Proceedings of a Workshop—in Brief

Convened September 29–30, 2025

Innovative Person-Centered Clinical Cancer Research

Proceedings of a Workshop—in Brief

Person-centered clinical research addresses questions that are informed by patient experiences, needs, and perspectives. It actively involves patients in all stages of research, including design, activation, enrollment, data collection, completion, and outcome reporting (Farah et al., 2023). This type of research can positively influence the outcomes of clinical research by providing a more accurate representation of real-world patient experiences, and in oncology, a person-centered approach is beneficial because treatments have such a profound impact on the daily lived experience of patients and their caregivers. Important strides have been made over the last several decades to capture and represent the patient voice in cancer clinical research. However, collection and analysis of patient-reported data can face challenges in terms of completeness, reliability, and validity, and there are continued opportunities to improve the design, conduct, and analysis of clinical trials through alignment with the perspectives, needs, and experiences of patients.

The National Cancer Policy Forum, in collaboration with the Forum on Drug Discovery, Development, and Translation of the National Academies of Sciences, Engineering, and Medicine, convened a workshop on September 29 and 30, 2025, to examine the challenges and explore potential opportunities to advance the conduct of innovative, person-centered clinical cancer research to improve patient outcomes. Workshop discussions focused on challenges and opportunities in enabling clinical cancer research and potential ways to support the oversight and regulation of novel person-centered clinical research methods. Speaker presentations and the workshop web-cast have been archived online.¹

Lawrence Shulman from the University of Pennsylvania presented the overarching goal of the workshop and emphasized a broad view of clinical cancer research that includes both pharmacological and nonpharmacological clinical trials and population-based research across the cancer care continuum. Observations from individual participants and suggestions to advance the conduct of innovative, person-centered clinical cancer research to improve patient outcomes are summarized in Boxes 1 and 2.

THE VALUE OF PERSON-CENTERED CLINICAL CANCER RESEARCH

Monica Bertagnolli, former director of the National Cancer Institute and National Institutes of Health and now at the Harvard Kennedy School of Government, commended the planning committee’s approach for considering person-centered clinical research within a broader context. Bertagnolli began by stating that the overarching goal for everyone who works on cancer is to

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eliminate suffering, by both preventing cancer and diagnosing and treating it effectively, with minimal or no adverse effects. She stated that although this goal is widely acknowledged, the various partners in cancer research and care, including patients, might each have different interpretations of what person-centered research should achieve. Bertagnolli said that ensuring that patients derive meaningful benefit from research requires consideration of the multiple perspectives and incentives contributed by everyone involved. She emphasized that when person-centered clinical research is governed by sound scientific principles, transparency, and effective communication, public trust in research and its application in medicine is strengthened. Bertagnolli said that achieving this goal requires sustained collaboration and coordination across disciplines, institutions, geographies, and communities. Finally, she emphasized that person-centered clinical research is an essential feature of a learning health system, in which science, incentives, and culture are all aligned to achieve continuous improvement, innovation, and quality in the nation’s health care system (Madara et al., 2025). “Everyone wins if we conduct research that is person centered,” she concluded.

Patients and Caregivers as Active Participants in Clinical Research

Julie Maués, a cancer survivor and co-founder of Guiding Researchers and Advocates to Scientific Partnerships, said that together, patients and researchers in clinical cancer research can advance more breakthroughs in cancer care. Maués said that patients have knowledge about their diseases and can offer valuable input into clinical research. She said that patients can provide insights into approaches that are practical and patient friendly and treatment challenges that are often ignored. In addition, actively engaging patients could expand public understanding and trust in science.

Ji Im from CommonSpirit Health added that mutual respect and co-learning between patients and researchers as partners in clinical research could also foster patients’ trust in the discovery process. As a cancer survivor, Im said that the process of navigating treatment options can be complex and emotionally taxing for patients. Navigating the health care system to seek high-quality cancer care is difficult in general, but it is extremely challenging for patients looking for clinical trials they might be eligible for, especially when experiencing symptoms or side effects. Eligibility for clinical trials is complex, given the numerous inclusion and exclusion criteria, which often leave patients out of the process. Financial and insurance concerns are also challenging. She said that health insurance companies have different policies that often vary by states and regions. To overcome these challenges, Im proposed four opportunities: exploring the use of clinical trial navigators who know the culture at the access points for patients; improving system-level collaboration among institutions to encourage transparent information sharing; harnessing artificial intelligence (AI) to expedite the eligibility process, especially because a patient’s eligibility evolves as new clinical trials become available; and developing and promoting policies that include better insurance coverage for patients’ initial consultations with the clinicians leading the clinical trial or drug design. “Without insurance coverage of the required initial visit to a clinician, the cost may be prohibitive for patients,” Im said.

