Ethical and legal considerations in social media research for health technology assessment: conclusions from a scoping review.

OBJECTIVES: The objective was to identify and describe the published guidance and current academic discourse of ethical issues and standards related to the use of Social Media Research for generating patient insights for the use by health technology assessment (HTA) or health policy decisions. METHODS: A scoping review of the literature was conducted in PubMed and Embase and identified 935 potential references published between January 2017 and June 2021. After title and abstract screening by three reviewers, 40 publications were included, the relevant information was extracted and data were collected in a mind map, which was then used to structure the output of the review. RESULTS: Social Media Research may reveal new insights of relevance to HTA or health policies into patient needs, patient experiences, or patient behaviors. However, the research approaches, methods, data use, interpretation, and communication may expose those who post the data in social media channels to risks and potential harms relating to privacy, anonymity/confidentiality, authenticity, context, and rapidly changing technologies. CONCLUSIONS: An actively engaged approach to ensuring ethical innocuousness is recommended that carefully follows best practices throughout planning, conduct, and communication of the research. Throughout the process and as a follow-up, there should be a discourse with the ethical experts to maximally protect the current and future users of social media, to support their trust in the research, and to advance the knowledge in parallel to the advancement of the media themselves, the technologies, and the research tools.

Using social media research in health technology assessment: stakeholder perspectives and scoping review.

OBJECTIVES: The aim of this initiative was to examine collaboratively, in a multi-stakeholder team (health technology assessment (HTA) practitioners with patient involvement expertise, health technology industry, patient advocates, health policy experts, patient engagement experts), whether evidence generated through social media research (SMR) fills current information gaps relating to insights on specific aspects of patient experiences, preferences, or patient needs and delivers additional value to HTA. METHODS: The framing of the project was done in a co-creative, deliberative multi-stakeholder process. Challenge and refinement happened through discussions with 25 independent stakeholders from HTA bodies, industry, academia, and patient advocacy. For critical themes identified during the framing phase, scoping literature reviews were performed including the state of methods and examples for the use of SMR in HTA. RESULTS: The framing and stakeholder discussions specified a set of expectations and requirements, and the scoping reviews revealed the current state of methods and usage of SMR in health-policy decision making. CONCLUSIONS: The project concluded that SMR can contribute new, relevant evidence to HTA. It is however recommended to evolve the science through defining best practices when planning, conducting, and using SMR and to conduct multi-stakeholder pilot SMR projects to address questions relevant to current HTAs and to validate and improve the proposed practices.

A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force.

Many qualitative and quantitative methods are readily available to study patient preferences in health. These methods are now being used to inform a wide variety of decisions, and there is a growing body of evidence showing studies of patient preferences can be used for decision making in a wide variety of contexts. This ISPOR Task Force report synthesizes current good practices for increasing the usefulness and impact of patient-preference studies in decision making. We provide the ISPOR Roadmap for Patient Preferences in Decision Making that invites patient-preference researchers to work with decision makers, patients and patient groups, and other stakeholders to ensure that studies are useful and impactful. The ISPOR Roadmap consists of 5 key elements: (1) context, (2) purpose, (3) population, (4) method, and (5) impact. In this report, we define these 5 elements and provide good practices on how patient-preference researchers and others can actively contribute to increasing the usefulness and impact of patient-preference studies in decision making. We also present a set of key questions that can support researchers and other stakeholders (eg, funders, reviewers, readers) to assess efforts that promote the ongoing impact (both intended and unintended) of a particular preference study and additional studies in the future.

Cost-Effectiveness of Erenumab for the Preventive Treatment of Migraine in Patients with Prior Treatment Failures in Sweden.

BACKGROUND: Migraine is a common neurological disease that disproportionately affects females and has a peak incidence during productive years, resulting in significant burden. OBJECTIVE: The aim of the study was to determine the cost effectiveness of erenumab for the preventive treatment of migraine. METHODS: A hybrid decision-tree plus Markov model was developed to evaluate the cost effectiveness of erenumab as a migraine treatment compared with best supportive care only for patients experiencing at least 4 monthly migraine days for whom at least two prior preventive treatments had failed. Clinical efficacy data were based on results from four randomized controlled trials of erenumab against placebo. The primary outcomes were costs, migraine days, and quality-adjusted life-years (QALYs). An incremental cost-effectiveness ratio (ICER) was estimated as the cost per QALY gained. The cost per migraine day avoided was also estimated, as were disaggregated direct and indirect costs. The analysis was conducted from Swedish societal and healthcare system perspectives based on total migraine, chronic migraine and episodic migraine populations, using a discount rate of 3% applied to both costs and health benefits and using year 2019 values. RESULTS: In the base-case deterministic analyses, erenumab treatment resulted in ICERs of Swedish krona (SEK) 34,696 (€3310) and SEK301,565 (€28,769) per QALY gained in the total migraine and episodic migraine populations, respectively. Erenumab was dominant in the chronic migraine population. In the total migraine population, the use of erenumab resulted in a net benefit to society of SEK81,739 (€7773) per patient, assuming a willingness-to-pay threshold of SEK300,000 (€28,528) per QALY. CONCLUSIONS: Our analysis suggests that erenumab is a cost-effective treatment for migraine with a willingness-to-pay threshold of SEK300,000 per QALY.

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.

Modelling the Cost-Effectiveness of Indacaterol/Glycopyrronium versus Salmeterol/Fluticasone Using a Novel Markov Exacerbation-Based Approach.

Purpose: Exacerbations drive outcomes and costs in chronic obstructive pulmonary disease (COPD). While patient-level (micro) simulation cost-effectiveness models have been developed that include exacerbations, such models are complex. We developed a novel, exacerbation-based model to assess the cost-effectiveness of indacaterol/glycopyrronium (IND/GLY) versus salmeterol/fluticasone (SFC) in COPD, using a Markov structure as a simplification of a previously validated microsimulation model. Methods: The Markov model included three health states: infrequent or frequent exacerbator (IE or FE; ≤1 or ≥2 moderate/severe exacerbations in prior 12 months, respectively), or death. The model used data from the FLAME study and was run over a 10-year horizon. Cycle length was 1 year, after which patients remained in the same health state or transitioned to another. Analysis was conducted from a Swedish payer's perspective (Swedish healthcare costs, converted into Euros), with incremental costs and quality-adjusted life-years (QALYs) calculated (discounted 3% annually). Results: At all post-baseline timepoints, IND/GLY was associated with more patients in the IE health state and fewer patients in the FE and dead states relative to SFC. Over a 10-year period, IND/GLY was associated with a cost saving of €1,887/patient, an incremental benefit of 0.142 QALYs, and an addition of 0.057 life-years, compared with SFC. Conclusion: This Markov model represents a novel cost-effectiveness analysis for COPD, with simpler methodology than prior microsimulation models, while retaining exacerbations as drivers of disease progression. In patients with COPD with a history of exacerbations in the previous year, IND/GLY is a cost-effective treatment option compared with SFC.

Economic evaluations in migraine: systematic literature review and a novel approach.

Background: For novel migraine therapies, economic evaluations will be required to understand the trade-offs between additional health benefit and additional cost. The purpose of this study was to conduct a systematic literature review (SLR) to identify previous economic evaluations in migraine from the United Kingdom or Irish perspective to critically appraise these evaluations and to propose, if necessary, a novel modelling approach that can be used for future economic evaluations of migraine therapies.Methods: An SLR was conducted to identify previous economic evaluations of preventive migraine treatments. Key opinion leaders were consulted to determine the criteria for a robust migraine economic evaluation. Economic evaluations identified in the SLR were appraised against these criteria, and a novel cost-effectiveness model structure was then proposed.Results: Eight records reporting on published economic evaluations were identified and critically appraised for general quality. Expert consultation provided 6 recommendations on the ideal model structure for migraine that is both clinically and economically meaningful. A decision-tree plus Markov structure was then developed as a cost-effectiveness model for migraine therapies where each health state is associated with a patient distribution across monthly migraine day (MMD) frequencies.Conclusions: Future migraine economic evaluations should allow for assessments across the full spectrum of migraine, a response-based stopping rule, and the estimation of benefits and resource costs based on MMD frequency. The approach proposed in this paper captures all of the desired elements for an economic evaluation of migraine therapy and is suitable to assess new migraine therapies.

The Clinical Effectiveness of Ranibizumab Treat and Extend Regimen in nAMD: Systematic Review and Network Meta-Analysis.

INTRODUCTION: Neovascular age-related macular degeneration (nAMD) is a chronic eye condition that causes severe deterioration of vision and ultimately blindness. Two vascular endothelial growth factor inhibitors are approved for nAMD treatment in Europe: ranibizumab and aflibercept. The European license for ranibizumab was updated with an individualized "treat and extend" (T&E) regimen, which involves more proactive treatment based on changes in best corrected visual acuity (BCVA) and/or anatomical outcomes. The aim of this publication is to compare the efficacy of the ranibizumab T&E regimen with other approved dosing regimens for nAMD on the basis of outcomes identified from a systematic review and subsequent NMA. METHODS: Following a systematic search of publications, to identify relevant studies, a repeated-measures network meta-analysis (NMA) was performed to estimate the relative effectiveness of ranibizumab T&E versus approved dosing regimens of ranibizumab and aflibercept. The analysis focused on licensed treatment regimens for nAMD. We examined mean change from baseline in BCVA on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. RESULTS: The systematic literature review identified 22,949 records, of which 23 studies were included in the NMA. At 12 months, the ranibizumab T&E dosing regimen vs ranibizumab pro re nata (PRN) was associated with small differences in change in BCVA, between 1.86 letter gain at 12 months and 2.35 letter gain at 24 months. A similar difference was observed in the aflibercept dosing regimen versus ranibizumab T&E ; 1.94 letter gain at 12 months and 3.31 letter gain at 24 months. All doses of ranibizumab and aflibercept showed similar effectiveness, and the differences between treatment options were not significant. CONCLUSION: This study used novel repeated-measures NMA to synthesize efficacy results when treatment effects were reported at multiple follow-up times. This repeated-measures NMA suggests that treating patients with the ranibizumab T&E regimen yields similar effectiveness compared to other approved ranibizumab and aflibercept dosing regimens for nAMD treatment. FUNDING: Novartis Pharmaceuticals UK Ltd, Surrey, UK.

Screening for gestational diabetes mellitus in primary versus secondary care: The clinical outcomes of a randomised controlled trial.

AIMS: To examine the clinical outcomes of screening for gestational diabetes mellitus (GDM) in primary care versus secondary care, in the Irish healthcare system. DESIGN AND METHODS: A parallel group randomised controlled trial (RCT) of screening for GDM in primary versus secondary care was used to examine (i) prevalence, (ii) gestational week of screen, (iii) time to access specialist care, and (iv) maternal and neonatal outcomes. In total 781 women were recruited for screening in primary care (n=391) or secondary care (n=390). RESULTS: The prevalence of GDM and gestational week of screen were similar in both locations. There was a trend towards a longer time to access diabetes care in primary care (24days) versus secondary care (19days), a difference of 5days (p=0.09). Women screened in primary care also showed a trend towards a higher rate of large for gestational age (LGA) infants (20%) than those screened in secondary care (14.7%), (p=0.09). There were no differences between groups in maternal outcomes. CONCLUSIONS: This RCT suggests that screening for GDM in secondary care may be associated with potentially faster time to access specialist antenatal diabetes care and possibly lower LGA rates. Further research is needed to clarify these findings and to improve the delay in accessing specialist care requires an urgent focus. Further research is needed to test these findings in other health systems.

A Prepregnancy Care Program for Women With Diabetes: Effective and Cost Saving.

