| 1. |
- Linge, Jennifer, 1990-
(author)
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Adverse Muscle Composition : Revisiting Sarcopenia in General Population and Liver Disease using Magnetic Resonance Imaging
- 2023
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Doctoral thesis (other academic/artistic)abstract
- Sarcopenia - from the Greek words 'sarx' (flesh) and 'penia' (loss) - was, when coined in 1989, a term denoting the decline in muscle mass and strength that occurs with aging. Such definition implies everyone suffers from sarcopenia to varying degrees, which naturally makes studying sarcopenia challenging. Early (practical) definitions of sarcopenia focused on identification of low muscle mass, while later definitions also include criteria of low muscle strength. Use of such definitions to study sarcopenia has shown that wasting is intensified in those suffering from metabolic diseases, and even more rapid in end-stage diseases. Although it is unknown whether sarcopenia accelerate disease or the other way around, detection of sarcopenia concurrent with other diseases clearly identifies a vulnerable subgroup of patients who may need more extensive care.In severe stages of liver disease, poor muscle health has been linked to higher morbidity and mortality, and may affect the outcome of liver transplantation. Sarcopenia is therefore recognized as an important factor that should affect both clinical decision-making and intervention in patients being evaluated for liver transplantation. However, sarcopenia is poorly understood (and commonly overlooked) in earlier stages of disease, where the potential of preventative care is greater. One challenge has been the prevalence of obesity in diseases that may precede more advanced disease, such as non-alcoholic fatty liver disease (NAFLD). Due to their larger body size, individuals with obesity need more muscle mass to maintain mobility function. Therefore, the threshold for what is considered ‘low muscle mass’ needs to be higher, or somehow adjusted for body size.This thesis started by applying the European definition of sarcopenia in 10,000 individuals aged 44-78 years volunteering for the UK Biobank imaging study. It was identified that current body size adjustments used to detect 'low muscle mass' were ineffective. The consequence of this was underdiagnosis of sarcopenia in individuals with overweight and obesity.Therefore, a more personalized muscle volume assessment, that was independent of body size, was developed with the aim to describe how much an individual is deviating from what is expected and address whether they have an 'adequate' amount of muscle volume - muscle volume z-score.Muscle volume was measured using magnetic resonance imaging and from the same images, muscle fat infiltration (indicating muscle quality) was also quantified. The first results indicated that muscle volume z-score and muscle fat infiltration were independently associated with mobility function and hospitalization, and that a combination of the two may identify the most vulnerable individuals. Therefore, thresholds were suggested to identify an adverse muscle composition (low muscle volume z-score combined with high muscle fat infiltration).Following studies investigated associations of adverse muscle composition with metabolic diseases, mobility function, and mortality in general population and NAFLD. Overall, the studies showed that adverse muscle composition was associated with increased morbidity and mortality independent of mobility function, and indicated that muscle composition assessment could provide clinically relevant information that may be useful in risk-stratification of heterogeneous disease populations like NAFLD.Today, the relevance of adverse muscle composition and potential clinical use cases are evaluated in the liver transplant setting through both European and American clinical studies.
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| 2. |
- Siverskog, Jonathan, 1988-
(author)
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Opportunity cost in healthcare priority setting
- 2022
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Doctoral thesis (other academic/artistic)abstract
- The resources available for the public provision of health care are not unlimited. Cost-effectiveness evidence on new healthcare interventions can help us prioritise in order to use scarce resources wisely, but to interpret cost-effectiveness evidence, it may appear as if we must make trade-offs between life and money. This is not so. If we are able to quantify the health improvements that resources would or could have generated in alternative use, a decision about providing or denying treatment can instead be framed as a trade-off between health gained and health forgone. In this thesis, I seek to provide a more robust basis for this way of reporting and interpreting cost-effectiveness evidence.In Chapter II, I discuss the definition of opportunity cost in economic evaluation. The opportunity cost of providing an intervention is what we must give up to provide it. More precisely, it is typically defined as the value of the best alternative forgone. In economic evaluation of health care, opportunity cost has been understood in terms of the least cost-effective, currently funded intervention, which should be displaced when funding new interventions subject to a fixed budget. I show that alternative uses forgone may be neither currently funded nor well-defined, which implies that we should not look to cost-effectiveness evidence on specific interventions for information on opportunity cost. Further, identifying a best alternative use assumes that priority setting is based on objectives that can be summarised into a single measure of value. If economic evaluation is used to inform trade-offs between one measure of value (e.g., quality-adjusted life years, QALYs) and other, unquantified objectives, I suggest that it would be more appropriate to define opportunity cost as value in expected alternative use.To quantify opportunity cost as health forgone, we need evidence on the health that resources would or could have generated in alternative use. In Chapter III, I use panel data on health spending and life expectancy in Swedish regions to estimate the marginal cost of producing a QALY. My findings imply that Swedish health care can produce health at a marginal cost of SEK 180,000 per QALY, which could be used as an expectation on how productive health spending would be in alternative use. I discuss methodological issues with this approach and identify some credibility problems with selection-on-observables strategies plaguing this and similar research to date. I address (some of) these problems by assessing coefficient stability and the causal mechanisms between healthcare resource use and health outcomes, using a second panel on hospital bed capacity and mortality. This analysis finds that health could be gained at a cost of SEK 420,000 per QALY by providing more hospital beds.To illustrate the role of this evidence in healthcare priority setting, Chapter IV considers how it could have been used to inform decision making in a case of pharmaceutical reimbursement. I propose that economic evaluation report cost-effectiveness evidence as QALYs forgone per QALY gained. This frames a decision about providing or denying treatment as a judgement on the relative priority of QALYs gained and QALYs forgone, which is more transparent about a trade-off between equity and efficiency than deciding whether the monetary cost per QALY is too high. Framing decisions as health gained versus health forgone could also lead to better decision making by making opportunity costs more salient to decision makers and the reason for sometimes denying costly treatments easier to communicate.In summary, cost-effectiveness evidence can be used to achieve the theoretical objective of health maximisation, but economic evaluations rarely report opportunity costs explicitly as health forgone. This thesis provides the practical means to be explicit and implications for the definition of opportunity cost and the interpretation of cost-effectiveness evidence when health maximisation is not the sole objective of healthcare priority setting.
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| 3. |
- Gruneau, Lina, 1994-, et al.
(author)
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Precision i hälsoekonomiska utvärderingsresultat och osäkerhet i prioriteringsbeslut
- 2022
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Reports (other academic/artistic)abstract
- Tandvårds- och läkemedelsförmånsverket (TLV) genomför på uppdrag av regeringen ett arbete med att utveckla metoder för hälsoekonomiska utvärderingar av precisionsmedicin och betalningsmodeller för avancerade terapiläkemedel (ATMP). TLV redovisar i sin andra rapport inom ramen för uppdraget ett antal nya möjliga metoder som kan användas i en hälsoekonomisk utvärdering för att värdera huruvida de priser som företagen efterfrågar är rimliga i förhållande till läkemedlens nytta. Eftersom hälsoekonomiska utvärderingar alltid är behäftade med osäkerhet i skattningarna är frågan om hur osäkerhet skall analyseras och beskrivas central för precisionsmedicin och ATMP då dataunderlagen ofta är knapphändiga. I en slutsats från ett tidigare arbete om utmaningarna med att utvärdera kostnader och hälsoeffekter inom ramen för precisionsmedicin konstaterades det att osäkerheten i skattningarna av kostnadseffektivitet kommer att öka när patientpopulationerna som utvärderas blir allt mindre. Vidare framgick det i de tidigare arbetena att TLVs ansats för att beskriva och analysera osäkerhet inte alltid är helt tydlig. Som en del i TLVs regeringsuppdrag ingår det att beskriva och tydliggöra osäkerheter i skattningar av kostnadseffektivitet samt hur dessa osäkerhet kan påverka osäkerhet i prioriteringsbeslut och den här rapporten är en del i det arbetet.Rapporten är fristående från den rapport som TLV skrivit inom ramen för regeringsuppdraget men har som mål att ge en kompletterande beskrivning av de osäkerheter som oftast föreligger när hälsoekonomiska utvärderingar utgör en del av ett underlag för prioriteringsbeslut. Förhoppningen är att denna rapport tillsammans med TLVs rapport ska stimulera till fortsatt diskussion om potentiella lösningar för att hantera utmaningar inte bara med precisionsmedicin och ATMP utan även med beslutsfattande under osäkerhet generellt inom hälso- och sjukvården.
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| 4. |
- Henriksson, Martin, Associate Professor, 1974-, et al.
(author)
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Hälsoekonomiska utvärderingsaspekter av precisionsmedicin och ATMP
- 2021
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Reports (other academic/artistic)abstract
- Tandvårds- och läkemedelsförmånsverket (TLV) har inom ramen för ett regeringsuppdrag undersökt hur hälsoekonomiska bedömningar för precisionsmedicin kan utvecklas samt utrett möjliga betalningsmodeller för gen- och cellterapier (ATMP). Inom ramen för detta uppdrag har CMT bistått TLVs utredning genom utrednings- och analysarbete kopplat till vissa av de frågeställningar som myndigheten valt att fokusera på i regeringsuppdraget. Föreliggande rapport redovisar resultatet av det arbetet. Syftet har varit att ge en tillräckligt detaljerad beskrivning av hälsoekonomisk utvärderingsmetod för att kunna problematisera hälsoekonomiska utvärderingsaspekter av precisionsmedicin och ATMP. Rapporten gör inte anspråk på att utreda alla aspekter kopplade till precisionsmedicin och ATMP och syftar inte till att ge några definitiva lösningar på de komplexa utmaningar som dessa utvärderingar innebär. Rapporten kan läsas fristående från TLVs huvudrapport men innehållet och de exempel som redovisas präglas av vad utredarna inom ramen för TLVs uppdrag har fokuserat på. Författarna är ensamt ansvariga för innehållet i den här rapporten.
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| 5. |
- Siverskog, Jonathan, 1988-, et al.
(author)
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The health cost of reducing hospital bed capacity
- 2022
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In: Social Science and Medicine. - : Elsevier. - 0277-9536 .- 1873-5347. ; 313
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Journal article (peer-reviewed)abstract
- In the past two decades, most high-income countries have reduced their hospital bed capacity. This could be a sign of increased efficiency but could also reflect a degradation in quality of care. In this paper, we use repeated cross-sections on mortality and staffed hospital beds per capita in all 21 Swedish regions to estimate the potential death toll from reduced bed capacity. Between 2001 and 2019, mortality and beds decreased across all regions, but regions making smaller bed reductions experienced on average greater decreases in mortality, equivalent to one less death per three beds retained. This estimate is stable to a wide range of specifications and to adjustment for potential confounders, which supports a causal interpretation. Our results imply that by providing one more bed, Swedish health care could produce about three quality-adjusted life years (QALYs) at a cost of SEK 400,000 (similar to US$40,000) per QALY. These findings could be informative about the marginal productivity of health care and support the credibility of empirical work attempting to estimate the opportunity cost of funding new healthcare interventions subject to a constrained budget.
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