Ricki Fairley, a breast cancer survivor and co-founder of Touch: The Black Breast Cancer Alliance said certain populations are underrepresented in clinical trials even though they are disproportionately affected by the burden of those diseases. For example, compared to non-Hispanic White women, non-Hispanic Black women with breast cancer have a 41 percent higher death rate, 39 percent higher risk of recurrence, and 71 percent higher relative risk of death, but they are underrepresented in breast cancer clinical trials (Fairley et al., 2024). Fairley and colleagues examined why Black women choose not to participate in clinical trials (Fairley et al., 2024) and found that about 95 percent of participants were aware of trials, but challenges such as financial expenses and mistrust of the medical system hindered participation. Although clinicians, community organizations, and breast cancer support groups were found to be trusted sources, more than half of participants indicated that they initiated discussions on clinical trials rather than their clinicians. Fairley said that it is critical to focus recruitment activities in settings where people receive their care and for clinicians to proactively discuss clinical trials with patients and provide them with adequate informa-

tion about the potential risks and benefits. Furthermore, Fairley said that her organization leverages technology through its website and social media to reach out to patients diagnosed with breast cancer and help to match them with potential clinical trials.

Susan Mazanec from Case Western Reserve University said that family caregivers are also essential partners in clinical cancer research and care. She noted that the experience of a cancer diagnosis and treatment extends beyond the patient to the social network of the patient, and she emphasized that it is critical to engage family caregivers as members of the health care team in early discussions about a patient’s clinical trial decisions. Family caregivers are key to maintaining the patient’s physical and mental well-being throughout cancer treatment, and although they are an integral part of the care team, they are rarely recognized and acknowledged. Mazanec said that when they are well integrated into the clinical research process, family caregivers can help to increase recruitment and enrollment rates in clinical research and reduce dropout rates. She added that clinicians have a responsibility to improve a caregiver’s level of health literacy and support them in their role. Mazanec suggested that researchers develop a systematic approach to obtain information from caregivers throughout and after the clinical trial process.

Kristin Carman from the Patient-Centered Outcomes Research Institute (PCORI) said that meaningful engagement of patients and caregivers in clinical cancer research throughout the entire process has a critical role in generating patient-centered evidence that matters, supporting better-informed health decisions, and achieving the ultimate goal of ensuring optimal health for all. Carman said that the pathway to achieving this goal starts with identifying the research questions and outcomes that matter to patients and their care partners; influencing the conduct of studies to enable more representative study samples; improving the quality of studies to broaden the relevance of the research; and providing information that is trusted by patients and care partners. She described PCORI’s Foundational Expectations for Partnerships in Research,² which guides awardees and applicants to improve engagement and strengthen partnerships, with the aim of making results more patient centered, relevant, and useful. Carman highlighted some areas where additional research is needed, including the development of more reliable and valid process and outcome measures of engagement; more effective engagement activities and approaches; more information about what works for whom, under what conditions, and with what resources; how to sustain partnerships; and more evidence for engagement throughout the research life cycle. Carman emphasized that when patients are made true partners in a clinical study, it increases the real-world relevance of the findings, facilitates integrating results into clinical settings, and augments dissemination of the results into the communities of interest.

CONSIDERATIONS FOR EMBEDDING PERSON-CENTERED PRINCIPLES ACROSS CLINICAL CANCER RESEARCH

Several participants reflected on lessons learned over the years in designing and operationalizing person-centered clinical cancer research.

Patient-Reported Outcome Measures (PROMs) and Meaningful Patient Outcomes

Patients may experience adverse drug effects during clinical cancer research. Ethan Basch from University of North Carolina at Chapel Hill said some of these, including nausea, changes in taste, shortness of breath, fatigue, and muscle pain, are highly subjective experiences that are best known by patients themselves. However, Basch said, these symptoms are often not directly reported by the patients but instead typically recorded through Common Terminology Criteria for Adverse Events (CTCAE), a library of individual items designed for clinicians or investigators.³ He noted that the CTCAE often cannot adequately convey the true patients experiences, because it is difficult for a clinician to fully understand the subjective experience of their patient (Atkinson et al., 2012; Basch et al., 2014). As a result, Basch said, this approach may underestimate the potential risks and overestimate the potential benefits. To enable patients to directly report their symptoms and adverse effects, the National Cancer Institute (NCI) oversaw developing a patient-reported version of CTCAE; data from its PRO-CTCAE tool⁴ are widely used in global drug development and begin-

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ning to be included in drug labels, Basch said. Using well-established methods, like PRO-CTCAE, for collecting and reporting adverse effects improves patient centeredness of clinical research. He added that the Food and Drug Administration (FDA) encourages industry sponsors to integrate PROs into clinical trials to improve the quality of data for making the best decisions about treatments (Kluetz et al., 2018). Basch also noted growing interest, including from the FDA, NCI, and Friends of Cancer Research to integrate PROs into early-phase drug trials to enable patient-centered dose selection.⁵

Bryce Reeve from Duke University School of Medicine discussed the importance of assessing symptoms and functioning in clinical cancer research to evaluate treatment benefit. Reeve said that FDA defines clinical benefit as a positive effect on how a patient feels, functions, or survives.⁶ He described a toolbox of measures for clinical outcome assessments (COAs), which include patient-reported, clinician-reported, observer-reported, performance, and digital measures. He emphasized that patient self-reporting is prioritized across these different types of measures. FDA has been promoting the use of COAs, including patient-reported outcomes.⁷ Reeve delineated a set of questions that he said are essential for a researcher to ask while designing a study to effectively capture meaningful clinical benefits:

• What symptoms and functional outcomes should be assessed?
• Who is the best source for this information?
• Is there an available, fit-for-purpose COA?
• How can the COA be integrated into the study to maximize data quality and value?
• What is the best way to summarize findings and make decisions?

Reeve said that “patients are not just simply participants in your study; they should be actively involved in deciding what the research questions should be.”

PROMs as Study Endpoints

Claire Piccinin from the European Organisation for Research and Treatment of Cancer (EORTC) said that its Quality of Life Group’s approach to patient-reported outcome (PRO) measurement has evolved considerably to align with regulatory and health technology assessment (HTA) standards.⁸ Piccinin said that the advancement of measurement science, more widespread implementation of PROMs, and significant evolution of innovative cancer therapies have resulted in the need to adopt more flexible approaches to PRO assessment to ensure that PROMs remain relevant and fit for purpose (Piccinin et al., 2025). The foundation of the EORTC measurement strategy is composed of two elements. The first is a core questionnaire that asks patients about their status regarding physical, role, emotional, social, and cognitive functioning and experience of symptoms including fatigue and pain, along with other generic quality of life (QOL) indicators. The second is a disease-, treatment-, or population-specific module that addresses targeted health-related QOL issues. Piccinin said that flexibility can be achieved through the addition of customized questionnaires (i.e., item lists) and open-ended questions to capture additional symptoms that are not covered by the selected measures. She added that the measurement strategy continues to evolve to ensure that measures remain meaningful and patient-centered. To enable PROs to be effectively used for regulatory decision making, a workshop⁹ was hosted by EORTC and the European Medicines Agency (EMA), which emphasized that PRO endpoints need to be treated the same as any other relevant clinical endpoint, by having well-defined research objectives, using appropriate PROMs, and ensuring rigorous methodological standards. The PRO data are used by both the EMA and national HTA bodies to inform risk–benefit assessment, product labeling, and cost effectiveness and reimbursement decisions. Piccinin said that ongoing col-

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laborations among clinicians, patients, regulatory agencies, and HTA bodies promote impactful use of PRO data. laborations among clinicians, patients, regulatory agencies, and HTA bodies promote impactful use of PRO data.

Vishal Bhatnagar from the Oncology Center of Excellence (OCE) at FDA said that PROMs are often collected only in late-stage or registration oncology clinical trials, but it is advantageous to measure PROs during early-phase trials and throughout the drug development process. Bhatnagar said that FDA has a dedicated set of resources to advance patient-focused drug development, much of which is driven by the authorization of the Prescription Drug User Fee Act¹⁰ and 21st Century Cures Act, which highlights the importance of patient-focused drug development. OCE has a designated core outcome set that should be collected in every cancer trial.¹¹ This includes disease symptoms, treatment related symptoms, physical and role functioning, and overall side effect impact. Bhatnagar said this list does not represent the full range of PROs but is the minimum set to understand patient experiences. He added that to increase the relevance of the data, it is important to collect information from patients at appropriate time points, such as when side effects are likely to occur. This is particularly key because that information could help in assessing safety, efficacy, and tolerability, including for dose escalation and expansion studies. For regulatory purposes, how the intended PRO data will be used ought to be articulated, for example, as additional supportive or exploratory data to inform overall benefits or risks or descriptive information for labeling. “PRO concepts should be well understood; instruments should be fit for purpose and well defined,” Bhatnagar said, adding that “well-collected and meaningful PRO information should be communicated to patients, caregivers, clinicians, health care payers, and regulators.”

Gregory Abel from Dana-Farber Cancer Institute and Harvard Medical School said that a range of bioethical frameworks, including principlism,¹² virtue ethics,¹³ utilitarianism,¹⁴ deontology,¹⁵ feminist ethics (ethics of care),¹⁶ and narrative ethics,¹⁷ offer guidance for collecting PROs and reporting findings of clinical research to participants (Childress and Beauchamp, 2022). Abel said that surrogate endpoints¹⁸ and PROs are increasingly used to assess efficacy in cancer clinical care, yet PRO collection and reporting is inconsistent, and data are also rarely included in primary publications or relegated to a supplement when included (Kemp and Prasad, 2017; Patel et al., 2024). He said that this lack of requirement by journals for reporting PROs could deprive researchers and patients of information needed to best assess the risk and benefit of a particular treatment. Also, informed consent may be violated when the findings are not reported (Blackstone et al., 2025). Abel said that understanding the collection and reporting of PROs in clinical research is crucial to holistically evaluating the benefits and burdens of new treatment. He urged journals to make it an expectation for researchers to report meaningful, well validated, and rigorously assessed PROs when designing and reporting results from clinical trials.

