K. Steen Carlsson

On‐demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome

Summary.  Using an 11‐year panel of 156 Norwegian and Swedish patients with severe haemophilia, and including retrospective case‐book data from birth, we compared the differences in the haemophilia‐related resource use between on‐demand and prophylactic treatment. Patients treated on‐demand had more surgery (arthrodeses, prostheses implantations and synovectomies) and more days lost from work. Median annual factor‐concentrate consumption among adults (18+) was 211 000 IU [interquartile range (IQR) 154 000–268 000] or 3 024 IU kg−1 year−1 for patients on prophylactic treatment and 55 000 IU (IQR 28 000–91 000) for on‐demand patients (780 IU kg−1 year−1). This was partly explained by the fact that the median dose per kg body weight was twice as great 28, (IQR 24–32) for prophylaxis compared with 14 (IQR 12–16) for on‐demand. Prescribed dose per kg body weight was found to be an important factor explaining the variation in total annual factor‐concentrate consumption per patient for both types of treatment. Other variables included in the panel‐data regression analysis were the number of weeks on secondary prophylaxis for on‐demand patients and age, body weight and type of haemophilia for children (0–17 years) on prophylaxis. Differences were consistently substantial and will affect both costs and benefits of the two treatment strategies.

Analysis of three outcome measures in moderate to severe psoriasis : a registry-based study of 2450 patients

Summary Background  As moderate to severe psoriasis is a systemic disease with large effects on health‐related quality of life, generic measures that include overall health, not only skin involvement, are necessary. Knowledge about the relationship between the generic preference‐based EuroQol 5D (EQ‐5D) and dermatology‐specific measures in psoriasis is limited.

Economic evaluation: what are we looking for and how do we get there?

Summary.  The interest in economic evaluation of alternative strategies for haemophilia treatment has increased through the years. Few studies have actually been undertaken, however, and most of them have been simple cost‐minimization or cost‐effectiveness analyses. From the perspective of the binational project ‘Treatment strategies for severe haemophilia − prophylaxis vs. on‐demand’, the present paper discusses the pros and cons of different methods for economic evaluation and their data requirements. Severe haemophilia is a rare disease that requires lifelong treatment. In addition, treatment has both short‐ and long‐term effects which are likely to differ between strategies. Accordingly, regardless of the chosen evaluation method, data requirements are non‐trivial. Hence, the various problems connected to the generation of data, as well as how they may be addressed, are also discussed.

The association between health utility and joint status among people with severe haemophilia A : findings from the KAPPA register

People with severe haemophilia A have reportedly impaired health related quality of life (utility) mainly due to recurrent bleeding, arthropathy and treatment burden.

Comparative burden of arthropathy in mild haemophilia: a register‐based study in Sweden

Mild haemophilia is a congenital bleeding disorder affecting males. The burden of arthropathy in mild haemophilia has not been comprehensively described.

Bleeding-related hospitalization in patients with von Willebrand disease and the impact of prophylaxis : Results from national registers in Sweden compared with normal controls and participants in the von Willebrand Disease Prophylaxis Network

Patients suffering from von Willebrand disease (VWD) have a variety of bleeding symptoms and require both outpatient care for treatment and, in more severe cases, hospitalization.

Surgery and survival in birth cohorts with severe haemophilia and differences in access to replacement therapy: The Malmö experience.

Persons with severe haemophilia require lifelong replacement therapy, prophylaxis, to prevent bleeding. Data describing long‐term outcomes of prophylactic treatment are scarce. The aim of this study was to investigate joint surgery and survival among persons with severe haemophilia with special attention to access to prophylaxis in the early years of life.

THU0636 Assessment of Functional Status and Health-Related Quality of Life Instruments in A Population-Based Cohort of Patients with Psoriatic Arthritis

