Co-creating discovery in basic science: Challenges and opportunities in patient-partnered research for rare liver disease

Abstract

Background:

Patient-partnered basic and translational research is the most productive and meaningful approach to developing informed research questions, generating high-quality data, and producing impactful work. While several organizations have published guidelines for patient-partnered research, few experiential reports in basic science are written from both the patient and researcher perspectives and comment on real-time practical challenges and approaches that ensure the establishment and maintenance of a non-tokenistic, organized, and successful research partnership with patient partners.

Methods:

We describe patient partners’ and researchers’ experiences working together in a basic science and translational research team studying the drivers of primary sclerosing cholangitis, a rare liver disease for which liver transplantation remains the only treatment to disrupt disease progression. We outline approaches used to support meaningful communication, collaboration, and shared output among project stakeholders, drawing on the perspectives of both basic science researchers and patient representatives involved in long-standing joint research projects.

Results:

Several practical elements support the success of patient partnership in basic and translational science, including structured communication, clear role definition, mutual respect, sustained engagement, and intentional mechanisms to support patient partners’ meaningful contributions. We highlight challenges and strategies used to address them in real time, offering insight into how patient–researcher partnerships can be maintained in an organized, authentic, and non-tokenistic way.

Conclusions:

This review highlights a patient-partnered research team's experiential insights to bridge the gap between published guidance and real-world practice, reinforcing how authentic patient–researcher partnerships can drive more inclusive and impactful basic and translational science.

Lay Summary

Involving patients as active partners in research leads to better questions, stronger data, and more meaningful results. While many organizations have created guidelines for including patients in research, there are still very few reports and reviews that describe what this looks like in real time, especially in basic science and translational research in rare liver disease. In this article, we share the experience of both patient partners and researchers working together on a project focused on primary sclerosing cholangitis, a rare liver disease for which liver transplant is the only treatment that can stop progression. We describe how we built and maintained a true partnership by using practical strategies for communication, collaboration, and shared decision making. This work offers lessons learned by both researchers and patient partners and shows how strong patient–researcher relationships can lead to better science and greater impact.

Introduction

Preclinical and basic science research programs investigating the molecular and cellular bases of disease are fundamental to the development of new therapies and the improvement of diagnostic and prognostic tools. Despite significant advances in preclinical research, there remains a persistent gap between discovery in the laboratory and successful clinical application, a phenomenon often referred to as the “valley of death” in translational research. This gap can result from misalignment between research priorities and patient needs, lack of effective communication, a paucity of feasibility considerations, and challenges in applying basic findings to real-world health contexts and clinical trials.

Increasingly, biomedical research organizations and funders are recognizing that engaging patients as partners throughout the research process (1,2), beyond participation as subjects or recipients of care, is essential to closing this translational gap. Patient partnership, broadly defined as meaningful collaboration with patients and their representatives in shaping research questions, study design and interpretation, and dissemination, while keeping the patient's experience at the centre, fosters research that is more relevant, ethical, and impactful (3). While patient engagement has been extensively studied and implemented in clinical and primary care settings (4,5), its integration into basic and translational science remains limited and under-developed.

The nature of basic science research introduces unique challenges to patient partnership, wherein the complexity and technical language of foundational biomedical research can create barriers to patient understanding and involvement. In basic science, research timelines tend to be longer, with less immediate clinical applicability, which may affect patient motivation and engagement. Additionally, differing expectations between scientists and patient partners regarding roles, contributions, and outcomes require careful navigation. To address these challenges, there is a need for specific frameworks and practical strategies tailored to the basic and translational science context, enabling sincere, sustained, and mutually beneficial collaborations.

This review draws on both a comprehensive examination of the literature and the first-hand experiences of patient partners and basic science/translational researchers investigating the pathogenesis of primary sclerosing cholangitis. The basic science researchers, Dr Diana Nakib and Dr Sonya MacParland, serve as members of the Canadian Institutes of Health Research (CIHR)-funded Primary Sclerosing Cholangitis Collaborative Autoimmune Network (PSC-CAN), the Developing Medicines through Open Science (DMOS) program by Conscience.ca, and the Chan Zuckerberg Initiative (CZI)-funded Patient-Partnered Collaborations for Single-Cell Analysis of Rare Inflammatory Pediatric Disease project entitled Understanding the Cellular Ecosystem of Primary Sclerosing Cholangitis. The patient partners, Mary Vyas and Rachel Gomel, serve as patient representative members in both of the above-listed research initiatives, sit on the governing board of PSC Partners Seeking a Cure Canada, and are directly affected by primary sclerosing cholangitis (PSC). This interdisciplinary partnership, which includes basic scientists, computational biologists, clinicians, and patient representatives, has led to the co-development of research programs investigating the cellular drivers of PSC, a rare autoimmune liver disease with a limited therapeutic landscape. The combined perspective provides unique insight into the successes and challenges of patient-partnered research in a preclinical setting and underscores the value of including patient voices from project inception through knowledge translation.

Here, we summarize key concepts, outline benefits and challenges, and present recommendations for establishing strong and effective patient partnerships in basic and translational biomedical research. We aim to offer a road map for researchers who seek to incorporate patient perspectives into foundational basic and translational science, to enhance the scientific relevance, ethical rigour, and translational potential of their work.

Defining Patient Engagement

A crucial initial step in incorporating patient engagement into research initiatives is to clearly define key terms and concepts that underpin patient-partnered research (Table 1). These definitions establish a shared language and framework essential for consistent communication among researchers, patients, and stakeholders.

Table 1:

Key terms and definitions in patient-partnered research

Term	Definition
Priority setting	The process of identifying and ranking research questions or areas based on their importance to patients, researchers, clinicians, and stakeholders. This collaborative approach ensures that the research agenda aligns with patient needs and preferences, ultimately leading to more relevant and impactful research outcomes.
Knowledge translation and dissemination	The process of ensuring that research findings are effectively communicated and applied in real-world settings to improve health outcomes. Dissemination involves actively sharing research results with stakeholders, including patients, health care providers, and policy-makers, in accessible formats.
Capacity building	The process of developing the skills, knowledge, and resources necessary for individuals and organizations to effectively engage in patient-oriented research. This includes training, mentorship, and providing support to ensure that patients can meaningfully contribute to research activities and decision-making processes.
Organizational requirements	The structures, policies, and practices that organizations must have in place to support patient engagement in research. This includes establishing collaborative frameworks, ensuring adequate resources for patient partners, and creating environments that promote inclusivity and mutual respect among all stakeholders involved in research.
Stakeholder meetings	Meetings that involve various parties interested in or invested in the research process, including patients, researchers, health care providers, and policy-makers. These meetings aim to facilitate communication, share perspectives, set priorities, and collaboratively make decisions regarding the direction of the research project.
Inclusivity/diversity	The practice of ensuring that a wide range of patient perspectives are represented in the research process. This involves actively seeking input from diverse patient populations, including those from marginalized or under-represented groups, to ensure that research findings are relevant and applicable to all segments of the population.
Evaluation	The systematic assessment of the processes and outcomes of patient engagement in research. This includes measuring the effectiveness of engagement strategies, understanding the impact of patient partnerships on research quality and relevance, and identifying areas for improvement in future projects.
Patient	An individual with personal experience of a health issue or condition. This term is inclusive of those who have directly experienced a health concern as well as informal caregivers, including family members and friends who support them.
Patient representative	An individual who takes on a formal role to speak on behalf of a broader patient community, commonly within health care organizations, patient advocacy organizations, research settings, or advisory committees. A patient representative typically has personal experience as a patient or caregiver; takes on responsibilities beyond their own care; commits time and effort to stay informed about broader patient concerns and health care developments; actively participates in decision-making processes, often providing input on research priorities, health care policies, or service improvements; and serves as a link between the patient community and health care institutions or research teams.

Table 1 outlines key terms, such as priority setting, which emphasizes collaborative identification and incorporation of research questions based on what matters most to patients and other stakeholders. Priority setting between the patient partners and researchers is a critical step in identifying and incorporating patient needs into developing research questions relevant to patients. This ensures research agendas are aligned with real-world needs in addition to scientific curiosity. Knowledge translation and dissemination refer to the active and consistent communication of findings in accessible formats, ensuring research outcomes benefit patients and inform clinical practice. Capacity building highlights the importance of equipping patients and researchers with the necessary skills and resources to participate meaningfully, which includes training and ongoing mentorship. Equally, organizational requirements address the infrastructure needed within research institutions to support patient engagement, including policies, resources, and an inclusive culture that respects patient contributions. Other terms, such as stakeholder meetings, inclusivity and diversity, and evaluation, further detail essential processes for maintaining effective, equitable, and accountable partnerships.

The definitions of patient versus patient representative distinguish between individuals with lived health experience and those who formally advocate for broader communities. Patient representatives often serve in organizational roles and bring a broader, community-wide perspective to research priorities, linking grassroots experiences with scientific inquiry, including organizations, such as PSC Partners Seeking a Cure Canada, Necrotizing Enterocolitis (NEC) Society—Building a World Without NEC, and the Huntington's Disease Coalition for Patient Engagement (HD-COPE).

Understanding patient engagement also requires recognition of the spectrum of involvement, from patient-targeted to patient-led research (Table 2). These categories represent gradations in how patients can be involved, reflecting differences in decision-making power, roles, and desired outcomes. The level of involvement must be agreeable for both the researcher and the patients and/or patient representatives, as this will set the expectations for the duration of the partnership.

Table 2:

Categories of patient engagement in research

Category	Level of engagement	Key characteristics	Key determining elements
Patient-targeted	Inform	Interventions designed for specific patient groups or subsets of patient groups	Involves marketing or outreach strategies to engage patients who fit certain profiles Less emphasis on individual patient preferences or experiences
Patient-centred	Consult/involve	Shared decision making by researchers and patient representatives	Focuses on understanding and respecting patient preferences and values in care decisions Aims to create a relationship that enhances patient satisfaction and outcomes by considering the whole person, including emotional, social, and psychological factors
Patient-partnered	Collaborate	Patients as equal partners in all stages of research	Actively involved in defining research questions, designing studies, and interpreting results Emphasizes co-design and co-creation of research initiatives, recognizing patients as equal partners as well as subjects Aims to ensure research is relevant to patient needs and experiences, ultimately improving the applicability of findings
Patient-led	Empower	Patients direct their own care and/or research initiatives	Encourages self-management and empowerment among patients in research Often involves grassroots movements where patients advocate for changes in health care practices or policies based on their lived experiences Patients lead the sponsoring and funding of research by using patient resources and fundraising and establishing bio-repositories run by patient groups

Ensuring Access, Fairness, and A Supportive Environment for All Stakeholders

At the time of project initiation and throughout the duration of a research project, it is crucial to engage diverse patient perspectives and to participate in in-depth project discussions. Diversity, equity, and inclusivity in patient engagement and research programs are often overlooked; however, it is essential to include patient voices that represent different backgrounds in terms of ethnicity, socio-economic status, age, and sex, as well as patients at different stages of disease progression. In our experience, conferences organized by patient representative organizations and local knowledge translation events have been invaluable opportunities for engaging in patient-led discussions of basic science questions.

However, to achieve this, incorporating accessibility practices is key, such as coordinating virtual meetings and live-streamed events with closed captioning, providing content summaries with different translated language options, and ensuring that physical spaces are equipped for accessibility standards. Additionally, knowledge translation events (expanded upon below) should be followed by distributing evaluation forms to patients and patient partners for their feedback, concerns, and suggestions.

Beyond representation, true inclusion requires actively addressing structural barriers and power imbalances that can exclude marginalized groups. This includes overcoming language and cultural barriers, improving health literacy, and acknowledging historical mistrust in research among some communities. Proactive recruitment efforts and sustained relationship building are necessary to engage under-represented patients meaningfully from the earliest stages of project development. Training in cultural competency frameworks adapted from medical and health professional education for researchers and patient partners can foster respectful and sensitive communication (6,7); in the Canadian context, this includes awareness of Indigenous data sovereignty and ethical engagement principles specific to First Nations, Métis, and Inuit communities. Engagement approaches should also be flexible, offering multiple modes of participation, such as in-person and virtual options or asynchronous feedback, to accommodate diverse needs and preferences.

Finally, it is important to implement evaluation frameworks to assess inclusive and equitable research practices and guide continuous improvement. Ethical principles, such as confidentiality, informed consent, and respect for patient autonomy, remain critical throughout all engagement activities, especially when working with diverse and vulnerable populations.

Project Development

The creation and development of the research questions in patient-partnered research is a collaborative, cyclical, and iterative process that depends on mutual respect and consistent communication among all stakeholders. Early in the project development process, research teams typically begin with an orientation meeting aimed to define key terms, outline experimental approaches to be used, and summarize prior publications and their impact in their respective fields. Knowledge translation and dissemination remain central tenets of patient engagement and early patient engagement in project development, and various training opportunities and resources are available (as described below) to support researchers in generating plain language research summaries (8,9). Deciding on a research question and shared research project will depend on several factors, including experimental and research feasibility and patient partner–led priority setting (10). The ideal project will lie at the intersection of the following two questions: (a) What questions are patients interested in? and (b) What are the experimental and operational capabilities of the researchers? This process will require iterative rounds of priority setting by the patient partners and researchers, as well as refinement of the research question, while taking into consideration experimental and financial feasibility, as well as institutional goals, scientific merit, impact, and key considerations described in Table 3.

Table 3:

Key questions to consider when prioritizing research projects

Category	Key questions to consider
Alignment with goals	How does this research align with organizational or institutional goals of all partners? Does it address an important disease burden or evidence gap? How will it contribute to improving patient outcomes, directly or indirectly?
Patient relevance	What research questions matter most to patients? How might this work impact patients’ quality of life and daily challenges?
Scientific merit	Is the question answerable with current technologies and methods? How original and innovative is the research question?
Feasibility	Do we have the necessary expertise, funding, and resources to conduct this study? What are potential barriers and/or risks?
Impact	What is the potential impact on the field and clinical practice? Could it reduce disease burden or improve treatments?
Stakeholder engagement	How will we ensure meaningful and ongoing involvement of patient partners? What training or support will patient partners need? How will we achieve diversity and inclusivity among patient voices?
Ethical considerations	Are there ethical concerns that must be addressed? How will we ensure the safety, privacy, and autonomy of patients and patient partners?
Timeline	What is the realistic timeline and are there time-sensitive milestones?
Collaboration potential	Can this foster interdisciplinary collaboration and partnerships?
Knowledge translation	How will findings be communicated in accessible formats to patients and the wider community? How might results inform future research and clinical care?

It is important to note that research questions may change throughout the duration of the research program and project in question, particularly in the context of discovery-based basic science research projects wherein future aims depend on early findings, such as in drug candidate identification. As such, project development and priority setting should remain an iterative and flexible process revisited each 6–12 months as new data and results are generated.

Training

Incorporating structured training initiatives for both researchers and patient partners is essential to cultivating a collaborative and supportive patient-partnered research program. For researchers, this should include training in cultural competence and knowledge translation, with an emphasis on using clear, accessible, and plain language when communicating research concepts and findings. This is particularly critical in basic science, where the complexity of scientific language can unintentionally alienate patient partners and hinder meaningful engagement. To empower patient partners in their role and enable sustained participation throughout the project life cycle, accessible resources and training opportunities should be provided to introduce key basic science principles, relevant literature, and expectations regarding patient engagement.

To ensure these practices are implemented meaningfully and consistently, it is important that a designated member of the research team be formally assigned to coordinate and lead patient partnership efforts. Without clear leadership and accountability, even well-intentioned engagement strategies risk remaining aspirational rather than operational. The partnership lead can act as a liaison, ensuring open communication, supporting training delivery, and fostering inclusive collaboration across all phases of the research process.

Canada's Strategy for Patient-Oriented Research (SPOR), established by the CIHR, facilitates both funding opportunities and a wide array of resources dedicated to supporting patient engagement in research (11). Similarly, Healthcare Excellence Canada provides a variety of resources and patient testimonies to further educate both researchers and patient partners on the experiences and potential pitfalls of patient engagement in research (12). Additionally, various hospitals across Canada have established patient partner training and engagement programs, such as the St. Michael's Hospital Partners in Research program and the Pride in Patient Engagement in Research (PiPER) at the University Health Network (UHN) (https://kite-uhn.com/piper), wherein researchers, patients, and patients’ families are encouraged to engage with available training materials.

Funding and Budgeting

Funding is a cornerstone of basic science research, and several funding institutions within Canada and internationally have included patient engagement as a requirement and a defined criterion for funding eligibility. There are three key categories of funding: governmental, independent, and pharmaceutical. Here, we will focus on governmental and independent funding sources that emphasize and prioritize patient engagement. For example, the CIHR in Canada and the National Institutes of Health in the US have both established funding opportunities that outline patient engagement as well as knowledge translation as key criteria for evaluation. Additionally, the Patient-Centered Outcomes Research Institute (PCORI), which is funded by the American government, provides targeted funding opportunities aimed at patient-centred clinical effectiveness research (CER), methodology studies, and knowledge translation of CER projects (13). Alternatively, independent sources of research funding include patient partner organizations, philanthropic organizations, and private institutes. For example, the Chan Zuckerberg Initiative has set patient engagement as a key priority in basic science research, specifically launching Requests for Applications (RFAs) dedicated to patient-partnered collaborations (1,2) and providing targeted training to patient representatives. Additionally, patient-partnered organizations, such as PSC Partners Seeking a Cure, annually launch RFAs for preclinical and clinical research projects that incorporate patient engagement. For example, PSC Partners Seeking a Cure has established the patient-led International Collaborative Research Network, which is focused on bringing together clinical and preclinical PSC researchers with patient partners to foster international collaborations and to identify and fund patient-partnered research projects through RFAs.

The compensation and allocation of funds to patient partners have been a critical development in budgetary requirements from funding agencies. However, it is important to consult the ethical guidelines of the funding opportunities, such as the Tri-Council Policy Statement: Ethical Conduct for Research Involving Humans—TCPS 2 (2018) by the CIHR (14,15), that clearly outline the difference between patient partners and patient participants and the ethical approach to compensation for each.

Organizational Requirements

Effective patient engagement in basic and translational research relies heavily on intentional organizational structures and practices that support ongoing collaboration. Establishing clear frameworks for communication, knowledge exchange, and feedback is essential to foster trust, transparency, and mutual respect among all stakeholders (Table 4). The following sections outline key organizational components, regular stakeholder meetings, knowledge translation events, and continuous evaluation that collectively create a foundation for meaningful and lasting patient partnerships throughout the research life cycle.

Table 4:

Scoping reviews and evaluations of patient partner involvement in preclinical research

Title	Article type	Main takeaway
Patient engagement in preclinical laboratory research: a scoping review (Fox et al, 2021) (16)	Scoping review	This review highlights the feasibility and effectiveness of patient engagement in preclinical research, with key activities occurring primarily at the education and priority-setting stages. Identified barriers include differences in knowledge and experience.
Preparing for patient partnership: a scoping review of patient partner engagement and evaluation in research (Bird et al, 2020) (22)	Scoping review	This review emphasizes the importance of shifting from supportive roles to active involvement of patient partners, which can lead to improved research outcomes and alignment with patient priorities.
Patient and public involvement in preclinical and medical research: evaluation of an established program in a discovery-based medical research institute (Smith et al, 2024) (23)	Evaluation	Evaluating a patient involvement program in discovery-based medical research institute revealed positive impacts on research processes but highlighted the need for more formal evaluations in preclinical settings.
Applying patient and public involvement in preclinical research: a co‐created scoping review (Carroll et al, 2022) (10)	Scoping review	Patient and public involvement has been effectively utilized in clinical research; its application in preclinical settings is still limited. The authors developed principles based on their findings to guide future patient-partnered efforts in preclinical research, emphasizing the need for more empirical evidence to support these practices.
Development and formative evaluation of patient research partner involvement in a multi-disciplinary European translational research project (Birch et al, 2020) (24)	Evaluation	The study employed mixed-methods surveys and virtual meetings to evaluate patient-partnered involvement impact from both researchers’ and patient research partners’ perspectives. Findings indicate that patient-partnered involvement positively influenced the research process, enhancing public engagement and the personal development of researchers.
Patient engagement in health research: perspectives from patient participants (Easley et al, 2023) (25)	Perspective	This article discusses the experiences and insights of members of a patient advisory committee involved in a large, multidisciplinary cancer research study called CanIMPACT over 6 years. The article emphasizes the value of patient engagement in research while highlighting the need for intentional efforts to ensure satisfactory involvement and meaningful contributions from patient partners.
Future directions for patient engagement in research: a participatory workshop with Canadian patient partners and academic researchers (Chudyk et al, 2024) (26)	Study	The study highlights the need for strong leadership and joint action between patient partners, academic researchers, and health and research institutions to make patient engagement a key component of the learning health system. It also emphasizes the importance of considering both individual and systemic factors in shaping the future of patient engagement in health research.

