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Ellen M Janssen,1 Sydney M Dy,2 Alexa S Meara,3 Peter J Kneuertz,4 Carolyn J Presley,5 John FP Bridges6,7 1Center for Medical Technology Policy, Baltimore, MD, USA; 2Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA; 3Department of Internal Medicine Division Of Rheumatology, The Ohio State University, College of Medicine, Columbus, OH, USA; 4Thoracic Surgery Division, Department of Surgery, The Ohio State University Wexner Medical Center, Columbus, OH, USA; 5Division of Medical Oncology, Department of Internal Medicine, The Ohio State University College of Medicine, Columbus, OH, USA; 6Department of Biomedical Informatics, The Ohio State University College of Medicine, Columbus, OH, USA; 7Department of Surgery, The Ohio State University College of Medicine, Columbus, OH, USACorrespondence: Ellen M JanssenResearch Director, Center for Medical Technology Policy, 401 East Pratt Street, Suite 631, Baltimore, MD 21202, USATel +1 443-222-8775Email ellen.janssen@cmtpnet.orgObjective: Increased treatment options and longer survival for lung cancer have generated increased interest in patient preferences. Previous studies of patient preferences in lung cancer have not fully explored preference heterogeneity. We demonstrate a method to explore preference heterogeneity in the willingness of patients with lung cancer and caregivers to trade progression-free survival (PFS) with side effects.Patients and Methods: Patients and caregivers attending a national lung cancer meeting completed a discrete-choice experiment (DCE) designed through a collaboration with patients. Participants answered 13 choice tasks described across PFS, short-term side effects, and four long-term side effects. Side effects were coded as a one-level change in severity (none-mild, mild-moderate, or moderate-severe). A mixed logit model in willingness-to-pay space estimated preference heterogeneity in acceptable tradeoffs (time equivalents) between PFS and side effects. The study was reported following quality indicators from the United States Food and Drug Administration’s patient preference guidance.Results: A total of 87 patients and 24 caregivers participated in the DCE. Participants would trade 3.7 month PFS (95% CI (CI): 3.3– 4.1) for less severe functional long-term treatment side effects, 2.3 months for less severe physical long-term effects (CI: 1.9– 2.8) and cognitive long-term effects (CI: 1.8– 2.8), 0.9 months (CI: 0.4– 1.4) for less severe emotional long-term effects, and 1.8 months (CI: 1.4– 2.3) for less severe short-term side effects. Most participants (90%) would accept treatment with more severe functional long-term effects for 8.4 additional month PFS.Conclusion: Participants would trade PFS for changes in short-term side effects and long-term side effects, although preference heterogeneity existed. Lung cancer treatments that offer less PFS but also less severe side effects might be acceptable to some patients.Keywords: non-small cell lung cancer, patient preferences, discrete choice experiment, heterogeneity, long-term side effects

An expansion in the availability of clinical drug trials for genetic neurodevelopmental conditions is underway. Delineating patient priorities is key to the success of drug development and clinical trial design. There is a lack of evidence about parent decision-making in the context of clinical drug trials for genetic neurodevelopmental conditions. We assessed parents' priorities when making a decision whether to enroll their child with fragile X syndrome (FXS) in a clinical drug trial. An online survey included a best-worst scaling method for parents to prioritize motivating and discouraging factors for child enrollment. Parents were recruited through the National Fragile X Foundation and FRAXA. Sequential best-worst with conditional logit analysis was used to determine how parents prioritize motivating and discouraging factors about trial enrollment decisions. Respondents (N = 354) were largely biological mothers (83%) of an individual with FXS who ranged in age from under 5 to over 21 years. The highest motivating factor was a trial to test a drug targeting the underlying FXS mechanism (coeff = 3.28, p

Ilene L Hollin,1 Anne EF Dimmock,2 John FP Bridges,3 Sonye K Danoff,4 Rebecca Bascom21Department of Health Services Administration and Policy, Temple University College of Public Health, Philadelphia, PA, USA; 2Division of Pulmonary, Allergy and Critical Care Medicine, Department of Medicine, Penn State University College of Medicine, Hershey, PA, USA; 3Departments of Biomedical Informatics and Surgery, Ohio State University College of Medicine, Columbus, OH, USA; 4Division of Pulmonary Critical Care Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USAPurpose: 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.Keywords: stated preference methods, discrete choice experiment, patient-centered outcomes research, benefit-risk trade-off

Tetsuhiro Chiba,1 Atsushi Hiraoka,2 Shigeru Mikami,3 Masami Shinozaki,4 Yukio Osaki,5 Masamichi Obu,6 Takamasa Ohki,7 Naoyuki Mita,8 Dianne Ledesma,8 Nariaki Yoshihara,8 Kathleen Beusterien,9 Kaitlan Amos,9 John FP Bridges,10 Osamu Yokosuka111Department of Gastroenterology and Nephrology, Chiba University Graduate School of Medicine, Chiba, Japan; 2Gastroenterology Center, Ehime Prefectural Central Hospital, Matsuyama, Ehime Prefecture, Japan; 3Department of Internal Medicine, Kikkoman General Hospital, Noda-shi, Chiba Prefecture, Japan; 4Department of Gastroenterology, Numazu City Hospital, Shizuoka, Japan; 5Department of Gastroenterology and Hepatology, Osaka Red Cross Hospital, Osaka, Japan; 6Department of Gastroenterology, Kimitsu Chuo Hospital, Kisarazu City, Chiba Prefecture, Japan; 7Department of Gastroenterology, Mitsui Memorial Hospital, Tokyo, Japan; 8Market Access, Bayer Yakuhin, Ltd., Tokyo, Japan; 9ORS Health, Washington DC, USA; 10Department of Biomedical Informatics and Department of Surgery, The Ohio State University College of Medicine, Columbus, OH, USA; 11Japan Community Health care Organization Funabashi Central Hospital, Funabashi, Chiba Prefecture, JapanPurpose: 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

Jui-Hua Tsai, Ellen Janssen, John FP Bridges Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA Abstract: 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. Keywords: patients’ perspectives, decision making, drug approval, patient engagement, patient organization, patients’ preference

John FP Bridges,1 Allison H Oakes,1 Crystal A Reinhart,2 Ernest Voyard,3 Bernadette O’Donoghue3 1Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA; 2Center for Prevention Research and Development, University of Illinois at Urbana-Champaign, Champaign, IL, USA; 3The Leukemia & Lymphoma Society, Rye Brook, NY, USA 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. Keywords: acute myeloid leukemia, stated-preference, best–worst scaling, patient-focused drug development, community engagement

John FP Bridges,1,2 Norah L Crossnohere,2 Anne L Schuster,1 Judith A Miller,3 Carolyn Pastorini,3,† Rebecca A Aslakson2,4,5 1Department of Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, 2Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, 3Patient-Centered Outcomes Research Institute (PCORI) Project, Baltimore, MD, 4Department of Anesthesiology and Critical Care Medicine, The Johns Hopkins School of Medicine, Baltimore, MD, 5Armstrong Institute for Patient Safety and Quality, The Johns Hopkins School of Medicine, Baltimore, MD, USA †Carolyn Pastorini passed away on August 24, 2015 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. Keywords: patient preference, patient participation, community participation, research design&nbsp

John FP Bridges,1 Kathleen Beusterien,2 Stephan Heres,3 Pedro Such,4 Joaquín Sánchez-Covisa,5 Anna-Greta Nylander,4 Elcie Chan,6 Anne de Jong-Laird5 1Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA; 2Outcomes Research Strategies in Health, Washington DC, USA; 3Department of Psychiatry and Psychotherapy, Technische Universität München, Munich, Germany; 4Global Medical Affairs, H. Lundbeck A/S, Valby, Denmark; 5Medical Department, Otsuka Pharmaceutical Europe Ltd., Wexham, UK; 6HEOR/Market Access, Otsuka Pharmaceutical Europe Ltd., Wexham, UK 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. Keywords: recent-onset schizophrenia, preferences, treatment goals

Ellen M Janssen,1 Daniel R Longo,2 Joan K Bardsley,3 John FP Bridges1 1Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, 2Department of Family Medicine and Population Health, Virginia Commonwealth University, Richmond, VA, 3MedStar Health Research Institute and MedStar Nursing, Hyattsville, MD, USA 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. Keywords: preference heterogeneity, stated-preference methods, preference heterogeneity, willingness-to-pay, choice experiment, educational attainment

Timothy N Showalter,1 Mark V Mishra,2 John FP Bridges31Department of Radiation Oncology, University of Virginia School of Medicine, Charlottesville, VA, USA; 2Department of Radiation Oncology, University of Maryland School of Medicine, 3Department ofHealth Care Policy and Management, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USAPurpose: 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.Keywords: prostate cancer, treatment, decision making, preferences, patient

We report the results of a study analyzing the proportion of theoretical and empirical articles in two core health economics journals. The Journal of Health Economics published 30% theory during the period 1982-1986, but by 1997-2001 the proportion had risen to 40% theory. Health Economics published 38% theory during 1992-1996, but the proportion fell to 32% theory during 1997-2001. In both journals articles were more likely to be published by men (78%), and published women were 50% less likely to publish theory than were men. Articles were more likely to be published by United States authors (54%), but United States authors were less likely to publish theory than authors from other countries. Compared to other disciplines, health economics published a higher proportion of theory than sociology, chemistry, and physics but less than economics and political science.

Portfolio theory is central to the analysis of risk in many areas of economics but is seldom used appropriately in health economics. This contribution examines the use of portfolio theory in the context of cost-effectiveness analysis (CEA). A number of modifications are needed to apply portfolio analysis to the economic evaluation of health care interventions. First, the method of reporting the results of a CEA, and consequently some of the underlying assumptions, needs to be modified. Second, portfolio theory needs to be expressed in terms of effects on individuals aggregated to a population. Finally, one needs to allow for the possibility of synergy between the various health interventions. This paper derives a general formula for a portfolio of health care interventions that allows for synergies between interventions where the population effects are aggregated from individual effects. A number of special cases are also derived to highlight the nature of the formulation of the modified portfolio theory. We conclude that, while modified portfolio theory adds a theoretical foundation to health care evaluations, it may not be operational until estimates of the correlation between interventions are available, and the question of uncertainty is resolved in health care evaluation. Also, while a synergy may be present at the individual level, when aggregated over a large population it may not be significant given the standard assumption of constant returns to scale. © Springer-Verlag 2002.