Common sense oncology values to action: a patient-led framework for randomized clinical trial design

Abstract

Background

Randomized controlled trials are the foundation of oncology drug development, yet increasing concern exists that some prioritize regulatory approval over outcomes that matter to patients. The Common Sense Oncology (CSO) initiative was founded to reorient research toward patient-valued endpoints. The CSO Patient Priorities Working Group sought to codevelop a pragmatic, patient-centered framework articulating values and actions to guide the design, conduct, and reporting of oncology randomized controlled trials.

Methods

A multiphase, mixed-methods process included literature review, thematic analysis, and international stakeholder engagement. Eight focus groups were held between April 2024 and January 2025 with 131 participants across 7 countries, including people with lived cancer experience, advocates, clinicians, and trialists. Feedback from surveys and structured discussions—quantitative ratings and qualitative insights—informed iterative framework refinement.

Results

The final framework defines 9 core values with associated actions: meaningful research question, patient partnerships, informed consent, meaningful benefit, generalizability, optimal comparator arm, minimal participant burden, timely feedback of results, and clear dissemination. All values received high endorsement (median 8-9 on a 9-point numeric rating scale [from 1 (least important) to 9 (most important)]). Qualitative analysis emphasized meaningful endpoints, transparent communication, equitable inclusion, fair compensation, and plain-language consent.

Conclusions

The CSO Values to Action Framework provides a validated, internationally informed structure for trialists, ethics boards, funders, and advocates. It offers a practical tool to align oncology research with what matters most to patients, promoting more ethical, inclusive, and relevant clinical trials.

Background

Common Sense Oncology (CSO) is a grassroots initiative founded in 2023 by an international consortium of clinicians, academics, and patient advocates to ensure cancer care and research are focused on outcomes that matter to patients.¹ The CSO movement is rooted in 3 major work streams: evidence generation, evidence interpretation, and evidence communication. As part of this broader vision, CSO has published a checklist outlining principles for the design, analysis, and reporting of phase 3 randomized clinical trials (RCTs).²

RCTs are the gold standard for evaluating cancer treatments and updating standard care.^1,3 However, some contemporary oncology RCTs have been criticized for prioritizing regulatory approval over outcomes that truly matter to patients.^4,5 In particular, a majority of registration trials in recent years have used putative surrogate primary endpoints that may not translate into meaningful benefits such as prolonged survival or improved quality of life.⁶ These trials also suffer from other biases that compromise the validity and generalizability of trial findings.⁶ The recently published CSO RCT checklist attempts to address these issues, including recommending the inclusion of patient input in trial design and reporting.

The patient voice has become more prominent in cancer research over the past decade. Patients are now increasingly recognized as end users of research who should be involved in all stages of the clinical trial process, from conception and design to ethics review, conduct, and dissemination of findings.⁷ Several organizations have developed disease-specific or clinical trial–specific charters or patient rights documents that advocate for the inclusion of patient perspectives in research. Examples include the Canadian Cancer Clinical Trials Stakeholder Charter,⁸ the Stakeholder-Centric Engagement Charter,⁹ and the European Cancer Patient’s Bill of Rights.¹⁰ Although important, these often lack specificity about actionable steps or measurable criteria that can be consistently applied across different settings.

In parallel with the development of the CSO RCT checklist, the Patient Priorities Working Group within CSO launched an engagement program to develop a patient-centered value framework that articulates specific values and actions to be embedded in the design, conduct, and reporting of oncology RCTs. The aim was to cocreate a pragmatic, values-based tool that could support patient-centered research design and be used by trialists, ethics boards, funders, and patient organizations alike. This framework was designed to be applicable across diverse cultural and socioeconomic contexts, with a focus on phase 3 trials.

Methods

A working group was formed from members of the CSO group and included individuals with lived experience of cancer, medical oncologists, patient advocates, clinical trial leaders, and researchers with expertise in patient engagement. The group undertook a multistep process to develop the framework. The protocol was submitted to the Queen’s University Research Ethics Board who granted an exemption.

