Participatory methods used in the evaluation of medical devices: a comparison of focus groups, interviews, and a survey.

BACKGROUND: Stakeholder engagement in evaluation of medical devices is crucial for aligning devices with stakeholders' views, needs, and values. Methods for these engagements have however not been compared to analyse their relative merits for medical device evaluation. Therefore, we systematically compared these three methods in terms of themes, interaction, and time-investment. METHODS: We compared focus groups, interviews, and an online survey in a case-study on minimally invasive endoscopy-guided surgery for patients with intracerebral haemorrhage. The focus groups and interviews featured two rounds, one explorative focussing on individual perspectives, and one interactive focussing on the exchange of perspectives between participants. The comparison between methods was made in terms of number and content of themes, how participants interact, and hours invested by all researchers. RESULTS: The focus groups generated 34 themes, the interviews 58, and the survey 42. Various improvements for the assessment of the surgical procedure were only discussed in the interviews. In focus groups, participants were inclined to emphasise agreement and support, whereas the interviews consisted of questions and answers. The total time investment for researchers of focus groups was 95 h, of interviews 315 h, and survey 81 h. CONCLUSIONS: Within the context of medical device evaluation, interviews appeared to be the most appropriate method for understanding stakeholder views since they provide a scope and depth of information that is not generated by other methods. Focus groups were useful to rapidly bring views together. Surveys enabled a quick exploration. Researchers should account for these methodological differences and select the method that is suitable for their research aim.

Dealing With Uncertainty in Early Health Technology Assessment: An Exploration of Methods for Decision Making Under Deep Uncertainty.

OBJECTIVES: In early stages, the consequences of innovations are often unknown or deeply uncertain, which complicates early health economic modeling (EHEM). The field of decision making under deep uncertainty uses exploratory modeling (EM) in situations when the system model, input probabilities/distributions, and consequences are unknown or debated. Our aim was to evaluate the use of EM for early evaluation of health technologies. METHODS: We applied EM and EHEM to an early evaluation of minimally invasive endoscopy-guided surgery (MIS) for acute intracerebral hemorrhage and compared these models to derive differences, merits, and drawbacks of EM. RESULTS: EHEM and EM differ fundamentally in how uncertainty is handled. Where in EHEM the focus is on the value of technology, while accounting for the uncertainty, EM focuses on the uncertainty. EM aims to find robust strategies, which give relatively good outcomes over a wide range of plausible futures. This was reflected in our case study. EHEM provided cost-effectiveness thresholds for MIS effectiveness, assuming fixed MIS costs. EM showed that a policy with a population in which most patients had severe intracerebral hemorrhage was most robust, regardless of MIS effectiveness, complications, and costs. CONCLUSIONS: EHEM and EM were found to complement each other. EM seems most suited in the very early phases of innovation to explore existing uncertainty and many potential strategies. EHEM seems most useful to optimize promising strategies, yet EM methods are complex and might only add value when stakeholders are willing to consider multiple solutions to a problem and adopt flexible research and adoption strategies.

Identifying the Conditions for Cost-Effective Minimally Invasive Neurosurgery in Spontaneous Supratentorial Intracerebral Hemorrhage.

Background: In patients with spontaneous supratentorial intracerebral hemorrhage (ICH), open craniotomy has failed to improve a functional outcome. Innovative minimally invasive neurosurgery (MIS) may improve a health outcome and reduce healthcare costs. Aims: Before starting phase-III trials, we aim to assess conditions that need to be met to reach the potential cost-effectiveness of MIS compared to usual care in patients with spontaneous supratentorial ICH. Methods: We used a state-transition model to determine at what effectiveness and cost MIS would become cost-effective compared to usual care in terms of quality-adjusted life-years (QALYs) and direct healthcare costs. Threshold and two-way sensitivity analyses were used to determine the minimal effectiveness and maximal costs of MIS, and the most cost-effective strategy for each combination of cost and effectiveness. Scenario and probabilistic sensitivity analyses addressed model uncertainty. Results: Given €10,000 of surgical costs, MIS would become cost-effective when at least 0.7-1.3% of patients improve to a modified Rankin Scale (mRS) score of 0-3 compared to usual care. When 11% of patients improve to mRS 0-3, surgical costs may be up to €83,301-€164,382, depending on the population studied. The cost-effectiveness of MIS was mainly determined by its effectiveness. In lower mRS states, MIS needs to be more effective to be cost-effective compared to higher mRS states. Conclusion: MIS has the potential to be cost-effective in patients with spontaneous supratentorial ICH, even with relatively low effectiveness. These results support phase-III trials to investigate the effectiveness of MIS.

Global Outcome Assessment Life-long after stroke in young adults initiative-the GOAL initiative: study protocol and rationale of a multicentre retrospective individual patient data meta-analysis.

INTRODUCTION: Worldwide, 2 million patients aged 18-50 years suffer a stroke each year, and this number is increasing. Knowledge about global distribution of risk factors and aetiologies, and information about prognosis and optimal secondary prevention in young stroke patients are limited. This limits evidence-based treatment and hampers the provision of appropriate information regarding the causes of stroke, risk factors and prognosis of young stroke patients. METHODS AND ANALYSIS: The Global Outcome Assessment Life-long after stroke in young adults (GOAL) initiative aims to perform a global individual patient data meta-analysis with existing data from young stroke cohorts worldwide. All patients aged 18-50 years with ischaemic stroke or intracerebral haemorrhage will be included. Outcomes will be the distribution of stroke aetiology and (vascular) risk factors, functional outcome after stroke, risk of recurrent vascular events and death and finally the use of secondary prevention. Subgroup analyses will be made based on age, gender, aetiology, ethnicity and climate of residence. ETHICS AND DISSEMINATION: Ethical approval for the GOAL study has already been obtained from the Medical Review Ethics Committee region Arnhem-Nijmegen. Additionally and when necessary, approval will also be obtained from national or local institutional review boards in the participating centres. When needed, a standardised data transfer agreement will be provided for participating centres. We plan dissemination of our results in peer-reviewed international scientific journals and through conference presentations. We expect that the results of this unique study will lead to better understanding of worldwide differences in risk factors, causes and outcome of young stroke patients.

Ethnic disparities in ischemic stroke, intracerebral hemorrhage, and subarachnoid hemorrhage incidence in the Netherlands.

BACKGROUND AND PURPOSE: Data on the incidence of stroke subtypes among ethnic minority groups are limited. We assessed ethnic differences in the incidence of stroke subtypes in the Netherlands. METHODS: A Dutch nationwide register-based cohort study (n=7 423 174) was conducted between 1998 and 2010. We studied the following stroke subtypes: ischemic stroke, intracerebral hemorrhage, and subarachnoid hemorrhage. Cox proportional hazard models were used to estimate incidence differences between first-generation ethnic minorities and the Dutch majority population (ethnic Dutch). RESULTS: Compared with ethnic Dutch, Surinamese men and women had higher incidence rates of all stroke subtypes combined (adjusted hazard ratios, 1.43; 95% confidence interval, 1.35-1.50 and 1.34; 1.28-1.41), ischemic stroke (1.68; 1.57-1.81 and 1.57; 1.46-1.68), intracerebral hemorrhage (2.08; 1.82-2.39 and 1.74; 1.50-2.00), and subarachnoid hemorrhage (1.25; 0.92-1.69 and 1.26; 1.04-1.54). By contrast, Moroccan men and women had lower incidence rates of all stroke subtypes combined (0.42; 0.36-0.48 and 0.37; 0.30-0.46), ischemic stroke (0.35; 0.27-0.45 and 0.34; 0.24-0.49), intracerebral hemorrhage (0.61; 0.41-0.92 and 0.32; 0.16-0.72), and subarachnoid hemorrhage (0.42; 0.20-0.88 and 0.34; 0.17-0.68) compared with ethnic Dutch counterparts. The results varied by stroke subtype and sex for the other minority groups. For example, Turkish women had a reduced incidence of subarachnoid hemorrhage, whereas Turkish men had an increased incidence of ischemic stroke and intracerebral hemorrhage compared with ethnic Dutch. CONCLUSIONS: Our findings suggest that Surinamese have an increased risk, whereas Moroccans have a reduced risk for all the various stroke subtypes. Among other ethnic minorities, the risk seems to depend on the stroke subtype and sex. These findings underscore the need to identify the root causes of these ethnic differences to assist primary and secondary prevention efforts.

Socioeconomic inequalities in stroke incidence among migrant groups: analysis of nationwide data.

