What do stakeholders expect from patient engagement: Are these expectations being met?

Abstract Background Meaningful patient engagement (PE) in medicines development and during the life cycle of a product requires all stakeholders have a clear understanding of respective expectations. Objective A qualitative survey was undertaken to understand stakeholder expectations. Design The survey explored 4 themes from the perspective of each stakeholder group: meaning, views, expectations and priorities for PE. Participants were grouped into 7 categories: policymakers/regulators; health‐care professionals (HCPs); research funders; payers/purchasers/HTA; patients/patient representatives; pharmaceutical/life sciences industry; and academic researchers. Results Fifty‐nine interviews were conducted across a range of geographies, PE experience and job seniority/role. There was consensus across stakeholders on meaning of PE; importance of promoting PE to a higher level than currently; need for a more structured process and guidance. There was little consensus on stakeholder expectations and roles. Policymakers/regulators were expected by others to drive PE, create a framework and facilitate PE, provide guidelines of good practice and connect stakeholders, but this expectation was not shared by the policymakers/regulators group. HCPs were seen as the link between patients and other stakeholders, but HCPs did not necessarily share this view. Discussion and conclusions Despite broad stakeholder categories, clear themes emerged: there is no “leader”; no stakeholder has a clear view on how to meaningfully engage with patients; there are educational gaps; and a structure and guidance for PE is urgently required. Given the diversity of stakeholders, there needs to be multistakeholder collaborative leadership. Effective collaboration requires consensus on roles, responsibilities and expectations to synergize efforts to deliver meaningful PE in medicines life cycle.


| BACKG ROU N D
There is a growing consensus across stakeholder groups of the importance of patient engagement (PE) in medicines development, and during the life cycle of a product ("medicines life cycle").
There are an increasing number of efforts to achieve this. [1][2][3][4][5][6] in the research and development setting especially has received much focus with the development of frameworks or guidance. [7][8][9][10][11][12][13] There are also guidance or frameworks at other milestones such as in health technology appraisal, benefit-risk assessment [14][15][16][17] and in value determination. 18 The issue of definition and terminology of PE and patient centricity has also highlighted the need for a common understanding to facilitate multistakeholder teamwork. [19][20][21][22][23] Crucially, there is a need for a practical PE model that can be assessed to demonstrate the value of PE, in terms that each stakeholder group recognizes, to encourage acceptance and implementation. 11,[24][25][26][27] A recurrent theme across all these examples is collaboration to reach the common goal. This requires core elements or principles to be agreed across groups, including recognition and alignment of the

| S TUDY DE S I G N AND PRO CE SS
The study was designed to explore 4 key themes from the perspective of each stakeholder (defined in Appendix S1) (i) meaning of PE in the context of patient-focused medicines development, (ii) views on, and value of PE, (iii) expectations of each stakeholder group-what each group believes their role to be and what each stakeholder group expects from other groups-and degree of alignment in expectations within and between stakeholder groups and (iv) next steps and priorities for PE ( Figure 1). were conducted and feedback used to refine interview questions and approach.

| Identification and categorization of interviewees
Stakeholders were grouped into 7 main categories: patients/patient representatives (termed "patients"); health-care professionals (HCPs); policymakers/regulators (termed "policy"; payers/purchasers (termed "payers"); pharma/life sciences industry (termed "industry"); academic researchers (termed "researchers"); and research funders. Note, definitions of stakeholder groups such as "policy" or "payers" may vary internationally. The categories and definitions of stakeholders were adapted from Deverka et al 7 (Appendix S1). Interviewees were identified by the SEWG and MMP using Quota and Snowball techniques to achieve a broad reach across geographies, experience of PE and job role.

| Geographical location
The target of one-third, respectively, of all interviewees to be from different geographical regions was mostly achieved within stakeholder groups (Figure 2).

| Level of experience in patient engagement
Participant's experience of PE was categorized as Experienced, Some Experience and No Experience based on the interviewees own perception. Good representation was achieved across all groups: the level of experience varied between stakeholder groups, and no stakeholder group represented just one single level of experience (Appendix S3).

| Seniority within the organization
The job seniority of interviewees per stakeholder group was assessed and categorized as Executive/Senior-level Officials, Mid-Level Managers and Other Professionals (according to the US Equal Employment Opportunity Commission, Appendix S2). This categorization was not relevant for the patient stakeholder group who were instead identified as either being a patient or representing a patient organization, regardless of seniority. Across the survey, representation was achieved from all seniorities; however, due to their limited availability, Chief Executive Officer (CEO) roles do not feature in all groups. Appendix S3 shows a summary of stakeholders included in the analysis to provide a context for the interviewee responses.
Interviewees were asked about: the meaning, views and importance of PE; relationships, roles and responsibilities of stakeholders in PE; and priorities and needs in PE.

| Meaning
"How would you define the phrase patient-focused medicines development?" (59 answered) Although stakeholders' definitions varied, the underlying sentiment was consistent across stakeholder groups that patient-focused medicine development is involving patients in every step. It was described by an industry interviewee as "… a process through which patients are part of the idea, design, execution and feedback loop of medicines development from pre-discovery through to launch of medicines onto the market."

| Meaning
"Does the term patient 'engagement' or 'involvement' best capture patients' needs at the heart of medicines development?" (45 answered) Although there was no clear preference towards the terminology and language used, stakeholders were aligned on the need to be clear what is meant regardless of nuances of language. Generally,

Definition
What does patient-focused medicines development mean?
Does 'patient engagement' or 'patient involvement' best capture patients' needs in medicines lifecycle?

