A review of the methods used to generate utility values in NICE technology assessments for children and adolescents

Objective: This review summarises and critically examines methods used to generate utilities for child and adolescent health states in previous NICE (National Institute for Health and Care Excellence) technology assessments. Methods: We identified all NICE Technology Appraisal (TA) and NICE Highly Specialised Technologies (HST) guidance where the licensed indication for the technology included people under 18 years. Results: The review includes 40 NICE technology assessments. Most assessments generated utility values using EQ-5D scored using the adult version of the EQ-5D either exclusively (n=16) or alongside other utility measures and direct elicitation methods of patient own utility (n=17) and seven did not use the EQ-5D. Ten assessments used both adult and children population-specific measures: Health Utilities Index Mark 2 (n=6); a children and adolescents specific preference-based measure for atopic dermatitis (n=1); the youth version of the EQ-5D (EQ-5D-Y) generated using mapping and valued using the UK EQ-5D adult tariff (n=2), and the EQ-5D-Y using the EQ-5D adult tariff (n=1). The cost-effectiveness analyses used age adjustment (utility subtractions, weights and published mapping formulae) from adult EQ-5D UK population norms to reflect general population or disease-free health for children and adolescents (n=9), and one assessment assumed full health (utility value of 1). Conclusion: The review found very limited use of child and adolescent population-specific measures to generate health state utility values for children and adolescents in technology assessments submitted to NICE. Often they use an adult specific measure to reflect the health of children

Estimating a Dutch value set for the paediatric preference-based CHU-9D using a discrete choice experiment with duration

Objective: This paper presents the development of the Dutch value set for the CHU-9D, a paediatric preference-based measure of quality of life that can be used to generate quality adjusted life years (QALYs). Methods: A large online survey was conducted using a discrete choice experiment (DCE) including a duration attribute with adult members of the Netherlands general population (n=1,276) who were representative in terms of age, gender, marital status, employment, education and region. Respondents were asked which of two health states they prefer, where each health state was described using the nine dimensions of the CHU-9D (worried, sad, pain, tired, annoyed, school work/homework, sleep, daily routine, able to join in activities) and duration. The data was modelled using conditional logit with robust standard errors to produce utility values for every health state described by the CHU-9D. Results: The majority of the dimension level coefficients were monotonic, leading to a decrease in utility as severity increases. However there was evidence of some logical inconsistencies particularly for the school work/homework dimension. The value set produced was based on the ordered model and ranges from -0.568 for the worst state to 1 for the best state. Conclusion: The valuation of the CHU-9D using online DCE with duration with adult members of the Dutch general population was feasible and produced a valid model for use in cost utility analysis. Normative questions are raised around the valuation of paediatric preference-based measures including the appropriate perspective for imagining hypothetical paediatric health states

Health status of adults with Short Stature: A comparison with the normal population and one well-known chronic disease (Rheumatoid Arthritis)

BACKGROUND: To examine the subjective health status of adults with short stature (ShSt) and compare with the general population (GP) and one well-known chronic disease, rheumatoid artritis (RA). In addition, to explore the association between age, gender, height, educational level and different aspects of health status of adults with short stature. METHODS: A questionnaire was mailed to 72 subjects with short stature registered in the database of a Norwegian resource centre for rare disorders, response rate 61% (n = 44, age 16–61). Health status was assessed with SF-36 version 2. Comparison was done with age and gender matched samples from the general population in Norway (n = 264) and from subjects with RA (n = 88). RESULTS: The ShSt sample reported statistically significant impaired health status in all SF-36 subscales compared with the GP sample, most in the physical functioning, Mean Difference (MD) 34 (95% Confidence Interval (CI) 25–44). The ShSt reported poorer health status in mental health, MD 11 (95% CI 4–18) and social functioning, MD 11 (95% CI 2–20) but better in role physical MD 13 (95% CI 1–25) than the RA sample. On the other subscales there were minor difference between the ShSt and the RA sample. Within the short stature group there was a significant association between age and all SF-36 physical subcales, height was significantly associated with physical functioning while level of education was significantly associated with mental health. CONCLUSION: People with short stature reported impaired health status in all SF-36 subscales indicating that they have health problems that influence their daily living. Health status seems to decline with increasing age, and earlier than in the general population

Health-related quality of life of child and adolescent retinoblastoma survivors in the Netherlands