Recruitment and Retention of Trial Participants

Recruitment and retention of patients in clinical trials are complex processes. Chanita Hughes-Halbert from University of Southern California said that a lot of work has been done on understanding challenges to participation to develop guidance and programs aimed at addressing these, such as access to care, awareness about clinical trials, and workforce diversity, to increase the enrollment and retention rates (Hughes-Halbert et al., 2016; McDonald et al., 2014; Reopell et al., 2023). Hughes-Halbert said that while enrollment rates have increased across all demographic groups in recent times, they are still not representative of the cancer incidence for dif-

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ferent populations (Farooq et al., 2025). Hughes-Halbert listed three questions that may be used to determine whether representation of diverse populations is adequate in clinical trials and across all cancer research (Corbie-Smith et al., 2004): is the proportion of groups consistent with representation in the catchment area?; is the number of minorities sufficient to allow subgroup analyses?; and does the enrollment reflect the distribution of disease risk and outcomes? Hughes-Halbert noted inefficiencies in recruitment and retention that start from the screening process and continue into enrollment and then to retention. She said that behavioral science disciplines could offer insights into how to address those inefficiencies by understanding what makes it easy or difficult for individuals to participate in cancer research and how to generate the evidence base for recruitment strategies (Gillies et al., 2021).

Ruma Bhagat from Genentech, a member of the Roche Group, said that offering clinical trials in rural and other underserved areas is a scientific imperative because it could ensure that research findings are generalizable and applicable to all patient populations, address health disparities and provide equal access to promising new therapies, accelerate patient enrollment, increase clinical trial efficiency, and reach new markets. She also noted a growing emphasis from public health advocates on increasing diversity in clinical trials. However, core challenges, including geographic, socioeconomic, language, and infrastructure and historical mistrust of the medical system, hinder this imperative. She cautioned about a tendency to equate “underresourced” areas with being rural, noting that urban underresourced communities may be near hospitals but still lack access. “Sponsors need to tailor solutions for both, not apply a one-size-fits-all model,” Bhagat said. To facilitate the expansion of clinical trial participation, it is critical to foster and build sustained collaboration and coordination across multiple disciplines and sectors and other locally trusted organizations.

Regulatory and Structural Considerations for Person-Centered Research

Shulman said that advancement in cancer treatment is, in part, the result of partnerships between the pharmaceutical industry and public entities. He said that while industry and public funders share many of the same goals, patient engagement and interests may be prioritized differently. He said that patients expect access to treatment that will give them the best chance for survival, best QOL possible, and affordable, high-quality care delivered safely and efficiently. Shulman said that publicly funded clinical trials often aim to prioritize optimized treatment for patients, focusing on survival, QOL, optimal dosing (often reducing dose and frequency without compromising efficacy to the benefit of the patient), and other research areas related to screening and prevention, pediatric cancer, rare cancer, and improving public health (Unger et al., 2025). In contrast, industry often prioritizes rapid accrual, efficient trials that generate high-quality data for initial regulatory approval to enable early drug commercialization, and then seek label changes for a broader patient population, which expands market share and creates shareholder value. He presented data on clinical trials designed to compare chemotherapy drugs from the same class to show how the differing aims of trials could influence breast cancer care delivery with industry sponsored trials prioritizing regulatory approval and not assessing optimal patient quality of life (Qi et al., 2013; Slamon et al., 2011; Sparano et al., 2015; Unger et al., 2025).

Shivaani Kummar from Oregon Health and Science University said that cancer treatment has evolved from the standard cytotoxic methods to targeted therapies, which has implications for Phase 1 clinical trials.¹⁹ While the trials still evaluate safety and tolerability, they also now assess whether a drug hits its intended target to show proof of mechanism and early signs of efficacy at the right dose. Kummar said that this has increased the complexity of early-phase trials, restricted patient eligibility, and increased burden for patients and study teams in conducting these studies. She added that the eligibility requirements for patients in Phase 1 trials also impose significant burden on patients. Patients may have

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a significant time commitment for numerous clinic visits and extensive biomarker sampling. She said that defining the right dose has also evolved. She noted that maximum tolerated doses are determined under the premise that “more is better,” but for targeted therapies, efficacy may not be directly related to higher doses (Hochmair et al., 2024), and different doses may be needed for different patient subgroups. Kummar added that to make early-phase trials more person centered, it is essential to foster collaboration across regulators, sponsors, researchers, contract research organizations (CROs), and patients to identify areas where changes could be made to reduce the burden in Phase 1 trials while still ensuring patient safety.

