About our Real World Evidence Project

Real World Evidence (RWE) is an increasingly important component of post-market surveillance and evaluation for medical devices. The FDA has identified active surveillance of large clinical datasets as a priority for medical device safety. Unfortunately, a variety of regulatory, technical, ethical, financial, and legal barriers have prevented widespread adoption of this approach. In pediatrics, the small market size, cost of trials and registries, lack of infrastructure, and complexities related to consent as individuals transition from childhood to adulthood, have compounded the issues faced by medical devices in general. The National Evaluation System for Health Technologies (NEST) was established in 2016 in order to “support the sustainable generation and use of timely, reliable, and cost-effective RWE throughout the medical device lifecycle, using Real-World Data (RWD).” Since its inception, NEST has sponsored and supported several small scale demonstration projects focused on developing better registries, operationalizing electronic health records (EHRs) and clinical data, and collecting Patient Reported Outcome measures (PROs) via mhealth platforms. These projects are intended to demonstrate impact and inform NEST’s overall strategy.

Building upon our robust IT infrastructure and technically adept teams, CHLA and CTIP are well positioned to support the FDA and NEST’s goals to generate, capture, and meaningfully use real world evidence for the surveillance and evaluation of pediatric medical devices.

Specific Aims:

  1. Build a prospective datamart of patients with medical devices that contains clinical, device, and patient generated data
  2. Operationalize that data into 4 broad categories: medical device reporting, active surveillance, clinical applications, and research applications
  3. Expand the database to include other CTIP institutions to demonstrate scalability
  4. Contribute data to national medical device surveillance systems

We propose to establish the Pediatric Device Patient Database (PD2), a first of its kind prospective database that contains clinical, device, and patient generated health data. The foundation of PD2 will be CHLA’s NIH-funded research data warehouse. The data warehouse contains all clinical data generated by our EHR on every patient since 2008; it includes 817,023 unique CHLA patients and hundreds of variables. This core of clinical data will be augmented by adding device data and metadata from our existing clinical registries and through manufacturer APIs, and through new data entry. Finally, in order to provide a comprehensive perspective, we will add disease and device-specific PRO data acquired through an mHealth platform developed by one of our partners, Qolty (https://qolty.com/).

PD2 will be made available to researchers, industry, and the FDA in order to facilitate retrospective and prospective research and post-market surveillance of pediatric patients with medical devices.  Because PD2 includes the entirety of structured and unstructured clinical data generated by patients, it will be able to answer a variety of clinical and regulatory questions. Furthermore, with clinical data updates every 24 hours, PD2 will be able to provide active clinical surveillance for unexpected events and provide early warnings for possible device malfunction or side effects. We will work closely with our compliance office and the FDA in order to facilitate Medical Device Reporting (MDR) using PD2 surveillance, for example by auto-generating MedWatch forms (form FDA 3500).