The Burden of Access in Duchenne Muscular Dystrophy in the US

Duchenne Muscular Dystrophy Clinical Trial

Official title:

The Burden of Access in Duchenne Muscular Dystrophy in the US. A Qualitative Assessment of the Impact of Access on the Lives of Families Affected by DMD and Their Healthcare Providers.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03951675
• Other study ID #: OCR21561
• Secondary ID: LHF0001
• Status: Completed
• Start date: June 18, 2019
• Est. completion date: May 28, 2020

Study information

• Verified date: August 2020
• Source: University of Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these burdens. Data from healthcare providers will be collected by an online survey and from patients/families by one on one telephone interview.

Description:

Patient/Parent Portion

In this non-interventional study, DMD patients / parents of DMD patients will be interviewed to gather qualitative input, in the patient's voice, regarding challenges associated with access to medications, services and medical equipment, and how these burdens impact quality of life.

Healthcare Provider Portion

In this non-interventional study, healthcare providers who see patients with DMD and who deal with insurance issues on behalf of DMD patients will participate in an online survey designed to determine the burden associated with access to medications, services and medical equipment.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 57
• Est. completion date: May 28, 2020
• Est. primary completion date: May 28, 2020
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

Inclusion Criteria:

Patient/Parent interviews

• Patients residing in the US who have been diagnosed with DMD who are age 18 years or older, or the parent / legal guardian of a person of any age who has been diagnosed with DMD,

• Have provide "Proof of DMD" to ensure that they are impacted by the disease,

• Who have provided sufficient information in the RSVP process to determine their functional status; ambulatory, transitional or non-ambulatory,

• State that they are the person who deals with insurance issues for the affected patient and,

• Who are able to understand and consent to participation in the study

Healthcare Provider survey

• Healthcare providers (physicians, nurse practitioners, and physician assistants) currently involved in the care of patients with DMD

• Are currently practicing in the US,

• Who have provided sufficient information in the survey screening to determine that they currently care for DMD patients,

• State that they and/or persons on their staff interface with insurance companies for DMD patients related to access to medications, services and/or medical equipment and,

• Who are able to understand and consent to participation in the study

Exclusion Criteria:

• There are no stated exclusion criteria in this study. Study population must meet all inclusion criteria in order to be deemed eligible to participate.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
United States	University of Florida	Gainesville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of Florida	Engage Health, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Burden frequency by functional category	The frequency that each burden is mentioned by a patient according to their functional category as measured by the VIGNOS scale. This is the number of times each burden is mentioned by a patient during their interview, assessed by each functional category.	Over 12 months
Secondary	Burden frequency by type of insurance	The frequency that each burden is mentioned by a patient according to their insurance coverage. This is the number of times each burden is mentioned by a patient during their interview, assessed by each type of insurance.	Over 12 months
Secondary	Life impact frequency by functional category	The frequency that each life impact is mentioned by a patient according to their functional category as measured by the VIGNOS scale. This is the number of times each life impact is mentioned by a patient during their interview, assessed by each functional category.	Over 12 months
Secondary	Life impact frequency by type of insurance	The frequency that each life impact is mentioned by a patient according to their insurance coverage. The number of times each life impact is mentioned by a patient during their interview, assessed by each type of insurance.	Over 12 months