BACKGROUND:Chronic pain is a highly prevalent disease, leading to high health care costs, impaired productivity, and decreased quality of life. An artificial intelligence (AI)-powered digital tool for self-management (PaindrainerTM) has been shown to be an effective treatment in chronic pain and could have potential to reduce the burden on costs and quality of life. OBJECTIVE:The objective of this study was to estimate the cost savings and quality-of-life gains associated with the use of an AI-powered digital tool for self-management of chronic pain. The study is a subsequent health economic analysis of previously published data. METHOD:Resource use and quality-of-life data were derived from a 1-arm, multicenter study in the United States. Differences between baseline and follow-up (6 and 12 weeks) were translated to an annual monetary estimate by using published literature, national statistics, and accepted threshold values for a quality-adjusted life year (QALY). RESULT:At 12 weeks, the use of an AI-powered digital tool was associated with a reduction in health care costs of $127 per user and an increase in annual productivity of $930 per user. In addition, the intervention was associated with an increase in health of 0.0275 QALYs, corresponding to a monetized value of $1375. CONCLUSION:The analysis of cost savings and quality-of-life gains associated with the use of an AI-powered digital tool for pain self-management demonstrates significant potential improvements in measured parameters of up to $2432 per person in total ($7380 per year, assuming 70% compliance), supporting this as an important treatment alternative.

Background and aimsArginase 1 deficiency (ARG1-D) is a ultrarare disease with manifestations that cause mobility and cognitive impairment that progress over time and may lead to early mortality. Diseases such as ARG1-D have a major impact also outside of the health care sector and the aim of this study was to estimate the current burden of disease associated with ARG1-D from a societal perspective.MethodsThe study was performed as a web-based survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, United Kingdom). The survey was distributed at participating clinics and included questions on e.g. symptoms (including the Gross Motor Function Classification System, GMFCS, and cognitive impairment), health care use, medication, ability to work, caregiving, and impact on health-related quality-of-life (HRQoL) using the EQ-5D-5L.ResultsThe estimated total mean societal cost per patient and year was 63,775 pound (SD: 49,944) pound. The cost varied significantly with both mobility impairment (from 49,809 pound for GMFCS level 1 to 103,639 pound for GMFCS levels 3-5) and cognitive impairment (from 43,860 pound for mild level to 99,162 pound for severe level). The mean utility score on the EQ-5D-5L for patients was 0.498 (SD: 0.352). The utility score also varied significantly with both mobility impairment (from 0.783 for GMFCS level 1 to 0.153 for GMFCS level 3-5) and cognitive impairment (from 0.738 for mild level to 0.364 for severe level).ConclusionsSimilar to other studies of rare diseases, the study is based on a limited number of observations. However, the sample appear to be reasonably representative when comparing to previous studies of ARG1-D. This study shows that ARG1-D is associated with a high societal cost and significant impact on HRQoL. Earlier diagnosis and better treatment options that can postpone or withhold progression may therefore have a potential for improved HRQoL and savings for the patient, caregiver, and society.

Background: Timely treatment of acute allergic reactions (AARs) is important to minimize reaction severity. Corticosteroid tablets dissolved in water are commonly used in mainstay treatment. A new oral film that dissolves on the tongue provides a faster and less cumbersome alternative to tablets for corticosteroid administration during AARs. This study evaluated patients’ preferences for attributes related to administration mode of corticosteroids in AARs. Methods: A web-based survey was sent to a sample from the adult Swedish population (≥18 years) with experience of corticosteroid treatment for AAR. We assessed the willingness to pay (WTP) for attributes related to corticosteroid treatment by applying a discrete choice experiment (DCE) approach. DCE attributes were administration mode, time to symptom relief, and price. The WTP for each attribute was derived using the attribute’s coefficient in a logistic regression analysis. We specified a forced choice (FC) and an unforced choice (UC) model. In the FC model, the respondents chose between 2 hypothetical treatments and in the UC model, between any of 2 hypothetical treatments and their current treatment. Results: The final study population included 348 subjects, of which 80% were women. All the evaluated DCE attributes were significant predictors for the treatment choice (p<.001). In the FC model, the incremental WTP for an oral film compared with tablets was 409 Swedish kronor (SEK [≈€36.7]), with no other factors considered. In the UC model, the incremental WTP for the oral film compared with tablets was 574 SEK (≈€51.7). After considering the value of the respondents’ current treatment, the WTP for the oral film decreased to 336 SEK (≈€30.3). The total WTP was reduced by 17 SEK (≈€1.5) per minute of shorter time to symptom relief. Subgroup analyses showed that people with circulatory symptoms and experience of swallowing difficulties related to allergy medication had higher WTP for the oral film than the average respondent. Conclusion: The findings show a substantial economic benefit of the oral film vs tablets for patients with AARs in Sweden. This result remained also after compensation for the full value of the patients’ current treatment.

