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  • The PROTEUS Consortium

    The PROTEUS Consortium

    Registry

    As the healthcare decision-making evolves, patient-reported outcomes (PROs) are increasingly getting attention as crucial tools for encapsulating the patient voice in clinical research and routine care. PROs provide instrumental insight into symptoms, treatment impact, and quality of life, directly from the patients’ perspective. Yet, the methods for PRO selection, measurement, and reporting are often inconsistent and ambiguous, restricting their utility for patients and other stakeholders. Bridging this gap is the key for patient-centred healthcare.(1, 2) To address this need, the Patient-Reported Outcomes Tools: Engaging Users & Stakeholders, i.e., PROTEUS Consortium was formed in 2018.(3, 4)

    The PROTEUS Consortium is a global partnership of organizations- including clinician groups, research networks, patient advocacy groups, regulatory bodies, and health systems, all united in their commitment to enhancing the use of PROs in both clinical trials and routine clinical practice.(3) The core mission of the Consortium is to enable access for stakeholders across the healthcare ecosystem to high-quality, consistent, relevant, and actionable PRO data to help inform treatment decisions.(3-6)

    The unique characteristic of the PROTEUS Consortium is its dual initiative model- PROTEUS-Trials and PROTEUS-Practice.(3, 6) PROTEUS-Trials emphasizes assisting researchers with embedding PROs systematically into clinical trial design, conduct, and reporting; while PROTEUS-Practice facilitates PRO integration into real-world clinical systems.(6) This dual model consists of practical tools and methods for adopting PRO systems within electronic health records (EHRs) and communicating the value of PROs to stakeholders across diverse care settings. These two branches of the Consortium enable robust PRO guidance in both research and applied practice.(3, 5, 6)

    Rather than developing separate frameworks, PROTEUS curates and modifies existing best-practice approaches, such as those from the USFDA, International Society for Quality of Life Research (ISOQOL), and the CONSORT-PRO Extension, into practical, user-friendly resources.(3) These consist of checklists, tutorials, guides, and implementation manuals customized for clinical researchers, sponsors, healthcare administrators, and even patient advocates. These materials are evidence-based, openly available, and developed to facilitate adoption without many difficulties, thus ensuring broad applicability across healthcare settings.(3-6)

    The Consortium emphasizes stakeholder engagement as it operates with more than 50 partner organizations to co-develop and enhance its resources, collecting real-world feedback through pilot programs, workshops, and collective ventures. This facilitates the robustness, contextual appropriateness, cultural sensitivity, and adaptability of the tool to various healthcare systems. The Consortium’s collective model enables trust while cultivating shared responsibility of the mission to uplift the patient voice in healthcare.(3-6)

    In the end, the PROTEUS Consortium is making headway by endorsing agreement on best practices for PRO application, creating a sustainable foundation for knowledge, and facilitating the adoption of patient-centred approaches worldwide. As healthcare systems progress towards more value-based and tailored care models, PROTEUS will act as a crucial facilitator that ensures active patient participation in research in shaping meaningful outcomes.

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    References

    1. Weldring T, Smith SM. Patient-Reported Outcomes (PROs) and Patient-Reported Outcome Measures (PROMs). Health Serv Insights. 2013 Aug 4;6:61-8.
    2. Miller D, Steele Gray C, Kuluski K, Cott C. Patient-Centered Care and Patient-Reported Measures: Let’s Look Before We Leap. Patient. 2015 Aug;8(4):293-9.
    3. Snyder C, Crossnohere N, King M, et al; PROTEUS-Trials Consortium. The PROTEUS-Trials Consortium: Optimizing the use of patient-reported outcomes in clinical trials. Clin Trials. 2022 Jun;19(3):277-284.
    4. Brundage M. The PROTEUS Consortium: What it is and what it offers to radiation oncology. April 2024. [Accessed on 13th June 2025] Available at: https://www.astro.org/blog/april-24/the-proteus-consortium
    5. Snyder C. “Proteus” Patient-reported Outcomes Tools: Engaging Users & Stakeholders. [Accessed on 13th June 2025] Available at: https://publichealth.jhu.edu/center-for-health-services-outcomes-research/research/patient-outcomes/proteus-patient-reported-outcomes-tools-engaging-users-stakeholders
    6. Snyder C, Brundage M, Crossnohere N; PROTEUS Leadership. The PROTEUS Consortium: Helping You Navigate the Use of Patient-Reported Outcomes in Clinical Trials and Clinical Practice. [Accessed on 13th June 2025] Available at: https://www.isoqol.org/the-proteus-consortium/

