Clinical trial NCT03951675: The Burden of Access in Duchenne Muscular Dystrophy in the US
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.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: University of Florida
Sponsor class: OTHER
Collaborators: name: Engage Health Inc.
First posted: 2019-05-15
Enrollment: 57
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: CROSS_SECTIONAL
Sampling method: PROBABILITY_SAMPLE
Study population: For 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

For Healthcare Provider Survey:

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

Brief 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Burden frequency by functional category
timeFrame: Over 12 months
Secondary outcomes:
measure: Burden frequency by type of insurance
timeFrame: Over 12 months
measure: Life impact frequency by functional category
timeFrame: Over 12 months
measure: Life impact frequency by type of insurance
timeFrame: Over 12 months
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida
state: Florida
Study officials:
affiliation: University of Florida
name: Kara Godwin, MSN,APRN
role: PRINCIPAL_INVESTIGATOR
References: