emotional, spiritual, or economic harm that outweighs the benefits of participating. Ideally, we should identify some additional benefit to patients, whether that is opportunity for capacity-building, learning, or system change.

Selecting and Engaging Patient Participants in an Ethical Way

There are two related parts to this challenge. First, there are many barriers to patient participation in HTA, meaning that only the most strongly motivated are likely to participate (Reference Fung8). Second, when only the most strongly motivated participate, ethical issues related to coercion and selection bias are introduced.

Barriers to patient participation in policy decision-making activities have been well-documented (9;10). Barriers include activities which take place during working hours, absent or limited remuneration for time (Reference Shah and Robinson11), as well as a requirement to interpret and respond to complex information (9;12). Other barriers include significant power differentials between patients and other participants (Reference Lopes, Carter and Street13), and the request to disclose potentially personal information to an audience who is not reciprocating with assistance, or similar levels of disclosure or vulnerability (Reference Shah and Robinson11). Considering these barriers, it is not surprising that most HTA patient participants tend to be of higher socio-economic status than the general population. By ignoring barriers to participation, we risk excluding all but the most motivated from the knowledge-sharing process (Reference Fricker14).

What inspires some patients to overcome these barriers, to give up their time and energy, and to assume the costs and risks inherent to participation? Research indicates that patients may be motivated to acquire meaningful social roles, overcome stigma, improve the health system, and achieve social justice for others (Reference de Freitas15). Beyond these reasons, we have met participants who are motivated by the unresolved tensions they carry from previous harm experienced within the healthcare system, and those who participate because they wish to access the technology being assessed. We have met patients who participate at their own cost and others who receive support from industry. All of these reasons for participation may motivate particular behaviours from the individual, prompting fear that bias or coercion may influence the information that person provides.

Instead of ignoring or aiming to eliminate bias, we re-conceptualize this issue, believing that the value of patient involvement lies in the unique experience and perspective each person brings (16;17). Indeed, we are asking for patient participation because of, not despite their personal experience. To do this, we must find ways to recognize the value of the variations in experience and perspective and incorporate these variations to support the production of a rich and useful HTA process. The approach of valuing the diversity of lived experience rather than seeking to minimize bias conflicts with the approach taken in areas of the HTA which rely on epidemiological methods and “objective” data (Reference Facey, Boivin and Gracia16). Others have suggested that each individual should carefully manage and support the overlap between their private preferences (self-interest) and understanding of what serves the wider public good (Reference Williamson18).

One strategy for achieving this is to borrow the concept of reflexivity from qualitative research (Reference Koch and Harrington19). Reflexivity is a way to identify, interrogate, and communicate the influence of personal perspective on qualitative research decision making. A reflexive conversation would be useful in HTA at the time of recruitment to check the fit between the person and the activity. Each participant would be informed of the nature and objectives of the activity and asked to reflect on their motivation to contribute, the perspectives they hope to convey during the activity, and the expectations they have for their involvement in the activity and its outcomes. This would not be a one-way conversation; the HTA team would also share their experience, goals, and conflicts relevant to the proposed activities. This conversation could be used to adapt or refine the activity to allow more transparent and effective patient engagement, and be included in analysis and reporting to improve the transparency of the results.

Increasing the accessibility and appeal of HTA activities may help ameliorate barriers to participation and the potential for selection bias and coercion when only the most motivated participate. Actions to reduce barriers to participation include: meaningful remuneration, activities outside of normal working hours, and attention to the physical and mental needs of people who may be sick, in pain, or living with functional limitations (Reference Robillard and Feng20). Constructing activities that do not require high levels of education or verbal eloquence, and those which feel emotionally and psychologically safer will increase accessibility. These actions follow the guiding principles of empowering patients to contribute by respecting and valuing their contributions, creating structures that mitigate potential risks, and ensuring transparency through the accessibility of information.

Decreasing the Influence of Power and Information Differentials between Patients and Other HTA Members

There are ethical issues stemming from the different types of information patients and other participants bring to an HTA collaboration and the resulting influence that information has within the assessment and decision-making process. For example, some participants bring clinical or economic research evidence to an HTA, whereas patients often bring information about their lived experience with the technology and health conditions. Differences in how this information is valued can contribute to power differentials between patients and other HTA participants, with the potential to undermine the contributions of patients.

Ethical patient involvement should consider how the HTA process will be responsive to the information provided by patients when it contradicts the outcomes suggested by economic and clinical evidence. In our experience, it is an enduring challenge for committees, decision makers, and researchers to compare and prioritize discrepant messages from clinical, economic, and lived experience information. The challenge of a holistic review of information is at the heart of the HTA endeavour, but all too often we have witnessed committees comment that, while the lived experience information is compelling, they must make their decisions based on the clinical or economic evidence. This can be upsetting to patients, who may feel superfluous and betrayed at the previous messages of how important their contributions are (21;22). They may wonder if the decision makers ever intended to take their testimony and evidence into account, or whether they are merely being used for their “patient status” rather than their insight (Reference Johannesen22).

If we are committed to ethical patient involvement, we must be responsive to the information provided by patients. Like other forms of evidence (including synthesized evidence from qualitative studies of patient values and experiences), information gathered through patient involvement activities should be considered essential and authoritative. Lived experience knowledge should not be privileged over other forms of evidence, but it should be equally considered. Hermeneutical marginalization, a type of epistemic injustice, occurs when interpretive resources are organized in a way that privileges one type of knowledge over another, or silences certain groups of people (Reference Medina23). It is disheartening but not surprising that evidence from the direct involvement of patients does not often compete with clinical or economic evidence produced, synthesized, reported, and interpreted by people who are deeply embedded in a particular evidentiary system. Dismantling the information differentials requires divergent forms of information to be presented in a way that is understandable and open to critique and response from all participants. This may mean translation of the findings from all HTA reports into plain language, so that those without specialized expertise may critique the assumptions or results of each report.