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  • Strategizing Optimal Collection and Use of Real-World Data from Expanded Access Programs

    Strategizing Optimal Collection and Use of Real-World Data from Expanded Access Programs
    Strategizing Optimal Collection and Use of Real-World Data from Expanded Access Programs

    Expanded Access Programs (EAPs) are special programs designed to provide patients with serious or life-threatening conditions access to investigational treatments that have not yet been approved by regulatory authorities, offering them a chance to receive potentially beneficial therapies when no other options are available. EAPs not only offer a lifeline to patients but also present an exceptional opportunity to gather Real-World Data (RWD). By strategically harnessing data from EAPs, researchers and healthcare stakeholders can unravel a wealth of insights that contribute to evidence-based decision-making and drive improvements in patient care. However, the successful collection and strategic utilization of data from EAPs require careful planning, ethical considerations, and methodological rigor.[1,2]

    Ethical data collection from EAPs is paramount to their usage as RWD sources. The collection of patient data from EAP must prioritize patient privacy, informed consent, and data security. Striking a balance between patients’ urgent medical needs and their rights as research subjects is crucial.[3]

    A comprehensive data collection strategy begins by defining the specific data elements to be collected from EAP participants. This could include demographic information, disease characteristics, treatment regimens, adverse events, and patient-reported outcomes. Collaborating with healthcare professionals and patient advocates can help identify relevant data points contributing to a holistic understanding of treatment outcomes.[4]

    Establishing a robust data collection infrastructure is pivotal. Electronic health records, patient registries, and digital health platforms can facilitate seamless and standardized data collection. Integrating data collection into routine clinical workflows minimizes the burden on healthcare providers and ensures data accuracy.[4]

    Incorporating patient-reported outcomes (PROs) in the EAPs empowers patients to contribute their perspectives on treatment experiences and quality of life. Digital tools and mobile apps can streamline the collection of PROs, providing real-time insights into patient well-being. Additionally, maintaining data quality and reliability is paramount to drawing meaningful insights from EAPs. Implementing data validation protocols, standardized data entry procedures, and regular data audits mitigate the risk of inaccuracies and enhance the credibility of the collected data.[4]

    RWD from EAPs can also shed light on healthcare resource utilization by providing insights on hospitalization rates, length of stay, and use of medical services. By using this data, healthcare systems can make informed decisions about resource allocation and patient care planning.[3,4]

    Challenges in EAP data collection and utilization are not uncommon. One challenge is the potential for selection bias due to the non-randomized nature of EAPs. Patients with specific characteristics might be more likely to participate, introducing bias into the data. To mitigate this, advanced statistical techniques, such as propensity score matching, can be employed. Additionally, data completeness can be an issue due to variations in healthcare settings and patient engagement levels. Implementing data quality checks and incentives for data submission can encourage comprehensive data collection.[3,4]

    The optimal collection and use of RWD from EAPs hold immense potential to shape healthcare decisions, policy formulation, and patient care. Collaborative efforts among healthcare professionals, researchers, patient advocates, and regulatory bodies are essential to harness the full potential of RWD. As technology advances and data collection methodologies evolve, the seamless integration of EAP data into evidence-based medicine promises a brighter future for patients seeking hope beyond conventional treatment options. By navigating the ethical considerations, implementing robust data collection strategies, and leveraging advanced analytical techniques, stakeholders can unlock the power of RWD to improve patient outcomes and advance medical knowledge.

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    References

    1. Polak TB, Cucchi DG, Schelhaas J, Ahmed SS, Khoshnaw N, van Rosmalen J, Uyl-de Groot CA. Results from Expanded Access Programs: A Review of Academic Literature. Drugs. 2023 May 18:1-1.
    2. Polak TB, Cucchi DG, van Rosmalen J, Uyl-de Groot CA, Darrow JJ. Generating evidence from expanded access use of rare disease medicines: challenges and recommendations. Frontiers in Pharmacology. 2022:1917.
    3. Polak TB, Cucchi DGJ, van Rosmalen J, Uyl-de Groot CA. Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals. BMJ Open. 2022 Jan 6;12(1):e052186.
    4. Polak TB, van Rosmalen J, Uyl-de Groot CA. Expanded Access as a source of real-world data: An overview of FDA and EMA approvals. Br J Clin Pharmacol. 2020 Sep;86(9):1819-1826.

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