Background
The pharmaceutical manufacturer (Sponsor) aimed to study the real-world effectiveness and usage of their medicine in patients with Immune Thrombocytopenia Purpura (ITP) and conducted an observational multicentre study to achieve this.
ITP is a blood disorder generally caused by an autoimmune disorder and is characterised by low platelet counts. It can be categorised into newly diagnosed ITP, persistent ITP and chronic ITP. It is caused by a combination of impaired platelet production and increased peripheral platelet destruction. Its clinical management is dependent on its severity. It has an estimated incidence rate of 5 out of 100,000 people.
The primary objective was to understand real-world usage and effectiveness of their drug to enhance the knowledge base regarding routine medical practice. The study data aimed to provide adequate real-world data to Healthcare Professionals and the ITP community.
Study Design
This study was designed to collect both retrospective and prospective data for each patient with a forward data collection period of 12 months. The focus was on prospectively collating data on usage and effectiveness alongside patient and clinical reported outcomes. The retrospective aspects of the study were dependent on information that was available in patients’ medical records.
This study enrolled 150 patients across 30-40 sites in the following European countries: UK, Germany, Netherlands, Norway, Czech Republic, Switzerland, Spain, Croatia and Italy.
Multiple patient-reported outcomes were collected for this study to understand various aspects of patient experience. These included EQ-5D-5L, ITP-PAQ, FACIT-Fatigue, PGIC, WPAI, MMAS-8 and TSQM-9. This entailed over 50 questions for participants to answer and was implemented electronically via a tablet at the clinic or on the patient’s own device. Friendly reminders and notifications were sent to patients through an automatic service linked with the expected data collection. This ensured increased data completion per patient.
Solution
Cisiv’s platform, Baseline Plus, enables linking the electronic data collection from sites with patient reported outcomes (e-PRO) and e-Consent.
A single integrated point of design platform allows for clear and concise data querying, management, and analysis.
The key considerations when implementing/designing an ePRO include:
i) Available guidelines from the PRO licensor about the look and feel for the PRO.
ii) Required translations
iii) Utilisation of templated ePROs
iv) User Interface (UI) considerations for web vs app, including the potential need for customisations
v) Key timelines for the client in terms of IRB approvals.
Cisiv’s in house study designers were able to work with the necessary guidelines for each PRO, identify any translation needs and consider user interface in web vs mobile applications. This ensured that the platforms were easy to use by both HCP and patients.
Outcome
The CRO and Sponsor were pleased with study implementation and the use of ease of the technology. The data gathering is currently ongoing.
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