Variables affecting pricing of orphan drugs: the Italian case

Background and aim Evidence on determinants of prices for orphan medicines is scarce and not available for Italy. The aim of this paper is to provide an evidence on variables affecting the annual treatment cost of orphan drugs in Italy, testing the hypothesis of a negative correlation with the dimension of the target population and a positive correlation with the added therapeutic value of the drug and the quality of the evidence of pivotal studies. Methods Drugs with a European orphan designation reimbursed in Italy in the last 6 years (2014–2019) were considered. Univariate, cluster analysis and multiple regression models were used to investigate the correlation between the annual treatment cost and, as explanatory variables, the dimension of the target population, the existence of Randomized Clinical Trials as a proxy of the quality of the pivotal studies, the added therapeutic value. Results In the univariate analysis prevalence and added therapeutic value, as expected, have a negative and positive correlation with cost respectively. The correlation with RCT is not significant. In the multivariate model, coefficients for prevalence and added value are confirmed but for the latter are not significant anymore. We also found, through an interaction analysis, that the existence of an RCT has a positive impact on annual treatment cost when the target population is very small. Conclusions Our results suggest that value arguments and sustainability (dimension of the target population and its impact on budget impact) issues are considered for orphan drugs pricing: the role played by sustainability is systematically supported by our results. A more transparent and reproducible price negotiation process for orphan drugs is needed in Italy. This paper has contributed to highlight the implicit drivers of this process.

Treatment cost of HIV/AIDS in Turkey

PurposeThe purpose of this study is to estimate the average cost of treatment and investigate the related parameters of HIV/AIDS among patients based on their annual treatment regime during the 2017 in Izmir.Design/methodology/approachThe average annual direct cost of an HIV patient's treatment was estimated for 2017 at four university hospitals in a retrospective study in Izmir, Turkey. Inclusion criteria included confirmed HIV infection, age = 18 years, visited one of the hospitals at least three times a year and with at least one CD4+ T cell count. The average annual treatment cost per patient was calculated using accounting data for 527 patients from the hospitals' electronic databases.FindingsThe mean treatment cost per patient was US$4,381.93. Costs for treatment and care were statistically significantly higher (US$5,970.55) for patients with CD4+ T cell counts of fewer than 200 cells/mm3 than for other patients with CD4+ T cell counts above 200 cells/mm3. The mean treatment cost for patients who were 50 years old or older (US$4,904.24) was statistically significantly higher than for those younger than 50 years (US$4,216.10). The mean treatment cost for female patients (US$4,624.92) was higher than that of male patients ($US4,339.72), although the difference was not statistically significant. The main cost driver was antiretroviral treatment (US$3,852.38 per patient), accounting for almost 88% of all costs. However, the high burden of antiretroviral treatment cost is counterbalanced by relatively low care and hospitalization costs in Turkey.Originality/valueThe paper contributes to the literature by providing average annual treatment cost of an HIV-infected patient in Turkey by using a comprehensive bottom up approach. Moreover, cost drivers of HIV treatment are investigated.

Clostridioides difficile Spores: Bile Acid Sensors and Trojan Horses of Transmission

The Gram-positive, spore-forming bacterium, Clostridioides difficile is the leading cause of healthcare-associated infections in the United States, although it also causes a significant number of community-acquired infections. C. difficile infections, which range in severity from mild diarrhea to toxic megacolon, cost more to treat than matched infections, with an annual treatment cost of approximately $6 billion for almost half-a-million infections. These high–treatment costs are due to the high rates of C. difficile disease recurrence (>20%) and necessity for special disinfection measures. These complications arise in part because C. difficile makes metabolically dormant spores, which are the major infectious particle of this obligate anaerobe. These seemingly inanimate life forms are inert to antibiotics, resistant to commonly used disinfectants, readily disseminated, and capable of surviving in the environment for a long period of time. However, upon sensing specific bile salts in the vertebrate gut, C. difficile spores transform back into the vegetative cells that are responsible for causing disease. This review discusses how spores are ideal vectors for disease transmission and how antibiotics modulate this process. We also describe the resistance properties of spores and how they create challenges eradicating spores, as well as promote their spread. Lastly, environmental reservoirs of C. difficile spores and strategies for destroying them particularly in health care environments will be discussed.

Adherence to COPD treatment increase: is it possible to reduce the cost of treatment?

Annotation. Chronic obstructive pulmonary disease (COPD) makes a significant economic burden for the patients, their families and for the healthcare systems of different countries in general. At the same time the level of adherence to basis therapy is 30–40%. Aim of the study is to determine cost effectiveness of the measures for adherence to COPD treatment increase. 156 patients with COPD (III–IV GOLD) were included into study. The duration of the study was 13 months and it consisted of 6 visits. During each visit the adherence to treatment was measured according to value of inhaler dose counter. The instruction regarding necessity to adhere to basis therapy regimen as well as training of correct inhaler use technique were conducted. The costs for the patient with COPD treatment were calculated for 1 year before the start of the patients’ participation in the study (first year) and during their participation in the study (second year). The average value and standard error of average value were calculated for all the parameters. The comparison of parameters of intergroup differences was made with use of T-test for dependent samples and criterion χ2. It is shown that the adherence to COPD treatment rose from 44.41±1.07% to 75.21±0.78%, i.e. by 30.8% (p<0.001). On the background of adherence increase total cost for the patients treatment due to COPD diminished from 20304.88 ± 976.68 UAH to 9258.13 ± 507.90 UAH, i.e. by 54.4% during the year. Thus, adherence to COPD treatment increase permits to diminish the annual treatment cost for this disease significantly (by 54.4%). Detection of patients' visits frequency to medical institution in order to maintain the level of adherence to treatment which was already achieved has the prospect of further research.

