Patients as Partners in Sickle Cell Disease Research in Africa: A Framework for Equitable Patient-Engaged Health Research

Abstract

Background

Patient and public involvement (PPI) in health research is gaining global momentum through initiatives such as INVOLVE (UK), the Patient-Centred Outcomes Research Institute (USA), and the Strategy for Patient-Oriented Research (Canada). However, its implementation in Africa remains limited and lacks context-specific guidance. To address this gap, the Sickle Africa Data Coordinating Center supported the development of the Patients as Partners in Sickle Cell Disease Research (SCD-PAPIR) Framework to guide meaningful patient engagement and involvement in SCD research in Africa.

Methods

An iterative, participatory approach was adopted. The process involved the creation of a SCD PAPIR working group comprising SCD patient advocates in 14 African countries; 18 virtual working group meetings, one public webinar, and three in-person workshops. The framework was co-created through facilitated discussions reflecting on prior engagement in SCD research as a patient partner, and best practices for effective patient–researcher collaborations. Patient-only sessions and leadership roles were integrated to ensure safe spaces and to minimise power imbalances.

Results

The SCD-PAPIR Framework positions SCD patients and their caregivers as citizen researchers. Key to the framework is the principle of subsidiarity, which affirms the agency and experiential knowledge of patients while calling for epistemic humility from researchers. Its core pillars include valuing patient expertise, transparent communication, resource sharing, patient empowerment, collective learning, amplification of patient voice, continuous feedback, and shared benefits. Best practices emphasise two-way communication, addressing power asymmetries, co-learning and patient empowerment, co-ownership of outputs, and formalised PAPIR structures.

Conclusion

The SCD-PAPIR Framework provides a contextually grounded model for patient-engaged research in Africa and contributes to efforts to decolonise health research by positioning patients as co-creators of knowledge, and not merely a data source. The effective implementation of the framework will require investment in institutionalising PAPIR in SCD research. Future work should focus on designing implementation toolkits, developing PPI training modules for researchers and patient advocates, and adapting the framework to other health conditions.

Key Points

Apply the principle of subsidiarity in patient engagement in health research by establishing research processes that recognise the experiential knowledge of persons living with sickle cell disease (SCD) and promote epistemic humility among both researchers and patient partners.

Institutionalise SCD patient engagement by embedding the Patients as Partners in Research (PAPIR) model in SCD research funding calls, ethics review considerations, and PAPIR training for SCD researchers and patient advocates.

Promote accountability in patient-engaged SCD research by monitoring the effectiveness of PAPIR activities and including SCD patient advocates in research advisory boards.

Introduction

Patient and public involvement (PPI) in health research is gaining global attention. Over the past two decades, there has been growing recognition of citizen science and the importance of ensuring that patients and research participants are not only treated as data points but also as stakeholders who can contribute meaningfully to the design, implementation, and translation of health research [1, 2]. Established PPI models such as INVOLVE in the United Kingdom [3]; the Patient-Centered Outcomes Research Institute (PCORI) in the USA [4]; and the Strategy Patient‐Oriented Research Initiative (SPOR) in Canada [5] have institutionalised patient engagement in research governance, funding, and dissemination. More recently, the Patients as Partners in Research (PAPIR) model has emerged as a PPI approach that embeds the lived experience and experiential knowledge of patients throughout the research continuum [6]. These models share a normative commitment to moving beyond paternalistic paradigms that position researchers as sole decision makers, and patients and the public as passive providers of health research data [2, 7, 8]. Instead, they emphasise collaborative approaches to priority setting, study design, implementation, and knowledge translation, particularly in research on chronic and rare diseases, where patients bring embodied knowledge of their health condition [1, 9]. While PPI overlaps conceptually with community engagement in its emphasis on building trust between researchers and participants, it goes further by advocating for research to be conducted with and for patients [7, 10].

The implementation of PPI research in low- and middle-income countries (LMICs) is rare [11]. Barriers include insufficient funding for patient engagement, a lack of trained PPI practitioners, and entrenched paternalistic healthcare systems that marginalise patient voices [12–14]. These normative barriers may outweigh practical challenges, such as limited financial and human resources, which often improve once institutional cultures and priorities evolve. Nonetheless, interest in PPI within African health research programmes is growing [15, 16], but context-specific frameworks to guide its implementation are lacking.