Step 1: Drafting the initial framework

We conducted a comprehensive review of published literature on patient frameworks in clinical trial design and cancer care using PubMed and supplemented with grey literature identified through web searches, references from included articles, and outreach to professional and patient advocacy networks. We reviewed existing charters and patient rights documents. Thematic analysis was performed to identify gaps in current frameworks and to inform the content of the charter. Two authors (C.J. and M.T.) developed an initial draft framework comprising value statements and action points to give effect to the values (Table S1), which was then reviewed and revised in collaboration with the broader author group.

Step 2: Initial focus group and major revision

Following initial drafting, one focus group was undertaken as a pilot. Focus groups were conducted online and began with a presentation of CSO’s mission and the draft framework. The first author (M.T.) facilitated a guided discussion of the values and action statements, drawing on survey feedback to explore areas of consensus and divergence. Participants were encouraged to elaborate on their survey responses and highlight any missing elements. Survey comments and focus group discussions were recorded and analyzed thematically.

For the New Zealand (NZ) pilot, an invitation was shared broadly among academic and patient advocate communities without restriction on registration or attendance. Prior to the focus group each participant received the draft framework and an online survey via SurveyMonkey. Respondents were to rate the importance of each proposed value and corresponding action on a 9-point numeric rating scale (1 = least important, 9 = most important). Respondents could provide free-text comments for each item and suggest edits or additional actions. Demographic data (age, gender, and country of residence) and connection to cancer were collected. No identifying data were collected, nor did we collect information about any patient’s specific diagnosis or treatment. To ensure responses reflected patient perspectives, we excluded survey responses from individuals who identified as researchers or clinicians.

The NZ pilot focus group was held in April 2024 with 59 attendees. This focus group identified the need for clearer language and refinement of the endpoints section, which led to a major revision of the framework. Therefore, we analyzed survey feedback from the first and subsequent groups separately. Because of the complexity of managing a large and diverse meeting we also elected to narrow future focus groups to smaller audiences focusing primarily on individuals with lived experience of cancer and advocates; clinicians and researchers were not included in future focus groups.

Step 3: Subsequent meetings focused on input from individuals with lived experience of cancer

Between May 2024 and January 2025, we held 7 more focus groups with participants recruited through national organizations in India, the United States, Canada, Europe, South Africa, and Brazil. Patient and advocate participants were recruited through national and regional organizations, including the US National Breast Cancer Coalition, the Canadian Cancer Trials Group (Canada), the European Organisation for Research and Treatment of Cancer (Europe), and others.

Seven focus groups (including the NZ pilot) were conducted in English; the Brazilian group used a translated Portuguese version of the survey. The premeeting survey, meeting structure, and facilitated discussion followed the same format as described above. After 8 focus groups, thematic saturation was reached.

Step 4: Finalizing the framework

Pilot responses were analyzed separately because of the major revision undertaken after the first focus group. Quantitative analysis of survey data involved descriptive statistics (medians, means, standard deviations, interquartile ranges, and coefficients of variation). Statistical comparisons were made using the Mann–Whitney U and Kruskal–Wallis tests, with Bonferroni corrections for multiple comparisons. Analyses were performed across subgroups based on age (younger than 45, 45-64, and 65 years and older), gender, country income level (high income vs low and middle income), and connection to cancer (survivor, living with metastatic disease, family member, or other with a request to specify).

Qualitative data from open-text survey responses and focus group transcripts were thematically analyzed to identify recurrent concepts and perspectives. The initial draft framework was revised based on these insights, with 9 agreed values and several action statements per value agreed, with saturation of feedback and high consensus reached. The final version of the charter was reviewed and approved by the CSO Patient Priorities Working Group and CSO leadership.

Results

A total of 59 eligible responses to the prefocus group questionnaire were collected from the NZ pilot (Table S2), and 72 from the other 7 focus groups. Participant characteristics are described in Table 1. Respondents were mostly located in the United States (26%), Canada (21%), India (21%), and Brazil (14%).

Table 1.

Participant characteristics (postpilot).

Characteristics	No. (%)
Gender
Female	52 (72)
Male	18 (25)
Missing	2 (3)
Age, y
Younger than 45	13 (18)
45-64	31 (43)
65 and older	26 (36)
Missing	2 (3)
Country of residence
Brazil	10 (14)
Canada	15 (21)
Ghana	1 (1)
Greece	1 (1)
Guyana	1 (1)
India	15 (21)
Nepal	1 (1)
South Africa	5 (7)
United Kingdom	3 (4)
United States	19 (26)
Missing	1 (1)
Connection to cancer
Survivor	36 (50)
Living with metastatic cancer	9 (13)
Family member	9 (13)
Othera	16 (23)

^aPatient advocates, people undergoing cancer treatment who may not self-identify as a cancer survivor.

Respondents located in high-income countries constituted slightly more than half (53%) of participants. Half (50%) of participants self-identified as cancer survivors, while 13% were living with metastatic cancer; 13% were family members, and 22% expressed a different connection to cancer, often in treatment.

The final version of the charter is presented in Table 2. The original draft framework had 8 core value statements. The final framework comprises 9 core values with associated actions, having added a value specific to informed consent. For the purposes of clarity, in the results below, we describe the core values relative to their final numbering in Figure 1.

Table 2.

Common Sense Oncology Values to Action Framework to support patient-centered oncology trials.

