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.

The evolution of patient-focused drug development and Duchenne muscular dystrophy.

Introduction: There is a groundswell of interest from patient, industry, and regulatory groups to rigorously and transparently integrate patient-voice into regulatory decision-making. Patient-focused drug development (PFDD) is an approach established by the US Food and Drug Administration to systematically incorporate patient experiences into drug development and evaluation. It has created a demand for scientific advancement to measure and integrate patient-voice into decision-making.Areas covered: This narrative review describes the evolving nature of advocacy-regulatory relations preceding PFDD, characterizes current PFDD and other patient-engagement activities, and explores future opportunities for patient participation along the drug development pipeline. We present Duchenne muscular dystrophy as a case study to illustrate how PFDD is being operationalized by patient groups and regulators using both verbal and written data sources.Expert opinion: PFDD represents the most widespread approach yet to integrate the patient voice as a source of evidence to inform regulatory decision-making. Regulatory approvals are just one frontier in drug development. On the horizon remain uncertainties in how patient experience can inform post-marketing surveillance, pricing, reimbursement, and health technology assessment. Patient-input may be particularly crucial to demonstrate the value of expensive first-generation rare disease treatments that confer meaningful benefits but do not meet traditional thresholds for cost-effectiveness.

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.

Collecting patient preference information using a Clinical Data Research Network: demonstrating feasibility with idiopathic pulmonary fibrosis.

Purpose: Rare diseases present challenges for accessing patient populations to conduct surveys. Clinical Data Research Networks (CDRNs) offer an opportunity to overcome those challenges by providing infrastructure for accessing patients and sharing data. This study aims to demonstrate the feasibility of collecting patient preference information for a rare disease in a CDRN, using idiopathic pulmonary fibrosis as proof of concept. Patients and methods: Utilizing a cohort of idiopathic pulmonary fibrosis (IPF) patients across a CDRN, a discrete choice experiment was administered via electronic and paper methods to collect patient preference information about benefits and risks of two therapeutic options. Survey data were augmented with data from electronic health records and patient-reported outcome surveys. Results: Thirty-three patients completed the preference experiment. The amount of choice attributable to a benefit of slowing of decline in lung function was 36%. Improving efficacy in terms of lung function was 2.16 times as important as improving efficacy in terms of shortness of breath. In terms of side effects, decreasing risk of gastrointestinal problems was 2.6 times as important as decreasing risk of sun sensitivity and 2.4 times as important as decreasing risk of liver injury. In terms of benefit-risk trade-offs, improving efficacy in terms of lung function was 1.6 times as important as decreasing risk of gastrointestinal problems. Conclusion: This study used IPF as a proof of concept to demonstrate the feasibility of collecting patient preference information in a CDRN. The network was advantageous to the study of patient preferences. Future research should continue to explore pathways for the collection and use of patient preference information across networks. The power of consolidated collection efforts may lead to the ability to use preference data to inform decision-making at the regional, specialty, or individual encounter level.

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.

Research as an event: a novel approach to promote patient-focused drug development.

Patient groups are increasingly engaging in research to understand patients' preferences and incorporate their perspectives into drug development and regulation. Several models of patient engagement have emerged, but there is little guidance on how to partner with patient groups to engage the disease community. Our group has been using an approach to engage patient groups that we call research as an event. Research as an event is a method for researchers to use a community-centered event to engage patients in their own environment at modest incremental cost. It is a pragmatic solution to address the challenges of engaging patients in research to minimize patients' frustration, decrease the time burden, and limit the overall cost. The community, the event, and the research are the three components that constitute the research as an event framework. The community represents a disease-specific community. The event is a meeting of common interest for patients and other stakeholders, such as a patient advocacy conference. The research describes activities in engaging the community for the purpose of research. Research as an event follows a six-step approach. A case study is used to demonstrate the six steps followed by recommendations for future implementation.

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.

Identifying and prioritizing concerns associated with prosthetic devices for use in a benefit-risk assessment: a mixed-methods approach.

OBJECTIVE: We identified and prioritized concerns reported by stakeholders associated with novel upper-limb prostheses. METHODS: An evidence review and key-informant engagement, identified 62 concerns with upper-limb prostheses with implantable components. We selected 16 concerns for inclusion in a best-worst scaling (BWS) prioritization survey. Focus groups and BWS were used to engage stakeholders at a public meeting on prostheses. In 16 BWS choice tasks, attendees selected the most and least influential concern when choosing an upper-limb prosthesis. Aggregate data were analyzed using choice frequencies and conditional logit analysis. Latent class analysis examined heterogeneity in priorities. Estimates were adjusted to importance ratios which indicate how influential each concern is in the decision making process. RESULTS: Forty-seven (47) stakeholders from diverse backgrounds completed the BWS survey (response rate 51%). On aggregate, the most influential concern was reliability of the device (importance ratio: 13%), and least influential was the concern of an outdated device (importance ratio: 1%). Latent class analysis identified two classes with approximately 50% of participants each. The first class was most influenced by effectiveness of the device. The second class was most influenced by minimizing risks. CONCLUSION: In this pilot, we identified heterogeneity in how participants prioritize concerns for upper-limb prostheses.

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.

Quantifying the treatment goals of people recently diagnosed with schizophrenia using best-worst scaling.

OBJECTIVE: This study seeks to quantify the treatment goals of people recently diagnosed with schizophrenia and explore their impact on treatment plan. METHODS: People aged 18-35 years with a confirmed diagnosis of schizophrenia within the past 5 years were surveyed in the UK, Germany, and Italy. Treatment goals were assessed via a validated best-worst scaling instrument, where participants evaluated subsets of 13 possible treatment goals identified using a balanced incomplete block design. Participants identified the most and least important goals within each task. Data were also collected on current treatment and preference for daily oral versus long-acting injectable (LAI) treatment. Hierarchical Bayes was used to identify preference weights for the goals, and latent class analysis was used to identify segments of people with similar goals. The segments were compared with the current treatment and preference for oral versus LAI treatment. RESULTS: Across 100 participants, the average age was 26 years, 75% were male and 50% were diagnosed within 2 years ago. Overall, preferences were most favorable for reduced disease symptoms, think clearly, reduced hospitalizations, reduced anxiety, and take care of self. A total of 61% preferred oral medication and 39% LAI. Two groups were identified with different treatment goals; 50% of participants emphasized clinical goals, including reduced disease symptoms (preference weight =19.7%), reduced hospitalizations (15.5%), and reduced anxiety (10.5%). The other 50% emphasized functional goals, including improved relationships with family/friends (11.4%), increased interest in work (10.6%), experiencing a fuller range of emotions (8.4%), and ability to socialize (7.5%). Those emphasizing functional goals were more likely to be on LAI (44% versus 26%; p=0.059) and preferred LAI (46% versus 32%; p=0.151). CONCLUSIONS: People with recent-onset schizophrenia may focus more on clinical goals or functional goals, a discussion of which may help facilitate patient engagement.

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.

Education and patient preferences for treating type 2 diabetes: a stratified discrete-choice experiment.

PURPOSE: Diabetes is a chronic condition that is more prevalent among people with lower educational attainment. This study assessed the treatment preferences of patients with type 2 diabetes by educational attainment. METHODS: Patients with type 2 diabetes were recruited from a national online panel in the US. Treatment preferences were assessed using a discrete-choice experiment. Participants completed 16 choice tasks in which they compared pairs of treatment profiles composed of six attributes: A1c decrease, stable blood glucose, low blood glucose, nausea, treatment burden, and out-of-pocket cost. Choice models and willingness-to-pay (WTP) estimates were estimated using a conditional logit model and were stratified by educational status. RESULTS: A total of 231 participants with a high school diploma or less education, 156 participants with some college education, and 165 participants with a college degree or more completed the survey. Participants with a college degree or more education were willing to pay more for A1c decreases ($58.84, standard error [SE]: 10.6) than participants who had completed some college ($28.47, SE: 5.53) or high school or less ($17.56, SE: 3.55) (p≤0.01). People with a college education were willing to pay more than people with high school or less to avoid nausea, low blood glucose events during the day/night, or two pills per day. CONCLUSION: WTP for aspects of diabetes medication differed for people with a college education or more and a high school education or less. Advanced statistical methods might overcome limitations of stratification and advance understanding of preference heterogeneity for use in patient-centered benefit-risk assessments and personalized care approaches.

