Priorities to Promote Participant Engagement in the Participant Engagement and Cancer Genome Sequencing (PE-CGS) Network.

BACKGROUND: Engaging diverse populations in cancer genomics research is of critical importance and is a fundamental goal of the NCI Participant Engagement and Cancer Genome Sequencing (PE-CGS) Network. Established as part of the Cancer Moonshot, PE-CGS is a consortium of stakeholders including clinicians, scientists, genetic counselors, and representatives of potential study participants and their communities. Participant engagement is an ongoing, bidirectional, and mutually beneficial interaction between study participants and researchers. PE-CGS sought to set priorities in participant engagement for conducting the network's research. METHODS: PE-CGS deliberatively engaged its stakeholders in the following four-phase process to set the network's research priorities in participant engagement: (i) a brainstorming exercise to elicit potential priorities; (ii) a 2-day virtual meeting to discuss priorities; (iii) recommendations from the PE-CGS External Advisory Panel to refine priorities; and (iv) a virtual meeting to set priorities. RESULTS: Nearly 150 PE-CGS stakeholders engaged in the process. Five priorities were set: (i) tailor education and communication materials for participants throughout the research process; (ii) identify measures of participant engagement; (iii) identify optimal participant engagement strategies; (iv) understand cancer disparities in the context of cancer genomics research; and (v) personalize the return of genomics findings to participants. CONCLUSIONS: PE-CGS is pursuing these priorities to meaningfully engage diverse and underrepresented patients with cancer and posttreatment cancer survivors as participants in cancer genomics research and, subsequently, generate new discoveries. IMPACT: Data from PE-CGS will be shared with the broader scientific community in a manner consistent with participant informed consent and community agreement.

Neoadjuvant therapy for resectable pancreatic ductal adenocarcinoma: The need for patient-centered research.

Pancreatic ductal adenocarcinoma is an aggressive cancer with high recurrence rates following surgical resection. While adjuvant chemotherapy improves survival, a significant proportion of patients are unable to initiate or complete all intended therapy following pancreatectomy due to postoperative complications or poor performance status. The administration of chemotherapy prior to surgical resection is an alternative strategy that ensures its early and near universal delivery as well as improves margin-negative resection rates and potentially improves long-term survival outcomes. Neoadjuvant therapy is increasingly being recommended to patients with pancreatic ductal adenocarcinoma, however, patient-centered research on its use is lacking. In this review, we highlight opportunities to focus research efforts in the domains of patient preferences, patient-reported outcomes, patient experience, and survivorship. Novel research in these areas may identify relevant barriers and facilitators to the use of neoadjuvant therapy thereby increasing its utilization, improve shared-decision making for patients and providers, and optimize the experience of those undergoing neoadjuvant therapy.

Patient preferences for attributes of tyrosine kinase inhibitor treatments for EGFR mutation-positive non-small-cell lung cancer.

Aim: EGFR-tyrosine kinase inhibitors (TKIs) vary in efficacy, side effects (SEs) and dosing regimen. We explored EGFR-TKI treatment attribute preferences in EGFR mutation-positive metastatic non-small-cell lung cancer. Materials & methods: Patients completed a survey utilizing preference elicitation methods: direct elicitation of four EGFR-TKI profiles describing progression-free survival (PFS), severe SE risk, administration; discrete choice experiment involving 12 choice tasks. Results: 90 participated. The preferred profile (selected 89% of times) had the longest PFS (18 months) and the lowest severe SE risk (5%). Patients would need compensation with ≥three-times longer PFS for severe SEs. Patients would accept ≤7 months PFS reduction for oral treatments versus intravenous. Conclusion: Patients preferred longer PFS but were willing to accept reduced PFS for more favorable SEs and dosing convenience.

Japanese patient preferences regarding intermediate to advanced hepatocellular carcinoma treatments.

Purpose: This study aimed to evaluate Japanese patient preferences regarding features of intermediate or advanced (Progressed) hepatocellular carcinoma (HCC) treatments: transarterial chemoembolization (TACE), hepatic arterial infusion chemotherapy (HAIC), and oral anti-cancer therapy. Methods: Patients with HCC, recruited from clinical sites and a patient panel in Japan, completed a cross-sectional web-based survey. Preferences were quantified using best-worst scaling, where patients identified the best and worst among 13 treatment features. Direct elicitation was used to identify preference for TACE, HAIC, or oral therapy, including the likelihood of trying each. Additional items asked for the willingness to try an oral medication that delays progression by six months but has an 8% or 21% risk of severe hand-foot skin reaction (HFSR). Results: The sample (N=119; 29 early stage; 90 Progressed) most preferred "oral medication", "artery branches plugged", and "prevents formation of new blood vessels", and least preferred "risk of liver damage" and "risk of catheter-related complications". Overall, 51%, 40%, and 8% preferred oral therapy, TACE, and HAIC, respectively (p<0.05), and the mean likelihood of trying each were 59%, 52%, and 35%, respectively (p<0.001). Patients with sorafenib or TACE experience most preferred what they had received; however, both groups were equally willing to try the other treatment. Patients preferring oral therapy favored "oral medication" over "artery branches plugged", "surgery is repeated as required when the cancer grows again", and "risk of liver damage", compared to those preferring TACE (p<0.05). Sixty-eight percent would probably try therapy with an 8% risk of severe HFSR, compared to 50% with a 21% risk. Conclusion: Treatment type, mode of action, and risks may drive HCC patient preferences. Such features likely should be incorporated into physician-patient interactions regarding treatment decision-making.

