Development of a disease-specific health utility score for chronic obstructive pulmonary disease from a discrete choice experiment patient preference study.

OBJECTIVES: While patient input to health technology assessment (HTA) has traditionally been of a qualitative nature, there is increasing interest to integrate quantitative evidence from patient preference studies into HTA decision making. Preference data can be used to generate disease-specific health utility data. We generated a health utility score for patients with chronic obstructive pulmonary disease (COPD) and consider its use within HTAs. METHODS: Based on qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance, and urinary incontinence. We employed a discrete choice experiment (DCE) and the random parameter logistic regression technique to estimate utility scores for all COPD health states. The relationship between patients' COPD health utility scores, self-perceived COPD severity, and EQ-5D-3L utility scores was analyzed, with data stratified according to disease severity and comorbidity subgroups. RESULTS: The COPD health utility score had face validity, with utility scores negatively correlated with patients' self-perceived COPD severity. The correlation between the COPD health utility scores and EQ-5D-3L values was only moderate. While patient EQ-5D-3L scores were impacted by comorbidities, the COPD health utility score was less impacted by comorbid conditions. CONCLUSIONS: Our COPD utility measure, derived from a DCE, provides a patient-centered health utility score and is more sensitive to the COPD health of the individual and less sensitive to other comorbidities. This disease-specific instrument should be considered alongside generic health-related quality of life instruments when valuing new COPD therapies in submissions to licensing and reimbursement agencies.

Development of an international template to support patient submissions in Health Technology Assessments.

OBJECTIVES: To develop an international template to support patient submissions in Health Technology Assessments (HTAs). This was to be based on the experience and feedback from the implementation and use of the Scottish Medicines Consortium's (SMC) Summary Information for Patient Groups (SIP). METHODS: To gather feedback on the SMC experience, web-based surveys were conducted with pharmaceutical companies and patient groups familiar with the SMC SIP. Semistructured interviews with representatives from HTA bodies were undertaken, along with patient group discussions with those less familiar with the SIP, to explore issues around the approach. These qualitative data informed the development of an international SIP template. RESULTS: Survey data indicated that 82 percent (18 of 22 respondents) of pharmaceutical company representatives felt that the SIP was worthwhile; 88 percent (15/17) of patient group respondents found the SIP helpful. Both groups highlighted the need for additional support and guidance around plain language summaries. Further suggestions included provision of a glossary of terms and cost-effectiveness information. Patient group interviews supported the survey findings and led to the development of a new template. HTA bodies raised potential challenges around buy-in, timing, and bias connected to the SIP approach. CONCLUSIONS: The international SIP template is another approach to support deliberative processes in HTA. Although challenges remain around writing summaries for lay audiences, along with feasibility considerations for HTA bodies, the SIP approach should support more meaningful patient involvement in HTAs.

How to integrate evidence from patient preference studies into health technology assessment: a critical review and recommendations.

Health technology assessment (HTA) agencies vary in their use of quantitative patient preference data (PP) and the extent to which they have formalized this use in their guidelines. Based on the authors' knowledge of the literature, we identified six different PP "use cases" that integrate PP into HTA in five different ways: through endpoint selection, clinical benefit rating, predicting uptake, input into economic evaluation, and a means to weight all HTA criteria. Five types of insight are distinguished across the use cases: understanding what matters to patients, predicting patient choices, estimating the utility generated by treatment benefits, estimating the willingness to pay for treatment benefits, and informing distributional considerations. Summarizing the literature on these use cases, we recommend circumstances in which PP can add value to HTA and the further research and guidance that is required to support the integration of PP in HTA. Where HTA places more emphasis on clinical outcomes, novel endpoints are available; or where there are already many treatment options, PP can add value by helping decision makers to understand what matters to patients. Where uptake is uncertain, PP can be used to estimate uptake probability. Where indication-specific utility functions are required or where existing utility measures fail to capture the value of treatments, PP can be used to generate or supplement existing utility estimates. Where patients are paying out of pocket, PP can be used to estimate willingness to pay.

Assessing physician preferences on future therapeutic options and diagnostic practices in non-alcoholic steatohepatitis.

BACKGROUND & AIMS: There is currently no data on physician preferences regarding future therapies for non-alcoholic steatohepatitis (NASH); this study explores these preferences and characteristics that are relevant to physician decision-making when choosing a potential therapy for a patient with NASH. The results were compared with those from a similar patient preference survey which was conducted in parallel. METHOD: Initial exploratory 30-minute telephone interviews were conducted to inform the design of a 15-minute quantitative online specialist physicians survey, containing direct questions and a preference survey. This was based on a best-worst scaling (BWS) experiment to assess the relative importance of different treatment characteristics (attributes), followed by several paired comparison questions to understand the preference for 5 hypothetical product profiles. RESULTS: The answers come from 121 physicians from Canada (n = 31), Germany (n = 30), the UK (n = 30) and the USA (n = 30). The primary driving element in NASH treatment decision-making was efficacy (49.23%), defined as "[hypothetical product] impact on liver status" and "[slowing of] progression to cirrhosis". Physicians reported the common use of non-invasive NASH diagnostic tests and 81% reported performing liver biopsy. In 57% of cases, physicians reported that "concerns related to the available diagnostic methods" limit the number of patients with biopsy-confirmed NASH. CONCLUSIONS: This first physician preference study reveals that efficacy will be the main driver for physicians in selecting future NASH drugs. The findings also confirm the widespread use of non-invasive diagnostic tests and the reluctance to perform confirmatory liver biopsy despite guideline recommendations, mainly due to limited therapeutic options and patient refusal. LAY SUMMARY: This study explores physician preferences in relation to future therapies for non-alcoholic steatohepatitis (NASH) and characteristics that are relevant to physician decision-making when choosing a potential therapy for a patient with NASH. The results of a short online survey completed by 121 specialist physicians determined that the primary factor that influences treatment decision-making is efficacy, and that a wide range of non-invasive techniques are used to diagnose NASH, while confirmatory liver biopsy is not performed by all physicians despite guideline recommendations.

Impact of cough and mucus on COPD patients: primary insights from an exploratory study with an Online Patient Community.

Background: Qualitative research provides real-life information on patients' condition and facilitates informed design of future clinical studies. Objective: We used Online Communities as a qualitative research tool to evaluate the effect of cough and mucus on COPD patients. Methods: Two 2-week Online Communities were run in parallel in the UK and in the USA, including COPD patients with persistent cough and excessive mucus. Patients anonymously posted their responses to pre-assigned tasks, supervised and guided by a trained moderator. Five themes around the impact of cough and mucus were explored with new questions posted every 2-3 days. On the final day, high-level conclusions were shared with patients for feedback. Data were analyzed following the principles of grounded theory. Results: Twenty COPD patients (UK, n=10; USA, n=10) participated in the Online Communities. We found that cough and mucus disrupted COPD patients' lives at functional, emotional, social and economic levels. Patients created daily rituals and adjusted their lifestyle to cope with the impact of these symptoms. Patients identified themselves with our conclusions and saw the Online Community as an effective forum to share their experiences. Conclusion: Findings of our study add to the body of evidence on the negative impact of COPD symptoms and unmet needs of these patients.

Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs.

Although patient preferences have been studied broadly for marketed products or around the time of submission to authorities and launch, patient preference studies have rarely been used during the early drug development phases. In this paper, we formulate three hypotheses supporting the use of patient preference studies in early product development: (1) integration of the patient perspective into the development process from phase 1 onwards will result in healthcare solutions with outcomes that best address patients' needs; (2) a structured process to build patient-based evidence involving partnerships between patients and other key stakeholders will improve alignment of development activities with the needs of patients; (3) quantitative patient preference research built on robust qualitative insights is necessary to strengthen development decisions in the interests of patients. To illustrate such a structured process, we describe qualitative insights research (social media analysis and online bulletin boards) and quantitative patient preference studies in dry eye disease and non-alcoholic steatohepatitis conducted during early product development by a pharmaceutical company to generate patient-based evidence. The outputs from such early patient preference studies are being used to inform patient reported outcome strategies, clinical development strategies, product design and delivery features, and form the basis for early dialog with regulators, health technology assessment (HTA) bodies and payers to ensure focus and alignment on patient-relevant endpoints. Furthermore, to discuss and theoretically substantiate our hypotheses, we review how different groups and organizations are working to embrace fully the patient perspective in product development and healthcare decision-making. The hypotheses are commensurate with the general trend toward patient-centered healthcare and the activities initiated by regulators, HTA agencies, and patient organizations. We advocate that all healthcare players should actively contribute to aligning on best practices concerning choice of methodologies and engage in multi-stakeholder dialog along the entire product development chain, to realize health technologies that best meet the needs of patients.

Defining the needs and preferences of patients with dry eye disease.

