Clinical trial NCT01705613: Cost-effectiveness of the Treatment of Attention Deficit/Hyperactivity Disorder With Methylphenidate Immediate-release in Brazil
Official title: Cost-effectiveness of the Treatment of Attention Deficit/Hyperactivity Disorder With Methylphenidate Immediate-release in Brazil
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Attention Deficit/Hyperactivity Disorder
Keywords: Attention Deficit/Hyperactivity Disorder
ADHD
Methylphenidate Immediate-release
Cost-effectiveness
Cost-utility
QALYs
Lead sponsor: Hospital de Clinicas de Porto Alegre
Sponsor class: OTHER
Collaborators: name: Conselho Nacional de Desenvolvimento Científico e Tecnológico
name: Coordenação de Aperfeiçoamento de Pessoal de Nível Superior.
First posted: 2012-10-12
Enrollment: 120
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: ECOLOGIC_OR_COMMUNITY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children and adolescents with indication of Attention Deficit/Hyperactivity Disorder treatment

Brief summary: The first objective is to measure the QALYs (Quality Adjusted Life Years) of Brazilian children and adolescents, submitted to methylphenidate immediate-release treatment for Attention Deficit Hyperactivity Disorder (ADHD). For this, the Health Utility Index scale (HUI) will be completed by the patient and/or family, in two moments of an observation study with immediate-release methylphenidate: at the baseline and 6th month of treatment in the follow-up. The results provides information to calculate the utility measure and determine the QALYs (Quality Adjusted Life Years). After this, for the cost-effectiveness of the treatment, it will be used a Markov model of decision analysis, and a Monte Carlo simulation with 10,000 random trials.

Subjects will be submitted to immediate-release methylphenidate treatment in a maximum dose of 0.5mg/kg/day. The total treatment monitoring is 06 (six) months, and patients will be evaluated at baseline, first, third and sixth month. The HUI rating scales are fulfilled by parents and patients (if \>12 years old only) at baseline and 6th month. The period for inclusion of new patients comprises from 01/01/2011 to 31/12/2013.

Detailed description: Currently, Brazil has no estimates for quality of life, and/or utilities for ADHD economy analysis. Such data is necessary to calculate the QALYs (Quality Adjusted Life Years). Thus, the investigators will implement the Health Utility Index tool (HUI) to be completed by the patient and/or family, in two moments of an observational study with immediate-release methylphenidate: at baseline and 6th month of treatment. The determination of the QALY as an outcome in the decision analysis is justified because HUI incorporates into a single measure morbidity and mortality. The results of the cost-effectiveness study will assist government agencies in the process of decision making.

To determine the cost-effectiveness of methylphenidate immediate release available in the Brazilian market, the investigators will use a Markov model of decision analysis. The development of the model and analysis of the results will adopt the standards established in the Methodological Guidelines of the Brazilian Ministry of Health, and the perspective will be of the Brazilian National Health System. Medications prices will be obtained through the Board of Market Regulation of Medicines (CMED) at the site of ANVISA (National Health Surveillance Agency). For the treatment strategy that provides greatest cost ratio it will be calculated the incremental cost-effectiveness, compared to the lower cost. To estimate more accurately the impact on life, a Monte Carlo simulation with 10,000 random trials will be conducted.

This is a one treatment group and observational study, with 140 patients, and no controls. Where indicated, it will be suggested the use of 10 mg methylphenidate in a maximum dose of 0.5 mg / kg / day. The total treatment monitoring is 06 (six) months, and patients will be evaluated at baseline, first, third and sixth month. The HUI rating scales will be fulfilled by parents and patients (if \>12 years old only) at baseline and 6th month. The period for inclusion of new patients comprises from 01/01/2011 to 31/12/2013.

Eligibility criteria: Inclusion Criteria:

* Symptoms of attention deficit/hyperactivity disorder with impairment
* Children and adolescents (age from 06 to 18 years old)
* absence of contraindication for the use of methylphenidate immediate release

Exclusion Criteria:

* Contraindication methylphenidate immediate release use
* Neurological diseases
* Discordance with the informed consent form
* IQ\<70
* Neurological diseases

Primary outcomes:
measure: Swanson, Nolan, and Pelham Rating Scale (SNAP-IV)
timeFrame: Baseline, 1th, 3rd and 6th month
measure: Health Utility Index
timeFrame: Baseline and 6th month
Secondary outcomes:
Other outcomes:
Locations:
city: Porto Alegre
country: Brazil
facility: Hospital de Clínicas de Porto Alegre
state: RS
Study officials:
affiliation: Hospital de Clinicas de Porto Alegre
name: Luis A Rohde, MD, PhD
role: STUDY_CHAIR
affiliation: Hospital de Clinicas de Porto Alegre
name: Carlos R Moreira Maia, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Maia CR, Stella SF, Wagner F, Pianca TG, Krieger FV, Cruz LN, Polanczyk GV, Rohde LA, Polanczyk CA. Cost-utility analysis of methylphenidate treatment for children and adolescents with ADHD in Brazil. Braz J Psychiatry. 2016 Mar;38(1):30-8. doi: 10.1590/1516-4446-2014-1516. Epub 2015 Sep 8.