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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Development and Economic Evaluation of a Patient-Centered Care Model for Children With Duchenne Muscular Dystrophy:
Titiksha Sirari1,2, Renu Suthar2, Amarjeet Singh3,4
1Lovely Professional University, Phagwara, India.
Insights
This study evaluates a patient-centered care model for Duchenne muscular dystrophy (DMD), aiming to improve quality of life and reduce the economic burden on families. Findings will inform care programs for DMD patients in low- and middle-income countries.
Area of Science:
- Medical Research
- Public Health
- Health Economics
Background:
- Duchenne muscular dystrophy (DMD) is a rare, progressive condition affecting boys, leading to reduced quality of life (QoL) and significant economic strain on families.
- Comprehensive care is lacking, exacerbating the impact of DMD on patients and caregivers.
- The economic burden includes substantial direct, indirect, and informal care costs.
Purpose of the Study:
- To evaluate the feasibility and efficacy of a patient-centered care (PCC) model for children with DMD.
- To empower families, enhance QoL, and alleviate the economic burden associated with DMD.
- To assess the impact of a multicomponent, patient-centric intervention in low- and middle-income countries (LMICs).
Main Methods:
- Quasi-experimental study enrolling 70 families with boys (aged 5-15) diagnosed with DMD.
- Two-phase study (preintervention and postintervention) comparing routine care with the PCC model.
- Intervention includes a telecare component with teleconsultation to reduce in-person visits; data collected on economic burden and QoL.
Main Results:
- Participant recruitment commenced in July 2021; the study is ongoing with completion expected by March 2023.
- Baseline data analysis is underway, with findings anticipated for publication in 2023.
- The study is collecting data on out-of-pocket expenses and QoL scores for patients and caregivers.
Conclusions:
- The research protocol outlines the study of a PCC model for DMD patients in LMICs.
- Evidence will be generated on the potential of this intervention to reduce economic burdens and improve QoL.
- Results may guide policymakers and healthcare professionals in India and other LMICs to develop comprehensive DMD care programs.
Background:
Duchenne muscular dystrophy (DMD) is a rare progressive muscular disease that primarily affects boys. A lack of comprehensive care for patients living with DMD is directly associated with a compromised quality of life (QoL) for those affected and their caregivers. This disease also has a huge economic impact on families as its treatment requires substantial direct, indirect, and informal care costs.
Objective:
This study presents a protocol developed to evaluate the feasibility and efficacy of a patient-centered care (PCC) model for children with DMD. The care model was designed with the aim to empower families, improve QoL, and reduce economic burden on their families.
Methods:
This study is planned as a quasi-experimental study that will enroll 70 consecutive families with boys (aged 5-15 years) with DMD visiting a tertiary care center. The study is being conducted in 2 phases (preintervention and postintervention phases, referred to as phase 1 and phase 2, respectively). During phase 1, the patients received routine care. The study is now in phase 2, with the intervention currently being administered. The intervention is based on the PCC model individualized by the intervention team. The model has a comprehensive DMD telecare component that includes teleconsultation as one of its key components to reduce in-person physician visits at the health facility. Teleconsultation is especially beneficial for late-ambulatory and nonambulatory patients. Data on economic burden are being collected for out-of-pocket expenses for both phases during in-person visits via telephone or messaging apps on a monthly basis. QoL data for patients and their primary caregivers are being collected at 3 time points (ie, time of enrollment, end of phase 1, and end of phase 2). Outcome measures are being assessed as changes in economic burden on families and changes in QoL scores.
Results:
Participant recruitment began in July 2021. The study is ongoing and expected to be completed by March 2023. The findings based on baseline data are expected to be submitted for publication in 2023.
Conclusions:
This paper outlines a research proposal developed to study the impact of a PCC model for patients with DMD in low- and middle-income countries (LMICs). This study is expected to provide evidence of whether a multicomponent, patient-centric intervention could reduce economic burdens on families and improve their QoL. The results of this study could guide policy makers and health professionals in India and other LMICs to facilitate a comprehensive care program for patients living with DMD. The economic impact of a rare disease is an important consideration to formulate or evaluate any health policy or intervention related to new treatments and financial support schemes.
Trial Registration:
Clinical Trials Registry India (ICMR-NIMS) CTRI/2021/06/034274; https://www.ctri.nic.in/Clinicaltrials/pmaindet2.php?trialid=56650.
International Registered Report Identifier (Irrid):
PRR1-10.2196/42491.
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