Background In addition to reduce joint destruction progress and pain, one goal in psoriatic arthritis (PsA) treatment is to improve functional status (FS) and health-related quality of life (HRQoL). Demand for data on patient-reported outcomes is partly driven by the needs of cost-effectiveness analyses for reimbursement decisions. Several outcome measures are used for assessing the complex impact of PsA, but there is need for more information from studies with parallel assessment of FS and HRQoL measures. Objectives To assess patient-reported functional status by HAQ (health assessment questionnaire) and by BASFI (Bath Ankylosing Spondylitis Functional Index), health-related quality of life by EQ-5D (EuroQol-five dimensions), by DLQI (Dermatology Life Quality Index) and by GH (general health) across sex, age, presence of psoriasis and drug treatment to explore trends of similarities. Methods A sub cohort of PsA patients from the Swedish SpAScania cohort (age≥15 years, spondyloarthritis between 2003 and 2007 in the Skåne region) was used. 1181/2003 eligible PsA patients (diagnosed with PsA once by rheumatologist/internist or at least twice by other physician) responded to a questionnaire in 2009. Sex, age, disease duration, presence of psoriasis, HAQ, BASFI, EQ-5D, DLQI, and GH regarding joint (GH-rs) and skin symptoms (GH-ss) were assessed. Information about biologic disease modifying antirheumatic drugs (bDMARDs) treatment during 2008–2011 was collected from the Swedish Prescribed Drug Register and from the Southern Swedish Arthritis Treatment Group Register. Differences between groups were tested by chi-squared test for categorical data and Students t-test for continuous data. Results Out of 1181 patients 43% were men. The mean age (standard deviation) was 58 (13) and 57 (17) years for men and women respectively. Mean disease duration was 13 (11) years. 77% of the patients reported psoriasis. bDMARD treatment during all 4 years was reported by 17% of the patients; 74% reported no use at all during the same period. Women reported significantly worse health across all six health status measures compared to men (Figure 1). Patients with irregular use of bDMARDs during the 4-year period reported significantly worse health compared to patients with use during the whole period or no use at all. There was a significant association between worse health and older age for HAQ, BASFI and GH-rs. Patients with psoriasis reported significantly worse health measured by DLQI and GH-ss. Conclusions While all outcome measures seemed to capture differences in patient perceptions by treatment not all were sensitive to expected differences as to reporting psoriasis or age. This highlight the value of letting the choice of instrument match the decision problem. For reimbursement decisions, measures picking up differences by treatment group and direct disease related aspects such as skin problems seem important. This is also important knowledge in cost-effectiveness analyses where choice of effectiveness measure may have large impact on the ratio between costs and effects. The finding that patients with irregular bDMARD treatment seem to have worse health than those with long-term regular or no bDMARD treatment at all will be further investigated. Disclosure of Interest None declared

FRI0368 Incremental Costs Per Patient for Psoriasis and Psoriatic Arthritis in a Population-Based Referent Cohort in Southern Sweden: Are there Clear Links to Psoriasis Morbidity?

Background There is a need for more detailed information on the resource use across different cost components for patients with psoriasis alone (PSO) and psoriasis patients with psoriatic arthritis (PsA) from a population-based perspective, especially after the introduction of biological treatment for these groups. Objectives To study annual incremental societal costs for patients with PSO and PsA compared to population-based referents free from PSO and PsA years 2008-2011. Methods Patients of any age were identified by ICD-10 codes associated with PSO and PsA using information from 1998 to 2007 in the Skåne Healthcare Register (SHR), which covers all healthcare utilization by the Skåne Region population (total N=1.2 million 2011). For each included PSO/PsA patient, three population-based referents, matched for age, sex and residential area, were selected. Data included demographics, all-cause healthcare utilization by cost components (primary care, secondary outpatient care, and inpatient care) and caregivers (physician, nurse, physiotherapist, other), drugs and productivity losses. Resource use and costs were retrieved from SHR (healthcare), Swedish Prescribed Drug Register (drugs) and Social Insurance Register (productivity losses). We calculated the mean annual cost per patient over four years (2008-2011) adjusted for cases and referents dropping out of the study (relocation from the region or death). The human capital method was used to value productivity losses. Costs were assessed from a societal perspective and expressed in Euros (2011 prices). Differences in costs were tested by Students t -test. A P-value less than 0.05 was considered statistically significant. Results We identified 15 283 patients (51% women, mean age (SD) 52 (19)) who fulfilled the inclusion criteria for PSO (n=12 562, 50% woman, mean age 52 (20)) or PsA (n=2 721, 56% women, mean age 54 (16)) and included 45 849 referents. Mean annual societal cost per patient with PSO and PsA was 56% higher compared to referents, € 11 146 vs. € 7 132 (p<0.001). The cost was 84% higher for PsA compared to PSO, € 17 853 vs. € 9 693 (p<0.001). Costs due to productivity losses represented the largest share of the annual mean societal costs in all groups with the highest share for PsA (59%). Almost 25% of the costs were attributable to inpatient care for individuals with PSO and referents. The corresponding figure for PsA was 12%. Annual mean drug cost represented 15% (biologics 10%) of the costs for PsA. The corresponding figures for PSO was 9% (biologics 1.5%) and for referents 7% (biologics <1%). Conclusions The annual mean incremental societal cost per patient was highest for PsA, mainly due productivity losses and biological treatment. Patients with PSO had higher costs for inpatient care compared to PsA patients. The impact of comorbidities on these costs should be further studied. Disclosure of Interest None declared