Arun Balakumaran from Pfizer said that in drug development trials, pharmaceutical companies generally prioritize patients’ needs, lived experiences, and outcomes that are meaningful to patients in addition to survival. He said that it is essential to balance meaningful outcomes with operational feasibility and regulatory acceptance. Regulatory pathways remain anchored in traditional endpoints, such as survival, and PRO measures require rigorous validation for regulatory acceptance. He said that person-centric measures could shape both clinical research and market success. Balakumaran said endpoints that provide patient benefits other than survival need to be considered, pointing out that health care payers demand evidence of both clinical and market value. For example, if a treatment regimen reduces functional decline and delays hospital stays, that benefits patients and also provides tangible financial benefits for payers. He suggested that tools to measure PROs do not need to overly burden patients or research study staff, noting that pragmatic clinical trials and AI offer opportunities to overcome these challenges but would require regulatory and community alignment to advance these innovations.

Examples of Innovative Clinical Trial Designs

Traditional clinical trials face numerous challenges, including multiple sponsors and CROs managing a full menu of dozens of trials and excess manual data entry, said Howard “Skip” Burris from the Sarah Cannon Research Institute. A more streamlined model, he said, simplifies the CRO functions for the trial sites by utilizing just-in-time study openings, remote monitoring practices, and no unnecessary repeat site initiation visits. The streamlined model also integrates an automated, real-time electronic health records to electronic data capture transfer step. The information that is entered into a patient’s record is immediately uploaded to the trial sponsor’s case report form, enabling the sponsor to look at the data and make decisions in real time. Automating this step, Burris noted, saves money and frees up study staff to do other things. Technology coupled with human expertise can accelerate prescreening potential participants to match them with suitable cancer clinical trials, Burris said. The process uses experts in molecular profiling who screen patient biomarker data, screeners who prescreen patients for select studies, and curators who compile all the data into the software platform.

Bhagat said that a decentralized approach to clinical trials also offers the opportunity to make clinical research more accessible, equitable, and patient centered, but the regulatory landscape prevents trial sponsors from fully leveraging the advantages of decentralized clinical trials. “Decentralization requires regulatory flexibility, strong partnerships, and community trust,” she said. An ecosystem approach that involves sustained partnerships among community members, sponsors, regulators, patient advocacy groups, health systems, and payers could avoid fragmented approaches and build sustainable infrastructure.

Pat LoRusso from Yale University said that more efforts are needed to diversify Phase 1 cancer clinical trials to ensure that the findings on safety and efficacy are generalizable across all populations (Dunlop et al., 2022; Kahn et al., 2022; Perni et al., 2021). LoRusso and colleagues implemented a hybrid decentralization model for Phase 1 recruitment.²⁰ Their model employed, wherever possible, trial components that could be executed closer to the patient’s home, using procedures that varied from the traditional cancer center early-phase model, where patients were always traveling to a centralized site. Before operationalizing the clinical trial, LoRusso and colleagues conducted a multilevel assessment using qualitative interview and online surveys to determine the unique facilitators and challenges for recruiting underrepresented patients to early-phase cancer clinical trials.

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They found that patients reported the need for more information about cancer etiology, treatment options, and trials; invitations to trials; help with their unmet social needs; and a trusting, positive relationship with the health system and research. They also found that clinicians reported the need for more time and support to have conversations about trials; ways to address patients’ social needs so that a trial does not burden them; more information about trials for which patients may be a fit; and patient and community trust to support the conversation. LoRusso said that nurse navigators presented information to patients in different ways to enhance understanding, including multimedia in various languages. She emphasized that identifying and overcoming social challenges and bringing the trial to the patient has increased recruitment for all patients.

Roy Herbst from Yale University said that clinical trials are separated from health care delivery, and traditional models typically have strict eligibility criteria and complicated regimens (Angus et al., 2024), which create challenges for patient enrollment. A model that integrates clinical trials into the standard practices of a medical system and is also precise and pragmatic could increase enrollment and speed (Grant and Goldberg, 2023). Herbst said that effective public–private partnerships are also critical to efficient person-centered clinical cancer research. He discussed two clinical trial initiatives to demonstrate the impact of such partnerships. First, the Lung Master Protocol (Lung-MAP) was launched in 2014 as a groundbreaking design aimed at transforming metastatic non-small-cell lung cancer (NSCLC) treatment through precision medicine, tailoring therapies to patients who are most likely to benefit (Herbst et al., 2015, 2024). Herbst said that instead of having to undergo multiple diagnostic tests to determine eligibility for many different studies, enrollees are tested just once according to a “master protocol” and assigned to one of multiple trial substudies, each testing a different drug from a different developer (Herbst et al., 2024). In 2019, Lung-MAP 2.0 was introduced to add substudies that evaluate treatment combinations in patients with acquired resistance to targeted therapy. In 2024, Lung-MAP 3.0 was introduced to expand genomic screening through a variety of commercial and academic next-generation sequencing platforms.²¹ “Lung-MAP has improved clinical trial diversity and made drug development faster and more collaborative,” Herbst said. The second initiative is the Lung Pragmatica Trial,²² which removes many of the challenges that prevent people from joining clinical trials through its pragmatic design and broad eligibility criteria. Launched in March 2023, it had accrued 838 patients across more than 2,500 sites in less than 2 years, encompassing a diverse U.S. representation (Reckamp et al., 2024). He said that this design allows for rapid enrollment of patients who are more representative of the population diagnosed with advanced NSCLC and may help broaden the reach of investigational therapies to patient populations often excluded from clinical research, leading to generalizable results. “Pragmatic trials are just one type of design; there are many areas of opportunity to be more thoughtful about how we design trials and source our data,” Herbst added.