Situating PPI practices within the local cultural, epistemological, and institutional contexts in which they would be implemented is important to ensuring their effectiveness. Uncritical adoption of existing PPI models developed in high-income countries risks undermining trust between patients and researchers in Africa. This is because many of the models assume the presence of strong advocacy networks, high levels of health literacy, empowered patient populations, and institutionalised mechanisms for PPI in health research [11, 17–20]. Such assumptions may not hold in many African settings where healthcare and research are opportunities for providing free or accessible care to many patients, yet there are also resource limitations for PPI and hierarchical relationships between clinicians/researchers and patients. Hence, applying these models without adaptation risks making them ineffective, irrelevant, or extractive.

To address this gap, the SickleInAfrica consortium, a pan-African sickle cell disease (SCD) research collaboration [21, 22], developed the SCD-PAPIR Framework to promote equitable and context-specific patient engagement in health research. SickleInAfrica operates across eight countries: Nigeria, Ghana, Mali, Tanzania, Zambia, Zimbabwe, Uganda, and South Africa. In its early phases, patient representatives from Ghana, Nigeria, South Africa, and Tanzania contributed to the co-development of informed consent and educational materials for the SickleInAfrica registry [23]. As the consortium expanded, patient representatives called for meaningful involvement in research processes, including defining research priorities [24], while also raising concerns about paternalistic researcher–patient dynamics and the absence of clear guidance on what meaningful engagement should entail.

This paper outlines the process of developing the SCD-PAPIR Framework, describes its core principles, and explores its broader implications for advancing equitable patient involvement in African health research. It also presents practical strategies for operationalising these principles through core pillars and best practices.

Method: Co-Creation of an African Sickle Cell Disease–Patients as Partners in Research (SCD-PAPIR) Framework

To develop a conceptual framework for equitably and meaningfully engaging patients as partners in SCD research in Africa, we adopted an iterative, participatory methodology. The process involved establishing the SCD-PAPIR Working Group (PAPIR-WG), holding 18 virtual meetings, convening one public webinar, and organising three in-person workshops in Cape Town (South Africa), Kampala (Uganda), and Bamako (Mali). Each stage was designed to build consensus through dialogue, reflection, and co-learning, by drawing on Afrocentric dialogical traditions that prioritise respectful interaction, collective problem solving, and the resolution of tensions between groups [25].

Formation of the SCD-PAPIR Working Group

The co-creation process began with the formation of the SCD-PAPIR Working Group (PAPIR-WG). The working group was initiated in 2022 by NSM, a SickleInAfrica Ethics, Legal, and Social Issues (ELSI) researcher, and received funding and logistic support from the Sickle Africa Data Coordinating Center (SADaCC) at the University of Cape Town, South Africa, and the Center for ELSI Research and Analysis (CERA) at Stanford University, USA. Initially, the group included eight SickleInAfrica researchers and one patient representative from each of the eight SickleInAfrica sites. Its objectives were to (i) define the expected degree of patient engagement in research, (ii) articulate the role of patient support groups within PAPIR, and (iii) identify pathways for meaningful and equitable patient involvement in SCD research in Africa.

Early meetings of the working group revealed sociocultural dynamics and power asymmetries that could have impacted open discussions. There were concerns among the eight patient advocates that feedback of patient engagement activities within SickleInAfrica might be perceived by the researchers as criticism of the SickleInAfrica consortium. They also expressed a hesitation to question researchers due to entrenched hierarchies within healthcare systems and broader cultural norms that emphasise deference to authority, especially as some SickleInAfrica researchers were also clinicians involved in their routine clinical care. In response, the following safeguards were introduced: (i) researchers (except NSM) were excluded from initial meetings so that the patient advocates could articulate their concerns without fear of reprisal; (ii) leadership of the working group was transferred to two patient representatives to reduce power imbalances and allow the patient advocates to direct the agenda setting for all meetings; and (iii) the group was expanded to include additional SCD patient advocates, including those not directly linked to SickleInAfrica but active in national and regional advocacy networks. The last safeguard created a ‘safe space’ as it minimised the possibility of researchers pinning specific recommendations or experiences to a particular patient advocate. The additional members were identified and invited to the working group by the patient advocates. This expansion increased geographic representation from 8 to 14 African countries and helped create a safer, more inclusive space for dialogue. Researchers were primarily from the SickleInAfrica ELSI WG, while independent oversight was provided through regular consultations with MS, an ELSI researcher unaffiliated with the consortium (Table 1).