CONTEXT: Only a minority of women with diabetes attend prepregnancy care service and the economic effects of providing this service are unclear. OBJECTIVE: The objective of the study was to design, put into practice, and evaluate a regional prepregnancy care program for women with types 1 and 2 diabetes. DESIGN: This was a prospective cohort and cost-analysis study. SETTING: The study was conducted at antenatal centers along the Irish Atlantic Seaboard. PARTICIPANTS: Four hundred fourteen women with type 1 or 2 diabetes participated in the study. INTERVENTIONS: The intervention for the study was a newly developed prepregnancy care program. MAIN OUTCOME MEASURES: The program was assessed for its effect on the risk of adverse pregnancy outcomes. The difference between program delivery cost and the excess cost of treating adverse outcomes in nonattendees was evaluated. RESULTS: In total, 149 (36%) attended: this increased from 19% to 50% after increased recruitment measures in 2010. Attendees were more likely to take preconception folic acid (97.3% vs 57.7%, P < .001) and less likely to smoke (8.7% vs 16.6%, P = .03) or take potentially teratogenic medications at conception (0.7 vs 6.0, P = .008). Attendees had lower glycated hemoglobin levels throughout pregnancy (first trimester glycated hemoglobin 6.8% vs 7.7%, P < .001; third trimester glycated hemoglobin 6.1% vs 6.5%, P = .001), and their offspring had lower rates of serious adverse outcomes (2.4% vs 10.5%, P = .007). The adjusted difference in complication costs between those who received prepregnancy care vs usual antenatal care only is €2578.00. The average cost of prepregnancy care delivery is €449.00 per pregnancy. CONCLUSIONS: This regional prepregnancy care program is clinically effective. The cost of program delivery is less than the excess cost of managing adverse pregnancy outcomes.

The cost-effectiveness of screening for gestational diabetes mellitus in primary and secondary care in the Republic of Ireland.

AIMS/HYPOTHESIS: The aim of the study was to assess the cost-effectiveness of screening for gestational diabetes mellitus (GDM) in primary and secondary care settings, compared with a no-screening option, in the Republic of Ireland. METHODS: The analysis was based on a decision-tree model of alternative screening strategies in primary and secondary care settings. It synthesised data generated from a randomised controlled trial (screening uptake) and from the literature. Costs included those relating to GDM screening and treatment, and the care of adverse outcomes. Effects were assessed in terms of quality-adjusted life years (QALYs). The impact of the parameter uncertainty was assessed in a range of sensitivity analyses. RESULTS: Screening in either setting was found to be superior to no screening, i.e. it provided for QALY gains and cost savings. Screening in secondary care was found to be superior to screening in primary care, providing for modest QALY gains of 0.0006 and a saving of €21.43 per screened case. The conclusion held with high certainty across the range of ceiling ratios from zero to €100,000 per QALY and across a plausible range of input parameters. CONCLUSIONS/INTERPRETATION: The results of this study demonstrate that implementation of universal screening is cost-effective. This is an argument in favour of introducing a properly designed and funded national programme of screening for GDM, although affordability remains to be assessed. In the current environment, screening for GDM in secondary care settings appears to be the better solution in consideration of cost-effectiveness.

Health related quality of life two to five years after gestational diabetes mellitus: cross-sectional comparative study in the ATLANTIC DIP cohort.

BACKGROUND: There is no consensus on the effect of gestational diabetes mellitus (GDM) on health-related quality of life (HRQOL) for the mother in the short or long term. In this study we examined HRQOL in a group of women who had GDM in the index pregnancy 2 to 5 years previously and compared it to a group of women with normal glucose tolerance (NGT) in the index pregnancy during the same time period. METHODS: The sample included 234 women who met International Association of Diabetes Study Groups (IADPSG) criteria for GDM in the index pregnancy and 108 who had NGT. The sample was drawn from the ATLATIC-DIP (Diabetes In Pregnancy) cohort - a network of antenatal centers along the Irish Atlantic seaboard serving a population of approximately 500,000 people. HRQOL was measured using the visual analogue component of the EQ-5D-3 L instrument in a cross-sectional survey. RESULTS: The difference in HRQOL between GDM and NGT groups was not significant when adjusted for the effects of the covariates. HRQOL was negatively affected by increased BMI and abnormal glucose tolerance post-partum in the NGT group. Moderate alcohol consumption was positively associated with HRQOL in the NGT group only. The negative association with smoking on HRQOL was substantially higher in the GDM group. CONCLUSIONS: A diagnosis of GDM does not appear to have an adverse effect on HRQOL, 2 to 5 years after the index pregnancy. On the contrary, its diagnosis might lead to the development of coping strategies, which, consequently attenuates the adverse effect of the subsequent acquisition of abnormal glucose tolerance post-partum on HRQOL. Women whose pregnancy was affected by GDM are more susceptible to the adverse effects on HRQOL of alcohol use and tobacco smoking.

Feasibility, acceptability and uptake rates of gestational diabetes mellitus screening in primary care vs secondary care: findings from a randomised controlled mixed methods trial.

AIMS/HYPOTHESIS: It is postulated that uptake rates for gestational diabetes mellitus (GDM) screening would be improved if offered in a setting more accessible to the patient. The aim of this study was to evaluate the proportion of uptake of GDM screening in the primary vs secondary care setting, and to qualitatively explore the providers' experiences of primary care screening provision. METHODS: This mixed methods study was composed of a quantitative unblinded parallel group randomised controlled trial and qualitative interview trial. The primary outcome was the proportion of uptake of screening in both the primary and secondary care settings. All pregnant women aged 18 years or over, with sufficient English and without a diagnosis or diabetes or GDM, who attended for their first antenatal appointment at one of three hospital sites along the Irish Atlantic seaboard were eligible for inclusion in this study. Seven hundred and eighty-one pregnant women were randomised using random permutated blocks to receive a 2 h 75 g OGTT in either a primary (n = 391) or secondary care (n = 390) setting. Semi-structured interviews were conducted with 13 primary care providers. Primary care providers who provided care to the population covered by the three hospital sites involved were eligible for inclusion. RESULTS: Statistically significant differences were found between the primary care (n = 391) and secondary care (n = 390) arms for uptake (52.7% vs 89.2%, respectively; effect size 36.5 percentage points, 95% CI 30.7, 42.4; p < 0.001), crossover (32.5% vs 2.3%, respectively; p < 0.001) and non-uptake (14.8% vs 8.5%, respectively; p = 0.005). There were no significant differences in uptake based on the presence of a practice nurse or the presence of multiple general practitioners in the primary care setting. There was evidence of significant relationship between probability of uptake of screening and age (p < 0.001). Primary care providers reported difficulties with the conduct of GDM screening, despite recognising that the community was the most appropriate location for screening. CONCLUSIONS/INTERPRETATION: Currently, provision of GDM screening in primary care in Ireland, despite its acknowledged benefits, is unfeasible due to poor uptake rates, poor rates of primary care provider engagement and primary care provider concerns. TRIAL REGISTRATION: http://isrctn.org ISRCTN02232125 FUNDING: This study was funded by the Health Research Board (ICE2011/03).

Attitudes towards legalising physician provided euthanasia in Britain: the role of religion over time.

Hastening the death of another whether through assisted suicide or euthanasia is the subject of intense debate in the UK and elsewhere. In this paper we use a nationally representative survey of public attitudes - the British Social Attitudes survey - to examine changes in attitudes to the legalisation of physician provided euthanasia (PPE) over almost 30 years (1983-2012) and the role of religious beliefs and religiosity in attitudes over time. Compatible questions about attitudes to euthanasia were available in the six years of 1983, 1984, 1989, 1994, 2005, and 2012. We study the trends in the support for legalisation through these time points and the relationship between attitudes, religious denomination and religiosity, controlling for a series of covariates. In total, 8099 individuals provided answers to the question about PPE in the six years of the study. The support for legalisation rose from around 76.95% in 1983 to 83.86% in 2012. This coincided with an increase in secularisation exhibited in the survey: the percentage of people with no religious affiliation increasing from 31% to 45.4% and those who do not attend a religious institution (e.g. church) increasing from 55.7% to 65.03%. The multivariate analysis demonstrates that religious affiliation and religiosity as measured by religious institution attendance frequency are the main contributors to attitudes towards euthanasia, and that the main increase in support happened among the group with least religious affiliation. Other socio-demographic characteristics do not seem to alter these attitudes systematically across the years. Our study demonstrates an increase in the support of euthanasia legalisation in Britain in the last 30 years coincided with increased secularisation. It does not follow, however, that trends in public support are immutable nor that a change in the law would improve on the current pragmatic approach toward hastening death by a physician adopted in England and Wales in terms of the balance between compassion and safeguards against abuse offered.

Preferences for physician services in Ukraine: a discrete choice experiment.

Evidence on preferences of Ukrainian consumers for healthcare improvements can help to design reforms that correspond to societal priorities. This study aims to elicit and to place monetary values on public preferences for out-patient physician services in Ukraine. The method of discrete choice experiment is used on a sample of 303 respondents, representative of the adult Ukrainian population. The random effect logit model with interactions provides the best fit for the data and is used to calculate the marginal willingness to pay (MWTP) for quality and access improvements. At a sample level, there is no clear preference to pay formally rather than informally or vice versa. We also do not find that visiting a general practitioner is preferred over direct access to a medical specialist. However, there are differences between population groups. Quality-related attributes of physician services appear important to respondents, especially the attitude of medical staff. Thus, interpersonal aspects of out-patient care should be given priority in decisions about investments in quality improvements. Other aspects, that is social quality and access, are important as well but their improvement brings fewer social gains. Measures should be taken to eradicate the informal payment channels and to strengthen the gate-keeping role of primary care.

Willingness and ability to pay for physician services in six Central and Eastern European countries.

Patient charges for physician services are implemented in high-income countries and often are considered in the Central and Eastern Europe (CEE). However, there is no evidence on the potential consumption effects of service charges in these countries. This study provides evidence on the potential impact of patient charges on the consumption of specialized physician services in six CEE countries: Bulgaria, Hungary, Lithuania, Poland, Romania, and Ukraine. We apply a semi-parametric survival analysis to stated willingness and ability to pay (WATP) in order to identify potential demand pools and their price, income and age semi-elasticity. Data are collected through a survey held in 2010 among representative samples of about 1000 respondents in each country. Our results suggest that median WATP in the studied countries is comparable to the cost of the services. The obtained demand pools appear to be theoretically valid and externally consistent. They provide information on the shares of population that would be WATP certain fee levels, and their heterogeneity across socio-demographic groups gives an idea about the population groups that will need to be exempted.

Willingness to pay for physician services at a primary contact in Ukraine: results of a contingent valuation study.

BACKGROUND: The existence of quasi-formal and informal payments in the Ukrainian health care system jeopardizes equity and creates barriers to access to proper care. Patient payment policies that better match patient preferences are necessary. We analyze the potential and feasibility of official patient charges for public health care services in Ukraine by studying the patterns of fee acceptability, ability and willingness to pay (WTP) for public health care among population groups. METHODS: We use contingent valuation data collected from 303 respondents representative of the adult Ukrainian population. Three decision points were separated: objection to pay, inability to pay, and level of positive non-zero WTP. These decisions were studied for relations with quality profiles of the services, and socio-demographic characteristics of the respondents and their households. RESULTS: The likelihood to object to pay is mostly determined by the quality characteristics of the services. Objection to pay is not related to corresponding behavior in real life. The likelihood of being unable to pay is associated with older age, lower income, and a larger share of household members with no income. The level of positive WTP is positively related to income (+7% per 1000 UAH increase in income) and is lower for people who visited a doctor but did not pay (-22%). CONCLUSIONS: Rather substantial WTP levels (between 0.9% and 1.9% of household income) for one visit to physician indicate a potential for official patient charges in Ukraine. User fees may cover a substantial share of personnel cost in the out-patient sector. The patterns of inability to pay support well designed exemption criteria based on age, income, and other aspects of economic status. The WTP patterns highlight the necessity for payments that are proportional to income. Other methodological and policy implications are discussed.