The Use of Real-World Evidence and Data in Clinical Research and Postapproval Safety Studies.

BACKGROUND:: The adoption and use of real-world evidence (RWE) is becoming increasingly important to drug development and patient safety.

METHODS:: The Tufts Center for the Study of Drug Development (CSDD) conducted a benchmark survey of pharmaceutical and biotechnology companies and contract research organizations in a number of areas that support real-world data (RWD) and evidence, including operations and performance areas. Data were gathered on organizational functions, staff, roles and responsibilities, and skill sets required. Also, current and future allocation of budgets and spending were examined as well as return on investment measures. A total of 30 unique companies responded to the survey.

RESULTS:: Nearly all respondents (29/30 companies) reported that their organizations had an RWE function and most companies indicated that their RWE functions were increasing in size (21 companies). From a postapproval regulatory and labeling perspective, there were two primary areas for company use of RWD to generate evidence: one for postapproval safety studies, including decreasing the severity of a label warning or to support risk evaluation and mitigation strategies (REMS) (12/22 companies; 55%), which allows for real-world patient population data to inform safety decisions; and the other for postmarketing studies (13/23 companies; 57%). Developing greater insight into therapeutic area needs, gaining market access, and greater understanding of drug effectiveness were the top measures identified for return on investment for use of RWE.

CONCLUSIONS:: Expanding the use of RWE in regulatory decision making and increasing uses of real-world data by sponsors will fill the gaps that are critically needed for drug development and safety.