International Society For Pharmacoeconomics And Outcomes Research Research Articles

Pharmacoeconomic analysis of type 2 diabetes mellitus and its microvascular complications

The international society for pharmacoeconomics and outcomes research (ISPOR) defines “Pharmacoeconomics as the field of study that evaluates the behaviour of individuals, firms and markets relevant to the use of pharmaceutical products, services and programs and which frequently focuses on the costs (inputs) and consequences (outcomes) of that use”. It is the description and analysis of the cost of drug therapy to health care systems and society. This prospective observation study was carried out for over a period of 6 months commencing from March 2014 to August 2014 among inpatients of General medicine and Nephrology departments of a tertiary care referral hospital in kerala.All diabetes mellitus patient treated in the Inpatient department of General medicine and Nephrology department during March-August 2014 were monitored, collect relevant data and entered into the data sheet. Based on the inclusion and exclusion criteria of the protocol approved by the IEC, patients belonging to the age group 40-70 of both sex were selected and enrolled for the study.

HIV partner services in Kenya: a cost and budget impact analysis study

BackgroundThe elicitation of contact information, notification and testing of sex partners of HIV infected patients (aPS), is an effective HIV testing strategy in low-income settings but may not necessarily be affordable. We applied WHO guidelines and the International Society for Pharmaco-economics and Outcomes Research (ISPOR) guidelines to conduct cost and budget impact analyses, respectively, of aPS compared to current practice of HIV testing services (HTS) in Kisumu County, Kenya.MethodsUsing study data and time motion studies, we constructed an Excel-based tool to estimate costs and the budget impact of aPS. Cost data were collected from selected facilities in Kisumu County. We report the annual total and unit costs of HTS, incremental total and unit costs for aPS, and the budget impact of scaling up aPS over a 5-year horizon. We also considered a task-shifted scenario that used community health workers (CHWs) rather than facility based health workers and conducted sensitivity analyses assuming different rates of scale up of aPS.ResultsThe average unit costs for HIV testing among HIV-infected index clients was US$ 25.36 per client and US$ 17.86 per client using nurses and CHWs, respectively. The average incremental costs for providing enhanced aPS in Kisumu County were US$ 1,092,161 and US$ 753,547 per year, using nurses and CHWs, respectively. The average incremental cost of scaling up aPS over a five period was 45% higher when using nurses compared to using CHWs (US$ 5,460,837 and US$ 3,767,738 respectively). Over the five years, the upper-bound budget impact of nurse-model was US$ 1,767,863, 63% and 35% of which were accounted for by aPS costs and ART costs, respectively. The CHW model incurred an upper-bound incremental cost of US$ 1,258,854, which was 71.2% lower than the nurse-based model. The budget impact was sensitive to the level of aPS coverage and ranged from US$ 28,547 for 30% coverage using CHWs in 2014 to US$ 1,267,603 for 80% coverage using nurses in 2018.ConclusionScaling aPS using nurses has minimal budget impact but not cost-saving over a five-year period. Targeting aPS to newly-diagnosed index cases and task-shifting to community health workers is recommended.

Danish translation and linguistic validation of the BODY-Q Chest Module

The aim of this study was to translate and linguistically validate the patient-reported outcome (PRO) instrument body-q chest module, designed to measure outcomes following chest contouring surgery. The BODY-Q Chest Module includes two scales that measure appearance of chest and nipples. The translation and validation were performed according to the guidelines from the world health organization (who) and the international society for pharmacoeconomics and outcomes research (ISPOR). This approach involved two independent forward translations, a backwards translation, an expert panel meeting and cognitive debriefing interviews with patients. Each step was undertaken with the aim of achieving a conceptual and culturally equal instrument. This process led to a linguistically validated and conceptually equivalent danish version of the body-q chest module. The forward translation resulted in several discrepant translations of items that were harmonized to form the backward translation. This translation included three items with conceptual differences that required further revision. The revised version presented at the expert panel meeting had six items that needed to be revised due to conceptual discrepancies. The cognitive debriefing interviews led to revision of one item. The practices from the who and ispor guidelines were essential to developing a translation that preserved the meaning of the content of the body-q chest module from the original development study. The translation and linguistic validation methods used in our study could be used for further translations and validation of pro instruments. These new scales have since been field-tested as part of an international psychometric study.