Monthly stakeholder meetings

A recent scoping review investigating patient engagement activities in preclinical research highlighted that most studies engaged patients primarily at the education or priority-setting stages and less throughout the duration of the project (16). In contrast, our experience shows that consistent monthly meetings with complete stakeholder representation, including full representation from patient partners, clinicians, research collaborators, and coordinators, have been central in the continuity of patient partner engagement throughout the duration of a project. These meetings facilitate regular communication, project updates, and opportunities for all stakeholders to provide input on decisions. They foster community building locally and internationally and create informal spaces for patient–researcher dialogue. Importantly, they also help offset knowledge imbalances and navigate the sensitive clinician–patient relationships in cases where the researcher is also a clinician providing patient care.

Knowledge translation events

Knowledge translation events targeting the broader patient community have been a cornerstone of our patient-partnered program, supporting mutual learning, dissemination, and capacity building. Recognizing the diversity within the PSC patient population, including both adults and children, we tailor events to engage these groups appropriately. These gatherings foster community building and create informal spaces for patient–patient and patient–researcher dialogue, deepening partnerships. A benefit of knowledge translation events is also to incentivize and encourage active engagement of patients in future research efforts, developing their interest, building trust, and encouraging willingness to participate in research.

To enhance accessibility and comprehension, we incorporate a patient interpreter role, in which the patient interpreter reviews researcher presentations beforehand to ensure clarity and the absence of unnecessary technical jargon. Basic science content can overwhelm audiences unfamiliar with biomedical research, so it is critical for researchers to adapt their communication accordingly. Resources and training programs dedicated to improving researchers’ skills in knowledge translation and communication are valuable supports for this work. Patient partners have reported that these knowledge translation events have served as a powerful gauge in assessing their own level of understanding of the science through attempts to communicate, translate, and interpret the science in lay language.

Evaluations and feedback

Continuous and systematic evaluation is vital to refining patient engagement approaches, gaining deeper insight into patient perspectives, and ensuring reproducibility in future collaborations. We regularly distribute evaluation forms and surveys following knowledge translation events to gather patient feedback on session content, format, and speakers. This feedback has informed improvements, such as simplifying scientific presentations, shortening talk durations, and increasing question-and-answer periods, often using anonymous online platforms to encourage open participation.

Best practices in patient-partnered research include the incorporation of internal assessments of the collaboration to determine strengths and weaknesses of the project (17). An example of an evaluation framework is the Public and Patient Engagement Evaluation Tool (PPEET), developed by McMaster University in Canada (18,19), which has been translated and applied internationally (20,21) and incorporates the following categories: scientific rigour, patient and public perspective, comprehensiveness, and usability (18,19).