Language Priorities
What are the priorities for all stakeholder groups?
Are there any skills or knowledge that would help stakeholders involve patients more meaningfully?

Views (ii) Importance
What is the importance of patient engagement to stakeholder groups now and what should it be?

Expectations (iii)
Relationships What are the current and desired relationships between stakeholders?

Roles
What do stakeholders think their own role and others' in patient engagement is?

Goals
Do stakeholders have different goals from patient engagement?

Industry perceptions
What are stakeholders' perceptions on the industry involving patients in medicines development?
interviewees cared less about terminology and more about function.
Each term (engagement, involvement, participation, activation, consultation) potentially has different nuances or interpretations. There may be cultural and geographical differences, as well as language barriers in the interpretation of the terms "engagement" and "involvement." A researcher interviewee noted that "…'engagement' in some languages may mean there is a fee for service."

| Views
"How important is patient engagement to your own stakeholder group now and how important should it be?" (44 answered) Overall, interviewees thought that PE should be more important than it is now and that their stakeholder group is not doing enough to address the needs of patients. The importance of PE to all groups was assessed in terms of its current level of importance and how important it should be in the future (on a scale of 1-10, with 1 being lowest and 10 highest level of importance). The current importance scored an average of 4.8, however when asked to assess what it should be this rose to 8.8 ( Figure 3). Interviewees recognized that PE is a key aspect to drug development, but the degree to which they were willing and able to accept an active role in PE differed. In addition, there were other agenda items such as cost and clinical effectiveness (payers/policy), medical education and scientific discussion (policy/industry), and the number of people whose quality of life can be improved (policy) that scored as more important than PE in certain stakeholder groups.

| Views
"What are your thoughts on patient engagement in medicines development in the industry right now?" (55 answered) There was an impression that there is an effort within industry to involve patients, but it is not being done well enough and more could be done.
Stakeholders were aware that patients are involved at clinical trial stage; they perceived a lack of PE at earlier stages of the drug development process, for example:

| Roles
"What is the role of each of the stakeholder groups?" (44 answered) Stakeholders' views on their role and the roles of others are summarized in Table 1

| Responsibility
"Do stakeholders have the same responsibility for patient engagement?" (48 answered). Fewer than half of interviewee votes (21 votes; 41%) supported the view that all stakeholders had equal responsibility. One-fifth of votes (10; 21%) were for stakeholders having "unequal responsibility," but with interviewees not being able to specify which stakeholder group should be most responsible and take the lead on PE. Where lead stakeholders were specified, industry (7 votes; 14%) and researchers (5 votes; 10%) were the most commonly cited (Figure 4), for example: Although industry and researchers were thought by all other stakeholders to have more responsibility in PE-neither group believed they have greater responsibility.

| Stakeholder expectations matrix
Stakeholders' views of relationships, roles, goals and responsibilities were analysed together (using grounded theory analysis 29

| Priorities
"What are the priority areas for your stakeholder group? And is there anything you think other groups should be focussing on?" (44 answered). Responses are summarized in Table 2.
Interviewee responses to priorities could be broadly grouped into 4 key themes: vision, values, strategy and execution. While "vision" was generally aligned, stakeholders' views became more disparate as PE moved along the continuum towards execution.
There was agreement and shared vision that having patients involved should be a priority, and there needs to be greater collaboration with all stakeholders. There were some discrepancies in the value of, or the perception of the value others place on, having patients involved in medicines development as some stakeholder F I G U R E 4 Stakeholder responsibility for PE † . † Based on 48 respondents, one interviewee indicated that responsibility fell with 3 groups and another that responsibility fell with 2 groups, and 46 interviewees indicated a single group giving an overall denominator of 51

| Needs
Are there any skill/capability or knowledge areas that you would

| DISCUSS ION/CON CLUS ION
We have surveyed a wide range of stakeholders in a qualitative study to identify common themes and perspectives amongst and within stakeholders. 28 Our findings confirm the common understanding of the priority of PE but also show where there is less alignment or lack of clarity of roles and expectations. They highlight 3 important  In conclusion, this qualitative multistakeholder survey builds on insights from others on the need to align expectations in PE. 11,23,28,32,33 Importantly, it highlights that there is no "leader"; no stakeholder group has a clear view on how to meaningfully engage with patients; there are educational gaps; and a structure and guidance for PE is urgently required. Given the diversity of stakeholders in PE, the potential for conflict of interest, and that different stakeholders may have different drivers for and requirements from PE, there needs to be cross-stakeholder collaboration-facilitated by platforms where stakeholders can connect and work together in a non-competitive way-to address these issues. Such collaboration will only be effective when there is understanding of (and consensus on) roles, responsibilities and expectations. This is essential if we are to synergize PE efforts, have realistic and achievable goals, and prevent misunderstanding and disappointments that can hamper even the most worthwhile endeavours. We hope that the findings from this survey will inform the essential conversations between stakeholders, facilitate alignment and deliver meaningful PE in medicines development.

ACK N OWLED G EM ENTS
The authors gratefully acknowledge the survey participants for their open and honest input and independent health-care consultancy Monmouth Partners (UK) who conducted the interviews and collated responses.

CO N FLI C T O F I NTE R E S T
Authors have no conflict of interests to disclose.