Cross-Cultural Adaptation, Validity, and Reproducibility of the Mediterranean Islands Study Food Frequency Questionnaire in the Elderly Population Living in the Spanish Mediterranean

The objective of this study was to perform cross-cultural adaptation of the Mediterranean Islands Study Food Frequency Questionnaire (MEDIS-FFQ) and to evaluate its reproducibility and validity in a population over 60 years of age in the Spanish Mediterranean. Three hundred forty-one people completed the food frequency questionnaire (FFQ), which was administered twice (FFQ1 and FFQ2) with nine 24-h dietary recalls (24-HDRs) over a nine-month period to assess its reproducibility and validity. Cross-cultural translation and adaptation were performed according to the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) guidelines and included direct translation, back-translation, and a pilot comprehension test. Reproducibility was evaluated with Pearson’s and interclass correlation coefficients. Validity was estimated using correlations between the FFQ food groups and the 24-HDR mean. The levels of agreement and misclassification were expressed as the proportions of individuals classified by comparing the estimated information from the FFQ2 and the 24-HDR. Reproducibility correlation coefficients ranged from r = 0.44 to r = 0.90. Validity indices ranged from 0.71 to 0.99. More than 80% of the subjects were classified in the same quartile on both instruments. The kappa statistic showed a moderate to high level of agreement (0.70–0.95) between the two instruments. In conclusion, the MEDIS-FFQ showed good reproducibility and validity in estimating the nutrient intake of the elderly population in the Spanish Mediterranean.

Linguistic and content validity of the norwegian patient-generated subjective global assessment

Rationale: The Patient-Generated Subjective Global Assessment (PG-SGA) is an instrument to screen and assess malnutrition and risk factors, and to triage for interventions. The Boxes (Weight, Food intake, Symptoms and Activities/function), i.e. the Short Form (SF), were designed to be completed by patients and the Worksheets by healthcare professionals (HCP). We translated and culturally adapted the original PG-SGA for the Norwegian setting, in concordance with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Principles. We aimed to test linguistic validity, i.e. comprehensibility and difficulty, and content validity (relevance) as perceived by cancer patients and HCP.

Assessing comfort in the epilepsy monitoring unit: Development of an instrument

Background/purposeSome patients perceive the experience of hospitalization in an epilepsy monitoring unit (EMU) to be an uncomfortable one. In order to provide comfort-enhancing measures, nurses need to assess comfort levels. For this purpose, Kolcaba developed the General Comfort Questionnaire (GCQ), which may be modified for usage in specific settings. The objective of this study was to develop an instrument to assess comfort of adult patients during hospitalization in an EMU, namely the Epilepsy Monitoring Unit Comfort Questionnaire (EMUCQ). MethodsThe GCQ was translated from American English into German following the guidelines of the International Society for Pharmacoeconomics and Outcome Research (ISPOR). Three native German speakers with good command of the English language did forward translations. An expert who grew up bilingual did a backtranslation. For use in an EMU, literature-based setting-specific items were added. In a qualitative-descriptive study, cognitive debriefing with 25 patients was conducted using cognitive interviews. Qualitative data analysis was based on the framework method. In a quantitative-descriptive study, nine clinical experts assessed content validity. ResultsFor setting-specific modification, 12 items pertaining to surroundings, feeling observed, and feeling afraid of a seizure were added to the translated GCQ. Based on the initial content validity rating, 26 items remained unchanged, 12 items underwent revisions, and 14 items were omitted. Eight items were put aside for a follow-up rating in the context of cognitive debriefing. Cognitive interviewing revealed problems regarding the interpretation of items and missing items. According to the results, 27 items remained unchanged, 11 items were reworded, and six items were added. The final content validity rating showed item-content validity indices (I-CVI) between .33 and 1, and an average CVI on a scale level (S-CVI/ave) of .84. ConclusionsEnhancing comfort is a fundamental nursing goal in demanding situations. Therefore, the contribution of nurses to the quality of individualized patient care is a substantial one. The EMUCQ is a valuable tool to support the assessment of comfort levels. The ISPOR guidelines proved to be useful to ensure high quality of the translated instrument. Using cognitive interviews enhanced the understandability of items and supported modification of the GCQ. At present, S-CVI/ave value of the EMUCQ is acceptable. Further testing is necessary.