Reporting Clinical Research Findings

Basch reported a clinical trial design used in a NCI cooperative group phase III trial (Alliance N1048) in which patient-reported symptomatic adverse events were shared in real-time with site investigators at the point of care to inform CTCAE reporting. He said that the process improved the precision of symptomatic adverse event reports, and better aligned CTCAE reports with the actual patient symptom experience (Basch et al., 2023).

Edgar Braendle from AVEO Oncology said that it is important to report trial results back to participants because it is a sign of respect for their contributions, builds trust and transparency, meets ethical and regulatory standards, improves public understanding of research, strengthens relationships to advocacy groups, and supports patient-centered care and future research. Sarah Greene, a cancer research advocate and health services researcher, as well as a cancer survivor, noted long-standing challenges in returning clinical research results to study participants. She said that key challenges include difficulties in contacting participants years later; reluctance of Institutional Review Boards to allow

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researchers to recontact participants; researchers’ reluctance or inability to produce plain-language summaries for participants; and lack of support by most current funding mechanisms. Jeff Yorio from Texas Oncology agreed and added that long-term follow-up and contact with clinical trial participants can be extremely challenging. Furthermore, he added, many patients with advanced disease would already know whether they responded to the treatment or not, and for those with a terminal illness, patients may no longer be alive when the information is reported.

Greene said that returning results to participants in a cancer clinical trial is very complex and nuanced. She suggested a few questions to consider as guiding principles: what results should be shared; in what form they should be shared, either individualized or aggregated; when they should be shared (e.g., are they clinically actionable or time sensitive); and who should deliver them. Underlying these questions are deeper ethical issues of what patients receive for giving up their time, energy, and data to participate in clinical research. She suggested that progress would require developing a universal approach focused on why, what, when, and how to share results, cognizant of the legal, privacy, and clinical implications. Greene said that it is essential to determine a timeline for returning results as a part of the trial development process and then routinely revisit it as the study unfolds. “If we start to get better at enhancing our organizational and research commitments to sharing results, I think that can increase trust in science and research,” Greene added.

Yorio and Braendle said that reporting results of clinical cancer research remains inconsistent, with large proportions of studies never making their results publicly available. One analysis of 12,240 cancer clinical trials on ClinicalTrials.gov over a 10-year period found that nearly 40 percent do not report the results to the public (Liu et al., 2021). Yorio said that out of the 60 percent that did so, 23 percent were reported only on ClinicalTrials. gov, 22 percent were published just in journal articles, and 16 percent were published online or also in journal articles. Furthermore, single-center, Phase 1, and industry-funded trials were less likely to have publicly reported results. Yorio said that negative or null results are typically not reported, even though they are essential to help refine treatment regimens to avoid repeated ineffective approaches. He encouraged the research community, trial sponsors, regulators, and journals to disseminate negative study results to patients and the broader community. Braendle said that even among most trials with publicly reported results, 20–50 percent remain unpublished within 2–5 years of completion (Chen et al., 2016). Also, discrepancies often arise between primary outcome data published in clinical trial registries and peer-reviewed journals (Alayche et al., 2023; TARG Meta-Research Group Collaborators, 2023). Furthermore, results that are returned to participants are often delayed, incomplete, and not plain-language summaries that could be easily understood (Chan et al., 2014; Speich et al., 2022). These issues contribute to transparency challenges.

Yorio explained that participants in clinical trials have varying motivations and concerns. One recent analysis reported that the five key motivations for patients to enroll were improved illness, access to new and effective treatments, discovery of genetic diseases in the family, free medications, and more attentive care and time with health clinicians (Tolunay et al., 2025). The five key concerns people raised were potential adverse effects of an experimental treatment, being treated as a “test subject,” receiving a placebo or an ineffective drug, potential for exploitation, and breach of medical information privacy (Tolunay et al., 2025). “We need to continue to do a better job of getting this information back to patients. These patients are giving their time, their life, everything to these clinical trials. We owe it to them to give it back,” Yorio added.

