Michela Tinelli

Shedding new light onto the ceiling and floor? A quantile regression approach to compare EQ-5D and SF-6D responses

An important issue in the measurement of health status concerns the extent to which an instrument displays lack of sensitivity to changes in health status at the extremes of the distribution, known as floor and ceiling effects. Previous studies use relatively simple methods that focus on the mean of the distribution to examine these effects. The aim of this paper is to determine whether quantile regression using longitudinal data improves our understanding of the relationship between quality of life instruments. The study uses EQ-5D and SF-36 (converted to SF-6D values) instruments with both baseline and follow-up data. Relative to ordinary least least-squares (OLS), a first difference model shows much lower association between the measures, suggesting that OLS methods may lead to biased estimates of the association, due to unobservable patient characteristics. The novel finding, revealed by quantile regression, is that the strength of association between the instruments is different across different parts of the health distribution, and is dependent on whether health improves or deteriorates. The results suggest that choosing one instrument at the expense of another is difficult without good prior information surrounding the expected magnitude and direction of health improvement related to a health-care intervention.

What determines patient preferences for treating low risk basal cell carcinoma when comparing surgery vs imiquimod?: a discrete choice experiment survey from the SINS trial

BackgroundThe SINS trial (Controlled Clinical Trials ISRCTN48755084; Eudract No. 2004-004506-24) is a randomised controlled trial evaluating long term success of excisional surgery vs. imiquimod 5% cream for low risk nodular and superficial basal cell carcinoma (BCC). The trial included a discrete choice experiment questionnaire to explore patient preferences of a cream versus surgery for the treatment of their skin cancer.MethodsThe self-completed questionnaire was administered at baseline to 183 participants, measuring patients’ strength of preferences when choosing either alternative ‘surgery’ or ‘imiquimod cream’ instead of a fixed ‘current situation’ option (of surgical excision as standard practice in UK). The treatments were described according to: cost, chance of complete clearance, side effects and appearance. Participants had to choose between various scenarios. Analysis was performed using a mixed logit model, which took into account the impact of previous BCC treatment and sample preference variability.ResultsThe analysis showed that respondents preferred ‘imiquimod cream’ to their ‘current situation’ or ‘surgery’, regardless of previous experience of BCC symptoms and treatment. Respondents were more likely to be worried about their cosmetic outcomes and side effects they might experience over and above their chance of clearance and cost. Those with no experience of surgery (compared with experience) valued more the choice of ‘imiquimod cream’ (£1013 vs £781). All treatment characteristics were significant determinants of treatment choice, and there was significant variability in the population preferences for all of them.ConclusionsPatients with BCC valued more ‘imiquimod cream’ than alternative ‘surgery’ options, and all treatment characteristics were important for their choice of care. Understanding how people with a BCC value alternative interventions may better inform the development of health care interventions.

Development, validation and application of a patient satisfaction scale for a community pharmacy medicines-management service.

Objective  To develop, validate and apply a scale to measure patient satisfaction in a randomised controlled trial of community pharmacy service.

Costs of a community pharmacist-led medicines management service for patients with coronary heart disease in England : Healthcare system and patient perspectives

BackgroundCoronary heart disease (CHD) is the most common cause of death in the UK. CHD cost the UK National Health Service (NHS) £3.5 billion in 2003. The economic impact of community pharmacists providing a medicines management service for patients with CHD has not been rigorously evaluated; the full economic costs of such interventions are rarely presented in the literature.ObjectiveTo examine the incremental costs of a 1-year community pharmacistled medicines management service for patients with CHD in the UK, from a healthcare system and patient perspective.Methods and participantsA cost-minimisation analysis was conducted alongside a multicentre randomised controlled trial. The primary study participants were patients with CHD identified from general practice computer records. Patients (intervention, n = 980; control, n = 500) from 38 general practices in nine geographical areas in the UK were included in the study.Intervention and outcomes measuresThe intervention consisted of a review of pharmaceuticals and lifestyle advice by pharmacists in their premises, with recommendations communicated to the patient’s GP. The main outcome measure was the incremental cost per patient in the intervention group compared with the control group. Annual costs (£, 2003/4 values) included the costs of the intervention (training and delivery costs), the usual costs of NHS treatment (costs of pharmaceuticals, GP and hospital visits) and costs borne by patients. Data were collected in the 12 months before and 12 months after the intervention.ResultsThe total NHS cost increased between baseline and follow-up in both groups (from £1243 to £1286 [3%] in the control group and from £1410 to £1433 [2%] in the intervention group).The greater cost in the intervention group largely reflects the additional cost of the pharmacist training and the time taken to deliver the intervention; the difference in costs between the intervention and control groups, after controlling for differences in costs at baseline at follow-up, was statistically significant (p = 0.001). The costs of pharmaceuticals was higher in the intervention group (£769.20 vs £742.3; p = 0.04).According to the sensitivity analysis, the intervention cost would need to decrease by 35% to achieve equivalence between costs in each arm of the trial. Difference to costs of patients and their carers at follow-up were not statistically significant.ConclusionsThe introduction of a 1-year pharmacist-led medicines management service is likely to increase the total cost of CHD treatment and prevention from the healthcare perspective, as the cost of the intervention outweighed the observed reduction in the cost of drugs in the intervention group. No changes in costs from the patient perspective were found.

Valuing Benefits to Inform a Clinical Trial in Pharmacy

BackgroundThe generic health-related quality-of-life (HR-QOL) utility measures the EQ-5D and SF-6D are both commonly used to inform healthcare policy developments. However, their application to pharmacy practice is limited and the optimal method to inform policy developments is unknown.ObjectivesOur objective was to test the sensitivity of the EQ-5D and SF-6D within pharmacy when measuring whether changes in health status or other co-variates at baseline affect the effectiveness of the intervention at follow-up. A further objective was to consider the implications of the findings for pharmacy research and policy.MethodsThe EQ-5D and SF-6D utility measures were employed within a randomized controlled trial (RCT) of community pharmacy-led medicines management for patients with coronary heart disease. The intervention covered a baseline visit with the potential for follow-up. Simultaneous quantile regression assessed the impact of the intervention on both EQ-5D and SF-6D measures at follow-up, controlling for baseline health, appropriateness of treatment, personal characteristics and self-reported satisfaction.ResultsNo statistically significant difference in HR-QOL across the intervention and control groups at follow-up was reported for either measure. Increased health gain was however associated with the baseline utility score (with the EQ-5D more sensitive for those in worse health) and the appropriateness of treatment, but not patient characteristics or self-reported satisfaction.ConclusionNeither generic measure detected a gain in HR-QOL as a result of the introduction of an innovative pharmacy-based service. This finding supports other work in the area of pharmacy, where health gains have not changed following interventions. Disease-specific utility measures should be investigated as an alternative to generic approaches such as the EQ-5D and SF-6D. Given that the RCT found an increase in self-reported satisfaction, broader measures of benefit that value patient experiences, such as contingent valuation and discrete-choice experiments, should also be considered in pharmacy.

A cluster randomised control trial to evaluate the effectiveness and cost-effectiveness of the Italian medicines use review (I-MUR) for asthma patients

