scholarly journals The IDeaS initiative: pilot study to assess the impact of rare diseases on patients and healthcare systems

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Ainslie Tisdale ◽  
Christine M. Cutillo ◽  
Ramaa Nathan ◽  
Pierantonio Russo ◽  
Bryan Laraway ◽  
...  

Abstract Background Rare diseases (RD) are a diverse collection of more than 7–10,000 different disorders, most of which affect a small number of people per disease. Because of their rarity and fragmentation of patients across thousands of different disorders, the medical needs of RD patients are not well recognized or quantified in healthcare systems (HCS). Methodology We performed a pilot IDeaS study, where we attempted to quantify the number of RD patients and the direct medical costs of 14 representative RD within 4 different HCS databases and performed a preliminary analysis of the diagnostic journey for selected RD patients. Results The overall findings were notable for: (1) RD patients are difficult to quantify in HCS using ICD coding search criteria, which likely results in under-counting and under-estimation of their true impact to HCS; (2) per patient direct medical costs of RD are high, estimated to be around three–fivefold higher than age-matched controls; and (3) preliminary evidence shows that diagnostic journeys are likely prolonged in many patients, and may result in progressive, irreversible, and costly complications of their disease Conclusions The results of this small pilot suggest that RD have high medical burdens to patients and HCS, and collectively represent a major impact to the public health. Machine-learning strategies applied to HCS databases and medical records using sentinel disease and patient characteristics may hold promise for faster and more accurate diagnosis for many RD patients and should be explored to help address the high unmet medical needs of RD patients.

2020 ◽  
Vol 222 (7) ◽  
pp. 1138-1144 ◽  
Author(s):  
Sarah M Bartsch ◽  
Elizabeth A Mitgang ◽  
Gail Geller ◽  
Sarah N Cox ◽  
Kelly J O’Shea ◽  
...  

Abstract Background The protection that an influenza vaccine offers can vary significantly from person to person due to differences in immune systems, body types, and other factors. The question, then, is what is the value of efforts to reduce this variability such as making vaccines more personalized and tailored to individuals. Methods We developed a compartment model of the United States to simulate different influenza seasons and the impact of reducing the variability in responses to the influenza vaccine across the population. Results Going from a vaccine that varied in efficacy (0–30%) to one that had a uniform 30% efficacy for everyone averted 16.0–31.2 million cases, $1.9–$3.6 billion in direct medical costs, and $16.1–$42.7 billion in productivity losses. Going from 0–50% in efficacy to just 50% for everyone averted 27.7–38.6 million cases, $3.3–$4.6 billion in direct medical costs, and $28.8–$57.4 billion in productivity losses. Going from 0–70% to 70% averted 33.6–54.1 million cases, $4.0–$6.5 billion in direct medical costs, and $44.8–$64.7 billion in productivity losses. Conclusions This study quantifies for policy makers, funders, and vaccine developers and manufacturers the potential impact of efforts to reduce variability in the protection that influenza vaccines offer (eg, developing vaccines that are more personalized to different individual factors).


Lupus ◽  
2018 ◽  
Vol 27 (8) ◽  
pp. 1247-1258 ◽  
Author(s):  
N McCormick ◽  
C A Marra ◽  
M Sadatsafavi ◽  
J A Aviña-Zubieta

Objective We estimated the incremental (extra) direct medical costs of a population-based cohort of newly diagnosed systemic lupus erythematosus (SLE) for five years before and after diagnosis, and the impact of sex and socioeconomic status (SES) on pre-index costs for SLE. Methods We identified all adults newly diagnosed with SLE over 2001–2010 in British Columbia, Canada, and obtained a sample of non-SLE individuals from the general population, matched on sex, age, and calendar-year of study entry. We captured costs for all outpatient encounters, hospitalisations, and dispensed medications each year. Using generalised linear models, we estimated incremental costs of SLE each year before/after diagnosis (difference in costs between SLE and non-SLE, controlling for covariates). Similar models were used to examine the impact of sex and SES on costs within SLE. Results We included 3632 newly diagnosed SLE (86% female, mean age 49.6 ± 15.9) and 18,060 non-SLE individuals. Over the five years leading up to diagnosis, per-person healthcare costs for SLE patients increased year-over-year by 35%, on average, with the biggest increases in the final two years by 39% and 97%, respectively. Per-person all-cause medical costs for SLE the year after diagnosis (Year + 1) averaged C$12,019 (2013 Canadian) with 58% from hospitalisations, 24% outpatient, and 18% from prescription medications; Year + 1 costs for non-SLE averaged C$2412. Following adjustment for age, sex, urban/rural residence, socioeconomic status, and prior year's comorbidity score, SLE was associated with significantly greater hospitalisation, outpatient, and medication costs than non-SLE in each year of study. Altogether, adjusted incremental costs of SLE rose from C$1131 per person in Year –5 (fifth year before diagnosis) to C$2015 (Year –2), C$3473 (Year –1) and C$6474 (Year + 1). In Years –2, –1 and +1, SLE patients in the lowest SES group had significantly greater costs than the highest SES. Unlike the non-SLE cohort, male patients with SLE had higher costs than females. Annual incremental costs of SLE males (vs. SLE females) rose from C$540 per person in Year –2, to C$1385 in Year –1, and C$2288 in Year + 1. Conclusion Even years before diagnosis, SLE patients incur significantly elevated direct medical costs compared with the age- and sex-matched general population, for hospitalisations, outpatient care, and medications.


2016 ◽  
Vol 34 (2_suppl) ◽  
pp. 81-81
Author(s):  
Tracy Li ◽  
Neal D. Shore ◽  
Maneesha Mehra ◽  
Mary Beth Todd ◽  
Ryan Saadi ◽  
...  