Developing and piloting an instrument to prioritize the worries of patients with acute myeloid leukemia.

BACKGROUND: Acute myeloid leukemia (AML) is a rapidly progressing blood cancer for which new treatments are needed. We sought to promote patient-focused drug development (PFDD) for AML by developing and piloting an instrument to prioritize the worries of patients with AML. PATIENTS AND METHODS: An innovative community-centered approach was used to engage expert and community stakeholders in the development, pretesting, pilot testing, and dissemination of a novel best-worst scaling instrument. Patient worries were identified through individual interviews (n=15) and group calls. The instrument was developed through rigorous pretesting (n=13) and then piloted among patients and caregivers engaged in this study (n=25). Priorities were assessed using best-worst scores (spanning from +1 to -1) representing the relative number of times that items were endorsed as the most and the least worrying. All findings were presented at a PFDD meeting at the US Food and Drug Administration (FDA) that was attended by >80 stakeholders. RESULTS: The final instrument included 13 worries spanning issues such as decision making, treatment delivery, physical impacts, and psychosocial effects. Patients and caregivers most prioritized worries about dying from their disease (best minus worst [BW] score=0.73), long-term side effects (BW=0.28), and time in hospital (BW=0.25). CONCLUSION: Community-centered approaches are valuable in designing and executing PFDD meetings and associated quantitative surveys to document the experience of patients. Expert and community stakeholders welcomed the opportunity to share their experiences with the FDA and strongly endorsed implementing this survey nationally.

A patient and community-centered approach selecting endpoints for a randomized trial of a novel advance care planning tool.

BACKGROUND: Despite a movement toward patient-centered outcomes, best practices on how to gather and refine patients' perspectives on research endpoints are limited. Advanced care planning (ACP) is inherently patient centered and would benefit from patient prioritization of endpoints for ACP-related tools and studies. OBJECTIVE: This investigation sought to prioritize patient-centered endpoints for the content and evaluation of an ACP video being developed for patients undergoing major surgery. We also sought to highlight an approach using complementary engagement and research strategies to document priorities and preferences of patients and other stakeholders. MATERIALS AND METHODS: Endpoints identified from a previously published environmental scan were operationalized following rating by a caregiver co-investigator, refinement by a patient co-investigator, review by a stakeholder committee, and validation by patients and family members. Finalized endpoints were taken to a state fair where members of the public who indicated that they or a loved one had undergone major surgery prioritized their most relevant endpoints and provided comments. RESULTS: Of the initial 50 ACP endpoints identified from the review, 12 endpoints were selected for public prioritization. At the state fair, 359 individuals prioritized the endpoints, the highest ranking of which was having a meaningful conversation with a physician before surgery (57%). CONCLUSION: Using a novel combination of engagement and research techniques provided the opportunity to understand which endpoints were most important to patients and families and fostered framework development clarifying the differential contributions of engagement and research. Results from this study ultimately changed the content and evaluation of the ACP video.

Live, Learn, Pass It on: A Patient Advocacy Engagement Project on the Lived Experience of Lung Cancer Survivors.

INTRODUCTION: The objective of this project was to engage survivor-advocates by describing their experiences living with lung cancer in an era of increasing treatment options. METHODS: This was a qualitative engagement project with participants from a lung cancer advocacy organization's survivor advisory board. Interviews were conducted, transcribed, and analyzed for stages and associated experiences using interpretive phenomenological analysis and elements of narrative analysis, in partnership with the patient advocacy organization. RESULTS: Of 27 engaged members, interviews were conducted with 19, mostly long-term survivors with stage 3 or 4 lung cancer. Within the quest for patient-centeredness, we identified 3 stages of the patient experience. The stage Live describes the journey of the lived experience; Learn describes the quest for knowledge, empowerment, and skills; and Pass it on describes making a difference through guiding others, building awareness, and community. CONCLUSIONS: Lung cancer survivor-advocates have an intertwined experience of their personal journey, the quest for knowledge, and developing advocacy. Future patient engagement can incorporate these findings into increasing the survivor-centeredness of partnerships and research, particularly for quality of life and shared decision-making.

Factors that influence patient preferences for prostate cancer management options: A systematic review.

PURPOSE: We performed a systematic review to evaluate evidence regarding factors that influence patient preferences for management options for localized prostate cancer. METHODS: We followed a prespecified search protocol (PROSPERO identifier CRD42014009173) to identify studies that evaluated patient preferences for prostate cancer management options for localized prostate cancer. We queried PubMed, the Cochrane Database of Systematic Reviews, Embase, Cumulative Index to Nursing & Allied Health Literature (CINAHL) Plus, and Econ-Lit databases. Two separate reviewers completed the article selection process and review, including coding of study characteristics. Study quality was scored according to the PREFS checklist, which consists of five criteria: Purpose, Respondents, Explanation, Findings, and Significance. Reviewers summarized the primary findings of each article included in the analysis. RESULTS: Of the 606 citations identified in the literature search, there were a total of 21 articles that met all selection criteria, reporting results for a total of 4,131 subjects. Themes identified in the studies included: the importance of patient perceptions of treatment efficacy and side effects; the influence of physician recommendations on patient decision-making; and the prioritization of concerns regarding treatment side effects among those men who prefer radiation therapy or active surveillance. The articles had an average PREFS score of 3.4 (standard deviation [SD] 1.0), which is similar to a recent study for breast cancer treatment preferences. CONCLUSION: This systematic review of factors that influence patient preferences for prostate cancer management options identified a small, but high quality, group of articles that satisfied the selection criteria. The available evidence suggests that interventions aimed at informing patients regarding the comparative effectiveness of prostate cancer management alternatives should include the influence of physician recommendations and family members' desires on patient decision-making.

Creating an advance-care-planning decision aid for high-risk surgery: a qualitative study.

BACKGROUND: High-risk surgery patients may lose decision-making capacity as a result of surgical complications. Advance care planning prior to surgery may be beneficial, but remains controversial and is hindered by a lack of appropriate decision aids. This study sought to examine stakeholders' views on the appropriateness of using decision aids, in general, to support advance care planning among high-risk surgery populations and the design of such a decision aid. METHODS: Key informants were recruited through purposive and snowball sampling. Semi-structured interviews were conducted by phone until data collected reached theoretical saturation. Key informants were asked to discuss their thoughts about advance care planning and interventions to support advance care planning, particularly for this population. Researchers took de-identified notes that were analyzed for emerging concordant, discordant, and recurrent themes using interpretative phenomenological analysis. RESULTS: Key informants described the importance of initiating advance care planning preoperatively, despite potential challenges present in surgical settings. In general, decision aids were viewed as an appropriate approach to support advance care planning for this population. A recipe emerged from the data that outlines tools, ingredients, and tips for success that are needed to design an advance care planning decision aid for high-risk surgical settings. CONCLUSIONS: Stakeholders supported incorporating advance care planning in high-risk surgical settings and endorsed the appropriateness of using decision aids to do so. Findings will inform the next stages of developing the first advance care planning decision aid for high-risk surgery patients.

Controlling liver cancer internationally: A qualitative study of clinicians' perceptions of current public policy needs.

BACKGROUND: Liver cancer is the fifth most common cancer in men and the seventh for women. Usually because of late diagnosis, the prognosis for liver cancer remains poor, resulting in liver cancer being the third most common cause of death from cancer. While some countries have treatment guidelines, little is known or understood about the strategies needed for liver cancer control internationally. OBJECTIVE: To explore leading liver cancer clinician's perceptions of the current public policy needs to control liver cancer internationally. METHODS: Key informant interviews were conducted with a range of liver cancer clinicians involved in policy in eleven countries. Interviews were digitally recorded, transcribed verbatim, translated (where necessary), de-identified and analyzed by two researchers using a constant comparative method. RESULTS: Twenty in-depth semi-structured interviews were conducted in: Australia, China, France, Germany, Italy, Japan, Spain, South Korea, Taiwan, Turkey and the United States. Nine themes were identified and cluster into three groups: 1) Promoting prevention via early risk assessment, focusing on viral hepatitis and other lifestyle factors; 2) Increasing political, public and medical community awareness; and 3) Improving funding for screening, liver cancer surveillance and treatment. CONCLUSION: This study is an important step towards developing an evidence-based approach to assessing preparedness for implementing comprehensive liver cancer control strategies. Evaluation mechanisms to assess countries' performance on the needs described are needed. Future research will concentrate of understanding how these needs vary across countries and the optimal strategies to improve the diagnosis and prognosis of patients with liver cancer internationally.

Can pharmacoeconomics and outcomes research contribute to the empowerment of women affected by breast cancer?

Breakthroughs in genetic testing have informed patients and physicians in the treatment of breast cancer; however, they have also added to the complexity of decision-making. Genetic testing for breast cancer susceptibility not only changes treatment and screening options, but also challenges the way in which interventions are evaluated. While comparative effectiveness and cost-effective analysis methods are now standard for evaluation at the societal level, technologies such as genetic testing require us to consider the role of patient preference, especially as we move towards more personalized approaches to medicine. In this review, we discuss the changing role of pharmacoeconomics and outcomes research by highlighting how the discipline could use patient preference methods, such as conjoint analysis, to promote shared decision-making and to empower breast cancer patients. By adopting these methods we could move our focus from what is best for payers or society to one that applies scientific methods to identify what is best for patients.