Therapeutic Areas

Therapeutic Area (TA) expertise becomes more and more important for clinical data scientists working
in the pharmaceutical industry as it is crucial for the understanding of patients’ needs and the
interpretation of analysed data.

For a profound knowledge of a TA, the following information is of critical importance for the clinical data
scientist:

• Description of Disease

• Demographics and Baseline Characteristics

• CDISC Standards and Therapeutic Area User Guides

• Agency Guidelines

• Study Design and Study Endpoints

• Data Challenges

Our PHUSE Educations teams is working on comprehensive overviews for various Therapeutic Areas,
which are referenced below.
Each comprehensive TA overview follows the structure as described below.

1 - Oncology

Description of Disease
A detailed description of a TA should include history, incidence, etiology and symptoms of a disease.
Where different indications and classifications are available, this information should also be added to
get a full picture of the disease.

Demographics and Baseline Characteristics

Each TA has its own demographics and baseline characteristics. The relationship between these
different patient groups and the endpoints typical for the TA could be presented easily by using
common data visualisation or graphic tools. On the one hand, this will result in a better understanding
of data especially for clinical data scientists new to a TA. On the other hand, these data could be used in
collaboration with statistical algorithms to be able to give predictions for upcoming studies (saving time
and costs) so the awareness of these opportunities will be of a great advantage during the clinical
development of a drug.

CDISC Standard TAs and Therapeutic Area User Guides (TAUGs)

On the CDISC webpage there is a list with different TAs and their corresponding user guides
(https://www.cdisc.org/standards/therapeutic-areas) available. These TA standards could be seen as the
extension of the foundational standards to represent data that pertains to special disease areas. TA
standards include disease-specific metadata, examples and guidance on implementing CDISC standards
for a variety of uses, including global regulatory submission. The nomenclature of the TAs found on the
CDISC webpage should be kept for the PHUSE Education TA sub-cluster, to be aligned with the official
standards.
Agency Guidelines (e.g. the FDA, PMDA, EMA)
The design of clinical studies often depends on the agency’s needs for registration of a drug. As these
guidelines usually provide a lot of information on target populations and endpoints of studies they could
serve as an excellent starting point to learn more about study set-up in this TA. In addition, guidelines
from other scientific medical organisations could be used if the agency guidelines are not very detailed.

Study Design and Study Endpoints (Safety and Efficacy)

As mentioned in the chapter above, there are quite often agency recommendations for study designs
(e.g. up-titration, down-titration, use of pre-existing data) and study endpoints (e.g. typical scales,
Patient Reported Outcomes (PROs)). Where there are trends identifiable, these should be highlighted
and listed under this chapter.

Data Challenges
As TAs quite often use the same study endpoints and data measurements, challenges with data could be
observed in more than one study. A detailed explanation of the challenge and the solution or work-
around to fix this issue would support data scientists new to this TA as they will pay attention to these
data from the beginning.

Reference Material
Recommended PHUSE educational material

Recommended Readings

Recommended Videos

Recommended Websites