Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis, Myasthenia Gravis, Sickle Cell Disease
Conditions
Keywords
Adherence, MAR-Scale, Rare disease, Observational
Brief summary
The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease, and to identify the top patient-reported reasons for non-adherence. Internal reliability of the MAR-Scale will also be assessed in each condition.
Interventions
OTHERMAR-Scale
The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.
Sponsors
The Touro College and University System
Xperiome
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* Have a confirmed (self-reported) diagnosis by a doctor or other licensed healthcare professional of one of the following conditions: * Cystic fibrosis * Hemophilia (A or B) * Idiopathic pulmonary fibrosis * Myasthenia gravis * Sickle cell disease * Have access to the internet * Are aged 18 years or above * Are comfortable reading and answering questions in English * Have an active prescription for at least one medication indicated to treat the relevant condition (self-reported) * NB: Individuals will be eligible even if the prescription has not been dispensed (filled) yet, and also if they have the medication(s), but are not actually taking it/them Are able and willing to provide consent electronically through the my.raremark.com platform
Exclusion criteria
* There are no
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. | Baseline | Percentage of respondents who indicated at least one reason for non-adherence in the past seven days. |
| To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. | Baseline | Tally of reasons for non-adherence. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients. | Baseline | Use the 200 responses collected from each condition to determine the Cronbach's alpha of the scale for each condition. |
Outcome results
None listed