Common Pitfalls and Strategies to Overcome Them

Power imbalances

A major challenge in patient-partnered research is the presence of unequal power dynamics between researchers and patient partners, which can lead to feelings of marginalization among patient contributors. When patient partners perceive that their input is undervalued or overshadowed by the academic or clinical expertise of researchers, meaningful engagement and open dialogue become difficult to maintain. Strategies to overcome and navigate possible power imbalances include fostering mutual respect, establishing guidelines and structure for meetings, ensuring shared decision-making processes through defining the collaborative partnership (see Table 2), and addressing stakeholder project expectations (see Table 3). It is important to establish processes that ensure all voices are consistently heard, such as through clear guidelines for discussion and dedicated time for feedback from patients and patient representatives. Shared decision making is another key practice that requires intentionality and consistency; explicit involvement of patient partners in research decisions at all stages, from setting project objectives to evaluating final outcomes, helps reaffirm the equal value of their perspectives and expertise.

Communication barriers

Language and terminology gaps often hamper constructive collaboration between researchers and patient partners. In some instances, the highly specialized vocabulary of biomedical science can alienate those who lack advanced training, preventing them from fully understanding research goals or the potential impact of proposed methodologies. Conversely, researchers may lack familiarity with best practices for communicating clearly with non-experts, while patient partners may not fully grasp the formal processes that govern academic research.

To overcome challenges associated with a lack of communication in researcher–patient partnerships, the use of plain language, free of jargon and specialized acronyms, serves as the cornerstone of effective communication. Providing concise summaries, visual aids, or infographics can further enhance accessibility. These elements help break down complex information into more digestible components. In addition, both researchers and patient partners benefit from structured orientation sessions that clarify expectations, terminology, and roles in the research process. Frequent check-ins to ensure comprehension and give space for questions can help maintain a shared understanding throughout the project.

Compensation issues

From the perspective of the scientist, the goals are to have clarity and fairness around compensating patient partners and to prevent disillusionment and frustration. Many researchers recognize the importance of reimbursing patient collaborators for their time and expertise even though formalized compensation policies can be unclear or difficult to navigate. Our patient partners have added a dimension to our understanding of the value of their partnership with researchers. They affirmed that their prime motivation in becoming a patient partner is a relentless sense of urgency and an unequivocal passion to do anything within their power to facilitate a cure. As an example, they described the early stages of a research initiative when there may be no grants, funding, or tangible outcomes. The feeling of being involved at the nascent stage of an initiative and partnering prior to obtaining funding for the research were far superior to being remunerated for their involvement.

In full consideration of the patient partners’ perspective, researchers and patient partners can co-develop a transparent and consistent compensation structure to reinforce that patient partners’ contributions are valued. It is beneficial to communicate at the outset of a project how and when individuals will be compensated, whether monetarily or through in-kind benefits, such as training, conference attendance, or relevant professional development. Being open to flexible or non-traditional compensation models (eg, travel subsidies, gift cards, child care support) can further show respect for diverse needs and circumstances. The CIHR has a published framework outlining approaches to patient compensation as a part of its Strategy for Patient-Oriented Research, which details the types of payments available and when each approach is appropriate (27).

Time constraints

Many patient partners face time constraints arising from health conditions, caregiving responsibilities, and personal obligations. When research meetings and deadlines are rigid and fail to consider these realities, patient engagement can wane, weakening the overall collaboration. To foster more inclusive participation, meetings should be scheduled with patient partners’ availability in mind, and flexible options, such as virtual attendance and asynchronous collaboration, should be consistently offered. Sharing materials before and after meetings, along with written follow-up summaries and action items, allows patient partners to stay informed and contribute meaningfully at their own pace. While these approaches enhance accessibility, they may also place added coordination demands on research teams. To support feasibility, assigning a dedicated team member to lead patient engagement efforts can help streamline communications, manage scheduling, and ensure continuity. In the event of missed meetings, offering personalized follow-up via phone calls can help maintain trust and connection, particularly for partners who prefer verbal updates. These thoughtful and flexible practices, grounded in respect for diverse needs, not only make participation more sustainable for patient partners but also help foster stronger, more enduring research relationships.

