Medication Adherence and Non-adherence in Adults With Rare Disease

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

Sponsors

Lead Sponsor: Raremark

Collaborator: The Touro College and University System

Source Raremark
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.

Overall Status Not yet recruiting
Start Date November 2020
Completion Date September 2022
Primary Completion Date September 2022
Study Type Observational
Primary Outcome
Measure 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. Baseline
To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases. 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. 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. 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. 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. 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. 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. 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. 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. 1 year
Secondary Outcome
Measure Time Frame
To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients. Baseline
Enrollment 1000
Condition
Intervention

Intervention Type: Other

Intervention Name: MAR-Scale

Description: The MAR-Scale is a 20-item questionnaire based on commonly reported reasons for non-adherence to medications.

Eligibility

Sampling Method: Non-Probability Sample

Criteria:

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.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: Accepts Healthy Volunteers

Overall Official
Last Name Role Affiliation
Elizabeth J Unni, BpharmMBAPhD Principal Investigator Touro College of Pharmacy
Overall Contact

Last Name: Pete Chan, BSc

Phone: +44 (0) 20 3920 9880

Email: [email protected]

Verification Date

September 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Arm Group

Label: Cystic fibrosis

Description: Patients aged 18+ and diagnosed with cystic fibrosis. Patients will answer the MAR-Scale once every three months for a year.

Label: Hemophilia A or B

Description: Patients aged 18+ and diagnosed with hemophilia A or B. Patients will answer the MAR-Scale once every three months for a year.

Label: Idiopathic pulmonary fibrosis

Description: Patients aged 18+ and diagnosed with idiopathic pulmonary fibrosis. Patients will answer the MAR-Scale once every three months for a year.

Label: Myasthenia gravis

Description: Patients aged 18+ and diagnosed with myasthenia gravis. Patients will answer the MAR-Scale once every three months for a year.

Label: Sickle cell disease

Description: Patients aged 18+ and diagnosed with sickle cell disease. Patients will answer the MAR-Scale once every three months for a year.

Patient Data Undecided
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

Source: ClinicalTrials.gov