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The Role of Real World Evidence in Clinical Development

Real world data may provide long term efficacy with economic assessment and may be useful in designing future randomized controlled trials and pragmatic clinical trials.

By leveraging the increasing amount of data captured and recorded electronically, the medical field is poised to drive a major evolution in pharmaceutical, biotechnology and medical device development. The Food & Drug Administration (FDA) defines real world evidence (RWE) as data regarding the usage, or the potential benefits or risks, of a drug (and other therapeutic and diagnostic products) derived from sources other than traditional clinical trials. The analysis of real world data (RWD), usually derived from multiple sources, leads to the creation of RWE, which may be used to define the benefits of a particular product and/or therapy.

The most common RWD sources include:

  • Electronic health records (EHRs)
  • Insurance claims databases
  • Claims and billing activities
  • Product and disease registries
  • Patient registries and patient-reported outcomes
  • Data gathered from other sources that can inform on health status, such as mobile devices
  • Peer reviewed journal articles
Breaking out of silos
In the past, data collected in clinical trials and RWD sources were either not accessible or highly bureaucratic to access. There have been several movements to make research data more transparent and accessible. The Yale University Open Data Access (YODA) Project, as an example, advocates for responsible sharing of research data, open science, and transparency. It's focused on improving the health of patients and informing science and greater public health.

Having a highly transparent and accessible data sources should improve science overall. Using RWE as supplemental data for regulatory, therapy and diagnostic product development and health care decision-making by governments and consumers alike can hopefully help to produce better clinical and economic outcomes in the future.

RWE in action
In a recent effort by Microsoft using real world data in the form of internet search records to predict incidences of pancreatic cancer. Microsoft looked at search content such as symptoms and they found that approximately 5-15 percent of the undiagnosed population was identified several months before a formal diagnosis was made. Early detection is crucial for a disease with a high mortality rate; we hope that advancements in RWE could further expand diagnostic medicine that is vetted by regulatory bodies around the world.

Medical devices
Medical devices already on the market have tremendous potential for RWE. Many of these devices record and store relevant clinical data that can be collected to learn about a product or therapy. Patients with implants, for example, already follow routine standard of care and is generally followed up by their physicians in a more structured way. Having these datasets aggregated and analyzed may lead to better therapy and lower costs.

The 21st Century Cures Act, passed in 2016, which places additional focus on the use of RWE data to provide support towards regulatory decision making including new drug applications (NDA) and premarket approval (PMA) submissions is an important step towards recognizing the role of RWE in healthcare decision making. With respect to this, it is important to note that not all RWD sources are usable, in order for it to be truly credible, RWD sources must be relevant, cleaned and harmonized.

A relevant case study involves transcatheter aortic valve replacement (TAVR) devices, which are medical devices that allow treatment of patients with diseased aortic valves who are judged too-high of a risk for conventional aortic valve replacement. The American College of Cardiology (ACC) and the Society of Thoracic Surgeons (STS), worked together with the FDA and the Centers for Medicare and Medicaid Services to create a new TAVR registry that builds on the pre-existing registries administered by both organizations.

By developing common data formats and data collection methods, the ACC and STS have expedited use of the registry to conduct prospective, randomized, post-market trials of different valve products. Evidence generated by the registry has already resulted in the FDA approval of expanded indications for the Sapien® (Edwards Lifesciences) Transcatheter Heart Valve. The collaboration of government, public and private industries culminated in this innovative approach and linked traditional clinical trial and real world data assessment. We believe that this approach has potential for many other areas of clinical development.

Want to know more about the impact that RWE can have on clinical trials? Contact EASi now.