Lack of institutional support

Institutions may not always provide the necessary frameworks, policies, support, or resources to facilitate meaningful patient engagement in research. In environments where patient partnership is not yet embedded as a standard practice, researchers and patient partners can struggle to secure funding, administrative support, or formalized guidance on best practices. As mentioned above, PiPER at UHN has been central in creating institutional support for promoting collaborative research practices that include patient voices; this leadership also provides official guidelines and resources for standardizing and normalizing inclusive research practices for patient engagement as well as offering opportunities to amplify patient partner experiences within the UHN. In cases where institutional support is not available, researchers and patient partners can access available online resources provided by PiPER, PCORI, and SPOR. Advocating for institutional recognition and dedicated resources is crucial to embed patient partnership as a core research principle and to promote pan-institutional adoption of patient-engaged research approaches. Having such structures in place validates the importance of patient-partnered research and offers tangible support to investigators and patient collaborators.

Conclusion

Patient-partnered approaches in basic and translational science offer unique opportunities to generate research that is scientifically robust and deeply relevant to patient communities. By embedding and prioritizing patient perspectives throughout the research process, from priority setting and project development to knowledge translation and dissemination, investigators can produce findings more likely to translate into meaningful clinical applications, foster trust with the patient community, and strengthen long-term relationships with those most affected by the disease.

The experiences highlighted in this review, drawn from the PSC-CAN and CZI Patient-Partnered Collaborations research programs, demonstrate how consistent stakeholder engagement, tailored training, and inclusive knowledge translation events can overcome many of the recognized barriers in preclinical research. Structured practices, such as regularly scheduled meetings that include patient representatives alongside researchers, sustained communication and troubleshooting, and formal evaluation frameworks, help maintain equitable power balances and promote continuous communication. These strategies can mitigate common pitfalls like knowledge gaps, power imbalances, and inconsistent involvement, ensuring that patient partners remain active contributors at every stage.

Despite growing evidence of feasibility and impact, patient-partnered basic science research is still maturing as a field. More formal evaluations, standardized frameworks, funding opportunities, and institutional policies will be essential to scale these principles widely. Future directions include refining metrics for measuring engagement outcomes, advancing training programs to equip both researchers and patients with the necessary collaborative skills, and reinforcing diversity, equity, and inclusion practices so that a broader range of patient voices inform research priorities. Overall, basic science research in Canada, and globally, can achieve significant progress by embracing genuine patient partnerships, aligning scientific innovation more closely with patient needs and ultimately expediting the pathway from bench to bedside.

Funding Statement

D Nakib has received doctoral and post-doctoral funding from the Canadian Institutes of Health Research (CIHR) (CGS-D and CAN TAP Talent Clinical Research Training Fellowship). This research is also funded by the University Health Network (UHN) Foundation, and the Developing Medicines through Open Science (DMOS) program by Conscience.ca, as well as grant numbers CZF2019-002429, CZF2021-237921, and CZF2022-316558 from the Chan Zuckerberg Initiative Donor-Advised Fund (DAF), an advised fund of Silicon Valley, and from CIHR grant HIT168002 (SA MacParland, GD Bader, I McGilvray, A Ricciuto).

Contributions:

Conceptualization, D Nakib, M Vyas, R Gomel, S MacParland; Funding Acquisition, S MacParland; Investigation, D Nakib, M Vyas, R Gomel, S MacParland; Methodology, D Nakib, M Vyas, R Gomel, S MacParland; Writing – Original Draft, D Nakib, M Vyas; Writing – Review & Editing, D Nakib, M Vyas, R Gomel, S MacParland.

Ethics Approval:

N/A

Informed Consent:

N/A

Registry and the Registration No. of the Study/Trial:

N/A

Data Accessibility:

N/A

Funding:

D Nakib has received doctoral and post-doctoral funding from the Canadian Institutes of Health Research (CIHR) (CGS-D and CAN TAP Talent Clinical Research Training Fellowship). This research is also funded by the University Health Network (UHN) Foundation, and the Developing Medicines through Open Science (DMOS) program by Conscience.ca, as well as grant numbers CZF2019-002429, CZF2021-237921, and CZF2022-316558 from the Chan Zuckerberg Initiative Donor-Advised Fund (DAF), an advised fund of Silicon Valley, and from CIHR grant HIT168002 (SA MacParland, GD Bader, I McGilvray, A Ricciuto).

Disclosures:

The authors have no conflicts of interest to declare.

Peer Review:

This article has been peer reviewed.

Animal Studies:

N/A