Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT03522623 |
| Other study ID # |
HS20851(H2017:189) |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
November 1, 2017 |
| Est. completion date |
May 1, 2020 |
Study information
| Verified date |
October 2018 |
| Source |
University of Manitoba |
| Contact |
WAEL EL-MATARY, MD |
| Phone |
2047871039 |
| Email |
welmatary[@]HSC.MB.CA |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
inflammation of the gastrointestinal tract. A recent Canadian study from found that Canada
has amongst highest incidence rates of childhood-onset IBD (10 per 100,000 for children
<16y). In 2012, Crohn's and Colitis Canada estimated that direct medical costs of IBD in
Canada were >$1 billion, and estimated indirect costs amounting to $1.8 billion. An American
study demonstrated the direct costs of caring for children with IBD was double those for
adults. Indirect health care costs in children with IBD have not been well-described. The
Canadian Gastro-Intestinal Epidemiology Consortium (CanGIEC) is a pan-Canadian network of new
and established IBD clinician-researchers and methodologists from 6 provinces experienced in
the use of health administrative data. CanGIEC is evaluating variation in care of children
with IBD, and will expand this research stream to assess direct and indirect cost of care.
This will involve a collaboration with the CIHR/CHILD Foundation Canadian Children IBD
Network (CIDsCaNN), which comprises an inception cohort of children diagnosed at all 12
pediatric IBD centres across Canada.
Hypothesis: Direct health costs are dominated by medication expenses (particularly
biologics), with resulting variation within and across provinces in costs and out-of-pocket
expenses to the families due to coverage disparity. Indirect costs include school and
parental absenteeism, and productivity losses.
Aims:
1. Determine the cost of care of children with IBD, incurred by caregivers,across Canada.
Costs include:
a Indirect costs - costs to the patient or family related to having the disease but not
to direct health care.
b. Out of pocket (OOP) - costs paid in cash or credit for health-related expenses not
covered by the public health or private insurance systems.
2. Determine the sociodemographic and disease characteristics associated with higher costs
Methods:
Population: Incident cases of IBD (<16y) over 12 months (est. enrollment 250-300).
Indirect and OOP disease-related costs will be determined with surveys conducted one year
following diagnosis and every 6mo for 2y. These will be conducted querying families on the
preceding 4 weeks including: school and work days missed, out-of-pocket expenses, distance
travelled to appointments, medications expenses incurred, and disability benefits collected.
Indirect costs will be calculated using the Human Capital Approach (gross income not earned
due to disease).
Description:
Specific objectives:
1. Determine the Indirect and out of pocket costs of care of children with IBD, incurred by
caregivers, funders, and the health system. Costs include:
1. Indirect costs - all costs related to the patient having the disease but not
related to direct health care costs. These include those paid by the patient,
family, insurance system, and public funding. These include but are not limited to
the costs of lost productivity hours due to missed work, missed school and
volunteering activities, employment or disability insurance costs.
2. Out of pocket (OOP) costs - those paid by the patient or family of the patient in
cash or credit for healthcare related expenses that are not covered by the public
health or insurance systems. These include but are not limited to medication
dispensing fees, parking costs for medical appointments, travel expenses for
medical appointments, special diet, alternative health treatments, and any other
incurred disease-related costs such as educational books and donations.
2. Determine the sociodemographic and disease characteristics associated with higher costs
Background:
Inflammatory bowel disease (IBD) is a group of disorders characterized by chronic
inflammation of the gastrointestinal tract with remissions and relapses. The two most common
subtypes are Crohn's disease (CD) and ulcerative colitis (UC). The incidence of IBD appears
to have risen over the last twenty years especially in the pediatric population.1-4 IBD is
often diagnosed in the second or third decades of life, and therefore can impact both
patients' social functioning and wellbeing, but also the family unit.. In addition, the fact
that patients are most frequently diagnosed while in school or soon after, can result in
significant cost to both the health system and to the economy. In 2012, the burden-of-illness
report from the Crohn's and Colitis Canada estimated that the direct medical costs of IBD in
Canada were over one billion dollars, primarily funded through the Canadian public healthcare
system.1 While many international studies examined IBD-related costs in adults, information
on direct and indirect health care costs resulting from a childhood diagnosis of IBD is not
readily available.
Review of the Canadian Literature in Adult IBD: Members of our group investigated the direct
hospital costs from the payer perspective for a cohort of 187 CD and 115 UC adult patients at
a tertiary care hospital in Manitoba, in 1994-1995. The mean cost of hospital admission per
medical case was C$2571 (95% CI, C$1801-C$3340) for CD and C$2186 (95% CI, C$1449-C$2922) for
UC. The mean cost per hospitalized surgical case was higher, with C$3427 (95% CI,
C$2728-C$4126) for CD and C$4635 (95% CI, C$3549-C$5726) for UC. Using the median values per
hospitalized patient, the medical cost was C$1664 for CD and C$1262 for UC; the surgical cost
was C$2546 for CD and C$3341 for UC. Surgery accounted for 50% of all hospital admissions,
58% of all hospital days, and 61% of all costs.5 The same investigators used administrative
data to estimate health care utilization in adults with IBD in Manitoba. They concluded that
the first 2 years from disease diagnosis were the most costly in terms of health care
utilization.6 Longobardi et al examined indirect costs in the form of work loss related to
IBD. Approximately 29% of adults with IBD reported labor force non-participation. The
investigators estimated IBD-related indirect costs attributable to nonparticipation in 1998
to be over C$104 million.7 In another study from Manitoba, an acceptable concordance between
patient-reported and administrative data-driven health care utilization.8 A high rate of
concordance was also reported in a recent study from the Netherlands.9 This specific question
of self-reported versus health records-reported health care utilization has never been
examined in pediatric IBD.