Evaluating Costs of Biologic Drugs for the Treatment of Moderate-to-Severe Psoriasis in the United States

Objective:To provide a contemporary comparative assessment of biologic drug costs and cost effectiveness for treating moderate-to-severe psoriasis in the United States.Methods:A literature search was conducted in PubMed on October 4, 2018 with the search terms of “psoriasis,” “biologic,” and “cost” with a filter of publication dates within the last 5 years (2014-2018). Studies included in this review were required to be conducted from a United States perspective.Results:Of the 16 studies retrieved, the costs associated with use of biologic drugs for the treatment of moderate-to-severe psoriasis were relatively high, with some drugs exhibiting sustained lower costs than others. Some comparative findings on the cost-effectiveness of biologic drugs based on the annual treatment cost per Psoriasis Area and Severity Index (PASI) 75 responder concluded that the annual treatment cost was lowest for brodalumab 210 mg at US$48 782; the cost was highest for ustekinumab 45/90 mg (US$87 243), followed by adalimumab 40 mg (US$82 655), ixekizumab 160 mg (US$77 957), and secukinumab 300 mg (US$75 671). The annual treatment cost per PASI 100 responder was also lowest for brodalumab at US$87 585. This pattern remained consistent in other cost studies, with brodalumab being the treatment with the lowest treatment costs when compared to other biologic drugs.Conclusion:Brodalumab was consistently observed to be the least costly treatment option among biologic drugs used to treat moderate-to-severe psoriasis in the United States, which is largely the result of its low-drug cost and high-PASI response rate.

Acute hand injury splinting – the good, the bad and the ugly

Injuries to the hand comprise 20% of all emergency department attendances, with an estimated annual treatment cost of over £100 million in the UK. The initial assessment and management of hand injuries is usually undertaken by junior staff, many of whom have little or no training or experience in splinting hand fractures. In the Department of Orthopaedic Hand Surgery, Morriston Hospital, we regularly observe patients presenting to the specialist hand fracture clinics having had initial management that shows no appreciation for the treatment objectives or the safe positions for splinting. This article aims to provide guidance for frontline staff on the management of hand fractures, with particular emphasis on the appropriate nonoperative care to avoid any unnecessary morbidity.

Evaluation of the cost-effectiveness of buprenorphine in treatment of chronic pain using competing EQ-5D weights

Background and aimsChronic pain is a life altering condition and common among elderly persons. The 7-day buprenorphine patch could be a suitable treatment for managing chronic pain of moderate severity in elderly patients in Sweden.The objective of this analysis was to investigate the cost-effectiveness of the 7-day buprenorphine patch, versus no treatment, in patients >50 years old who suffer from moderate pain in a health economic perspective. An additional aim was to evaluate how the cost-effectiveness is affected by the choice of EQ-5D weights.MethodsThe annual treatment cost and the potential gains in health-related quality of life (HRQoL) of buprenorphine, compared to no treatment, were evaluated. Original EQ-5D data were collected from four clinical reference studies at baseline and at the final visit. Treatment effects on HRQoL were then assessed using both UK and Swedish EQ-5D weights. Annual treatment costs were calculated based on costs of physician visits and pharmaceuticals.The optimal treatment dose was 10-15 μg/h and the analysis was hence performed on both a 10- and a 15 μg/h buprenorphine patch.ResultsThe analysis of buprenorphine treatment resulted in improved HRQoL in all reference studies, irrespective of choice of EQ-5D weight set. The change in quality adjusted life years (QALYs) varied with a gain of 0.042-0.118 using the UK weights and 0.020-0.051 with the Swedish weights. The average annual treatment cost was SEK14454 for the 10μg/h patch and SEK17 017 for the 15 μg/h patch, while cost for the no-treatment alternative was SEK 9 960. The base case incremental cost-effectiveness ratios (ICER) with the UK weights were SEK 40000-SEK 170000 and SEK 90000-SEK 350000 when applying the Swedish weights. The corresponding ICER-span in the sensitivity analysis was SEK 15 000-SEK 400 000 when applying the UK weights and SEK 30 000-SEK 840 000 with the Swedish weights (SEK 100 is about €11).ConclusionsThe results imply that the 7-day buprenorphine patch may be a cost-effective treatment of moderate chronic pain in patients over 50 years of age. The UK and the Swedish EQ-5D weights generated vastly different HRQoL estimates but buprenorphine remains cost-effective regardless choice of weight set.

Biologics in the Treatment of Psoriasis: Clinical and Economic Overview

Background: The use of biologic medications for psoriasis is a recent therapeutic advance. Objective: To review clinical and economic data for biologic therapies in the treatment of chronic plaque psoriasis. Methods: Published meta-analyses and additional literature review identified randomized controlled trials. Analyses included the number needed to treat (NNT), the cost in Canadian dollars for the first year of treatment per the Canadian product monograph, and the estimated cost per responder achieving a 75% reduction in Psoriasis Area and Severity Index score (PASI 75). Results: Pooled NNTs were 1.3 (infliximab), 1.5 (ustekinumab 90 mg), 1.6 (adalimumab and ustekinumab 45 mg), 2.3 (etanercept), 4.1 (efalizumab), and 5.6 (alefacept). The annual treatment cost per patient was $19,825 to $37,600. The cost per patient achieving a PASI 75 response at 12 weeks ranged from $8,330 (adalimumab) to $71,371 (alefacept). Conclusion: This analysis suggests favorable cost and benefits of biologic psoriasis therapies, particularly adalimumab, infliximab, and ustekinumab.