Braendle noted the different audiences to be considered when reporting clinical trial results and that each one needs to be addressed using the appropriate medium, such as plain-language summaries, clinical trial registries, peer-reviewed publications, direct communication to patients, and accessible online platforms. He emphasized that posting results to registries or providing plain-language summaries does not preclude publication in peer-reviewed journals.

Braendle explained that the pharmaceutical industry has to comply with regulatory requirements for clini-

cal trials. He said that because most Phase 2 and 3 trials are multinational and multisite, the companies must obey not only U.S. regulations and policies but also those in every country where they conduct research, and the frameworks vary among countries. For example, in Europe, companies are required to not only report results to clinical trial registries within 12 months of the study’s end but also provide lay summaries that are accessible, clear, and patient centric for the protocol synopsis.²³ Braendle said other countries have similar regulations as result of the 2025 World Health Organization guidance on plain-language summaries for clinical trial results.²⁴ He said many companies have created public-facing portals where patients can access lay summaries in multiple languages. Braendle said it is important to make reporting results to patients a standard part of clinical trials, and he encouraged patients’ involvement to develop timely, accessible, nontechnical, and culturally sensitive plain-language summaries. Furthermore, it is important to respect participant privacy and autonomy. “Participants should have the option to opt in and opt out of receiving results,” he said.

Whether collecting PROs or returning clinical research results to participants, the information needs to be meaningful, relevant, and actionable to them, said Lori Minasian from NCI. She said that because clinical trials can be highly complex and the results can be highly nuanced and difficult to convey in very simple language, what can be reported back to participants may depend on the study design. For example, Minasian said that interventional clinical trials are designed around specific clinical questions in which an intervention is expected to modify a disease state or condition, and patients need to understand the hypothesis, anticipated outcomes, and objectives to give consent to participate. She said that the trials also collect additional data and specimens for exploratory research that participants may consent to as well. She mentioned that this creates different scenarios for returning results. Minasian said that historically, cancer treatment trials often did not do so, because patients had advanced disease and many did not live to see the end of the trial. However, treatment regimens have improved, and more cancer survivors want to be informed and engaged. She said that several groups within the NCI-sponsored National Clinical Trials Network have developed trial summaries, and patient advocates have been involved in reviewing and shaping these. In addition, cancer prevention and screening trials typically enroll healthy participants who frequently want to know information about the results. Furthermore, she added that for these trials, it is necessary to carefully develop a coordinated communication plan, from recruitment through the end of the study. Communication may include newsletters, e-mails, and end-of-study letters.

Minasian illustrated the complexity of returning results with three specific clinical trials. First, the Breast Cancer Prevention Trial²⁵ demonstrated positive findings (reduced incidence of breast cancer), so investigators wanted to offer the drug to all participants before FDA approval. Participants received individualized letters indicating whether they were on tamoxifen or placebo, and placebo recipients were offered the drug. However, the clinical consequences for genetic test results were unknown in 1992, and the anonymization of blood samples made the return of individual BRCA results to trial participants not possible. Second, the Selenium and Vitamin E Cancer Prevention Trial²⁶ showed no benefit and a nonsignificant signal for harm, so investigators needed to tell participants to stop the intervention but also asked them to continue to be followed in the study. The researchers worked closely with the Participant Advisory Board to effectively communicate the results to participants before announcing them to the public. Third, the National Lung Cancer Screening Trial²⁷ demonstrated a 20 percent reduction in lung cancer mortality from CT screening, so letters were distributed to all participants providing the results and offering CT scans to control participants. However, some participants learned about the results from the press before the letter reached them. Thus, she said, returning results requires balancing scientific and ethical considerations.

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Leveraging Technology to Enhance Person-Centered Clinical Cancer Research

For many community clinics and small hospitals, especially those in underserved and remote locations, it can be challenging for clinicians to keep up with evolving options for cancer clinical trials, Burris said. LoRusso added that the work required of clinicians to become experts in clinical trials in addition to their existing workload could be untenable. Technology offers opportunities to make clinicians aware of trial availability and potential patient eligibility, but Burris explained that data managers and resource nurses are still needed to get information about available trials to patients in a timely and efficient manner. Cleo Ryals from Flatiron Health noted that technology can also be a resource to disseminate clinical trial results to a broader population. She said that plain-language summaries could be posted on social media platforms to engage with nontraditional scientific populations. This could help build public trust and overcome misinformation, she said.

PUTTING PERSON-CENTERED CLINICAL CANCER RESEARCH INTO ACTION

Deborah Collyar from Patient Advocates in Research said that progress has been made in advancing person-centered clinical cancer research over the past 3 decades, though not nearly fast enough to satisfy the needs of patients, their families, and their caregivers. As a cancer survivor, Collyar said that patients value real-world data collection, but they also want real-world answers that can help them live their best lives.