Value 1: Meaningful questionsClinical trial research in oncology should focus on answering treatment questions that are meaningful to patients and their communities.
Action 1a: Define unmet needs	Researchers, in collaboration with patients, caregivers, and advocates,a should identify and clearly define unmet needs, considering not only treatment gaps but also cost, treatment burden, and care delivery. Trials should seek to improve patients’ quality of life, enhance their overall well-being, and promote equitable access to care.
Value 2: Patient partnershipPatients are essential partners in research.
Action 2a: Codesign with patients	Studies should be codesigned with a diverse group of patients, including trained patient advocates and representatives from Indigenous and underrepresented communities. Engagement approaches should be tailored to varying levels of advocacy experience, ensuring that all involved voices are heard and valued, regardless of expertise.
Action 2b: Involvement at all stages	Patient advocates, including those with lived experience, should be involved at every stage of clinical trials, from design to interpretation and dissemination of results.
Action 2c: Informed participation	Patients and advocates should be sufficiently informed and supported to enable meaningful patient engagement, participation, and ensuring that their perspectives are integrated into all aspects of the trial process.
Value 3: ConsentA high-quality informed consent process is necessary to help people make their own informed and voluntary decisions.
Action 3a: Participant understanding	Informed consent in clinical trials must ensure that participants fully understand the study, including its risks and benefits, using clear, plain language that is culturally sensitive and accessible.
Action 3 b: Plain language	Informed consent documents should present trial details using plain language and simple explanations, with additional layers of information available for those who seek more technical details.
Action 3c: Ongoing updates	Informed consent should be an ongoing process, with participants regularly updated on any changes and risks and encouraged to ask questions. Patient advocates should be involved in reviewing consent materials to ensure clarity and cultural relevance.
Value 4: Meaningful benefitsTrials should assess whether new treatments provide clinically meaningful benefits, such as meaningfully improved survival or quality of life.
Action 4a: Quality of life	Quality of life, measured through patient-reported tools, should be a key outcome in all trials, especially those aiming to change standard practice or using surrogate endpoints.b
Action 4b: Endpoint validation	Overall survival is a key endpoint in advanced disease trials; if surrogate endpoints are used, they must be validated or justified.
Action 4c: Follow-up	Early stage trials using disease-free survival as a primary endpoint should conduct long-term follow-up for overall survival, late-onset toxicities, and quality of life to help inform patient treatment decisions.
Action 4d: Reporting overall survival	Progression-free survival (PFS),c although it may have intrinsic value for some patients with advanced cancer, should not replace clinically meaningful outcomes like overall survival or quality of life. Trials using PFS as a primary endpoint should also measure and report on overall survival and quality of life.
Action 4e: Appropriate sizing	Trials should be designed with an appropriate number of participants to ensure reliable results, avoiding studies that are too small to detect meaningful changes in outcomes or too large to yield results that are statistically significant but not clinically meaningful.
Value 5: GeneralizabilityClinical trials should be designed so that the results are generalizable to the patient community who might receive the treatment in the future.
Action 5a: Representative sample	Trial entry criteria should reflect diverse populations, including age, race, ethnicity, socioeconomic status, and disease characteristics, aiming for a sample that mirrors the future treatment population.
Action 5b: Minimize exclusions	Exclusions because of comorbidities and age should be minimized, with clear justification for any medically necessary exclusions.
Action 5c: Inclusive participation	Efforts should focus on increasing the participation of Indigenous, minority, and low-income groups through culturally sensitive and ethically appropriate methods, including trust-building initiatives. Researchers should make intentional efforts to conduct trials and include participants in lower-income countries and communities.
Action 5d: Reporting on inclusivity	Researchers should report on methods to increase inclusivity, sharing successes and challenges to ensure transparency and continuous improvement.
Value 6: Best comparatorControl arms of studies should always include best available standard care to ensure ethical and meaningful comparisons with the new treatment.
Action 6a: Standard of care	Patients should always receive current best care in a trial control arm. The use of inferior treatments as control arms can skew trial results and make new treatments appear more effective than they truly are.
Action 6b: Postprotocol therapy	Researchers should make every effort to ensure that trial participants can access the best available postprotocol therapy,d reflecting real-world practice. This therapy should be described in the report of trial results and provided whenever possible.
Action 6c: Standardization of care	Researchers should ensure that best available care is standardized across all sites and countries participating in the trial, taking into account the limitations and variations in health-care resources and accessibility.
Action 6d: Crossover	Crossovere to the best available care, when appropriate, should be allowed for participants in the control arm if the trial shows that the new treatment is more effective.
Value 7: Minimal burdenTaking part in trials should carry the least necessary additional burden for patients.
Action 7a: Trial-related procedures	The additional burden of trial-related procedures should be minimized wherever possible. All additional procedures must have a clear purpose for safety or benefit.
Action 7b: Compensation	Participants should receive compensation, preferably upfront, for direct expenses incurred by taking part in trials, including travel, parking, and lodging when applicable. Compensation should be fair and equitable.
Value 8: Feedback to participantsReporting trial results to participants respects their contribution, ensuring transparency and fostering trust in the research process.
Action 8a: Dissemination of results	Participants and, if appropriate, their families should be asked if they want to receive results of the trial. Respect for patient preferences should be maintained.
Action 8b: Accessible results	For participants opting to receive trial results, aggregate results should be provided in a timely and accessible manner, ensuring they have a complete understanding of the findings.
Action 8c: Communicating benefits	Results should clearly state the absolute benefits of the treatment and explain how these benefits may impact patient outcomes that matter to them.
Value 9: Clear dissemination of resultsClear and timely communication of trial results enhances understanding of optimal care within the community.
Action 9a: Public reporting	Trial results should be made publicly available in a timely manner.
Action 9b: Reporting benefits and risks	Researchers should prioritize communicating patient benefits and risks in clear, accessible language including statistical or technical endpoints.
Action 9c: Plain language reporting	Trial results should be conveyed in plain language that is easily understood by patients and communities, emphasizing the benefits and potential risks to help patients make informed decisions.
Action 9d: Plain language summaries	Plain-language summaries of trial results should be made available wherever trial results are shared, including in peer-reviewed journal articles, scientific conferences, and other platforms that are accessible and usable by the patient advocacy community.

^aA patient advocate is someone personally affected by a disease who represents and is accountable to a patient-led, patient-centered constituency. They are knowledgeable, trained, and prepared to actively participate in scientific and medical decision making.

^bA surrogate endpoint is a measure that stands in for a more important result. It is something that can be easily measured during a trial, like how much a tumor shrinks, but it does not directly show whether the treatment improves things that matter most to patients, such as how long they live or their quality of life.

^cPFS is a surrogate endpoint that measures the length of time a patient’s cancer does not get worse after starting treatment. It means the cancer has not spread or grown, but it does not necessarily mean the patient is feeling better or living longer.

^dPostprotocol therapy refers to any treatment a patient receives after the official clinical trial treatment has ended. This can include additional treatments that are given based on the patient’s condition or the doctor’s recommendation after the trial period.

