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The Burden of Access in Duchenne Muscular Dystrophy in the US

key information

study id #: NCT03951675

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

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.

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT03951675

last updated: June 24, 2019

study details

start date: June 18, 2019

estimated completion: June 2020

phase of development: N/A

size / enrollment: 130

study 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.

primary outcomes:

  • Burden frequency by functional category [ Time Frame: Over 12 months ]
    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.

secondary outcomes:

  • Burden frequency by type of insurance [ Time Frame: Over 12 months ]
    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.
  • Life impact frequency by functional category [ Time Frame: Over 12 months ]
    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.
  • Life impact frequency by type of insurance [ Time Frame: Over 12 months ]
    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.

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 contacts

sponsor: University of Florida

contacts: Stephanie M Salabarria BHSc, 3522736582, ssalabarria@ufl.edu

investigators: Kara Godwin, MSN, APRN

locations: United States

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