OBJECTIVE: Dry eye disease is a multifactorial chronic disease, leading to ocular discomfort and visual disturbance with a substantial impact on quality of life. Therefore, the patient's perspective should be taken into account early in the drug development process. We have developed a step-by-step methodology based on the self-explicated conjoint approach to assess the needs and preferences of patients with moderate-to-severe dry eye disease. METHODS AND ANALYSIS: Following a literature review and social media listening (step 0), qualitative phone call interviews were conducted with 12 patients (step 1). Patients' responses underwent content analysis and were coded, quantified and displayed as charts. Based on the emerging trends and attributes identified as relevant in steps 0 and 1, a quantitative online questionnaire was designed and conducted with 160 patients across four countries (step 2). RESULTS: The online questionnaire was rated as easy/very easy to understand by 60% of respondents, 62% rated the survey as easy/very easy to complete and 71% rated it as interesting/very interesting. Treatment satisfaction was the most important aspect for patients, and the three most relevant attributes were as follows (with the most important indexed to 100%): 'treatment effectiveness on symptoms of dry eyes' (100%), 'frequency of treatment use' (96%) and 'how the treatment works' (95%). CONCLUSION: Our methodology was well received by patients, and the results will help inform future clinical trial development and discussions with health technology assessment bodies and regulators on unmet needs and product attributes that are of most value to patients with dry eye disease.

Understanding Patient Preferences and Unmet Needs in Non-alcoholic Steatohepatitis (NASH): Insights from a Qualitative Online Bulletin Board Study.

INTRODUCTION: The aim of this work was to understand how patients with non-alcoholic steatohepatitis (NASH) perceive their disease, unmet needs, and expectations regarding future treatment through online bulletin board (OBB) qualitative research. METHODS: OBB is an asynchronous online qualitative market research tool that provides an open forum for interactive discussion among participants. Patients with NASH were recruited via physician referral and completed a screener questionnaire to ensure their eligibility and willingness to participate. A trained moderator managed the discussion that allowed open answers and responses to other participants' posts. Patient responses were analyzed using a combination of different qualitative analytical tools. RESULTS: The OBB ran for 4 days and included 16 patients (n = 8, UK; n = 8, US) with NASH (fibrosis stages F1-F3) and comorbidities including diabetes/prediabetes (n = 9) and obesity (n = 12). The key insights were (1) patients with NASH have a poor understanding of the disease, its progression, and management-they feel a lack of adequate educational support from their physicians; (2) diagnosis of NASH is incidental in most cases, mainly because patients fail to spontaneously associate their signs or symptoms with their liver condition; (3) comorbidities (obesity and diabetes) are more concerning to patients than NASH; and (4) patients perceive that NASH impacts their social life and work performance in more advanced stages. CONCLUSIONS: This OBB provided valuable patient insights into NASH disease perception and management and revealed unmet need areas. In light of no approved therapies, these patient insights can inform early drug development strategies and stakeholder discussions on NASH. FUNDING: Novartis Pharma AG, Basel.

Monitoring of Extraction Efficiency by a Sample Process Control Virus Added Immediately Upon Sample Receipt.

When analysing food samples for enteric viruses, a sample process control virus (SPCV) must be added at the commencement of the analytical procedure, to verify that the analysis has been performed correctly. Samples can on occasion arrive at the laboratory late in the working day or week. The analyst may consequently have insufficient time to commence and complete the complex procedure, and the samples must consequently be stored. To maintain the validity of the analytical result, it will be necessary to consider storage as part of the process, and the analytical procedure as commencing on sample receipt. The aim of this study was to verify that an SPCV can be recovered after sample storage, and thus indicate the effective recovery of enteric viruses. Two types of samples (fresh and frozen raspberries) and two types of storage (refrigerated and frozen) were studied using Mengovirus vMC0 as SPCV. SPCV recovery was not significantly different (P > 0.5) regardless of sample type or duration of storage (up to 14 days at -20 °C). Accordingly, samples can be stored without a significant effect on the performance of the analysis. The results of this study should assist the analyst by demonstrating that they can verify that viruses can be extracted from food samples even if samples have been stored.

Behavioral needs of the transition cow and considerations for special needs facility design.

Improved building designs come from a better understanding of the behavioral needs of the dairy cow. The costs to provide for these needs in the facility must be offset by improved milk production, health, and longevity. Research is still required to more fully understand the health implications of many building design considerations and their impact on disease. Perhaps the most important end result of an improved environment for the transition cow, however, is an improvement in animal well-being. Better buildings that accommodate the behavioral needs of cows present "win-win" situations where dairy cattle thrive and work is more enjoyable. This results in an improved image for the industry, greater consumer confidence in the quality and safety of the final food product, and a prosperous dairy industry.

Using herd records to monitor transition cow survival, productivity, and health.

There is no single monitor that can fully characterize the success of a transition cow management program in a herd. Rather we must rely on a group of key monitors. Table 5 outlines the key indices and targets that we use in herd investigations. By using these monitors, effective transition cow programs can be differentiated from problematic ones, and many of the problems can be resolved for the good of the herd owners, dairy laborers, and most of all, the cows. Development of more sophisticated monitors and software with stronger epidemiologic structure will allow for better analysis of programs in the future.