Table 1.
Overview of research concepts and questions that remain underrepresented in clinical cancer research today (Kempf et al., 2017; Lacombe et al., 2017; Lacombe et al., 2019b). The conclusions which may emerge from studies that explore such topics are highly relevant for clinical practice and are shown for a given treatment A.
| Research concept | Research question | Possible conclusions |
|---|---|---|
| Combination | How and to which extent should the new therapeutic intervention be combined with other existing treatments? | Treatment A should or should not be combined with treatment B for optimal effectiveness |
| Sequence | In which sequence does the new therapeutic intervention have to be applied when combined with additional therapies? | Treatment A is best given before, after or at the same time as treatment B |
| Comparison | How well does the new therapeutic intervention perform compared to alternative treatments? | Treatment A is better or worse than or noninferior to standard-of-care treatment B |
| Performance in real-world patients | How will the new therapeutic intervention perform in patient populations that were excluded from clinical trials? | Specific patient subpopulations may experience better, worse, or similar outcomes when given treatment A compared to the sample of participants included in the clinical studies |
| Treatment duration | How long does the new therapeutic intervention have to be applied to achieve the desired effects? | Treatment A should be administered for as long the patient lives or may be discontinued after a certain amount of time with no effect on disease outcomes |
| Dosing | What is the lowest dose at which the new therapeutic intervention can be given without negatively impacting treatment outcomes? | The dosage of treatment A should remain unchanged or may be lowered with no effect on therapeutic outcomes |
| Long-term outcomes | How do the efficacy, effectiveness and safety of the new therapeutic intervention evolve over a longer period of time? | The efficacy, effectiveness, and/or safety of treatment A may remain stable or decrease over time |
| Patient-relevant outcomes | How does the new therapeutic intervention perform in terms of patient-relevant outcome measures? | Treatment A may or may not significantly improve patients' perceived health status, quality of life, or overall survival |