Clinical trial NCT04541875: Medication Adherence and Non-adherence in Adults With Rare Disease
Official title: A Longitudinal, Digital Study Using the Medication Adherence Reasons Scale (MAR-Scale) to Identify the Reasons for Non-adherence to Medications in Rare Disease
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Cystic Fibrosis
Hemophilia A
Hemophilia B
Idiopathic Pulmonary Fibrosis
Myasthenia Gravis
Sickle Cell Disease
Keywords: Adherence
MAR-Scale
Rare disease
Observational
Lead sponsor: Xperiome
Sponsor class: OTHER
Collaborators: name: The Touro College and University System
First posted: 2020-09-09
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Anyone who fits the eligibility criteria and gives their consent to take part in this study.

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.


Eligibility 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.

Primary outcomes:
measure: To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
timeFrame: Baseline
measure: To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
timeFrame: 3 months
measure: To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
timeFrame: 6 months
measure: To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
timeFrame: 9 months
measure: To use the MAR-Scale to determine the extent of non-adherence to specific medications indicated to treat a range of rare diseases.
timeFrame: 1 year
measure: To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
timeFrame: Baseline
measure: To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
timeFrame: 3 months
measure: To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
timeFrame: 6 months
measure: To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
timeFrame: 9 months
measure: To use the MAR-Scale to identify the top patient-reported reasons for non-adherence to specific medications indicated to treat these rare diseases.
timeFrame: 1 year
Secondary outcomes:
measure: To perform psychometric analysis of the MAR-Scale questionnaire based on data collected from rare disease patients.
timeFrame: Baseline
Other outcomes:
Locations:
Study officials:
affiliation: Touro College of Pharmacy
name: Elizabeth J Unni, BpharmMBAPhD
role: PRINCIPAL_INVESTIGATOR
References: