Module 2 General Info + Unmet Need - Definitions & Questions

Translational research

Moving knowledge and discoveries gained from basic science to clinical and community settings. Often described as "bench-to-bedside" and "bedside-to-community" research.

Translational research continuum

A bidirectional continuum that moves discoveries between basic science, humans, patients, clinical practice, and the community.

Team science

Collaboration among different types of knowledge and expertise needed to move between translational phases.

Unidisciplinary

Team science involving a single discipline.

Multidisciplinary

Team science that is additive, complementary, independent, and sequential.

Interdisciplinary

Team science that is interactive and combines and integrates different disciplines.

Transdisciplinary

Team science that is holistic, transcends disciplinary perspectives, and creates new methodological or conceptual frameworks.

Unmet medical need

A condition whose treatment or diagnosis is not adequately addressed by available therapy.

Immediate unmet need

A need involving a defined population, such as treatment for a serious condition with no or limited treatment.

Long-term unmet need

A need affecting society over time, such as addressing resistance to antibacterial drugs.

Ways a new drug can address an unmet medical need

Treat a condition with no existing treatment or provide benefits that existing treatments do not, such as improved outcomes, decreased toxicity, fewer drug interactions, targeting an underserved population, or greater cost-effectiveness.

Two dimensions of unmet need

The per-patient level of need and the number of patients experiencing that level of need.

Priority when addressing unmet need

Priority may be given to patients with no other treatments or to patients expected to receive the largest health gain.

Triple-negative breast cancer

Breast cancer characterized by absence of estrogen receptor, absence of progesterone receptor, and no HER2 overexpression.

TNBC incidence

TNBC represents approximately 10-20% of invasive breast cancers.

TNBC prognosis

TNBC has a relatively poorer prognosis than major breast cancer subtypes, with peak recurrence risk within 3 years and increased mortality rates for 5 years after diagnosis.

TNBC risk associations

TNBC is associated with younger age at diagnosis, more advanced disease, higher grade, high mitotic indices, family history of breast cancer, and BRCA1 mutations.

PARP inhibitors in TNBC

Used against BRCA1/2-mutant TNBCs, representing a small subset of TNBC patients.

Estrogen-driven breast cancer therapies

Tamoxifen/SERMs, aromatase inhibitors, and fulvestrant.

HER2-driven breast cancer therapies

Trastuzumab, pertuzumab, and lapatinib.

What is translational research?

Moving knowledge and discoveries gained from basic science to clinical and community settings.

What are the four flavors of team science?

Unidisciplinary, multidisciplinary, interdisciplinary, and transdisciplinary.

Which type of team science is additive, complementary, independent, and sequential?

Multidisciplinary.

Which type of team science combines and integrates different disciplines?

Interdisciplinary.

Which type of team science transcends disciplinary perspectives?

Transdisciplinary.

What is an unmet medical need?

A condition whose treatment or diagnosis is not adequately addressed by available therapy.

How can a new drug address an unmet medical need?

By treating a condition with no existing treatment or providing benefits current treatments do not provide.

What are examples of benefits that could make a new treatment address an unmet need?

Improved treatment outcomes, decreased toxicity, fewer drug interactions, targeting an underserved population, or greater cost-effectiveness.

What are the two dimensions of unmet need?

The per-patient level of need and the number of patients with that level of need.

What defines triple-negative breast cancer?

ER−, PR−, and HER2−.

Approximately what percentage of invasive breast cancers are TNBC?

10-20%.

Which TNBC patients may potentially benefit from PARP inhibitors?

Patients with BRCA1/2 mutations.