[This corrects the article DOI: 10.1016/j.waojou.2024.100896.].

Background:Acute allergic reactions (AARs) occur shortly after exposure to an allergen, and the severity is on a continuum. Systemic corticosteroids (CS) are mainstay treatment of moderate to severe AARs, whereas those at risk of the most severe AARs (ie, anaphylaxis) are also recommended prescription of epinephrine autoinjectors. There is limited research on the impact of AARs not fulfilling the criteria for anaphylaxis. We have characterized a sample with a history of moderate to severe AARs and evaluated their self-reported disease burden (ie, daily life impact, anxiety, and treatment impediments). Methods:Survey study of adults with experience of AARs treated with CS. Participants recruited from a web-based panel and using social media were asked to complete a questionnaire related to their allergy and experience of AARs. The results were summarized for the whole sample and across subgroups with and without prescription of epinephrine. Results:The final study sample included 387 participants (80% women, mean age 41), of which 129 (33%) had at some point been prescribed epinephrine. The most common symptoms were respiratory (80%) and skin (78%) manifestations, and the mean (standard deviation, SD) self-rated severity score (scale from 0 [very mild] to 10 [very severe]) of the most recent AAR was 6.1 (2.0). More than 80% had experience of AARs interrupting daily activities and 50% of AARs that had limited work/studies or participation in leisure activities. Most of the respondents reported some degree of anxiety related to AARs and 43% had feared for their lives. Moreover, difficulties swallowing allergy medicine at an AAR was experienced by 26% and not having the medicine available when needed by 66%. Participants with prescription of epinephrine experienced more severe AARs than those without such prescription (mean [SD] severity 6.8 [2.1] vs 5.8 [1.8], p < 0.0001); however, also those without epinephrine prescription reported considerable anxiety and impact on daily life and to a similar degree as those with prescription. Conclusions:In this sample, subjects with experience of AARs treated with CS showed a considerable disease burden with anxiety and interruption on daily life, as well as problems related to access to, and swallowing of, medication. Although respondents with epinephrine prescription had more severe disease, a high disease burden was also evident among those without epinephrine. The study increases the knowledge of people with moderate to severe AARs, a patient population that has previously been underrepresented in the research literature.

Arginase 1 deficiency (ARG1-D) is an ultrarare, metabolic disease which may cause spastic paraplegia, cognitive deficiency, seizures, and ultimately severe disability. The aim of this study was to assess disease burden in ARG1-D by performing a cross-sectional survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, and the United Kingdom). Patients were enrolled at participating clinics and data were collected using a web-based questionnaire. The findings indicate that there is a significant share of patients who experience severe cognitive and mobility impairment but also that there is a considerable variance in symptom severity among patients. Disease management was mostly in line with treatment guidelines and self-reported adherence to treatment was reported to be high among a majority although following diet restrictions was perceived as difficult. However, despite this, since a large share of patients experienced severe cognitive and mobility impairment an unmet need among this patient population is suggested. The introduction of disease-modifying therapies and early identification and diagnosis may help alleviate the disease burden associated with ARG1-D in the future.