She said that a notable advancement is the acknowledgment of the expanded role of patients across the entire life cycle of some clinical trials, from design to reporting of results. Others include the adoption of alternative clinical trial designs and approaches, integration of PROs, and the move to engage community members to build trust. In addition, the process has moved toward incorporating plain language and health literacy principles. For example, some language in common usage—such as “patients failed treatment”—places responsibility for the lack of response on the patients, which is both inaccurate and emotionally burdensome. Instead, she suggested saying, “the treatment failed the patients.” She said that to make trials more person centered, it is essential to articulate the goals for each trial and explain why they are the best ones. She advocated for assessing the goals of clinical cancer research through a lens of economic, therapeutic, and social value, adding that this would require a major cultural and operational shift toward patient-centered research. “Patients like me don’t want more treatment, we want better treatment,” Collyar said.

Richard Schilsky from University of Chicago said that U.S health care system remains fragmented rather than coordinated. The misalignment limits the inclusion of all people in clinical trials. Schilsky said one challenge that cancer researchers have faced when including PRO endpoints in a trial has been to integrate them meaningfully into trial design, rather than the more common practice of treating them as an add-on. “We are not going to move the needle … on making these trials more accessible, simpler, easier, more generalizable if we keep doing the same thing and then try to tweak it around the edges to make it a little bit more pragmatic. Let’s start with pragmatic designs and then retreat if it is absolutely necessary,” he said.

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DISCLAIMER This Proceedings of a Workshop—in Brief was prepared by Francis Amankwah, Michael Zierler, and Sharyl Nass as a factual summary of what occurred at the workshop. The statements made are those of the rapporteurs or individual workshop participants and do not necessarily represent the views of all workshop participants; the planning committee; or the National Academies of Sciences, Engineering, and Medicine.

PLANNING COMMITTEE Gwen Darien (Co-Chair), Patient Advocate Foundation; Lawrence N. Shulman (Co-Chair), University of Pennsylvania; Gideon Blumenthal, Merck; S. Gail Eckhardt, Baylor College of Medicine; Roy S. Herbst, Yale University; Randy A. Jones, University of Virginia; Shivaani Kummar, Oregon Health and Science University; Larissa Nekhlyudov, Brigham and Women’s Hospital; Harvard Medical School; Dana-Farber Cancer Institute; Megan O’Meara, Pfizer; Cleo A. Ryals, Flatiron Health; Richard L. Schilsky, University of Chicago; Ann Taylor, formerly at AstraZeneca; Robert A. Winn, Massey Comprehensive Cancer Center, Virginia Commonwealth University. The National Academies’ planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. Responsibility for the final content rests entirely with the rapporteurs and the National Academies.

REVIEWERS To ensure that it meets institutional standards for quality and objectivity, this Proceedings of a Workshop—in Brief was reviewed by Ethan Basch, University of North Carolina at Chapel Hill; Esther Krofah, Milken Institute; Julia Maués, Guiding Researchers and Advocates to Scientific Partnerships (GRASP); and Jeff Yorio, Texas Oncology. Kirsten Sampson-Snyder, National Academies of Sciences, Engineering, and Medicine, served as the review coordinator.

SPONSORS OF THE NATIONAL CANCER POLICY FORUM This workshop was supported by Contract No. 75D30121D11240, Task Order No. 75D30124F00042, and Contract No. 75N98024D00037, Task Order No. 75N98025F0002, with the Centers for Disease Control and Prevention and National Cancer Institute/National Institutes of Health, respectively, and by the American Association for Cancer Research; American Cancer Society; American College of Radiology; American Society of Clinical Oncology; Association of American Cancer Institutes; Association of Cancer Care Centers; AVEO Oncology, Cancer Center at Illinois; Flatiron Health; Lilly, Merck; National Comprehensive Cancer Network; Novartis Oncology; Oncology Nursing Society; Partners In Health; Patient Advocate Foundation; Pfizer Inc.; and Vibrant Health. Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project.

STAFF National Cancer Policy Forum; Francis Amankwah, Director; Anna Adler, Research Associate; Torrie Brown, Program Coordinator; Makeda Haughton, Senior Program Assistant; Julie Wiltshire, Senior Finance Business Partner; and Sharyl Nass, Senior Program Director, Health Care and Public Health. Forum on Drug Discovery, Development, and Translation; Carolyn Shore, Director.

SUGGESTED CITATION National Academies of Sciences, Engineering, and Medicine. 2026. Innovative Person-Centered Clinical Cancer Research: Proceedings of a Workshop—in Brief. Washington, DC: National Academies Press. https://doi.org/10.17226/29369.

For additional information regarding the workshop, visit https://www.nationalacademies.org/projects/HMD-HCS-25-03/event/44567.

Copyright 2026 by the National Academy of Sciences. All rights reserved.