Estimating costs for patients with Duchenne muscular dystrophy in France
Estimation of Non-Reimbursable Direct Medical Costs, Direct Non-Medical Costs, and Indirect Costs for Patients With Duchenne Muscular Dystrophy in France
This study looks at the healthcare costs for boys with Duchenne muscular dystrophy in France to see how much families have to pay out of pocket.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 171 (estimated) |
| Ages | 1 Year to 99 Years |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 1 site (Paris) |
| Trial ID | NCT06861270 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder affecting predominantly males. It aims to analyze the healthcare utilization and associated costs for DMD patients in France by utilizing data from national health databases. The study will characterize patient demographics and care pathways, specifically looking at costs that are not reimbursed under existing healthcare schemes. By understanding these costs, the study seeks to provide a clearer picture of the financial burden faced by DMD patients and their families.
Who should consider this trial
Good fit: Ideal candidates for this study are patients of any age diagnosed with DMD for more than six months who are included in the national health database.
Not a fit: Patients currently participating in interventional clinical studies or those who object to data collection will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the economic impact of DMD, potentially influencing healthcare policies and support systems for affected patients.
How similar studies have performed: While there may be limited data on similar studies in France, the approach of analyzing healthcare costs in chronic conditions has been utilized successfully in other contexts.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Paris
- AP-HP Hôtel Dieu — Paris, France (Recruiting)
Study contacts
- Study coordinator: Isabelle Durand-Zaleski, Professor
- Email: isabelle.durand-zaleski@aphp.fr
- Phone: 0140274143
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.