BACKGROUND AND PURPOSE: Low socioeconomic status has been linked to high incidence of stroke in industrialized countries; therefore, reducing socioeconomic disparities is an important goal of health policy. The evidence on migrant groups is, however, limited and inconsistent. We assessed socioeconomic inequalities in relation to stroke incidence among major ethnic groups in the Netherlands. METHODS: A nationwide register-based cohort study was conducted (n=2 397 446) between January 1, 1998, and December 31, 2010, among ethnic Dutch and ethnic minority groups. Standardized disposable household income was used as a measure of socioeconomic position. RESULTS: Among ethnic Dutch, the incidence of stroke was higher in the low-income group than in the high-income group (adjusted hazard ratio, 1.18; 95% confidence interval, 1.16-1.20). Similar socioeconomic inequalities in stroke incidence were found among Surinamese (1.36; 1.17-1.58), Indonesians (1.15; 1.03-1.28), Moroccans (1.54; 0.97-2.43), Turkish (1.19; 0.97-1.46), and to a lesser extent among Antilleans (1.24; 0.84-1.84). When compared with ethnic Dutch, the incidence of stroke was lower in Moroccans, similar in Turkish, but higher in Surinamese among all income groups. The incidence of stroke was higher in Indonesian low- and high-income groups than in their ethnic Dutch counterparts. Among Antilleans, the risk of stroke was higher than ethnic Dutch but only in the low-income group. CONCLUSIONS: Our findings reveal socioeconomic inequalities in stroke incidence among all ethnic groups. Reduction of socioeconomic inequalities in stroke incidence among all ethnic groups may lead to a major public health improvement for all. Policy measures tackling socioeconomic inequalities should take into account the increased risk of stroke among ethnic minority populations.

Comparison of telephone and face-to-face assessment of the modified Rankin Scale.

BACKGROUND: A structured interview improves the reliability of the modified Rankin Scale (mRS), a commonly used functional outcome scale in stroke trials. Telephone interview is a fast and convenient way to assess the mRS grade, but its validity is unknown. We assessed the validity of a telephone interview in patients who had had an aneurysmal subarachnoid haemorrhage (SAH) by comparing it with a face-to-face assessment. METHODS: Eighty-three SAH patients were interviewed twice, once face-to-face and once by telephone, by 2 of 5 observers who used a structured interview to assess the mRS grade. Intermodality agreement was measured using weighted kappa statistics. To check for systematic differences between face-to-face and telephone assessment the Wilcoxon test for matched pairs was used. RESULTS: Agreement between telephone and face-to-face assessment was perfect in 47 (57%) patients. A difference of 1 level occurred in 31 (37%) patients and this was almost equally distributed over the grades of the mRS. Weighted kappa was 0.71 (95% CI 0.59-0.82). Telephone assessment did not result in a consistently more or less favourable grade than face-to-face assessment (Wilcoxon test for matched pairs, p = 0.33). CONCLUSIONS: Telephone assessment of the mRS with a structured interview has a good agreement with face-to-face assessment and can thus be used reliably in the setting of a clinical trial.

Assessment of the contribution of the external carotid artery to brain perfusion in patients with internal carotid artery occlusion.

BACKGROUND AND PURPOSE: The purpose of this study was to prospectively investigate the contribution of the ipsilateral external carotid artery (ECA) to cerebral perfusion in patients with internal carotid artery occlusion. METHODS: Institutional Review Board approval and informed consent were obtained. Thirty functionally independent patients (24 men, 6 women; mean age, 63 years) with an angiographically proven unilateral internal carotid artery occlusion and transient or minor disabling ischemic attacks ipsilateral to the side of the internal carotid artery occlusion were included. Grading of ECA collateral flow was performed with intraarterial digital subtraction angiography. The contribution of the ECA to regional cerebral blood flow was assessed with selective arterial spin labeling MRI. Differences in regional cerebral blood flow were analyzed with Student t test. RESULTS: Twenty percent of the patients had ECA Grade 0 collateral flow (no filling of ophthalmic artery), 20% Grade 1 (filling of carotid siphon), and 60% Grade 2 (filling of anterior and/or middle cerebral artery) as demonstrated on digital subtraction angiography. Although in the Grade 1 group, the ECA supplied a smaller region of the brain compared with the Grade 2 group, the mean regional cerebral blood flow of the perfusion territory supplied by the ECA is similar (P=0.70) in the Grade 1 group (mean+/-SD 57+/-16 mL/min/100 g) and the Grade 2 group (60+/-12 mL/min/100g). CONCLUSIONS: In patients with symptomatic internal carotid artery occlusion, focal brain regions may strongly depend on the contribution to cerebral perfusion of the ECA ipsilateral to the side of the internal carotid artery occlusion, even in patients with limited ECA collateral supply as demonstrated on digital subtraction angiography.