Overview of program

At the end of 2018, the FDA released a draft framework for a new Real-World Evidence (RWE) Program. The FDA proposes to use the RWE Program to guide generation of data in support of approval for new indications or to help support post approval study requirements. It is proposed to be used by both the FDA’s drug and biologic review programs. This whitepaper highlights the scope of this program, its opportunities, gaps and implications for the pharmaceutical industry.


Real World Data (RWD) refers to patient data that is collected through a variety of sources such as Electronic Health Records (EHRs), medical claims and billing data, data from product and disease registries, patient-generated data (includes in-home-use settings and mobile devices).

Real World Evidence (RWE) is clinical evidence obtained from the analysis of RWD that providing information about usage, risks and benefits of a medical product derived from sources other than traditional randomized clinical trials.

Scope of the RWE Program Framework

The FDA’s RWE program will assess inclusion of RWD to support approval of new indications and to help to support or satisfy post-approval study requirements. The following may be included:

a. Addition or modification of an indication- change in dose, dose regimen, route of administration

b. Addition of a new population

c. Addition of comparative effectiveness or safety data

d. Any other post approval requirements

RWD can be used to supplement traditional clinical trial data and improve the efficiency of these trials. Trials within the RWE Program scope can be one of two types:

  • Hybrid: traditional clinical trial supplemented with RWD from EHR, medical claims or pharmacy databases

  • Pragmatic: contains some elements which closely resemble routine clinical practice

The hybrid trial approach may include the use of RWD as a control arm in a traditional clinical trial.

Observational studies are anticipated within the pragmatic approach. The FDA intends to use an existing guidance, the Pharmacoepidemiologic Guidance, to evaluate epidemiologic studies as applicable for hypothesis generating pragmatic studies.