Applying stated-preference methods to improve health systems in sub-Saharan Africa: a systematic review.

INTRODUCTION: Sub-Saharan African health systems must balance shifting disease burdens with desires for robust institutions. Stated-preference methods have been applied extensively to elicit health care workers' preferences and priorities for rural practice. This systematic review characterizes the range of their applications to African health systems problems. Areas covered: A PRISMA protocol was submitted to PROSPERO. Six databases were queried for peer-reviewed articles using quantitative stated-preference methods to evaluate a health systems-related trade-off. Quality was assessed using the PREFS checklist. Seventy-seven articles published between 1996 and 2017 met review criteria. Methods were primarily choice-based: discrete-choice experiments (n = 46), ranking/allocation techniques (n = 21), conjoint analyses (n = 7), and best-worst scaling (n = 3). Trade-offs fell into six 'building blocks': service features (n = 27), workforce incentives (n = 17), product features (n = 14), system priorities (n = 14), insurance features (n = 4), and research priorities (n = 1). Five countries dominated: South Africa (n = 11), Ghana (n = 9), Malawi (n = 9), Uganda (n = 9), and Tanzania (n = 8). Discrete-choice experiments were of highest quality (mean score: 3.36/5). Expert commentary: Stated-preference methods have been applied to many health systems contexts throughout sub-Saharan Africa. Studies examined established strategic areas, especially primary health care for women, prevention and treatment of infectious diseases, and workforce development. Studies have neglected the emerging areas of non-communicable diseases.

Using clinical data to predict high-cost performance coding issues associated with pressure ulcers: a multilevel cohort model.

OBJECTIVE: Hospital-acquired pressure ulcers (HAPUs) have a mortality rate of 11.6%, are costly to treat, and result in Medicare reimbursement penalties. Medicare codes HAPUs according to Agency for Healthcare Research and Quality Patient-Safety Indicator 3 (PSI-03), but they are sometimes inappropriately coded. The objective is to use electronic health records to predict pressure ulcers and to identify coding issues leading to penalties. MATERIALS AND METHODS: We evaluated all hospitalized patient electronic medical records at an academic medical center data repository between 2011 and 2014. These data contained patient encounter level demographic variables, diagnoses, prescription drugs, and provider orders. HAPUs were defined by PSI-03: stages III, IV, or unstageable pressure ulcers not present on admission as a secondary diagnosis, excluding cases of paralysis. Random forests reduced data dimensionality. Multilevel logistic regression of patient encounters evaluated associations between covariates and HAPU incidence. RESULTS: The approach produced a sample population of 21 153 patients with 1549 PSI-03 cases. The greatest odds ratio (OR) of HAPU incidence was among patients diagnosed with spinal cord injury (ICD-9 907.2: OR = 14.3; P < .001), and 71% of spinal cord injuries were not properly coded for paralysis, leading to a PSI-03 flag. Other high ORs included bed confinement (ICD-9 V49.84: OR = 3.1, P < .001) and provider-ordered pre-albumin lab (OR = 2.5, P < .001). DISCUSSION: This analysis identifies spinal cord injuries as high risk for HAPUs and as being often inappropriately coded without paralysis, leading to PSI-03 flags. The resulting statistical model can be tested to predict HAPUs during hospitalization. CONCLUSION: Inappropriate coding of conditions leads to poor hospital performance measures and Medicare reimbursement penalties.

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.

The changing role of economic evaluation in valuing medical technologies.

Economic evaluation is established within health-technology assessment but is challenged by those wanting to use economic evaluation to inform pricing and/or incorporate nontraditional sources of value and the views of diverse stakeholders. The changing role of economic evaluation in (formally or informally) assessing prices/values in four jurisdictions (UK, Australia, Germany and USA) is detailed and the authors propose a taxonomy of factors impacting the value of medical technology spanning clinical utility (effectiveness, safety/tolerability and quality of evidence), consumer demand (consumer preferences, process utility and unmet need), economic incentives (innovation, option value and market competition) and the societal perspective (social justice, social values and national interest). The authors suggest that multicriteria decision analysis methods grounded in hedonic-pricing theory can facilitate the valuing/pricing of medical technologies. The use of such an approach is hindered by a paucity of relevant educational opportunities, vested interests and aversion to placing prices/values on health.

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.