Steps to Derive and Justify Novel Outcomes in Real World Evidence Studies Using Claims or Electronic Health Records

Speaker(s)

Discussion Leader: Mary E Ritchey, PhD, MSPH, FISPE, CERobs Consulting, LLC, Philadelphia, PA, USA
Discussants: Cynthia J Girman, DrPH, FISPE, CERobs Consulting, LLC, Wrightsville Beach, NC, USA; Josephine Norquist, MS, Merck & Co., Inc, West Point, PA, USA

PURPOSE:

Regulators and payers are amenable to use of novel algorithmic or composite real world evidence (RWE) outcomes for clinical endpoints. However, the evidentiary requirements to justify the adequacy of these novel endpoints are not clearly defined. This workshop will review potential approaches to develop and justify measurement properties of novel RWE outcomes for use as clinical endpoints within studies.

DESCRIPTION:

Attendees will obtain a working knowledge of methods for RWE outcome validation as well as for developing health measurements, drawing from the paradigm of clinical outcome assessments (COAs).

Dr. Ritchey will introduce the importance of rigorous development and validation of outcome measurements in studies using RWE (15 min.). Ms. Norquist will discuss traditional approaches to developing health measurements, including COAs beginning with identifying relevant and feasible concept of interest and derivation of measures relevant to patients, and she will review what constitutes good measurement properties and interpretation for COAs that are meaningful to patients (15 min.). Dr. Girman will propose steps for development and justification to support use of outcomes that are fit-for-purpose for specific RWE study objectives (15 min).

The audience will participate in discussion of a case example (15 min) which will include digital handouts. This interactive workshop will be valuable to researchers studying novel outcomes in RWE studies.

Code

304

Topic

Study Approaches