The Burden of Access in Duchenne Muscular Dystrophy in the US

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.

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.

Study Overview

• Status: Completed
• Conditions: Duchenne Muscular Dystrophy

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.

• Study Type: Observational
• Enrollment (Actual): 57

Contacts and Locations

Study Locations

• United States
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 99 years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• 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

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Individuals Living with DMD; 90 patients/parents
Healthcare Providers to Patients with DMD; 40 healthcare providers

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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

Collaborators and Investigators

• Sponsor: University of Florida
• Collaborators: Engage Health Inc.
• Investigators: Principal Investigator: Kara Godwin, MSN,APRN, University of Florida

Study record dates

Study Major Dates

• Study Start (Actual): June 18, 2019
• Primary Completion (Actual): May 28, 2020
• Study Completion (Actual): May 28, 2020

Study Registration Dates

• First Submitted: May 14, 2019
• First Submitted That Met QC Criteria: May 14, 2019
• First Posted (Actual): May 15, 2019

Study Record Updates

• Last Update Posted (Actual): August 6, 2020
• Last Update Submitted That Met QC Criteria: August 4, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: OCR21561; LHF0001 (Other Identifier: WIRB)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No