The traditional health economic analysis is limited to estimating the impact on the treated patient. As vaccines are usually aimed at preventing infectious diseases, they may be associated with additional values for the non-treated wider population. Although there are valid reasons for treating vaccines differently, and a wide support for a broader perspective in the literature (i.e., beyond the net costs and health gain related to the outcome for the vaccinated individual), it remains unclear to what extent the Health Technology Assessment (HTA) agencies accept and apply a broader perspective. The purpose of this study is to examine and discuss what type of consequences are relevant for a health economic analysis of vaccines and which consequences are considered by HTA agencies. The study includes a strategic review of literature and HTA decisions in Sweden and other countries, online round-table discussions with stakeholders in Sweden, and a basic estimation of the value of a COVID-19 vaccination in Sweden. The study shows that, other than herd effect, broader economic consequences for the population are generally not included in the economic evaluation of vaccines. Also, all economic consequences for the treated patient (production loss) and caregiver (health loss) are not always considered. The perspective chosen can have a major impact on the outcome of the analysis. A vaccine for COVID-19 is estimated to provide a value of €744-€956 per dose when using a societal perspective including broader consequences for the population. Providing a complete and appropriate picture of the value of vaccination is of importance to allocate resources efficiently, to provide incentives for vaccine development, and to show the cost of delaying decisions to implement a new vaccine.

Phenylketonuria (PKU) is a metabolic disorder leading to a deficiency in the metabolism of phenylalanine (Phe). Elevated Phe levels in the blood, tissue and brain may lead to emotional, cognitive, and physical symptoms in adults. To control blood Phe levels, most individuals with PKU need to follow a strict, life-long Phe-restricted diet. The main objective of this study was to estimate the impact of PKU-related disease symptoms and dietary restrictions on health-related quality of life (HRQoL). This study was designed as a web-based survey using the methods of Time Trade-Off (TTO) and Discrete Choice Experiment with duration (DCETTO) in a survey of the Swedish general population and population with PKU. Data were collected using questionnaires for TTO diet (n = 509), TTO symptoms (n = 507), and DCETTO (n = 1117). The disutility of diet restrictions ranged from 0.050 for a partially restricted diet without medical food to 0.193 for a fully restricted diet with medical food in the TTO (DCE: 0.043–0.108). The disutility of experiencing symptoms (emotional, cognitive and physical) ranged from 0.148 for mild symptoms to 0.593 for severe symptoms in the TTO (DCE: 0.122–1.522). The study shows that both diet and symptoms have a significant negative impact on HRQoL in PKU and that utility estimates are largely consistent across methods and samples.

This paper compares the value per statistical life (VSL) in the context of suicide prevention to that of prevention of traffic fatalities. We conducted a contingent valuation survey with questions on willingness to pay (WTP) in both contexts by administering a web questionnaire to 1038 individuals aged 18 to 80. We conjectured that WTP for a given impact on the number of fatalities would be lower for suicide prevention because suicide, at least to some degree, is the result of individuals’ own decisions. However, this hypothesis was not supported by the within- or between-sample estimates of WTP or by responses to direct questions. Hence, no support is provided for the use of a lower valuation of the impact of suicide prevention than for risk-reducing programs in other fields, such as traffic safety. This implies that the same VSL should be used for evaluating suicide prevention interventions and for risk-reducing programs in other policy areas and funds for the prevention of fatalities should be directed to the area with the lowest cost per life saved.

Antibiotikaresistens har utnamnts till ett av de storsta framtida hoten mot manniskans halsa. Allt fler bakterier utvecklar resistens (motstandskraft) mot ett eller flera typer av antibiotika i huvudsak till foljd av en hog anvandning. Forutom att begransa och effektivisera anvandningen av existerande antibiotika finns det darfor ett stort behov av att utveckla nya antibiotika for att sakerstalla fortsatt effekt mot bakterier som idag ar multiresistenta. Trots detta gar utvecklingen av nya antibiotika mycket langsamt och flera lakemedelsbolag har valt att lagga ned sin utveckling. Detta beror huvudsakligen pa bristande lonsamhet vilket i sin tur beror dels pa att priset ar lagt da flera varden inte raknas in, dels pa att volymen ar lag da antibiotika anvands mycket restriktivt. Syftet med denna rapport ar att redovisa forskning kring olika metoder for vardering och betalning av antibiotika samt utarbeta ett forslag till metoder och betalningsmodeller i en svensk kontext. Rapporten visar att det finns forslag pa att skatta vardet av antibiotika med hjalp av dynamisk modellering som tillater simulering av breda effekter pa hela befolkningen (t ex minskad smittspridning, fordrojd resistensutveckling, beredskap i handelse av storre utbrott) och ett pilottest med denna metod pagar for narvarande i Storbritannien. Denna modell fangar dock inte samtliga varden av antibiotika. For att losa problemet med laga incitament till foljd av en restriktiv anvandning (och darmed lag betalning) foresprakas atgarder fran det offentliga som antingen sanker kostnaden for utvecklingen av nya antibiotika (push) eller hojer intakten for antibiotika som natt marknaden (pull). De flesta ar overens om att det idag kravs incitament for att fa antibiotika att na marknaden och det rader en allt storre konsensus om att detta bor ske via en ersattning som ar loss-kopplad fran anvandningen (de-linkage) och ges i samband med att ett antibiotikum nar marknaden, sa kallad marknadsintradesbeloning eller Market Entry Reward (MER). Vi foreslar en vardebaserad ”Abonnemangsmodell for Antibiotika” inom ramen for ett internationellt samarbete (till exempel EU + USA). En sadan modell ska bygga pa tre delar; (1) en internationell antibiotikafond med inbetalning fran bidragande lander baserat pa BNP, (2) preferensbaserade vikter for antibiotikans vardedrivare baserat pa experimentella studier bland befolkningen, samt (3) en oberoende Antibiotikakommitte som bestammer ersattningens storlek baserat pa det nya lakemedlets profil och preferensbaserade vikter for antibiotika. I vantan pa en full-skalig tillampning av en sadan modell inom ramen for ett internationellt samarbete foreslar vi att Sverige tar initiativ till en pilot for att ta fram preferensbaserade vikter for antibiotikans vardedrivare.

The outbreak of the pandemic COVID-19 (Coronavirus) has resulted in various international and national strategies, including non-pharmaceutical interventions (NPIs) such as social distancing and travel bans, which have purportedly mitigated the health loss due to the pandemic but also given rise to a severe economic crisis. Both factors, the pandemic and the NPIs, can be expected to have an impact on the Health-Related Quality-of-Life (HRQoL) of the population. The objective of this study was to estimate the impact on HRQoL of the Swedish adult population during the outbreak of the COVID-19 pandemic. A web-based survey was sent to randomised samples of the adult Swedish population before the outbreak of the pandemic in Sweden in February 2020 (n=1,016) and during the outbreak of the pandemic. The first wave pandemic data was collected in April 2020 (n=1,003), one-month after the outbreak and, the second wave data was collected in January 2021 (n=1,013), after 10-months living under the pandemic. HRQoL was measured using the EQ-5D-5L in the pandemic surveys, whereas the Visual Analogue Scale (VAS) was used in all surveys. The results suggested a reduction in average HRQoL as measured by VAS in the adult Swedish population, with 0.059 points reduction in VAS in April 2020 and 0.074 points reduction in January 2021, compared to the pre-pandemic measurement in February 2020. The loss in HRQoL was significant among respondents in the working age population (<65 years), suggesting that the social and economic impact of NPIs were the primary drivers for this specific cohort. Findings of this study supports a wide public health perspective and future HRQoL measurements at the population level throughout the pandemic. Published: Online September 2021