To assess health-related quality of life (HRQoL) in children (8-11 years) and adolescents (12-18 years) who survived retinoblastoma (RB), by means of the KIDSCREEN self-report questionnaire and the proxy-report version. This population-based cross-sectional study (participation rate 70%) involved 65 RB survivors (8-18 years) and their parents. Child/adolescents' and parents' perception of their youth's HRQoL was assessed using the KIDSCREEN, and the results were compared with Dutch reference data. Relations with gender, age, marital status of the parents, and visual acuity were analyzed. RB survivors reported better HRQoL than did the Dutch reference group on the dimensions "moods and emotions" and "autonomy". Increased ratings of HRQoL in RB survivors were mainly seen in perceptions of the younger children and adolescent girls. RB survivors with normal visual acuity scored higher on "physical well-being" than visually impaired survivors. Age was negatively associated with the dimensions "psychological well-being", "self-perception" (according to the child and parent reports) and "parent relations and home life" (according to the child). "Self-perception" was also negatively associated with visual acuity (according to the child). Only parents of young boys surviving RB reported lower on "autonomy" than the reference group, and parents of low visual acuity and blind RB survivors reported higher on "autonomy" than parents of visually unimpaired survivors. Survivors' perceptions and parents' perceptions correlated poorly on all HRQoL dimensions. RB survivors reported a very good HRQoL compared with the Dutch reference group. The perceptions related to HRQoL differ substantially between parents and their children, i.e. parents judge the HRQoL of their child to be relatively poorer. Although the results are reassuring, additional factors of HRQoL that may have more specific relevance, such as psychological factors or coping skills, should be explore

A cluster randomised controlled trial and evaluation and cost-effectiveness analysis of the Roots of Empathy schools-based programme for improving social and emotional well-being outcomes among 8- to 9-year-olds in Northern Ireland

Background:There is growing consensus regarding the importance of attending to children’s social and emotional well-being. There is now a substantial evidence base demonstrating the links between a child’s early social and emotional development and a range of key longer-term education, social and health outcomes. Universal school-based interventions provide a significant opportunity for early intervention in this area and yet the existing evidence base, particularly in relation to their long-term effects, is limited.Objectives and main outcomes:To determine the effectiveness and cost-effectiveness of Roots of Empathy (ROE), a universal school-based programme that, through attempting to enhance children’s empathy, seeks to achieve the following two main outcomes: improvement in prosocial behaviour and reduction in difficult behaviour.Design:A cluster randomised controlled trial and an economic evaluation. A total of 74 primary schools were randomly assigned to deliver ROE or to join a waiting list control group. Seven schools withdrew post randomisation and a further two withdrew before the immediate post-test time point. Children (n = 1278) were measured pre test and immediately post test, and then for 3 years following the end of the programme. Data were also collected from teachers and parents.Setting and participants:The intervention schools delivered ROE to their Year 5 children (aged 8–9 years) as a whole class.Intervention:ROE is delivered on a whole-class basis for one academic year (October–June). It consists of 27 lessons based around the monthly visit from a baby and parent who are usually recruited from the local community. Children learn about the baby’s growth and development and are encouraged to generalise from this to develop empathy towards others.Results:Although it was developed in Canada, the programme was very well received by schools, parents and children, and it was delivered effectively with high fidelity. ROE was also found to be effective in achieving small improvements in children’s prosocial behaviour (Hedges’ g = 0.20; p = 0.045) and reductions in their difficult behaviour (Hedges’ g = –0.16; p = 0.060) immediately post test. Although the gains in prosocial behaviour were not sustained after the immediately post-test time point, there was some tentative evidence that the effects associated with reductions in difficult behaviour may have remained up to 36 months from the end of the programme. These positive effects of ROE on children’s behaviour were not found to be associated with improvements in empathy or other social and emotional skills (such as emotional recognition and emotional regulation), on which the trial found no evidence of ROE having an effect. The study also found that ROE was likely to be cost-effective in line with national guidelines.Conclusions:These findings are consistent with those of other evaluations of ROE and suggest that it is an effective and cost-effective programme that can be delivered appropriately and effectively in regions such as Northern Ireland. A number of issues for further consideration are raised regarding opportunities to enhance the role of parents; how a time-limited programme such as ROE can form part of a wider and progressive curriculum in schools to build on and sustain children’s social and emotional development; and the need to develop a better theory of change for how ROE works.Trial registration:Current Controlled Trials ISRCTN07540423.Funding:This project was funded by the National Institute for Health Research (NIHR) Public Health Research programme and will be published in full in Public Health Research; Vol. 6, No. 4. See the NIHR Journals Library website for further project information

PROMISE: first-trimester progesterone therapy in women with a history of unexplained recurrent miscarriages - a randomised, double-blind, placebo-controlled, international multicentre trial and economic evaluation

BACKGROUND AND OBJECTIVES: Progesterone is essential to maintain a healthy pregnancy. Guidance from the Royal College of Obstetricians and Gynaecologists and a Cochrane review called for a definitive trial to test whether or not progesterone therapy in the first trimester could reduce the risk of miscarriage in women with a history of unexplained recurrent miscarriage (RM). The PROMISE trial was conducted to answer this question. A concurrent cost-effectiveness analysis was conducted. DESIGN AND SETTING: A randomised, double-blind, placebo-controlled, international multicentre study, with economic evaluation, conducted in hospital settings across the UK (36 sites) and in the Netherlands (nine sites). PARTICIPANTS AND INTERVENTIONS: Women with unexplained RM (three or more first-trimester losses), aged between 18 and 39 years at randomisation, conceiving naturally and giving informed consent, received either micronised progesterone (Utrogestan(®), Besins Healthcare) at a dose of 400 mg (two vaginal capsules of 200 mg) or placebo vaginal capsules twice daily, administered vaginally from soon after a positive urinary pregnancy test (and no later than 6 weeks of gestation) until 12 completed weeks of gestation (or earlier if the pregnancy ended before 12 weeks). MAIN OUTCOME MEASURES: Live birth beyond 24 completed weeks of gestation (primary outcome), clinical pregnancy at 6-8 weeks, ongoing pregnancy at 12 weeks, miscarriage, gestation at delivery, neonatal survival at 28 days of life, congenital abnormalities and resource use. METHODS: Participants were randomised after confirmation of pregnancy. Randomisation was performed online via a secure internet facility. Data were collected on four occasions of outcome assessment after randomisation, up to 28 days after birth. RESULTS: A total of 1568 participants were screened for eligibility. Of the 836 women randomised between 2010 and 2013, 404 received progesterone and 432 received placebo. The baseline data (age, body mass index, maternal ethnicity, smoking status and parity) of the participants were comparable in the two arms of the trial. The follow-up rate to primary outcome was 826 out of 836 (98.8%). The live birth rate in the progesterone group was 65.8% (262/398) and in the placebo group it was 63.3% (271/428), giving a relative risk of 1.04 (95% confidence interval 0.94 to 1.15; p = 0.45). There was no evidence of a significant difference between the groups for any of the secondary outcomes. Economic analysis suggested a favourable incremental cost-effectiveness ratio for decision-making but wide confidence intervals indicated a high level of uncertainty in the health benefits. Additional sensitivity analysis suggested the probability that progesterone would fall within the National Institute for Health and Care Excellence's threshold of £20,000-30,000 per quality-adjusted life-year as between 0.7145 and 0.7341. CONCLUSIONS: There is no evidence that first-trimester progesterone therapy improves outcomes in women with a history of unexplained RM. LIMITATIONS: This study did not explore the effect of treatment with other progesterone preparations or treatment during the luteal phase of the menstrual cycle. FUTURE WORK: Future research could explore the efficacy of progesterone supplementation administered during the luteal phase of the menstrual cycle in women attempting natural conception despite a history of RM. TRIAL REGISTRATION: Current Controlled Trials ISRCTN92644181; EudraCT 2009-011208-42; Research Ethics Committee 09/H1208/44. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 20, No. 41. See the NIHR Journals Library website for further project information

Informing the NHS Outcomes Framework : evaluating meaningful health outcomes for children with neurodisability using multiple methods including systematic review, qualitative research, Delphi survey and consensus meeting

