Dataset Information

Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

ABSTRACT: Concerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.We conducted a retrospective, propensity score matched study of publicly-listed orphan companies. Cases were defined as holders of orphan drug market authorisation in Europe or the USA between 2000-12. Control companies were selected based on their propensity for being orphan drug market authorisation holders. We applied system General Method of Moments to test whether companies with orphan drug market authorization are valued higher, as measured by the Tobin's Q and market to book value ratios, and are more profitable based on return on assets, than non-orphan drug companies.86 companies with orphan drug approvals in European (4), USA (61) or both (21) markets were matched with 258 controls. Following adjustment, orphan drug market authorization holders have a 9.6% (95% confidence interval, 0.6% to 18.7%) higher return on assets than non-orphan drug companies; Tobin's Q was higher by 9.9% (1.0% to 19.7%); market to book value by 15.7% (3.1% to 30.0%) and operating profit by 516% (CI 19.8% to 1011%). For each additional orphan drug sold, return on assets increased by 11.1% (0.6% to 21.3%), Tobin's Q by 2.7% (0.2% to 5.2%), and market to book value ratio by 5.8% (0.7% to 10.9%).Publicly listed pharmaceutical companies that are orphan drug market authorization holders are associated with higher market value and greater profits than companies not producing treatments for rare diseases.

SUBMITTER: Hughes DA

PROVIDER: S-EPMC5074462 | biostudies-other | 2016

REPOSITORIES: biostudies-other

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Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

Hughes Dyfrig A DA Poletti-Hughes Jannine J

PloS one 20161021 10

<h4>Background</h4>Concerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.<h4>Methods</h4>We conducted a retrospective, propensity score matched study of publicly-listed orphan companies. Cases were defined as holders of orphan drug market authorisation in Europe or the USA between 2000-12. Control companies were selected based on their propensity for being orphan drug market authorisati ...[more]

PMID: 27768685

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Project description:IntroductionRecommendations for venous thromboembolism and deep venous thrombosis (DVT) prophylaxis using graduated compression stockings (GCS) is historically based and has been critically examined in current publications. Existing guidelines are inconclusive as to recommend the general use of GCS.Patients/Methods: 24 273 in-patients (general surgery and orthopedic patients) undergoing surgery between 2006 and 2016 were included in a retrospectively analysis from a single center. From January 2006 to January 2011 perioperative GCS was employed additionally to drug prophylaxis and from February 2011 to March 2016 patients received drug prophylaxis alone. According to german guidelines all patients received venous thromboembolism prophylaxis with weight-adapted LMWH. Risk stratification (low risk, moderate risk, high risk) was based on the guideline of the American College of Chest Physicians. Data analysis was performed before and after propensity matching (PM). The defined primary endpoint was the incidence of symptomatic or fatal pulmonary embolism (PE). A secondary endpoint was the incidence of deep venous thromboembolism (DVT).ResultsAfter risk stratification (low risk n = 16 483; moderate risk n = 4464; high risk n = 3326) a total of 24 273 patient were analyzed. Before to PM the relative risk for the occurrence of a PE or DVT was not increased by abstaining from GCS. After PM two groups of 11 312 patients each, one with and one without GCS application, were formed. When comparing the two groups, the relative risk (RR) for the occurrence of a pulmonary embolism was: Low Risk 0.99 [CI95% 0.998-1.000]; Moderate Risk 0.999 [CI95% 0.95-1.003]; High Risk 0.996 [CI95% 0.992-1.000] (p > 0.05). The incidence of PE in the total group LMWH alone was 0.1% (n = 16). In the total group using LMWH + GCS, the incidence was 0.3% (n = 29). RR after PM was 0.999 [CI95% 0.998-1.00].ConclusionIn comparison to prior studies with only small numbers of patients our trial shows in a large group of patients with moderate and high risk developing VTE we can support the view that abstaining from GCS-use does not increase the incidence of symptomatic or fatal PE and symptomatic DVT.

Project description:BackgroundPrevious studies showed bacterial lysates were effective for pediatric asthma. However, evidence of polyvalent bacterial lysate Qipian is lacking.MethodsIn this real-world retrospective cohort study, data of children with asthma, aged six months to 14 years old, attending to Jiangxi Provincial Children's Hospital from January 2021 to April 2022, prescribed routine treatment for asthma plus Qipian (Qipian group) or not (control group) were extracted. To minimize the impact of confounders on the outcomes, baseline characteristics were utilized to perform propensity score matching through a multivariable logistic regression model. After matching, asthma control, exacerbation, etc. were compared.ResultsTotally, 795 patients were included (337 in the Qipian group and 458 in the control group), with 278 pairs (556 patients) matched. Most baseline characteristics were well-balanced. The proportion of males were 68.3% and 70.1% in the two groups. The Qipian group favored better asthma control, with more "controlled" [3-month: 257 (92.4%) vs. 240 (86.3%); 6-month: 246 (88.5%) vs. 235 (84.5%)], and fewer "poorly/very poorly controlled" patients, compared with the control group (P=0.004 and 0.025, respectively). Patients in the Qipian group had lower risks of exacerbation. Incidence rate ratios (IRR) for any exacerbation were 0.56 [95% confidence interval (CI): 0.33 to 0.93] in the 3-month period and 0.83 (95% CI: 0.55 to 1.26) in the 6-month period. IRR for severe exacerbations were 0.09 (95% CI: 0.01 to 0.71) in the 3-month period and 0.20 (95% CI: 0.06 to 0.70) in the 6-month period (compared to the control group). Qipian significantly reduced the cumulative dose of short-acting beta-agonist (3-month: 3.22±10.37 vs. 8.08±16.71 mg; P<0.001; 6-month: 6.56±16.23 vs. 11.81±24.41 mg; P=0.002). There was no difference in incidences of respiratory tract infection or fever due to respiratory tract infection between the two groups. Numbers of antibacterial agent prescription were fewer in the Qipian group compared to the control group (3-month: 0.67±1.16 vs. 1.04±1.45; P=0.001; 6-month: 1.14±1.69 vs. 1.51±2.12; P=0.023).ConclusionsAccording to this retrospective study, Qipian may be effective for improved pediatric asthma control. Safety profile and mechanisms of action of Qipian need further investigation. Further randomized controlled trials are warranted to confirm our results.

Project description:BackgroundCytokine release syndrome (CRS) plays a pivotal role in the pathophysiology and progression of Coronavirus disease-2019 (COVID-19). Therapeutic plasma exchange (TPE) by removing the pathogenic cytokines is hypothesized to dampen CRS.ObjectiveTo evaluate the outcomes of the patients with COVID-19 having CRS being treated with TPE compared to controls on the standard of care.MethodologyRetrospective propensity score-matched analysis in a single centre from 1st April to 31st July 2020. We retrospectively analyzed data of 280 hospitalized patients developing CRS initially. PSM was used to minimize bias from non-randomized treatment assignment. Using PSM 1:1, 90 patients were selected and assigned to 2 equal groups. Forced matching was done for disease severity, routine standard care and advanced supportive care. Many other Co-variates were matched. Primary outcome was 28 days overall survival. Secondary outcomes were duration of hospitalization, CRS resolution time and timing of viral clearance on Polymerase chain reaction testing.ResultsAfter PS-matching, the selected cohort had a median age of 60 years (range 32-73 in TPE, 37-75 in controls), p = 0.325 and all were males. Median symptoms duration was 7 days (range 3-22 days' TPE and 3-20 days controls), p = 0.266. Disease severity in both groups was 6 (6.6%) moderate, 40 (44.4%) severe and 44 (49%) critical. Overall, 28-day survival was significantly superior in the TPE group (91.1%), 95% CI 78.33-97.76; as compared to PS-matched controls (61.5%), 95% CI 51.29-78.76 (log rank 0.002), p<0.001. Median duration of hospitalization was significantly reduced in the TPE treated group (10 days vs 15 days) (p< 0.01). CRS resolution time was also significantly reduced in the TPE group (6 days vs. 12 days) (p< 0.001). In 71 patients who underwent TPE, the mortality was 0 (n = 43) if TPE was done within the first 12 days of illness while it was 17.9% (deaths 5, n = 28 who received it after 12th day (p = 0.0045).ConclusionAn earlier use of TPE was associated with improved overall survival, early CRS resolution and time to discharge compared to SOC for COVID-19 triggered CRS in this selected cohort of PS-matched male patients from one major hospital in Pakistan.

Project description:ObjectivesThis study investigated patterns in health service usage among older adults with dementia and matched controls over a 10-year span from 5 years before until 5 years after diagnosis.DesignPopulation-based retrospective matched case-control study.SettingAdministrative health data of individuals in Saskatchewan, Canada from 1 April 2008 to 31 March 2019.ParticipantsThe study included 2024 adults aged 65 years and older living in the community at the time of dementia diagnosis from 1 April 2013 to 31 March 2014, matched 1:1 to individuals without a dementia diagnosis on age group, sex, rural versus urban residence, geographical region and comorbidity.Outcome measuresFor each 5-year period before and after diagnosis, we examined usage of health services each year including family physician (FP) visits, specialist visits, hospital admissions, all-type prescription drug dispensations and short-term care admissions. We used negative binomial regression to estimate the effect of dementia on yearly average health service utilisation adjusting for sex, age group, rural versus urban residence, geographical region, 1 year prior health service use and comorbidity.ResultsAdjusted findings demonstrated that 5 years before diagnosis, usage of all health services except hospitalisation was lower among persons with dementia than persons without dementia (all p<0.001). After this point, differences in higher health service usage among persons with dementia compared to without dementia were greatest in the year before and year after diagnosis. In the year before diagnosis, specialist visits were 59.7% higher (p<0.001) and hospitalisations 90.5% higher (p<0.001). In the year after diagnosis, FP visits were 70.0% higher (p<0.001) and all-type drug prescriptions 29.1% higher (p<0.001).ConclusionsFindings suggest the year before and year after diagnosis offer multiple opportunities to implement quality supports. FPs are integral to dementia care and require effective resources to properly serve this population.

Project description:BackgroundMobile-based interventions appear to be promising in ameliorating huge burdens experienced by patients with type 2 diabetes. However, it is unclear how effective mobile-based interventions are in glycemic management of patients with type 2 diabetes based on real-world evidence.ObjectiveThis study aimed to evaluate the effectiveness of a mobile-based intervention on glycemic control in patients with type 2 diabetes based on real-world population data.MethodsThis retrospective, propensity score-matched cohort study analyzed longitudinal data from a clinical electronic health database. The study population included 37,913 patients with type 2 diabetes at cohort entry between October 1, 2016, and July 31, 2018. A total of 2400 patients were matched 1:1, using propensity score matching, into the usual care and mobile health (mHealth) groups. The primary outcomes of glycemic control included control rates of glycated hemoglobin (HbA1c), fasting blood glucose (FBG), and postprandial 2-hour blood glucose (P2BG). Mean values and variation trends of difference with 95% CI were the secondary outcomes. The general linear model was used to calculate repeated-measures analyses of variance to examine the differences between the two groups. Subgroup and sensitivity analyses were performed.ResultsOf the 2400 patients included in the analysis, 1440 (60.00%) were male and the mean age was 52.24 years (SD 11.56). At baseline, the control rates of HbA1c, FBG, and P2BG in the mHealth and usual care groups were 45.75% versus 47.00% (P=.57), 38.03% versus 32.76% (P=.07), and 47.32% versus 47.89% (P=.83), respectively. At the 3-, 6-, 9-, and 12-month follow-ups, the mHealth group reported higher control rates of HbA1c than did the usual care group: 69.97% versus 46.06% (P<.001), 71.89% versus 61.24% (P=.004), 75.38% versus 53.44% (P<.001), and 72.31% versus 46.70% (P<.001), respectively. At the four follow-up sessions, the control rates of FBG in the mHealth and usual care groups were statistically different: 59.24% versus 34.21% (P<.001), 56.61% versus 35.14% (P<.001), 59.54% versus 34.99% (P<.001), and 59.77% versus 32.83% (P<.001), respectively. At the four follow-up sessions, the control rates of P2BG in the mHealth group were statistically higher than in the usual care group: 79.72% versus 48.75% (P<.001), 80.20% versus 57.45% (P<.001), 81.97% versus 54.07% (P<.001), and 76.19% versus 54.21% (P=.001), respectively. At the four follow-up sessions, the percentages of HbA1c reduction in the mHealth group were 8.66% (95% CI 6.69-10.63), 10.60% (95% CI 8.66-12.54), 10.64% (95% CI 8.70-12.58), and 8.11% (95% CI 6.08-10.14), respectively. At the four follow-up sessions, the percentages of P2BG reduction in the mHealth group were 8.44% (95% CI 7.41-10.73), 17.77% (95% CI 14.98-20.23), 16.23% (95% CI 13.05-19.35), and 16.91% (95% CI 13.17-19.84), respectively. Starting from the sixth month, the mean HbA1c and P2BG values in the two groups increased slightly.ConclusionsThis mobile-based intervention delivered by a multidisciplinary team can better improve glycemic control rates of patients with type 2 diabetes than usual care. These effects were best sustained within the first 6 months. Starting from the sixth month, intensive management needs to be conducted to maintain long-term effectiveness of the mobile-based intervention.

Dataset Information

Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

Publications

Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

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