^eCrossover in a clinical trial happens when participants who were originally assigned to a treatment group (like a control or standard treatment group) are allowed to switch to a different treatment group, typically the experimental treatment, if it shows to be more effective. This often happens if the new treatment has been proven to work better, especially after the trial results show clear benefits. The idea is to give patients in the control group access to the potentially better treatment once it has been shown to be effective. However, crossover is not always allowed, especially if it could affect the trial’s results or if the new treatment has not yet proven to be better.

Figure 1.

Stacked bar chart of participant responses. *Value 2 was later split into 2 values (partnership and informed consent), and therefore numbering was altered in the final framework. Abbreviations: DFS = disease-free survival; OS = overall survival; PFS = progression-free survival.

The core values include meaningful research questions, patient partnerships, informed consent, meaningful benefit, generalizability, best comparator arms, minimal added burden on participants, timely feedback of trial results, and clear dissemination of results. All values received high endorsement, with median importance scores of 8-9 on a 9-point numeric rating scale.

The lowest median score was for the “patient exclusions” action, which also had the highest proportion of scores 4-6. There were few statements that had any participants rank values or action as “not important” (score 1-3); these were predominantly on the diversity, exclusions, trial burden, and compensation actions.

Quantitative results showed strong endorsement for the importance of all value and action statements; summary importance rating scores are summarized diagrammatically in Figure 1 and detailed in Table 3.

Table 3.

Quantitative results of draft framework (excludes pilot data).

	Meaningful questions		Partnership
	Value 1	Action 1	Value 2a	Action 1	Action 2	Action 3
Median	9	9	9	9	9	9
stdev	0.73	0.89	1.52	1.05	1.45	1.33
Average	8.7	8.5	8.1	8.5	8.1	8.3
quartile 1	9.0	8.0	7.0	8.0	8.0	8.0
quartile 2	9.0	9.0	9.0	9.0	9.0	9.0
quartile 3	9.0	9.0	9.0	9.0	9.0	9.0
IQR	0.0	1.0	2.0	1.0	1.0	1.0
Min	5	6	1	4	3	2
Max	9	9	9	9	9	9
Coefficient of variation	8%	11%	19%	12%	18%	16%

	Meaningful benefits
	Value 4	Action 1	Action 2	Action 3	Action 4	Action 5
Median	9	9	9	9	9	8
stdev	0.50	0.98	1.32	1.35	1.53	1.49
Average	8.8	8.5	8.1	8.2	8.0	7.9
quartile 1	9.0	8.0	7.0	8.0	8.0	7.0
quartile 2	9.0	9.0	9.0	9.0	9.0	8.0
quartile 3	9.0	9.0	9.0	9.0	9.0	9.0
IQR	0.0	1.0	2.0	1.0	1.0	2.0
Min	7	5	4	1	1	2
Max	9	9	9	9	9	9
Coefficient of variation	6%	12%	16%	16%	19%	19%

	Generalizability
	Value 5	Action 1	Action 2	Action 3	Action 4
Median	9	9	8	9	8.5
stdev	1.46	1.44	1.79	1.62	1.62
Average	8.1	8.1	7.4	8.0	8.0
quartile 1	8.0	8.0	6.5	8.0	8.0
quartile 2	9.0	9.0	8.0	9.0	8.5
quartile 3	9.0	9.0	9.0	9.0	9.0
IQR	1.0	1.0	2.5	1.0	1.0
Min	2	3	1	1	1
Max	9	9	9	9	9
Coefficient of variation	18%	18%	24%	20%	20%

	Best comparator			Minimal burden
	Value 6	Action 1	Action 2	Value 7	Action 1	Action 2
Median	9	9	9	9	9	9
stdev	1.02	0.88	1.12	1.16	1.39	1.49
Average	8.4	8.5	8.3	8.3	8.3	8.0
quartile 1	8.0	8.0	8.0	8.0	8.0	7.0
quartile 2	9.0	9.0	9.0	9.0	9.0	9.0
quartile 3	9.0	9.0	9.0	9.0	9.0	9.0
IQR	1.0	1.0	1.0	1.0	1.0	2.0
Min	5	5	5	5	3	3
Max	9	9	9	9	9	9
Coefficient of variation	12%	10%	13%	14%	17%	19%