Table 1.

Summary of stakeholders involved in the co-creation process

Stakeholder group	Affiliation	Description
Patient representatives (n = 8)	SickleInAfrica sites	Initial working group members selected from Consortium-affiliated sites
Patient advocates (n = 11)	Non-SickleInAfrica sites (Cameroon, Côte d’Ivoire, Democratic Republic of the Congo, The Gambia, Malawi and Kenya)	Invited by SickleInAfrica-affiliated patients; involved in SCD advocacy in Africa
Additional patient representatives (n = 10)	Non-affiliated to SickleInAfrica but based at SickleInAfrica operating countries	Nominated by initial members from their countries but not previously involved in SickleInAfrica activities
Researchers (n = 8)	SickleInAfrica Consortium (ELSI group and researchers)	Researchers involved in ethics and patient engagement activities
External advisor (n = 1)	Independent (non-SickleInAfrica)	Provided external feedback and reflections to guide the process

ELSI Ethics, Legal, and Social Issues, SCD sickle cell disease

Patient-Led Framework Development Through Online Meetings

Over 18 months, the PAPIR-WG held fortnightly Zoom meetings structured around three thematic phases: (i) reflecting on past involvement in SCD research, (ii) identifying recurrent barriers to effective collaboration, and (iii) defining principles and pillars for equitable patient–researcher partnerships. Meetings were guided by open-ended questions (Fig. 1) to encourage reflection, dialogue, and knowledge co-construction. Discussions were documented by a rotating scribe, and iterative drafts of the framework were developed in real time. Although English was the primary working language, two French-speaking patient advocates with good English proficiency participated actively. The draft framework was shared with researchers for feedback and jointly refined in subsequent meetings and workshops.

Fig. 1.

Guiding questions to facilitate discussions during the sickle cell disease (SCD) Patients as Partners in Research (PAPIR) meetings

Webinar on Patients as Partners in SCD Research

Before engaging more formally with researchers in the PAPIR WG, the patient representatives convened a public webinar on Patients as Partners in SCD research in Africa. The draft framework was presented at the webinar alongside contributions from two SickleInAfrica researchers: the Chair of the SickleInAfrica Steering Committee (LT) and the Principal Investigator of the SickleInAfrica Zimbabwe site (PK), who highlighted patient engagement initiatives within the consortium and emphasised the importance of integrating patient experience into governance, research priority setting, amplifying patient voices beyond SickleInAfrica, and in supporting advocacy for SCD research funding in Africa (https://www.youtube.com/watch?v=Dm099qh51YY&t=4316s). Feedback from this session (Table 2) was incorporated into the draft framework

Table 2.

Key messages from the SCD-PAPIR in Africa public webinar

Patient-engaged research	Content of the SCD PAPIR Framework
Conceptualisation	PAPIR considers SCD patients as citizen researchers Recognises SCD patient-partners as co-creators of knowledge
Foundational principles	Subsidiarity (nothing about us without us)
	Inclusivity (including patient voices in research planning and implementation)
	Equality (patients and researchers collaborate as equals, with defined responsibilities
	Respect (recognition of patients’ experiences and expertise in all research aspects)
	Shared decision making (patients and researchers jointly make decisions that affects the partnership)
	Mutual trust (through transparency, accountability, and consistent communication)
	Reciprocity (value patient contributions through compensation and shared benefits
Expectations of patient-partners	Provide feedback on research protocols, identify priorities
	Engage in community education and awareness
	Advocate with policymakers to prioritise SCD in public health programmes
Responsibilities of researchers	Enable patient partners access to other SCD stakeholders e.g. policymakers, biopharma
	Incorporate patient feedback into research
	Platform and amplify patient advocacy voices

PAPIR Patients as Partners in Research, SCD sickle cell disease

Refinements to the Framework: In-Person Workshops and Online Meetings

The final framework refinement took place through a series of joint in-person and virtual engagements, including: (i) a half-day session at the 10th SickleInAfrica Meeting in Kampala, Uganda (October 2023); (ii) a 3-day workshop in Cape Town, South Africa (November 2023); (iii) a half-day session at the 11th SickleInAfrica Meeting in Bamako, Mali (May 2024); and (iv) three additional virtual PAPIR WG meetings. These engagements addressed researcher expectations for patient engagement, conceptualising the PAPIR model, identifying barriers to equitable partnerships, and co-developing the core principles, pillars, and best practices of the framework.