A population-based surveillance cohort from Alberta examined predictors of costs for adults
with UC who were hospitalized for a flare or colectomy between 2001 and 2009. Median
hospitalization cost for UC flare, emergent colectomy and elective colectomy were: C$5,499,
C$23,698 and C$14,316, respectively. Adjusted hospitalization costs increased approximately
6% annually. Use of infliximab was an independent predictor of increased costs.10 Economic
data for a small adult sample hospitalized for Crohn's disease in Alberta, assessed costs two
years before and after infliximab therapy. Total health care resource use and direct health
care costs were compared for patients with or without fistulae. In the year following
initiation of infliximab therapy, there were significant decreases in health care use,
reflected in total hospital days, inpatient and outpatient colonoscopies and major
surgeries.11 Review of Pediatric IBD Research: Using a collection of American HMO
administrative data, Kappelman et al described the healthcare utilization and direct costs
associated with IBD in insured American children and adults with IBD. Overall, healthcare
utilization was higher in children with IBD compared to adults and in patients with CD
compared to those with UC. Mean annual direct health costs for CD and UC were US$8,265 and
US$5,066 respectively. In children, direct costs were approximately double that of .12 In a
cross-sectional study Sin et al estimated the out of pocket (OOP) costs in parents of 150
children with IBD in California through surveys. Over 28% of those parents estimated their
annual OOP costs to be more than US$1,000. Uncontrolled, severe, and frequently relapsing IBD
correlated with higher OOP costs.13 In another retrospective cross-sectional analysis from
Canterbury, New Zealand, the annual total costs per patient for pediatric CD were NZ$14,375
with direct and indirect costs comprising NZ$12,583 and NZ$1,792, respectively. Based on
these data, the investigators estimated the annual total direct and indirect costs of CD
across New Zealand to be approximately NZ$25.9 million.14 In a single center study, Wu et al,
examined non-drug costs associated with outpatient infliximab infusions in children with IBD.
The total annual drug cost was approximately $393,000 in 2011. For direct costs related to
infliximab infusions, more than 77% of the total health care costs were related to personnel
(e.g., nursing), facility operations, and laboratory costs. Only 23% of the total costs were
related to the actual infliximab drug costs.15
Rationale:
In its 2012 report on the burden of IBD in Canada, Crohn's and Canada estimated that direct
medical costs for IBD were C$1.2 billion, and that indirect costs were C$1.6 billion.
Accordingly, the total annual costs for IBD in Canada were approximately C$2.8 billion. It is
well-recognized that IBD causes significant financial burden on the economy, which is likely
rising due to increased prevalence of the disease and the increasing use of high-cost
biologic medications. Disease-related costs in Canadian children with IBD have never been
studied. Therefore, we will conduct a national Canadian study examining IBD-related health
costs, a crucial step to plan for proper allocation of resources and for future health
services for Canadian children with IBD.
The cohort of recently developed CIHR/CHILD Foundation Canadian Children IBD Network
(CIDsCaNN) includes about 1000 IBD patients. This network comprises 12 pediatric IBD centres
across Canada tasked with the creation of an inception cohort of all children diagnosed with
IBD. Biological and clinical information, including medication utilization, disease phenotype
and disease severity is collected, with longitudinal follow-up.
Methods:
Source Population: Incident cases of pediatric IBD (<17y) in (CIDsCaNN) centres enrolled over
a 12 month period or after (estimated enrollment 250-300 over a period of 12 months).
Patients will be expected to be followed for a minimum of 2 years Study Design: A prospective
cohort study will be conducted to determine disease-related costs using a series of
cross-sectional surveys (Appendix). Surveys will be conducted at 6-18 months following the
diagnosis then every 6 months for 2 years through the CIDsCaNN web portal, querying families
on activity in the 4 weeks prior to the survey: school (patient) and work (parents) days
missed, out-of-pocket expenses, distance travelled to care appointments, medications expenses
incurred, and disability benefits collected. If participants are unable to complete the
survey online, research assistant will give them the survey in a paper format to be
completed.
Duration of costs covered: The survey will collect direct (6 months period), indirect and OOP
costs in the 4 weeks preceding the survey (4 surveys over the study period) Indirect and OOP
costs will be calculated in Canadian dollars, and adjusted for inflation using an appropriate
inflationary measure (such as the Consumer Price Index (CPI)). Indirect costs will be
calculated using the Human Capital Approach (gross income not earned due to disease).20 All
costs will be adjusted for inflation using an inflationary measure such as CPI for health and
personal care, by province (March 2016) 21.
Predictors of increased costs will be determined, with variables assessed to include: gender,
age, pediatric IBD centre, family income, parents level of education, region of residence,
disease duration, disease phenotype using Paris modification of Montreal classification,
clinical activity at diagnosis and one year after diagnosis as measured by pediatric Crohn's
activity index for CD (PCDAI) and pediatric UC activity index for UC (PUCAI), and adherence
to IBD medications.
Statistical Analysis: Descriptive statistics will be reported as means with standard
deviations (SD), medians with interquartile range (IQR), or proportions where appropriate.
Two-part models will be estimated, in which the likelihood of incurring any expenditures and
the natural logarithm of conditional expenditures will be estimated separately by logistic
and ordinary least-squares models.
