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Liquidation of sponsor
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| Name | Class |
|---|---|
| The Touro College and University System | OTHER |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cystic fibrosis | Patients aged 18+ and diagnosed with cystic fibrosis. Patients will answer the MAR-Scale once every three months for a year. |
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| Hemophilia A or B | Patients aged 18+ and diagnosed with hemophilia A or B. Patients will answer the MAR-Scale once every three months for a year. |
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| Idiopathic pulmonary fibrosis | Patients aged 18+ and diagnosed with idiopathic pulmonary fibrosis. Patients will answer the MAR-Scale once every three months for a year. |
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| Myasthenia gravis | Patients aged 18+ and diagnosed with myasthenia gravis. Patients will answer the MAR-Scale once every three months for a year. |
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| Sickle cell disease | Patients aged 18+ and diagnosed with sickle cell disease. Patients will answer the MAR-Scale once every three months for a year. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MAR-Scale | Other | The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications. |
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| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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Anyone who fits the eligibility criteria and gives their consent to take part in this study.
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| Name | Affiliation | Role |
|---|---|---|
| Elizabeth J Unni, BpharmMBAPhD | Touro College of Pharmacy | Principal Investigator |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D006467 | Hemophilia A |
| D002836 | Hemophilia B |
| D054990 | Idiopathic Pulmonary Fibrosis |
| D009157 | Myasthenia Gravis |
| D000755 | Anemia, Sickle Cell |
| D035583 | Rare Diseases |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| 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 |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D040181 | Genetic Diseases, X-Linked |
| D011658 | Pulmonary Fibrosis |
| D017563 | Lung Diseases, Interstitial |
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006453 | Hemoglobinopathies |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |