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出境医 / 临床实验 / The Burden of Access in Duchenne Muscular Dystrophy in the US
The Burden of Access in Duchenne Muscular Dystrophy in the US
Study Description
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.
| Condition or disease |
|---|
| 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 Design
| Study Type : | Observational |
| Actual Enrollment : | 57 participants |
| Observational Model: | Case-Control |
| Time Perspective: | Cross-Sectional |
| 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. |
| Actual Study Start Date : | June 18, 2019 |
| Actual Primary Completion Date : | May 28, 2020 |
| Actual Study Completion Date : | May 28, 2020 |
Arms and Interventions
| Group/Cohort |
|---|
|
Individuals Living with DMD
90 patients/parents
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Healthcare Providers to Patients with DMD
40 healthcare providers
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Outcome Measures
Primary Outcome Measures :
- 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 Outcome Measures :
- 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.
Eligibility Criteria
| Ages Eligible for Study: | 18 Years to 99 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | Yes |
| 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
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.
Contacts and Locations
Locations
| United States, Florida | |
| University of Florida | |
| Gainesville, Florida, United States, 32610 | |
Sponsors and Collaborators
University of Florida
Engage Health, Inc.
Investigators
| Principal Investigator: | Kara Godwin, MSN,APRN | University of Florida |
| Tracking Information | |||||
|---|---|---|---|---|---|
| First Submitted Date | May 14, 2019 | ||||
| First Posted Date | May 15, 2019 | ||||
| Last Update Posted Date | August 6, 2020 | ||||
| Actual Study Start Date | June 18, 2019 | ||||
| Actual Primary Completion Date | May 28, 2020 (Final data collection date for primary outcome measure) | ||||
| Current Primary Outcome Measures |
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.
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| Original Primary Outcome Measures | Same as current | ||||
| Change History | |||||
| Current Secondary Outcome Measures |
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| Original Secondary Outcome Measures | Same as current | ||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||
| Descriptive Information | |||||
| Brief Title | 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. | ||||
| 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. |
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| Study Type | Observational | ||||
| Study Design | Observational Model: Case-Control Time Perspective: Cross-Sectional |
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| Target Follow-Up Duration | Not Provided | ||||
| Biospecimen | Not Provided | ||||
| 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 |
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| Condition | Duchenne Muscular Dystrophy | ||||
| Intervention | Not Provided | ||||
| Study Groups/Cohorts |
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| Publications * | Not Provided | ||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status | Completed | ||||
| Actual Enrollment |
57 | ||||
| Original Estimated Enrollment |
130 | ||||
| Actual Study Completion Date | May 28, 2020 | ||||
| Actual Primary Completion Date | May 28, 2020 (Final data collection date for primary outcome measure) | ||||
| Eligibility Criteria |
Inclusion Criteria: Patient/Parent interviews
Healthcare Provider survey
Exclusion Criteria:
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| Sex/Gender |
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| Ages | 18 Years to 99 Years (Adult, Older Adult) | ||||
| Accepts Healthy Volunteers | Yes | ||||
| Contacts | Contact information is only displayed when the study is recruiting subjects | ||||
| Listed Location Countries | United States | ||||
| Removed Location Countries | |||||
| Administrative Information | |||||
| NCT Number | NCT03951675 | ||||
| Other Study ID Numbers | OCR21561 LHF0001 ( Other Identifier: WIRB ) |
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| Has Data Monitoring Committee | No | ||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement |
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| Responsible Party | University of Florida | ||||
| Study Sponsor | University of Florida | ||||
| Collaborators | Engage Health, Inc. | ||||
| Investigators |
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| PRS Account | University of Florida | ||||
| Verification Date | August 2020 | ||||