Translation and cross-cultural adaptation of eight pediatric PROMIS® item banks into Spanish and German.

The Patient-Reported Outcome Measurement Information System (PROMIS®) is a National Institutes of Health (NIH)-funded initiative to develop reliable, valid, and normed item banks to measure health. We describe the first large-scale translation and cross-cultural adaptation effort to German and Spanish of eight pediatric PROMIS item banks: Physical activity (PAC), subjective well-being (SWB), experiences of stress (EOS), and family relations (FAM). We utilized methods outlined in the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) PRO Translation Task Force recommendations. Ten professional translators performed a translatability assessment and generated forward translations. Forward Translations were compared within a country and cross-culturally to identify problems and to produce a consensus-derived version, which was then back translated, evaluated, and revised where necessary. Reconciled versions were evaluated in cognitive interviews with 126 children before finalization. Eight resulting pediatric PROMIS® item banks were translated: Two PAC banks (22 total items), three SWB banks (125 total items), two EOS banks (45 total items), and one FAM bank (47 total items). Up to 92% of all items raised no or only minor translation difficulties, 0-5.6% were difficult to translate. Up to 20% item revisions were necessary to ensure conceptual equivalence and comprehensibility. Cognitive interviews indicated that 91-94% of the final items were appropriate for children (8-17years). German and Spanish translations of eight PROMIS Pediatric item banks were created for clinical trials and routine pediatric health care. Initial translatability assessment and rigorous translation methodology helped to ensure conceptual equivalence and comprehensibility. Next steps include cross-cultural validation and adaptation studies.

PHP193 - Findings from the 2018 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Student Interest Survey

PHP193 - Findings from the 2018 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Student Interest Survey

PRM124 - How is Real World Data Currently Used by Industry Professionals?

Randomized controlled trials may not provide enough information to resolve payer uncertainty around the benefit of an intervention in clinical practice and therefore the requirement for real-world evidence (RWE) to support healthcare decision making is growing. The objective of this survey was to understand pharmaceutical industry professionals’ views on the importance of and current use of RWE within their organisation. An electronic web-based survey was conducted at the 20th Annual European Congress of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) in November 2017. Industry professionals visiting the exhibit stand of the author organisation, a specialist RWE consultancy, were invited to complete a descriptive survey. A charity donation was offered for each completion. Data were anonymised, aggregated and reported following the conference. Twenty-three participants completed the survey with most (70%) employed by large or medium sized pharmaceutical companies. Almost two-thirds (61%) commissioned multi-country rather than single-country RWE studies to meet their evidence requirements. The most commonly reported markets requiring RWE studies included Europe (70%), America (30%) and the Nordic countries (22%). Participants reported the most important areas for using RWE to support brand value messages as: ‘the identification of unmet need’ (78%), ‘patient reported quality of life data’ (78%) and the ‘impact on healthcare resource utilisation’ (78%). Almost two thirds (65%) of participants stated that the use of RWE has changed the reimbursement of one or more of their brands. The results of this exploratory survey suggest that industry professionals recognise RWE as important in supporting brand value messages and report a direct positive impact of RWE on product reimbursement status. RWE should be considered as a key component of a product’s value proposition.

Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group

BackgroundSuccessful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated. ObjectiveTo develop for the first time a catalog of primary impediments to RD research and HTA, and to describe the cause and effect of individual challenges. MethodsChallenges were identified by an international 22-person expert working group and qualitative outreach to colleagues with relevant expertise. A broad range of stakeholder perspectives is represented. Draft results were presented at annual European and North American International Society for Pharmacoeconomics and Outcomes Research (ISPOR) congresses, and written comments were received by the 385-strong ISPOR Rare Disease Review Group from two rounds of review. Findings were refined and confirmed via targeted literature search. ResultsResearch-related challenges linked to the low prevalence of RDs were categorized into those pertaining to disease recognition and diagnosis, evaluation of treatment effect, and patient recruitment for clinical research. HTA-related challenges were classified into issues relating to the lack of a tailored HTA method for RD treatments and uncertainty for HTA agencies and health care payers. ConclusionsIdentifying and highlighting diverse, but interrelated, key challenges in RD research and HTA is an essential first step toward developing implementable and sustainable solutions. A collaborative multistakeholder effort is required to enable faster and less costly development of safe, efficacious, and appropriate new RD therapies that offer value for money.

Translation and cross-cultural adaptation into Brazilian Portuguese of the Mood and Feelings Questionnaire (MFQ) - Long Version.

Introduction Major depressive disorder (MDD) is prevalent among young people, with a high incidence during adolescence. It is, therefore, important to have reliable instruments to capture the construct of depression in this population. The objective of the present work is to describe the process of translation and cultural adaptation of the Mood and Feelings Questionnaire (MFQ) - Long Version, into Brazilian Portuguese. Method We followed the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) guidelines for translation and cultural adaptation, including the steps of preparation, forward translation, reconciliation, back-translation, back-translation review, harmonization, cognitive debriefing, review of cognitive debriefing results and finalization, proofreading and final report. Cognitive debriefing was conducted in a sample of adolescent patients and their respective caregivers at mental health clinics affiliated with the Brazilian public health system. Results Results suggest that the items were well understood and that the MFQ seems to be an appropriate instrument for use with Brazilian adolescents and caregivers. Conclusions The Brazilian Portuguese MFQ - Long Version constitutes an adequate tool for the assessment of depression among adolescents. Future studies are required to evaluate psychometric properties of the instrument.

Forgotten Joint Score – Portuguese translation and cultural adaptation of the instrument of evaluation for hip and knee arthroplasties

ObjectiveTo translate and adapt culturally to Brazilian Portuguese the Forgotten Joint Score (FJS) patient-reported outcome questionnaire. MethodsForty-five patients in the postoperative period (3–12 months) of total knee and hip arthroplasty were asked to answer the Br FJS questionnaire, translated into Portuguese based on the guidelines of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). ResultsTwenty-three patients completed the questionnaire correctly, suggesting changes when pertinent. In the first round of answers, it was observed that 20% had difficulty in understanding the expression “joint awareness.” In further harmonization of the questionnaire, it was decided to change the term “awareness” for “remember.” After this change no difficulty was observed in understanding for more than 85% of patients. ConclusionThe FJS questionnaire was translated and culturally adapted to Brazilian Portuguese. Additional studies are underway to compare the reproducibility and validity of the Brazilian translation to other questionnaires already established for the same outcome.

Determinants of health-related quality of life in international graduate students

IntroductionInternational graduate students often experience additional levels of stress due to acculturation. Given the impact of stress on health outcomes (both physical and mental), this study examined the health-related quality of life (HRQoL) in international graduate students to determine its association with acculturative stress, perceived stress, and use of coping mechanisms. MethodsA cross-sectional, self-administered survey was designed and sent to 38 student chapters within the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) student network. HRQoL [physical component summary (PCS) and mental component summary (MCS)] was measured using the 12-item Short Form (SF-12) while coping mechanisms were assessed using the Brief COPE Scale. Acculturative and perceived stress were assessed using the Acculturative Stress Scale for International students [ASSIS] and Graduate Stress Inventory-Revised (GSI-R), respectively. Demographic and personal information (e.g. age, religion) were also collected. Descriptive statistics (mean ± SD and frequency) and hierarchical multiple regression analysis were conducted. ResultsThe average PCS and MCS were 60 ± 9 and 44 ± 13, respectively, indicating that while the physical health was above the United States (US) general population norm (50), mental health scores were lower. Findings from the hierarchical multiple regression showed that perceived and acculturative stress significantly predicted mental health. Acculturative stress was also a significant predictor of physical health. ConclusionThe results from this study support the hypothesis that international students in the US experience both perceived and acculturative stress that significantly impacts their HRQoL. Universities should consider providing education on stress reduction techniques to improve the health of international graduate students.

A Systematic Review of Discrete-Choice Experiments and Conjoint Analysis Studies in People with Multiple Sclerosis

Multiple sclerosis (MS) is a chronic disabling, inflammatory, and degenerative disease of the central nervous system that, in most cases, requires long-term disease-modifying treatment (DMT). The drugs used vary in efficacy and adverse effect profiles. Several studies have used attribute-based stated-preference methods, primarily to investigate patient preferences for initiating or escalating DMT. To conduct a systematic review of attribute-based stated-preference studies in people with MS to identify common methods employed and to assess study quality, with reference to the specific challenges of this disease area. We conducted a systematic search for studies related to attribute-based stated-preference and MS in multiple databases, including Cochrane and MEDLINE. Studies were included if they were published in a peer-reviewed journal, were on the topic of MS, and used a survey methodology that measured stated preferences for attributes of a whole. Analysis was conducted using narrative synthesis and summary statistics. Study quality was judged against the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) conjoint analysis checklist. We identified 16 relevant articles reporting 17 separate studies, all but one focusing on DMTs. Most studies were discrete-choice experiments. Study quality was generally high, but we recommend the following: (1) that consideration of sample sizes be improved, (2) that survey design choices be justified and documented, (3) that qualitative approaches for attribute and level selection be incorporated to better involve patients, and (4) that reporting of experimental practice be improved. The effects of DMTs on reproduction and the impact of how risk and uncertainty are presented were identified as neglected research topics. The ISPOR conjoint analysis checklist was found to be unsuitable for the assessment of study quality. Attribute-based stated preference is a useful method with which to examine the preferences of people with MS in their choice of DMT. However, further research embracing the methodological recommendations identified, particularly greater use of qualitative methods in attribute development, is needed.

PP51 Updating Canadian Pharmaceutical Budget Impact Analysis Guidelines

Introduction:The Canadian BIA guideline was published by the Patented Medicine Prices Review Board (PMPRB) in 2007. Our initial systematic literature review of national and international BIA guidelines showed that a number of new recommendations relating to BIA model structure, input data and reporting format have been adopted in other jurisdictions such as UK, Australia, Poland, Ireland, Belgium, France and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). The main objective of the present study was to conduct a comparative review of national, international and Canadian Federal, provincial and territorial BIA guidelines and provide a list of new recommendations related to the BIA key elements which have not been discussed or included in the Canadian PMPRB BIA guidelines.Methods:BIAs guidelines were searched in databases such as MEDLINE, EMBASE, Cochrane, and the gray literature including regulatory agency websites. An Excel-based data abstraction form was designed in order to highlight differences between recommendations related to the BIA key elements provided by PMPRB, provincial, and other national and international BIA guidelines.Results:Twelve guidelines were reviewed in detail. Sixty percent of the recommendations were new or were different from recommendations in the Canadian PMPRB BIA guidelines. They related to BIA key elements such as perspective, target population, costing, presenting results, data sources and handling the uncertainty.Conclusions:The present literature review is the initial step towards updating the Canadian BIA guidelines. This study presents a comparative review of key elements in BIA among different guidelines and provides a list of relevant practical recommendations for the improvement of the Canadian BIA guidelines. The new methodologic advancements and recommendations that were identified are being presented to Canadian stakeholders for their opinion and feedback prior to the development of a proposed new set of Canadian guidelines.

Real-World Characteristics and Treatment Patterns in Patients with Urticaria Initiating Omalizumab in the United States.

Omalizumab is indicated for the management of chronic idiopathic urticaria (CIU) in patients aged 12 years or older with persistent hives that are not adequately controlled by H1 antihistamines. While its safety and efficacy in CIU patients have been evaluated in multiple clinical trials, real-world use of omalizaumab in CIU has not been well characterized. To assess demographics, clinical characteristics, and treatment patterns of CIU patients who initiated omalizumab to better understand the usage of this agent in CIU management in the real world. This retrospective cohort study used medical and pharmacy claims data in the United States from the HealthCore Integrated Database to identify patients with CIU newly treated with omalizumab (≥ 4 omalizumab claims within 6 months of the initial claim) between March 21, 2014, and October 31, 2015 (study intake period). The index date was defined as the date of the first claim for omalizumab during the study intake period. Demographic and clinical characteristics were described for patients treated with omalizumab, as were treatment patterns associated with omalizumab and concomitant medications associated with CIU treatment. Descriptive and inferential statistics were reported. The Kaplan-Meier method was used to examine omalizumab treatment patterns. This study included 298 omalizumab-treated patients (mean [SD] age of 43.5 [13.64] years; 70.8% female); approximately 84% were seen by an allergist/immunologist. All patients had ≥ 12 months of continuous enrolment and a subset of 138 patients had ≥ 18 months of follow-up. For patients with ≥ 12 months of post-index follow-up, 12.1% (n = 36), 28.5% (n = 85), and 32.9% (n = 98) discontinued omalizumab within the 6-month, 12-month, and the entire post-index periods (mean 530 days), respectively; the mean number of days patients were continuously treated with omalizumab was 443.1 (95% CI = 425.0-461.3); the probabilities of continuous treatment (95% CI) were 0.879 (0.836-0.911), 0.711 (0.656-0.759), and 0.647 (0.585-0.703) for the 6-, 12-, and 18-month post-index periods, respectively. For the 98 patients who discontinued omalizumab during the entire post-index period, 28.6% restarted omalizumab after the first discontinuation within the post-index period (mean time from first discontinuation to first restart=329 days). Use of medications such as oral corticosteroids, montelukast, cyclosporine, and prescription H1 and H2 antihistamines decreased during the 1- to 6-month and 7- to 12-month post-index periods compared with those within the 6-month pre-index period. In this cohort of CIU patients who were newly prescribed omalizumab, the majority were treated by allergists/immunologists as expected, and approximately 60% of patients continued on therapy beyond 18 months. Concomitant medication use decreased after omalizumab initiation. These data on the real-world use of omalizumab for CIU may help to better inform decision-making processes for health care payers by quantifying omalizumab and concomitant medication treatment patterns over a longer time frame relative to previous studies. This study was sponsored by Novartis Pharmaceuticals, which provided funding support for the conduct of the study. Kavati, Ortiz, and Paknis are employees of Novartis Pharmaceuticals. Ke, Wertz, Huang, Wang, Willey, and Stephenson are employees of HealthCore, an independent research organization that received funding from Novartis Pharmaceuticals for the conduct of this study. Beck is an employee of the University of Rochester Medical Center, who was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports grants from Genentech, outside the currently submitted work. Bernstein is affiliated with Bernstein Clinical Research Center, which was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports receiving grants and personal fees from Novartis Pharmaceuticals, grants and personal fees from Genentech outside of the submitted work, and is an author on the Joint Task Force for Practice Parameters for Urticaria and the GALEN international guidelines for urticaria under preparation. Selected study data were presented in a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 22nd Annual International Meeting on May 20-24, 2017, in Boston, MA. A poster based on this dataset was presented at the 2017 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting on October 26-30, 2017, in Boston, MA.

Real-World Characteristics and Treatment Patterns in Patients with Urticaria Initiating Omalizumab in the United States.

Omalizumab is indicated for the management of chronic idiopathic urticaria (CIU) in patients aged 12 years or older with persistent hives that are not adequately controlled by H1 antihistamines. While its safety and efficacy in CIU patients have been evaluated in multiple clinical trials, real-world use of omalizaumab in CIU has not been well characterized. To assess demographics, clinical characteristics, and treatment patterns of CIU patients who initiated omalizumab to better understand the usage of this agent in CIU management in the real world. This retrospective cohort study used medical and pharmacy claims data in the United States from the HealthCore Integrated Database to identify patients with CIU newly treated with omalizumab (≥ 4 omalizumab claims within 6 months of the initial claim) between March 21, 2014, and October 31, 2015 (study intake period). The index date was defined as the date of the first claim for omalizumab during the study intake period. Demographic and clinical characteristics were described for patients treated with omalizumab, as were treatment patterns associated with omalizumab and concomitant medications associated with CIU treatment. Descriptive and inferential statistics were reported. The Kaplan-Meier method was used to examine omalizumab treatment patterns. This study included 298 omalizumab-treated patients (mean [SD] age of 43.5 [13.64] years; 70.8% female); approximately 84% were seen by an allergist/immunologist. All patients had ≥ 12 months of continuous enrolment and a subset of 138 patients had ≥ 18 months of follow-up. For patients with ≥ 12 months of post-index follow-up, 12.1% (n = 36), 28.5% (n = 85), and 32.9% (n = 98) discontinued omalizumab within the 6-month, 12-month, and the entire post-index periods (mean 530 days), respectively; the mean number of days patients were continuously treated with omalizumab was 443.1 (95% CI = 425.0-461.3); the probabilities of continuous treatment (95% CI) were 0.879 (0.836-0.911), 0.711 (0.656-0.759), and 0.647 (0.585-0.703) for the 6-, 12-, and 18-month post-index periods, respectively. For the 98 patients who discontinued omalizumab during the entire post-index period, 28.6% restarted omalizumab after the first discontinuation within the post-index period (mean time from first discontinuation to first restart=329 days). Use of medications such as oral corticosteroids, montelukast, cyclosporine, and prescription H1 and H2 antihistamines decreased during the 1- to 6-month and 7- to 12-month post-index periods compared with those within the 6-month pre-index period. In this cohort of CIU patients who were newly prescribed omalizumab, the majority were treated by allergists/immunologists as expected, and approximately 60% of patients continued on therapy beyond 18 months. Concomitant medication use decreased after omalizumab initiation. These data on the real-world use of omalizumab for CIU may help to better inform decision-making processes for health care payers by quantifying omalizumab and concomitant medication treatment patterns over a longer time frame relative to previous studies. DISCLOSURES This study was sponsored by Novartis Pharmaceuticals, which provided funding support for the conduct of the study. Kavati, Ortiz, and Paknis are employees of Novartis Pharmaceuticals. Ke, Wertz, Huang, Wang, Willey, and Stephenson are employees of HealthCore, an independent research organization that received funding from Novartis Pharmaceuticals for the conduct of this study. Beck is an employee of the University of Rochester Medical Center, who was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports grants from Genentech, outside the currently submitted work. Bernstein is affiliated with Bernstein Clinical Research Center, which was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports receiving grants and personal fees from Novartis Pharmaceuticals, grants and personal fees from Genentech outside of the submitted work, and is an author on the Joint Task Force for Practice Parameters for Urticaria and the GALEN international guidelines for urticaria under preparation. Study concept and design were primarily contributed by Kavati, Ortiz, and Paknis, with assistance from the other authors. Huang, Wang, and Ke took the lead in data collection, with assistance from Wertz, Willey, and Stephenson. Data interpretation was performed by Ke, Wertz, and Willey, assisted by the other authors. The manuscript was written by Stephenson, Ke, and Wang, along with Willey, Wertz, and Huang, and revised by Bernstein and Beck, with assistance from the other authors. Selected study data were presented in a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 22nd Annual International Meeting on May 20-24, 2017, in Boston, Massachusetts. A poster based on this dataset was presented at the 2017 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting on October 26-30, 2017, in Boston, Massachusetts.

The joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making: an interview with Marc Berger.

Marc L Berger, MD, is a retired, part-time consultant. He recently became Chair of the Real World Evidence Advisory Board for SHYFT Analytics. Over a 25-year industry career, Marc has held senior-level positions at Pfizer, Inc., OptumInsight, Eli Lilly and Company, and Merck & Co., Inc. His professional activities have included serving on committees for Center for Medicare and Medicaid Services (CMS), Agency for Healthcare Research and Quality (AHRQ), Patient-Centered Outcomes Research Institute (PCORI), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), Drug Information Association (DIA), and the editorial advisory boards of several journals. Marc has written or co-written more than 100 peer-reviewed articles, book chapters, and other publications on a range of topics including health services research, outcomes research, health economics, health policy, and the analysis of real-world data.

MON-P187: Translation and Cross-Cultural Adaptation of the Scored Patient-Generated Subjective Global Assessment (PG-SGA) To the Thai Setting

Rationale: The Scored Patient-Generated Subjective Global Assessment (PG-SGA) is a multidimensional tool to assess malnutrition and risk factors. We aimed to translate and cross-culturally adapt the original PG-SGA for the Thai setting and test its linguistic validity and intra-rater reliability in cancer patients. Methods: The Thai version of the PG-SGA was developed according to the Principles of Good Practice for the Translation and Cultural Adaptation Process for the Patient Reported Outcomes by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In 50 cancer patients and 50 healthcare professionals, linguistic validity of the Thai PG-SGA was assessed by Scale Comprehensibility Index (S-CI) and Scale Difficulty Index (S-DI) ), using a 4-point scale. Relevance was assessed in professionals only, by Scale Content Validity Index (S-CVI). In addition, intra-rater reliability (test-retest within 72 hours of admission; Intraclass Correlation Coefficient (ICC) and weighted kappa (κ)) were assessed. Results: The Thai PG-SGA showed excellent comprehensibility (S-CI=0.99) and difficulty (S-DI=0.95) as perceived by patients. It also showed excellent comprehensibility (S-CI= 0.92) and borderline acceptable difficulty (S-DI=0.79) as perceived by professionals. Relevance of the Thai PG-SGA in assessing malnutrition was considered excellent (S-CVI=0.95). Agreement between numerical scores was good to excellent (ICC=0.95) and agreement between PG-SGA categories was very good (weighted κ=0.95). Conclusion: The Thai version of the PG-SGA was considered very easy to complete by patients. Professionals evaluated it as very comprehensible, relevant, and borderline acceptable in difficulty to complete. It is a reliable tool for assessment of malnutrition and risk factors in cancer patients.

Good Practices for Real-World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR-ISPE Special Task Force on Real-World Evidence in Health Care Decision Making.

Real-world evidence (RWE) includes data from retrospective or prospective observational studies and observational registries and provides insights beyond those addressed by randomized controlled trials. RWE studies aim to improve health care decision making. The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the International Society for Pharmacoepidemiology (ISPE) created a task force to make recommendations regarding good procedural practices that would enhance decision makers' confidence in evidence derived from RWD studies. Peer review by ISPOR/ISPE members and task force participants provided a consensus-building iterative process for the topics and framing of recommendations. The ISPOR/ISPE Task Force recommendations cover seven topics such as study registration, replicability, and stakeholder involvement in RWE studies. These recommendations, in concert with earlier recommendations about study methodology, provide a trustworthy foundation for the expanded use of RWE in health care decision making. The focus of these recommendations is good procedural practices for studies that test a specific hypothesis in a specific population. We recognize that some of the recommendations in this report may not be widely adopted without appropriate incentives from decision makers, journal editors, and other key stakeholders.