Results: The SCD-PAPIR Framework

The WG defined the SCD-PAPIR model as an inclusive, collaborative approach to patient-engaged research that positions persons living with SCD and/or their caregivers as equal partners and citizen researchers. There was consensus that the lived experiences and experiential knowledge of SCD patients and their caregivers can directly inform and improve research priority setting, study design, data collection, analysis, knowledge translation, and dissemination of findings. To strengthen its practical utility, the working group identified key research processes in which patient participation is necessary and required (Fig. 2).

Fig. 2.

Examples of research tasks that should include the patient partner in sickle cell disease (SCD) research

A recurrent theme across all discussions was mistrust, both between researchers and patient advocates, and within SCD patient support/advocacy groups. The patient advocates in the WG highlighted concerns such as poor communication, lack of clarity regarding roles, limited feedback on research outcomes, and restricted access to data for advocacy purposes. Some reported tensions with their support groups, who accused them of withholding opportunities offered by researchers. The researchers described challenges arising from limited familiarity with research processes by the SCD patient advocates, which sometimes caused misaligned expectations. Both groups, however, agreed on the need for respectful dialogue, solidarity, mutual trust, transparency, and consistent communication as the foundation of productive collaborations. They acknowledged the patient–researcher dyad as central to these efforts and that the breakdown of trust can result from actions or inactions on the part of either researchers or patient advocates.

These reflections informed a set of practical recommendations, including financial compensation for patient partners, the involvement of patient representatives in scientific advisory boards, and the adoption of patient engagement methods that affirm respect, interdependence, and collective learning.

Foundational Principles for SCD PAPIR Model

Key to the SCD-PAPIR is the idea that decisions should be made at the most immediate or local level possible, and by those most affected by the outcomes. This principle affirms the agency, legitimacy, and experiential expertise of people living with SCD, positioning them not as passive beneficiaries but as co-creators of research. Subsidiarity requires epistemic humility from researchers. This principle guided the overall development process of the framework (Fig. 3).

Fig. 3.

Foundational principles for PAPIR in SCD research. PAPIR Patients as Partners in Research, SCD sickle cell disease

Core Pillars of the SCD PAPIR Model

Equitable patient–researcher partnerships require more than intent; they demand a shared foundation of guiding principles supported by core pillars that sustain collaboration (Fig. 4). These pillars provide a practical roadmap for embedding meaningful patient engagement across the SCD research continuum.

Fig. 4.

Core pillars of SCD patient engagement in health research in Africa

Best Practices for Patient-Engaged SCD Research

Building on the foundational principles and core pillars, the WG developed a set of best practices to guide SCD patient–researcher partnerships (Table 3). These practices emphasise patient comfort, inclusive communication, recognition of power imbalances, provision of resources and capacity building, and fair recognition of contributions through authorship, advocacy opportunities, and shared benefits.

Table 3.

Best practices and strategies for operationalisation

Best practice	Description	Examples
Patient comfort	Create inclusive spaces where patient partners feel safe, respected, and empowered to participate	Begin meetings with check-ins; host patient-only sessions for candid discussions
Establish communication channels	Maintain open two-way communication using clear and accessible language	Share regular project updates; schedule dedicated feedback sessions
Recognise and address power imbalances	Acknowledge and work to mitigate unequal power relations	Rotate facilitation roles; include patient partners in decision making and governance structures
Provide resources	Equip patient partners with tools and knowledge, and financial compensation to support engagement	Provide training on basics of research, offer stipends, travel support, or internet bundles
Reward contributions and share benefits	Ensure patient partners are acknowledged and benefit from their participation in research	Involve patients in dissemination and advocacy activities; co-author publications, and support advocacy efforts

Discussion

Patient engagement models developed in high-income countries such as the INVOLVE, PCORI, and SPOR [3–5] have formalised PPI in health research. However, the PPI experience in Africa seems limited or less documented. The SCD-PAPIR Framework represents an important step in contextualising and operationalising PPI for African settings. It articulates principles for equitable patient engagement in SCD research while providing actionable strategies for implementation. A central strength of the framework lies in its co-creation process, which prioritised dialogue, reflexivity, and patient voice, ensuring it reflects both theoretical commitments and the lived realities of SCD patient advocates and researchers in Africa.

