Clinical trial NCT06861270: Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France
Official title: Estimation of Non-Reimbursable Direct Medical Costs, Direct Non-Medical Costs, and Indirect Costs for Patients With Duchenne Muscular Dystrophy in France
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2025-03-06
Enrollment: 171
Sex: ALL
Minimum age (years): 1
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study will focus on patients with Duchenne muscular dystrophy, whatever their age, and included in the BNDMR. This database brings together data on all patients treated in the rare disease expert centres approved by the DGOS, and shows a gross total of 2,028 patients with DMD on 1st March 2022.

Brief summary: Duchenne muscular dystrophy (DMD) is a chronic neuromuscular disorder affecting approximately 150 to 200 newborns annually, predominantly males, and is characterized by progressive muscle atrophy and weakness due to a complete absence of dystrophin. DMD presents a severe phenotype, with life expectancy typically extending into the third decade. Data on healthcare utilization and associated costs for DMD patients, particularly within the French healthcare context, remain limited. The EPARDYS study aims to examine the care pathways of these patients using data from the Banque Nationale des Données Maladies Rares (BNDMR) and the Système National des Données de Santé (SNDS), enabling a detailed characterization of patient demographics, healthcare utilization patterns, and associated costs.

However, only direct costs eligible for reimbursement under the Affection Longue Durée (ALD 100%) scheme can be evaluated. Additional resources are consumed by DMD patients, particularly for medical care that falls outside the scope of reimbursement. Furthermore, the high level of dependency in these patients may lead to the need for home adaptations or other expenditures outside the healthcare domain but linked to the disease. Informal caregiving also plays a significant role.

These additional family-incurred costs must be considered. The CouDuMyo study will aim to estimate all relevant cost components from a societal perspective, considering both minors and adults, whether they attend school or work. The medico-social aspect of care, along with the collection of data on aids and benefits received, will offer insights into the nature of the support available to families in France. The knowledge gained regarding non-reimbursable, disease-related costs according to patient characteristics will provide a comprehensive view of the financial burden borne by patients and their caregivers. This assessment is particularly relevant in the context of emerging therapies, which may reduce these associated costs. The main objective will be to describe the resource utilization associated with DMD care that is not eligible for reimbursement, including direct medical costs not covered, medico-social expenses, direct non-medical costs, and indirect costs.


Eligibility criteria: The patients identified are those present in the BNDMR and with an up-to-date address for their follow-up centre.

Inclusion criteria

* Patients of any age with DMD diagnosed more than 6 months ago;
* Patient included in the BNDMR, not objecting to the collection of his or her data (see BNDMR 'Agreement to be contacted for a protocol') and alive at the time of his or her request to participate in the study.

Exclusion Criteria:

* Patient taking part in an interventional clinical study with changes to usual management (see BNDMR 'Patient taking part in a protocol');
* Patient and/or main carer objecting to the use of their data;
* Patient and/or main carer whose understanding of the French language does not allow them to complete the questionnaire.

Primary outcomes:
measure: Average annualised cost per patient/family.
timeFrame: 1 year
Secondary outcomes:
measure: Different types of costs identified for managing the disease
timeFrame: from disease identification to questionnaire's completion, up to 98 years
measure: Costs based on the severity of the disease
timeFrame: from disease identification to questionnaire's completion, up to 98 years
measure: Determinants of the identified costs
timeFrame: from disease identification to questionnaire's completion, up to 98 years
Other outcomes:
Locations:
city: Paris
country: France
facility: AP-HP Hôtel Dieu
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Isabelle Durand-Zaleski, Professor
role: STUDY_DIRECTOR
References: