Background
The pharmaceutical manufacturer (Sponsor) wanted to understand the effectiveness of their medication in the real-world. For this purpose, they sponsored an observational study on the real-world effectiveness of their medication among patients with Exocrine Pancreatic Insufficiency (EPI) due to chronic pancreatitis (CP).
CP is a pathologic fibro-inflammatory disease that results in the destruction of pancreatic parenchyma or pancreatic duct distortion. It has a prevalence rate of 42 per 100,000 people in the United States. CP can result in EPI which can cause maldigestion, weight loss and impaired quality of life. This study explored the real-world effectiveness of their long-time approved medication in patients with EPI due to CP. They were interested in this because of the limited information on the impact of the medication in the real-world setting. This study was designed to aid commercial strategy and inform how the drug should be prescribed in routine medical practice.
Study objectives and design
The study was designed as a prospective, multi-centre observational study. The recruitment objective was for 90 patients across multiple sites in one country, with data collection happening over a 12-month period. The focus was specific to adult patients and included 4 patient-reported clinical assessments. These included an assessment of EPI symptoms (using the pancreatic exocrine insufficiency questionnaire symptom domain and questions around stool frequency and consistency), an assessment of health-related quality of life using PEI-Q impacts domain, an assessment of co-morbidities of anxiety, depression and fatigue using PROMIS questionnaires and an assessment of nutritional status. The partner CRO took the lead in designing and validating the nutritional status assessment as there was no established COA.
The schedule for outcomes assessments was at the initial visit, 1 month follow up and 3 months follow up.
Cisiv’s solution
We configured Baseline Plus, our proprietary platform for observational research, to the requirements of this patient reported outcomes study.
The platform was designed from the ground up, in order to meet the needs of late phase observational research. Baseline Plus uses a modern user interface and a series of workflows to support the end-user with their responses. Baseline Plus is a single platform that links the clinical data from the site to the patient e-Consent and their clinical outcomes assessment (eCOA). This meant the CRO did not have to reconcile the different databases post-hoc. Our study designers are experienced in observing necessary guidelines for each PRO, translation needs, and consider the user interface in web vs mobile applications. This ensured that the platforms were easy to use for both HCPs and patients.
The configuration was efficient, and the team of designers was able to design the study in a few weeks.
Implementation
Patients were invited to complete eConsent and ePROs in the study via a simple invitation from the clinician at the site. Patients also had the option to complete paper consent and paper PROs – the latter which would be transcribed into the system by the site. Once invited, the patients received an email which explained the expected data entry process over the course of the study. They also received an email following their first visit to prompt them to complete the questionnaire within 7 days. Patients had the option to use mobile phone apps or the web platform for this study.
Outcomes
The CRO and Sponsor were pleased with the study implementation and the ease of the technology. The data collection is currently ongoing. Publications pending.