BackgroundThe economic burden of asthma, which relates to the degree of control, is €5 billion annually in Italy. Pharmacists could help improve asthma control, reducing this burden. This study aimed to evaluate the effectiveness and cost-effectiveness of Medicines Use Reviews provided by community pharmacists in asthma.MethodsThis cluster randomised, multi-centre, controlled trial in adult patients with asthma was conducted in 15 of the 20 regions of Italy between September 2014 and July 2015. After stratification by region, community pharmacists were randomly allocated to group A (trained in and delivered the intervention at baseline) or B (training and delivery 3 months later), using computerised random number generation in blocks of 10. Each recruited up to five patients, with both groups followed for 9 months.The intervention consisted of a systematic, structured face-to-face consultation with a pharmacist, covering asthma symptoms, medicines used, attitude towards medicines and adherence, recording pharmacist-identified pharmaceutical care issues (PCIs). The primary outcome was asthma control, assessed using the Asthma-Control-Test (ACT) score (ACT ≥ 20 represents good control). Secondary outcomes were: number of active ingredients, adherence, cost-effectiveness compared with usual care. Although blinding was not possible for either pharmacists or patients, assessment of outcomes was conducted by researchers blind to group allocation.ResultsNumbers of pharmacists and patients enrolled were 283 (A = 136; B = 147) and 1263 (A = 600; B = 663), numbers completing were 201 (A = 97; B = 104) and 816 (A = 400; B = 416), respectively. Patients were similar in age and gender and 56.13% (458/816) had poor/partial asthma control. Pharmacists identified 1256 PCIs (mean 1.54/patient), mostly need for education, monitoring and potentially ineffective therapy. Median ACT score at baseline differed between groups (A = 19, B = 18; p < 0.01). Odds ratio for improved asthma control was 1.76 (95% CI 1.33–2.33) and number needed to treat 10 (95% CI 6–28). Number of active ingredients reduced by 7.9% post-intervention (p < 0.01). Adherence improved by 35.4% 3 months post-intervention and 40.0% at 6 months (p < 0.01). The probability of the intervention being more cost-effective than usual care was 100% at 9 months.ConclusionsThis community pharmacist-based intervention demonstrated both effectiveness and cost-effectiveness. It has since been implemented as the first community pharmacy cognitive service in Italy.Trial registrationTRN: ISRCTN72438848 (registered 5th January 2015, retrospectively).

Value of treatment of headache patients and need to improve headache patients’ journey

The European Brain Council recently unveiled the report BThe Value of Treatment for Brain Disorders,^which highlights the need for more investment into research on neurological and mental diseases and the wide disparities between and within countries relating to treatments, detection, and intervention. More than 165 million Europeans are living with brain disorders such as epilepsy, headache, Alzheimer’s disease, depression, and multiple sclerosis; the burden on national health budgets is staggering—rising to more than 800 billion euro a year in direct and indirect costs such as lost earnings and lost tax revenues. Governments and politicians concerned over the cost of headache care for very large numbers of people fail to recognize a fundamentally important aspect of the economics of headache disorders: untreated, they are a huge financial drain. The high disability leads to massive losses in productivity. The costs of treating headache may be high, but would be dwarfed by the savings made from recovered lost work time if the resources were allocated to treat headache disorders appropriately [1, 2]. For this reason, implementation of good headache healthcare is likely to be cost-saving. Our first priority to improve patients’ care should be to demonstrate the value of treating headache in these terms, so that policy will recognize the benefits of addressing these healthcare and educational failures. The Bcare pathway^ for most European people with headache is a series of dead ends. Many who would benefit from professional care find it unavailable, fragmentary, or difficult to access. Where headache services exist, they tend to be focused in specialist headache clinics, delivering high-end multidisciplinary care with very limited capacity. Such clinics are needed by the minority with complex disorders, but cannot serve this purpose when inundated by people who could be effectively treated in primary care—as most people needing headache care require neither specialist expertise nor investigations. Contrariwise, one in every three people receiving care for migraine in Russia and Spain, and one in every four in Luxemburg, does so from specialists [3]. Lack of knowledge among healthcare providers is a problem sewn in medical schools: worldwide, only four hours are committed to headache disorders in formal undergraduate medical training lasting 4–6 years. Poor awareness of headache disorders exists similarly among the general public: headache disorders are not perceived by the public as serious as they do not cause death and are not contagious. Consequently, headaches are often trivialized as Bnormal^ and seen in those who complain of them as merely an excuse to avoid responsibility. On a political level, many governments, even if aware of it, do not acknowledge the substantial burden of headache on society. Healthcare for headache obviously comes at a cost: large numbers of people need treatments, together with advices on correct usage, and delivery of these requires organized healthcare. Good healthcare can greatly reduce the burden of headache, but it persists—principally because healthcare systems that should provide this care do not exist or fail to reach many who need it [3]. The roots of this failure mostly lie in education failure, at every level, but * M. Leonardi matilde.leonardi@istituto–besta.it