Background: The identification of suitable outcome measures will improve the evaluation of integrated NHS care for the large number of children affected by neurodisability, and has the potential to encourage the provision of more appropriate and effective health care. This research sought to appraise the potential of patient-reported outcome measures (PROMs) for children and young people with neurodisability. Aim: This research aimed (i) to identify key outcomes of health care for children with neurodisability, beyond morbidity and mortality, from the perspectives of children, parents and professionals, (ii) to critically appraise existing generic multidimensional PROMs, and (iii) to examine whether or not the key outcomes might be measured by existing PROMs. We also sought agreement on a definition of neurodisability. Methods: Data were gathered in three main ways, (i) a systematic review identified eligible generic multidimensional PROMs and peer-reviewed studies evaluating psychometric performance using English-language questionnaires. Studies were appraised for methodological quality and psychometric performance was appraised using standard criteria. (ii) Focus groups and interviews with children and young people with neurodisability, and separately with parents, sought to identify important outcomes of NHS care, and their feedback on example PROM questionnaires. (iii) An online Delphi survey was conducted with a multidisciplinary sample of health professionals to seek agreement on appropriate NHS outcomes. In addition, we convened a consensus meeting with a small nominal group of young people, parents and professionals, the group sought agreement on a core set of important health outcomes. Results: From the systematic review, we identified 126 papers that reported eligible evidence regarding the psychometric performance of 25 PROMs. Evidence of psychometric robustness was more favourable for a small number of PROMs: KIDSCREEN (generic), DISABKIDS (chronic-generic) and Child Health Utility 9D (preference-based measure). The Pediatric Quality of Life Inventory and KINDL offer both self-report and a proxy report version for a range of age bands, but evidence of their psychometric performance was weaker. Evidence was lacking in one or more respects for all candidate PROMs, in both general populations and those with neurodisability. Proxy reporting was found generally to be poorly correlated with self-report. Focus groups and interviews included 54 children and young people, and 53 parents. The more important health outcomes were felt to be communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, parents of children with intellectual impairment identified behaviour, toileting and safety as important outcomes. Participants suggested problems with the face validity of example PROM questionnaires for measuring NHS care. In the Delphi survey, 276 clinicians from a wide range of professions contributed to at least one of four rounds. Professionals rated pain, hearing, seeing, sleep, toileting, mobility and communication as key goals for the NHS but also identified treating neurological symptoms as important. Professionals in the Delphi survey and parents working with the research team agreed a proposed definition for neurodisability. The consensus meeting confirmed overlap between the outcomes identified as important by young people, parents and professionals, but not complete agreement. Conclusions: There was agreement between young people, parents and professionals regarding a core suite of more important health outcomes: communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, behaviour, toileting and safety were identified as important by parents. This research suggests that it would be appropriate to measure these constructs using PROMs to assess health care. None of the candidate PROMs in the review adequately captures all of the identified constructs, and there is inadequate evidence that candidate PROMs are psychometrically robust for use across children with neurodisability. Further consultation with young people, families and professionals is warranted to support the use of PROMs to measure NHS outcomes. Research to test potential PROMs with different age groups and conditions would be valuable

Erikoiskaupan alan mikroyrityksen liiketoiminnan kehittäminen nykyaikaisen kassajärjestelmän avulla

Opinnäytetyön aiheena oli selvittää, miten erikoiskaupan alan mikroyrityksen liiketoimintaa voidaan kehittää nykyaikaisen kassajärjestelmän avulla. Tavoitteena oli saada selville, miten kassajärjestelmä tukee mikroyrityksen eri prosesseja, ja toimintoja. Idea työn aiheesta syntyi kirjoittajan omasta kiinnostuksesta alaa kohtaan. Erityisesti taloudellisesti vaikeina aikoina keinot sitouttaa mikroyritysten muutenkin niukkaa pääomaa entistä tuottavammin, ja tavat kehittää yritysten liiketoimintaa nykyajan vaatimusten mukaiseksi, muodostuvat monelle mikroyritykselle elinehdoiksi. Autioituvat kaupunkikeskustat kielivät talouden taantuman lisäksi siitä, että yrityksissä ei ole pystytty vastaamaan ajan hengen mukaisiin tarpeisiin, vaan monissa tapauksissa on kangistuttu vanhoihin kaavoihin. Tämä opinnäytetyö pyrkii osaltaan tutkimaan nykyaikaisia toimintamalleja, ja esittämään asian raporteissa siten, että saadaan kokonaiskäsitys aiheesta, niin että kuka tahansa opinnäytetyötä lukeva pienyrittäjä voisi asian omaksua, ja hyödyntää opinnäytetyössä käsiteltyjä asioita pohtiessaan oman yrityksensä jatkuvaa kehittämistä. Opinnäytetyö on jaettu teoriaosaan, jossa käydään hieman tarkemmin läpi, millaisia prosesseja, tai toimintoja erikoiskaupan alan mikroyrityksissä voidaan kassajärjestelmän avulla kehittää, sekä syvennytään mahdollisimman käytännönläheisesti tapoihin hyödyntää näitä keinoja arkisessa elävässä elämässä. Opinnäytetyön suuri aihekokonaisuus on jaettu kolmeen eri osa-alueeseen, keskittyen varastonhallintaan, asiakashallintaan, ja verkkokauppaan. Empiirisessä osassa on kuvattu erään mikroyrityksen aitoa tarvetta kehitykselle, ja keinoa tämän tarpeen ratkaisemiseksi. Empiirinen osuus on toteutettu teemahaastatteluna. Case-yritykselle otettiin käyttöön uusi kassajärjestelmä, jonka eri toimintoja, ja mahdollisia tapoja hyödyntää niitä, on esitelty opinnäytetyön raportissa.The purpose of this thesis was to examine how micro-enterprise business in specialized shop field can be improved with a modern POS-system (Point of Sale). The goal was to find out how the POS-system can support various processes and operations of a small business. The idea of the topic was established from the writer´s own interest among the field of POS-systems. Ways of indenting capital, which they have already quite a little in mid 2010s, and ways of improving business to come up to nowadays demand, become to vital conditions for many micro-enterprise businesses, especially during the financially difficult times in the market economy. Besides the economic depression, city´s getting more and more quiet are telling their own story of enterprises which couldn´t come up to demand of the time we live because they just did everything just like they had always done. The target of this thesis was to find out nowadays business models, and manifesting those especially for entrepreneurs, so they can have some ideas to assist their own business planning. This thesis was split in two parts. There is a theoretic part, where is searched more accurately what kind of processes or operations small businesses working in specialized shop field can improve with POS-system, and where also is solutions how to use those tools in daily business life. The theoretic part was split in three parts, focusing in warehouse management, customer relationship management, and e-commerce. In the empirical part, there is described a real life micro-entrepreneur, which had a need for business improvement, and to solve a problem. The empirical part of the thesis has been made by using a theme interview. There has been described several kinds of functions and methods to use them from the POS-system, which has been taken to real life use for the case-company

Lapset mielikuvien luojina tv-mainonnassa

Opinnäytetyön aiheena oli selvittää miten lapsia käytetään mielikuvien luojina tv-mainonnassa. Tavoitteena oli saada selville miten lasten avulla luodaan mielikuvia, mitä syitä mainostajilla on käyttää lapsia mainonnassa, mitä lapset saavat mainostaa ja millä tavalla sekä kuinka paljon lapsia käytetään tv-mainonnassa. Aihe työlle tuli omasta mielenkiinnosta lapsia ja mielikuvamainontaa kohtaan. Aihe on yleisesti ih-misiä kiinnostava ja antaa tietoa mainosten tekijöille, mainosten katsojille ja mainok-siin osallistuvien lapsien vanhemmille. Työn teoriaosuudessa käsiteltiin kolmea päälukua, jotka koostuvat tv-mainonnasta, mielikuvamainonnasta ja lainsäädännöstä. Työssä perehdyttiin tv-mainontaan mai-nonnan muotona, tv-mainonnan etuihin ja haasteisiin, mielikuvien syntymiseen ja mielikuvien tavoitteisiin. Laki osiossa kerrottiin kuluttajansuojalain määräämistä ra-joituksista lapsien käyttöön tv-mainonnassa. Empiirinen osio tehtiin mainosanalyysina, harkinnanvaraisella näytteellä, jossa valit-tiin 4 televisiomainosta, joissa esiintyi lapsia. Analyysissa käytettiin lomaketta, joka on työssä liitteenä. Mainoksia analysoitiin lomakkeen pohjalta. Lomakkeessa arvioi-tiin muun muassa kohderyhmää, missä osassa lapset olivat mainoksessa, oliko perus-teltu syy käyttää lapsia mainoksessa, millaisia mielikuvia lapset luovat mainokseen, mainoksen vaikutus tuotemielikuvaan ja mainoksen huomioarvoa. Kaikki havainnot ja mielipiteet ovat omia päätelmiä mainoksista. Tutkimustarkoitus oli kuvaileva, jossa on tarkoituksena vastata kysymyksiin, mitä, miten ja millainen. Mainoksia on pyritty kuvailemaan mahdollisimman puolueettomasti. Analyysiin valituista mainoksista kolme on kotimaisia ja yksi on ulkomaalainen. Tutkimustulosten perusteella lapsia käytetään melko yleisesti televisiomainoksissa näyttelijöinä. Mainosanalyysissa olevista mainoksista suurin osa oli suunnattu naisille. Mainoksien tuotteet olivat sellaisia, joita naiset eniten kotitalouksiin ostavat. Lasten käyttö mainoksissa lisää naisten kiinnostusta tuotteita kohtaan. Arvioitavissa mainoksissa lapsilla oli hyvin suuri rooli. Missään mainoksista ei esitetty suoraa os-tokehotusta. Kaikki mainokset oli tehty hyvän maun rajoissa, eikä niissä pilkattu lap-sia. Mainoksien lapset olivat luonnollinen osa mainosta, kuten kuluttajansuojalaki määrää. Mainosten huomioarvo televisiossa oli hyvä. Kaikissa mainoksissa oli jokin tarina ja juoni, jota ihmiset jäivät seuraamaan. Mainokset olivat erilaisia, kuin perin-teiset ”tämä on hyvä tuote, osta tämä” mainokset. Kaikki arvioitavat mainokset yrit-tivät myydä tuotetta mielikuvien ja lasten avulla