Medication Adherence and Non-adherence in Adults With Rare Disease

Study Overview
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Brief Summary
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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.

Official Title
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A Longitudinal, Digital Study Using the Medication Adherence Reasons Scale (MAR-Scale) to Identify the Reasons for Non-adherence to Medications in Rare Disease

Conditions
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Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis, Myasthenia Gravis, Sickle Cell Disease

Intervention / Treatment
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* Other: MAR-Scale


Participation Criteria
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Eligibility Criteria
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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 exclusion criteria. Any member of a Raremark community will be able to take part in this study if they meet the inclusion criteria and can provide their informed consent.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cystic fibrosis<br>Patients aged 18+ and diagnosed with cystic fibrosis. Patients will answer the MAR-Scale once every three months for a year. | Other: MAR-Scale<br>* The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.<br>|
| Hemophilia A or B<br>Patients aged 18+ and diagnosed with hemophilia A or B. Patients will answer the MAR-Scale once every three months for a year. | Other: MAR-Scale<br>* The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.<br>|
| Idiopathic pulmonary fibrosis<br>Patients aged 18+ and diagnosed with idiopathic pulmonary fibrosis. Patients will answer the MAR-Scale once every three months for a year. | Other: MAR-Scale<br>* The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.<br>|
| Myasthenia gravis<br>Patients aged 18+ and diagnosed with myasthenia gravis. Patients will answer the MAR-Scale once every three months for a year. | Other: MAR-Scale<br>* The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.<br>|
| Sickle cell disease<br>Patients aged 18+ and diagnosed with sickle cell disease. Patients will answer the MAR-Scale once every three months for a year. | Other: MAR-Scale<br>* The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. | Percentage of respondents who indicated at least one reason for non-adherence in the past seven days. | Baseline | 
| To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. | Percentage of respondents who indicated at least one reason for non-adherence in the past seven days. | 3 months | 
| To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. | Percentage of respondents who indicated at least one reason for non-adherence in the past seven days. | 6 months | 
| To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. | Percentage of respondents who indicated at least one reason for non-adherence in the past seven days. | 9 months | 
| To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. | Percentage of respondents who indicated at least one reason for non-adherence in the past seven days. | 1 year | 
| To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. | Tally of reasons for non-adherence. | Baseline | 
| To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. | Tally of reasons for non-adherence. | 3 months | 
| To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. | Tally of reasons for non-adherence. | 6 months | 
| To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. | Tally of reasons for non-adherence. | 9 months | 
| To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases. | Tally of reasons for non-adherence. | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients. | Use the 200 responses collected from each condition to determine the Cronbach's alpha of the scale for each condition. | Baseline | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Adherence, MAR-Scale, Rare disease, Observational