Using IMPrESS to guide policy change in multiple sclerosis

The International MultiPlE Sclerosis Study (IMPrESS) studied the significant impact of multiple sclerosis (MS) on the health and well-being of both people with the disease and their caregivers, along with its broader socioeconomic impact. Results confirmed that there is an urgent need to achieve better outcomes for people with MS. This paper uses results from the IMPrESS to present new international evidence on the socioeconomic burden of MS and discuss the merits of a likely paradigm shift in the management of MS towards the use of better (and more accurate) diagnostic follow-up to monitor disease progression and the earlier use of disease-modifying treatments (DMTs) to achieve better clinical, quality-of-life and socioeconomic results for individuals.

Value assessment of disease-modifying therapies for Relapsing-Remitting Multiple Sclerosis: HTA evidence from seven OECD countries

This study systematically compares HTA recommendations on a number of disease-modifying therapies for patients with Relapsing-Remitting Multiple Sclerosis. We analysed publicly available HTA reports for nine medicine-indication pairs across seven OECD countries using a methodological framework enabling systematic analysis of HTA recommendations. The analysis was conducted based on a number of value dimensions, including clinical and economic variables, as well as several other dimensions of value beyond cost-effectiveness. The material was qualitatively and quantitatively coded following the different stages of HTA decision-making process. Fifty-seven medicine-indication pairs were assessed across the study countries. Of those, eight medicine indication-pairs reported diverging HTA recommendations. Although HTA recommendations were based on the same evidence submitted in most cases, significant variations were identified in interpretation and acceptance of evidence resulting in different uncertainties raised and different ways of addressing them. Uncertainties arose both in terms of the clinical and the economic evidence, including the design of key trials or the data quality in economic models. Beyond costs and effects, additional dimensions of value had an impact in the direction of recommendations, however with different magnitude across countries. We show that there is heterogeneity across countries in HTA for evaluating DMTs for RRMS with a lack of standardised methods in evaluating clinical and economic evidence and the use of social value judgments to inform decision-making.

Novel pharmacist-led intervention secures the minimally important difference (MID) in Asthma Control Test (ACT) score: better outcomes for patients and the healthcare provider

Introduction A key priority in asthma management is achieving control. The Asthma Control Test (ACT) is a validated tool showing a numerical indicator which has the potential to provide a target to drive management. A novel pharmacist-led intervention recently evaluated and introduced in the Italian setting with a cluster randomised controlled trial (C-RCT) showed effectiveness and cost-effectiveness. This paper evaluates whether the intervention is successful in securing the minimally important difference (MID) in the ACT score and provides better health outcomes and economic savings. Methods Clinical data were sourced from 816 adult patients with asthma participating in the C-RCT. The success of the intervention was measured looking at the proportion of patients reaching MID in the ACT score. Different levels of asthma control were grouped according to international guidelines and graded using the traffic light rating system. Asthma control levels were linked to economic (National Health Service (NHS) costs) and quality-adjusted life years outcomes using published data. Results The median ACT score was 19 (partially controlled) at baseline, and 20 and 21 (controlled) at 3-month and 6-month-follow up, respectively (p<0.01). The percentage of patients reaching MID at 3 and 6 months was 15.8% (129) and 19.9% (162), respectively. The overall annual NHS cost savings per 1000 patients attached to the shift towards the MID target were equal to €346 012 at 3 months and increased to €425 483 at 6 months. Health utility gains were equal to 35.42 and 45.12 years in full health gained, respectively. Discussion The pharmacist-led intervention secured the MID in the ACT score and provided better outcomes for both patients and providers.