	Feedback of results
	Value 8	Action 1	Action 2	Action 3
Median	9	9	9	9
stdev	1.20	0.95	1.38	0.93
Average	8.6	8.6	8.4	8.6
quartile 1	9.0	9.0	8.0	9.0
quartile 2	9.0	9.0	9.0	9.0
quartile 3	9.0	9.0	9.0	9.0
IQR	0.0	0.0	1.0	0.0
Min	1	4	1	3
Max	9	9	9	9
Coefficient of variation	14%	11%	16%	11%

	Clear dissemination
	Value 9	Action 1	Action 2	Action 3
Median	9	9	9	9
stdev	0.84	1.28	0.82	0.92
Average	8.5	8.3	8.6	8.5
quartile 1	8.0	8.0	9.0	8.0
quartile 2	9.0	9.0	9.0	9.0
quartile 3	9.0	9.0	9.0	9.0
IQR	1.0	1.0	0.0	1.0
Min	5	2	5	5
Max	9	9	9	9
Coefficient of variation	10%	16%	9%	11%

^aValue 2 was split into values 2 and 3 at the final drafting phase. Numbering has been updated to reflect the final version after this point.

Qualitative feedback summary

Value 1: Meaningful questions

Recommendations were made for clarifying the definition of unmet needs and broadening this to include accessibility, affordability, and psychosocial burdens. The importance of engaging patients, caregivers, and advocates early and continuously in trial design was noted, ensuring that research aligns with what is meaningful to the patient community. Cultural sensitivity and trial access (reducing barriers to participation) were also mentioned.

Value 2: Patient partnership; and Value 3: Informed consent

These were originally 1 value and were split into 2 in the final framework to separate informed consent as a stand-alone value.

Participants stressed that patients and patient advocates should be included in all stages of clinical trials, from design to interpretation, with patients to be seen as key partners, not just passive participants. Inclusion of underserved groups and diverse populations was strongly supported. Participants emphasized the need for education and training, ensuring patients and advocates have the resources to meaningfully contribute to the trial design process.

Participants emphasized the need for plain language in informed consent documents. Excessive technical details were seen as a barrier to understanding. One participant pointed out that “excess information can create confusion” and “too much detail can obscure the core message.” Participants suggested that information be layered, with a basic summary of key points up front and more detailed information available for those who want it. Participants wanted ongoing communication about trial updates.

Value 4: Meaningful benefit

Although there was strong overall agreement, action 4 and 5 had the highest variability of all action statements (Table 3).

Participants voiced concerns about reliance on surrogate endpoints. Multiple participants emphasized the importance of long-term follow-up to assess the long-term outcomes and toxicities of treatments.

Measuring patient-reported outcomes was considered important; however, several open-ended comments reflect some concern about the effectiveness and reliability of current patient-reported outcomes, particularly in capturing true patient experiences and the toxicity of treatments, as well as the challenge of interpreting quality-of-life measures in a way that truly reflects patient well-being.

Some respondents accepted putative surrogate endpoints like progression-free survival (PFS) for provisional approvals, with a call for confirmatory trials. Related to this, 2 comments referred to requiring mandatory confirmatory trials when a drug is approved based on a surrogate endpoint.

There was a call to clarify “long-term follow-up,” especially in early stage disease. Some felt that the concept of “right-sizing” studies could be more explicitly defined and that the use of terms like overall survival and PFS might be difficult for laypersons and patients to fully grasp.

Value 5: Generalizability

The quantitative data for value 5 showed generally broad agreement on the importance of inclusive recruitment in clinical trials, particularly for underrepresented groups.

Culturally sensitive, ethical recruitment practices were emphasized, as well as increased transparency in how underrepresented populations are included. Some concerns were raised regarding practical challenges in reaching and engaging these groups, particularly in underserved regions. Additionally, the issue of exclusions for comorbidities was mentioned multiple times, with some feedback suggesting that exclusion criteria should be minimized unless there is a clear medical justification.

Value 6: Best comparator

Control arms of studies should always include best available standard of care.

Concordance for value 6 was high quantitatively; this was reflected qualitatively as well. Participants stressed that inferior treatments in control arms should not occur.

Some participants sought clearer definitions of what constitutes the “best” available treatment, with a suggestion that protocols should explicitly state what the standard of care is. This was emphasized particularly in varying health-care systems across different global regions. The cost of treatments in different regions was identified as a barrier, particularly for lower-income populations. The importance of ensuring access to postprotocol therapies at affordable prices, especially in resource-limited settings, was emphasized. The term postprotocol therapy was not clear to all, and recommendations were made for use of clearer language.

Value 7: Minimal burden

Participants called for clear purpose and justification for trial procedures to avoid unnecessary burdens on participants. Participants stressed how important it was to provide full disclosure about potential burdens, such as the frequency of clinic visits and any additional procedures or side effects.

Several participants stressed the importance of fair compensation for direct expenses like travel and lodging, with calls for compensation to be upfront rather than after the trial. Equitable, transparent compensation was a key concern. There was a concern regarding lack of compensation for indirect costs, particularly for lost wages because of trial-related appointments. These costs were burdensome and should inform fair compensation.

Concerns were raised regarding the vagueness of fair compensation and whether it truly reflects the real financial burden on participants. Some feedback indicated that the terms least necessary burden and fair compensation should be clarified to provide more precise guidelines.

Value 8: Feedback to participants

Key feedback included the need for timely and accessible communication, with many participants stressing that patients should be the first to receive results. Several comments highlighted the need to respect patient preferences regarding the communication of trial results. Some participants noted that patients’ desires for receiving results might change over time and that handling results should be sensitive to individual circumstances.

A recurring theme was the need for positive and negative results to be shared in a timely manner. One participant suggested adding a value about stating that publishing all clinical trial results and making data available were necessary to reduce duplicative research and information silos, ensuring better accessibility and utilization of existing findings.

Value 9: Clear dissemination of results

Plain language summaries were seen as important, as was making trial results accessible to patients and communities. One participant suggested breaking communication of results by target audience: (1) trial participants; (2) patient groups; (3) clinical and research community; and (4) the public. Clear communication of absolute benefits was widely supported.

Differences according to respondent characteristics

There were some minor differences by gender. The median score for value 5 (generalizability) was 9 (IQR = 8-9) in female respondents and 8 (IQR = 5-9) in male respondents (P = .008). Value 7 (minimal burden; action: minimize burden) was also given a median score of 9 for female respondents (IQR = 8-9) and 8 for male respondents (IQR = 6.25-9) (P = .035), as was value 9 (clear dissemination of results; action: plain-language summaries: median = 9, IQR = 9-9, vs median = 8, IQR = 7-9; P = .010), respectively.

The fair compensation action of value 7 was rated slightly more highly by people aged younger than 45 years (median score = 9, IQR = 8.25-9) than those aged 45-60 years (median score = 8, IQR = 6-9), potentially reflecting greater financial insecurity by younger-aged working people (P = .024). No other differences were seen by age.

Respondents from low- and middle-income countries (LMICs) scored value 4 (meaningful benefits) slightly more highly than high-income country respondents (median score = 9 vs 8, P = .023). The action statement around fair compensation was also rated slightly more highly by participants from LMICs than high-income countries (median = 9, IQR = 9-9, vs median = 8, IQR = 7-9) (P = .047).

Summary of changes to the framework based on focus group feedback

Minor changes in language were made for clarity and emphasis, for example from “patients and advocates” to people with lived experience. In 2(b), the engagement statement was broadened to reflect the inclusive audience participation. The use of surrogate endpoints was an item of discussion in the initial pilot, and as a result we clarified the statement about PFS as a primary endpoint in 4(d), which was revised to state that “progression-free survival (PFS), while it may have intrinsic value for some patients with advanced cancer, should not replace clinically meaningful outcomes like overall survival or quality of life. Trials using PFS as a primary endpoint should also measure and report on overall survival and quality of life,” highlighting that outcomes based on survival or quality of life, or validated as surrogates for these, were preferred to other endpoints (including nonvalidated putative surrogates). We strengthened action statements from general to specific with subsequent revisions. In the third iteration, a new stand-alone value on informed consent was incorporated, emphasizing plain language, the ongoing nature of consent, and advocate review of materials. We strengthened and expanded language on compensation to include upfront payments and coverage of parking, travel, and so forth. More emphasis was added to statements on statistical power and appropriate trial design. We incorporated actions to reflect new suggestions offered for building trust with underrepresented groups, conducting trials in low-income countries, and being transparent about efforts to improve inclusivity. Finally, we expanded and provided greater detail on communication and dissemination of results to include guidance on plain language summaries, accessibility of information across multiple platforms, and emphasizing clarity over technical endpoints.

