RWE, Lu

Control element in controlled experimental study

fully controlled

Control element in drug vs. placebo

control is placebo

Control element in head to head study

control is another drug

Control element in cross-over study

control is self

Drug vs. placebo, head to head, and cross-over studies are all examples of a _____ _____ _____

controlled experimental study

Two types of observational studies

descriptive, analytic

The 2 descriptive studies are _____ and _____. The 2 analytic studies are _____ and _____

Case report/series, cross-sectional; case-control, cohort

The difference between efficacy and effectiveness can be described as:

_____ aims to measure degree of success in a highly controlled environment

while _____ aims to measure success in real-world conditions

Efficacy, effectiveness

Real world evidence and real world data _____ (does/does not) come from controlled FDA approval trials

does not

_____ infers a degree of actionability whereas _____ are just the facts.

evidence, data

It is important to use RWE and RWD for analysis which is: _____, _____, _____, _____

better representation, more diverse, lower cost, faster data acquisition

Information consisting of RWE and RWD largely comes from _____ studies making it more cost effective

observational

_____ is more broad without manipulation and low internal validity but does have high external validity. _____ is more selective utilizing randomization with high internal validity but limited external validity.

RWE, RCT

Sources of RWD: any data other than _____ _____ data

clinical trial

Policy makers do not use RWD/RWE because of:

lack of expertise

21st Century Cures Act (2016) amended FD&C’s use of _____, so it can now be used for FDA drug approvals

RWE

21st Century Cures Act contains 2 core contents:

• _____ FDA’s approval and authorization for drugs and devices

• Enhance primary medical research and encourage medical _____

Accelerate, innovation

List what can be done with the FDA’s RWE program:

• additional _____

• _____

• changes in: _____, _____, drug labeling

indications, administration, dosage, usage

Synthetic control arms, or _____ studies utilizing RWD to provide RWE, can be used for FDA approval

observational

_____ data refers to data sets that store variables (demographics, drug lists, patient vitals, family hx) in the same, consistent format

Structured data

_____ data includes text-heavy documents, emails, videos, images, and audio files. Common examples include social media posts, PDFs, and customer support call recordings.

unstructured

Name a possible solution to understand unstructured data.

natural language processing