Vacciner har egenskaper som staller andra krav pa metoder for vardebaserad prissattning (VBP) an vardering av traditionella lakemedel eller traditionella medicinska teknologier. Vacciner avser ofta smittsamma sjukdomar och da forekommer externa effekter, som flockimmunitet, och indirekta konsekvenser, sasom serotypersattning, antibiotikaresistens och foljdsjukdomar i storre utstrackning an for andra medicinska teknologier. Vacciner ar exempel pa primarpreventiva atgarder men skiljer sig fran manga andra typer av primarpreventioner genom att vacciner ofta riktar sig till mycket stora populationer. Det innebar att aven mycket sma sannolikheter for biverkningar kan fa betydande halsokonsekvenser och stor uppmarksamhet som kan leda till betydande foljsamhetsproblem och minskad tackning med minskad/forsenad flockimmunitet och forlorat varde av vaccinationen. En annan konsekvens av att vacciner ofta riktar sig mot att forebygga smittsamma sjukdomar hos stora populationer ar att komparatorn, d v s alternativet till vaccin, kan vara annan icke medicinsk prevention som inte ingar i vara sjukvardsbudgetar. Alla ickemedicinska atgarder som vidtagits for att begransa smittspridningen under pagaende covid-19 pandemin ar goda illustrationer. Det handlar om varden som harror fran uppgiven produktion, fritid, fysisk isolering, makroekonomiska konsekvenser mm. Syftet med denna rapport ar att undersoka vilken evidens som ar relevant i samband med halsoekonomiska utvarderingar av vaccin samt vilken evidens som efterfragas av olika intressenter. Rapporten visar att vaccin pa manga satt skiljer sig fran traditionella medicinska teknologier och att det darfor finns skal att kartlagga klinisk och halsoekonomisk effekt pa ett satt som fullt ut tar hansyn till dess specifika egenskaper. Detta forutsatter modellering av epidemiologi och halsoekonomi, storre acceptans for intermediara effektmatt, samt eventuellt alternativa metoder och angreppssatt sasom t ex betalningsvilja. Det finns en bred acceptans for modellering i saval Sverige som andra lander. Flera intressenter i Sverige staller sig dessutom positiva till att inkludera bredare konsekvenser.

The value of a statistical life (VSL) is a widely used measure for the value of mortality risk reduction. As VSL should reflect preferences and attitudes to risk, there are reasons to believe that it varies depending on the type of risk involved. It has been argued that cancer should be considered a "dread disease," which supports the use of a "cancer premium." The objective of this study is to investigate the existence of a cancer premium (for pancreatic cancer and multiple myeloma) in relation to road traffic accidents, sudden cardiac arrest, and amyotrophic lateral sclerosis (ALS). Data were collected from 500 individuals in the Swedish general population of 50-74-year olds using a web-based questionnaire. Preferences were elicited using the contingent valuation method, and a split-sample design was applied to test scale sensitivity. VSL differs significantly between contexts, being highest for ALS and lowest for road traffic accidents. A premium (92-113%) for cancer was found in relation to road traffic accidents. The premium was higher for cancer with a shorter time from diagnosis to death. A premium was also found for sudden cardiac arrest (73%) and ALS (118%) in relation to road traffic accidents. Eliminating risk was associated with a premium of around 20%. This study provides additional evidence that there exist a dread premium and risk elimination premium. These factors should be considered when searching for an appropriate value for economic evaluation and health technology assessment.

Migraine is a disabling, chronic neurological disease leading to severe headache episodes affecting 13.2% of the Swedish population. Migraine leads to an extensive socio-economic burden in terms of healthcare costs, reduced workforce and quality of life (QoL) but studies of the health-economic consequences in a Swedish context are lacking. The objective of this study is to map the health-economic consequences of migraine in a defined patient population in terms of healthcare consumption, production loss and QoL in Sweden. The study is based on data from a web-based survey to members in the Swedish patients’ association suffering from migraine. The survey was conducted in May 2018 and included people with migraine aged 18 years or older. The survey included questions on health resource consumption, lost production resulting from migraine-related absenteeism and presenteeism, and QoL as measured by the EuroQol 5 dimensions questionnaire (EQ-5D-5 L) and the Headache Impact Test (HIT-6). The results are presented in yearly costs per patient and losses in quality adjusted life years (QALYs). The results are based on answers from 630 individuals with migraine and are presented by number of migraine days per month. The total cost per patient and year increased with the number of migraine days per month (p < 0.001) and varied between approximately €5000 for those with less than 3 migraine days per month and €24,000 per year for those with 21–28 migraine days per month. Production loss represented the main part of the costs, approximately 80%. The average loss in QALYs per year also increased with the monthly number of migraine days (p = 0.023). Migraine leads to significant societal costs and loss of quality of life. There appears to be an unmet need and a potential for both cost savings and QoL benefits connected with a reduction in the number of migraine days.

The value of a quality-adjusted life-year (QALY) and the value of a statistical injury (VSI) are important measures within health economics and transport economics. Several studies have, therefore, estimated people’s willingness to pay (WTP) for these estimates, but most results show scale insensitivity. The ‘original’ chained approach (CA) is a method developed to mitigate this problem by combining the contingent valuation (CV) with standard gamble (SG). In contrast to the version of the CA applied by the previous research of the WTP for a QALY, the original version allows the value of major health gains to be estimated without having the respondents express their WTP directly. The objective of this study was to estimate the value of a QALY and VSI in the context of non-fatal road traffic accidents using the original CA to test if the approach, applied to a wide range of health gains, is able to derive valid estimates and a constant value of a QALY which the previous research has not been able to show. Data were collected from a total of 800 individuals in the Swedish adult general population using two web-based questionnaires. The values of a QALY based on trimmed estimates were close to constant at €300,000 irrespective of the size of the QALY gain. The study shows that the original CA method may be a valid method to estimate the value of a QALY and VSI for major health losses. It also supports the use of a higher threshold value for a QALY than that which is currently applied by several health technology assessment agencies in different countries.

For the assessment of value of new therapies in healthcare, Health Technology Assessment (HTA) agencies often review the cost per quality-adjusted life-year (QALY) gained. Some HTA agencies accept a higher cost per QALY gained when treatment is aimed at prolonging survival for patients with a short expected remaining lifetime, a so-called end-of-life (EoL) premium. The objective of this study is to elicit the existence and size of an EoL premium in cancer. Data was collected from 509 individuals in the Swedish general population 20–80 years old using a web-based questionnaire. Preferences were elicited using subjective risk estimation and the contingent valuation (CV) method. A split-sample design was applied to test for order bias. The mean value of a QALY was MSEK4.8 (€528,000), and there was an EoL premium of 4–10% at 6 months of expected remaining lifetime. Using subjective risk resulted in more robust and valid estimates of the value of a QALY. Order of scenarios did not have a significant impact on the WTP and the result showed scale sensitivity. Our result provides some support for the use of an EoL premium based on individual preferences when expected remaining lifetime is short and below 24 months. Furthermore, we find support for a value of a QALY that is above the current threshold of several HTA agencies.

Introduction: Trastuzumab is part of the standard treatment for HER2-positive breast cancer. The aim of this study was to estimate the societal value of trastuzumab administered through subcutaneous (SC) injection compared to intravenous (IV) infusion.Methods: Female patients with HER2-positive breast cancer receiving SC or IV trastuzumab were consecutively enrolled from five Swedish oncology clinics from 2013 to 2015. Data on time and resource utilization was collected prospectively using patient and nurse questionnaires. Societal costs were calculated by multiplying the resource use by its corresponding unit price, including direct medical costs (pharmaceuticals, materials, nurse time, etc.), direct non-medical costs (transportation) and indirect costs (production loss, lost leisure time). Costs were reported separately for patients receiving trastuzumab for the first time and non-first time ("subsequent treatment").Results: In total, 101 IV and 94 SC patients were included in the study. The societal costs were lower with SC administration. For subsequent treatments the cost difference was (sic)117 (IV (sic)2099; SC (sic)1983), partly explained by a higher time consumption both for nurses (14 min) and patients (23 min) with IV administration. Four IV and 16 SC patients received trastuzumab for the first time and were analysed separately, resulting in a difference in societal costs of (sic)897 per treatment. A majority of patients preferred SC to IV administration.Conclusion: SC administration resulted in both less direct medical costs and indirect costs, and was consequently less costly than IV administration from a societal perspective in a Swedish setting. (C) 2016 Elsevier Ltd. All rights reserved.