Discussion

The CSO Values to Action Framework is unique among other patient-rights centric documents in that it has been specifically designed and tested with people with lived experience of cancer in multiple countries and across differing socioeconomic background and cultural contexts. It outlines core values and actions applicable to trial planning and conduct.

All values and actions received high median ratings, showing strong alignment with patient priorities. Value 1 (meaningful questions) and value 3 (meaningful benefits) were rated especially highly.

Respondents from LMICs rated items related to burden, inclusion, and compensation more highly than those from high-income countries, likely reflecting greater financial vulnerability and higher relative costs of care in these settings.^11,12 Despite small differences (typically 8 vs 9), overall alignment was strong. Therefore, we conclude that the values and actions were consistently regarded as important across patient demographics and the countries and income settings we tested.

Our framework stands alongside other work that demonstrates that patients prefer endpoints that translate to real-world experience such as improved longevity or impact on quality of life over radiologic endpoints, which have less intrinsic value.^13-16 This finding should encourage trialists and research ethics boards to include measures such as overall survival or quality of life as the primary endpoints in late-phase studies and require strong explanations and justifications when surrogates take precedence.

Overall, the CSO Values to Action Framework has now been tested in focus groups in many countries and differing income settings. Although some minor variance is seen between gender, age, and income setting, importance was rated very high across all value and action statements.

The CSO Values to Action Framework represents an internationally informed structure that can be applied by trialists, ethics boards, funders, and advocacy groups when considering the design of, and participation in, RCTs. It offers a practical tool to align oncology research with what matters most to patients, promoting more ethical, inclusive, and relevant clinical trials.

Author contributions

Michelle Tregear, PhD (Conceptualization; Data curation; Formal analysis; Methodology; Supervision; Validation; Writing—original draft; Writing—review & editing), Haydeé Verduzco-Aguirre, MD, MHPE (Conceptualization; Formal analysis; Investigation; Methodology; Writing—original draft; Writing—review & editing), Brian Shkabari, BHSc (Data curation; Formal analysis; Project administration; Validation; Writing—review & editing), Rachel Koven, MSc (Conceptualization; Methodology; Validation; Writing—review & editing), Henry Scowcroft, MBioch, MSc (Formal analysis; Investigation; Validation; Visualization; Writing—review & editing), Viji Venkatash, BA (Conceptualization; Formal analysis; Investigation; Validation; Writing—review & editing), Michael Brundage, MD, MSc (Conceptualization; Formal analysis; Investigation; Methodology; Supervision; Validation; Visualization; Writing—review & editing), Runcie Chidebe, MSc (Conceptualization; Investigation; Methodology; Validation; Visualization; Writing—review & editing), Elizabeth Eisenhauer, MD (Conceptualization; Formal analysis; Methodology; Validation; Writing—review & editing), Bishal Gyawali, MD, PhD (Conceptualization; Investigation; Methodology; Supervision; Validation; Writing—review & editing), Bhawna Sirohi, MBBS, FRCP (Conceptualization; Formal analysis; Investigation; Methodology; Validation; Writing—review & editing), Christopher Booth, MD, FRCPC (Data curation; Formal analysis; Investigation; Methodology; Supervision; Validation; Writing—review & editing), and Christopher Jackson, FRACP (Conceptualization; Data curation; Investigation; Methodology; Project administration; Supervision; Validation; Writing—original draft; Writing—review & editing)

Supplementary material

Supplementary material is available at JNCI: Journal of the National Cancer Institute online.

Funding

This project had no external funding.

Conflicts of interest

B.G. is a JNCI associate editor and co-author on this paper and was not involved in the editorial review process or decision to publish the manuscript. All other authors have no conflicts to declare.

Data availability

The data underlying this article are available in the article and in its online supplementary material.