Key to the SCD-PAPIR Framework is the principle of subsidiarity, which affirms the legitimacy of patient knowledge and calls for epistemic humility from SCD researchers. It discourages tokenistic forms of patient engagement and health research and promotes shared decision making and co-learning between persons living with SCD and researchers. This aligns with global PPI efforts [26–29] and resonates with Afrocentric philosophies such as Ubuntu, which emphasise relationality, solidarity, reciprocity, substantive representation, and consensus-driven decision making [30, 31]. Importantly, the framework demonstrates that while the goals of PPI may be universal, strategies must be context-specific and reflect the realities of African health and research systems.

The framework also contributes to ongoing efforts to decolonise health research [32]. It challenges extractive health research practices that treat patients as passive data sources and instead positions them as co-creators of knowledge. This shift is both ethical and epistemological. It values the embodied expertise of persons living with SCD and advances a more participatory approach to the production of scientific knowledge. Early applications of the PAPIR model within SickleInAfrica, such as in the newborn screening project [33] and the malaria chemoprophylaxis study, demonstrate how embedding patient perspectives can improve study design, enhance participant enrolment, and strengthen peer support and counselling for SCD. The framework also responds to the ethical imperative of returning value to participants in African health research, especially for genetic conditions [20, 34, 35]. Too often, research outcomes for genetic diseases fail to reflect patient priorities or provide direct benefit to communities. By embedding subsidiarity, solidarity, knowledge sharing, patient empowerment, and reciprocity into its principles, the PAPIR framework operationalises pathways for ensuring that SCD research gives voice to persons living with the condition, generates tangible value for patients, and sustains long-term patient–researcher partnerships.

As interest in participatory SCD research grows, these principles must be supported by concrete actions, including research policy reforms, funding mechanisms, and institutional practices that embed patient partnership as standard practice rather than the exception. Practical steps include adapting ethics review processes to integrate PAPIR standards, creating dedicated funding streams for PPI in SCD research, developing PAPIR training programmes for researchers and patient advocates, ensuring fair compensation for patient partners, and embedding patients in governance structures.

Limitations and Future Directions

The SCD-PAPIR framework is a useful and evolving PPI research tool. A key limitation in its development is that many of the working group members had prior exposure to patient engagement concepts through SCD advocacy or research initiatives. This may have contributed to some of the framework principles mirroring those in other PPI models. Future work should explore whether alternative principles would emerge when co-developing frameworks with patients and researchers who have little or no prior PPI experience. Nonetheless, the strength of the SCD-PAPIR framework does not lie in proposing entirely new principles, but in adapting and operationalising them within African health and research systems. Its relevance is in its contextual sensitivity and co-creation process.

Further research is needed to assess the adaptability of the framework in other disease areas and settings, particularly where patient organisations are informal or absent. Priorities include developing toolkits and measurable indicators for implementation, building patient and researcher capacity through PAPIR-aligned training modules, and embedding the framework within institutional and national research governance systems. Future directions will also include a qualitative secondary analysis of recorded SCD PAIR WG sessions to provide insights into how patients and researchers negotiated aspects of the framework.

Funding

Open access funding provided by University of Cape Town. This work was jointly funded through a pilot grant to NSM from the Centre for ELSI Resources and Analysis (CERA), Stanford University (NHGRI grant U24 HG010733-01) to establish the PAPIR WG; as well as financial support from the Sickle Africa Data Coordinating Center (SADaCC), University of Cape Town, through a grant to AW (NHLBI: U24HL135600). Both CERA and SADaCC receive funding from the NIH. NSM and AW receive funding from the National Institute of Mental Health (Award number U01MH127692) for the project Public Understanding of Big Data in Genomics Medicine in Africa (PUBGEM-Africa) under the Harnessing Data Science for Health Discovery and Innovation in Africa (DS-I Africa) programme. The content of this article is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

Declarations

Author Contributions

Conceptualisation: NSM. Funding acquisition: NSM and AW; methodology: NSM; framework development: all authors; project administration: NSM VN, MS; supervision: NSM, MS AW; validation; all authors; writing—original draft: NSM; writing—review and editing: all authors.

Data Availability

The data that support the development of the framework are available from the Sickle Africa Data Coordinating Center, but restrictions that were applied in relation to the ethics of the current study apply to the availability of these data, so some of it is not publicly available. However, the data are available from the authors upon reasonable request and with the required ethics approval and consent from the members of the PAPIR WG.

Conflict of Interest

The authors have no competing interests to declare that are relevant to the content of this article.