81 Background: The objective of this study was to estimate the direct medical costs of secondary metastases in prostate cancer (PC) patients initially diagnosed with locoregional disease. Methods: We used data from the United States Surveillance, Epidemiology, and End Results (SEER) cancer registry linked to Medicare claims to identify a cohort of PC patients initially diagnosed with locoregional disease between 2000 and 2011, who were age ≥ 66 at diagnosis, and who first had a diagnosis of metastasis (index date) ≥ 4 months after PC diagnosis (cases). We matched each case to up to four controls (patients without metastasis) on baseline patient characteristics to assess the incremental impact of developing metastasis. A “match date” corresponding to the index date of the case was constructed for each control. Cases and controls were followed from up to 12 months before and up to 12 months after metastasis. Medicare claims were used to calculate the average total cost per month in the cases and controls. Monthly costs also were stratified according to place and type of service, e.g. inpatient, outpatient, and by whether costs pertained to cancer services, i.e. chemotherapy or androgen deprivation therapy (ADT), other services with an ICD-9 code for cancer, or other care. Results: The cohort consisted of 10,370 cases and 39,200 controls, with a mean age at baseline of 79 years in both groups. The median time to subsequent metastasis (cases) was 37 months and 85% had bone metastasis. In the control group, total costs remained stable throughout the observation period, averaging $1,354/month before and $1,173 12/month after matched index date. In the cases, costs were similar to the controls from -12 to -6 months before the index date. Thereafter, medical costs rose sharply to $11,982 in the month of diagnosis of metastasis (index date), and then declined but stayed > $2,000/month higher than the control for the remainder of the observation period. Expenses from inpatient, physician services, outpatient, and skilled nursing facility accounted for the largest proportions of total costs. Conclusions: Developing metastasis in Medicare PC patients results in substantial additional costs to the healthcare system.


2009 ◽  
Vol 51 (2) ◽  
pp. 180-184 ◽  
Author(s):  
Bradley C. Martin ◽  
Timothy S. Church ◽  
Ron Bonnell ◽  
Rami Ben-Joseph ◽  
Ted Borgstadt

Author(s):  
Wenjia Chen ◽  
Hamid Tavakoli ◽  
J FitzGerald ◽  
Padmaja Subbarao ◽  
Turvey Stuart ◽  
...  

Background: Quantifying age trends in healthcare costs of pediatric asthma leads to better understanding of the natural history of the disease and informed decision-making on the allocation of healthcare resources. Methods: We identified children with incident asthma from the health administrative data of British Columbia, Canada (Jan 1998 to Dec 2015), and followed them from their first diagnosis of asthma or wheezing until age 18. We estimated direct medical costs (in 2016 Canadian dollars [$]), including inpatient and outpatient encounters and pharmacy costs, attributed to asthma (primary outcome) and other respiratory diseases (secondary outcome). We assessed the impact of sex and socioeconomic status on age trends, adjusting for calendar effect. Results: The final analysis included 44,552 children with asthma (62% boys). From age 0 to 18, costs of asthma/wheezing and other respiratory conditions decreased from $1,036 to $29/child-year, and from $1,145 to $31/child-year, respectively. Children under 3 years of age incurred 4–fold higher costs for asthma/wheezing and other respiratory conditions. In particular, costs of asthma hospitalizations were 10 times higher in this age group compared to older children. Age trends were generally similar between sex groups and across socioeconomic status. However, medication costs for asthma/wheezing decreased in boys, whereas those in girls declined during childhood but increased during adolescence. Conclusions: The highest costs of pediatric asthma are concentrated in children younger than 3. Age trends were generally consistent between sex and across socioeconomic status.


2020 ◽  
Vol 49 (10) ◽  
pp. 731-741
Author(s):  
Gwyneth J Lim ◽  
Yan Lun Liu ◽  
Serena Low ◽  
Keven Ang ◽  
Subramaniam Tavintharan ◽  
...  

Introduction: This was a retrospective cross-sectional study to assess the impact of chronic kidney disease (CKD) and its severity in Type 2 diabetes mellitus (T2DM) on direct medical costs, and the effects of economic burden on CKD related complications in T2DM in Singapore. Methods: A total of 1,275 T2DM patients were recruited by the diabetes centre at Khoo Teck Puat Hospital from 2011–2014. CKD stages were classified based on improving global outcome (KDIGO) categories, namely the estimated glomerular filtration rate (eGFR) and albuminuria kidney disease. Medical costs were extracted from the hospital administrative database. Results: CKD occurred in 57.3% of patients. The total mean cost ratio for CKD relative to non-CKD was 2.2 (P<0.001). Mean (median) baseline annual unadjusted costs were significantly higher with increasing CKD severity—S$1,523 (S$949), S$2,065 (S$1,198), S$3,502 (S$1,613), and S$5,328 (S$2,556) for low, moderate, high, and very high risk respectively (P<0.001). CKD (P<0.001), age at study entry (P=0.001), Malay ethnicity (P=0.035), duration of diabetes mellitus (DM; P<0.001), use of statins/fibrates (P=0.021), and modified Diabetes Complications Severity Index (DCSI) (P<0.001) were positively associated with mean annual direct medical costs in the univariate analysis. In the fully adjusted model, association with mean annual total costs persisted for CKD, CKD severity and modified DCSI. Conclusion: The presence and increased severity of CKD is significantly associated with higher direct medical costs in T2DM patients. Actively preventing the occurrence and progression in DM-induced CKD may significantly reduce healthcare resource consumption and healthcare costs. Keywords: Chronic kidney disease, costs, endocrinology, nephrology


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