. 2020 Feb 13;10(2):17. doi: 10.1038/s41408-020-0273-x

Table 2.

Key requirements for long-term treatment approaches.

Requirement	Specific needs for continuous therapy and maintenance treatment
Efficacy/effectiveness	•Agents/regimens must be active. •Further long-term treatment options are needed that are efficacious across patient subgroups, including those with high-risk disease¹⁷, for whom longer-term treatment is a particular requirement to achieve sustained disease control. •Additional options are also needed that have demonstrated real-world feasibility and effectiveness, with no impact on feasibility or efficacy of subsequent treatment at relapse. •Given the heterogeneity of MM, long-term treatments incorporating multiple drugs with differing mechanisms of action may be required for prolonged disease control in specific patient subgroups⁹.
Tolerability/safety	•Must be able to be tolerated for a prolonged period with little-to-no cumulative or chronic toxicity or substantive adverse impact on patients’ QoL.
Minimal treatment burden	•Minimal treatment burden through convenience of administration is important, highlighting the preference for all-oral treatment options that avoid the patient and caregiver burden associated with repeat parenteral administration. •Indeed, patient preference for all-oral vs. injectable proteasome inhibitor-based treatment has been reported in the relapsed/refractory setting¹⁴. •All-oral regimens have been shown to have lower economic burden of illness, less activity impairment, lower productivity loss, and a trend towards greater convenience than injectable regimens in the frontline setting^15,16. •A minimal treatment and toxicity burden is also important in the context of patients potentially otherwise preferring a treatment-free interval.

Requirement

Specific needs for continuous therapy and maintenance treatment

Efficacy/effectiveness

•Agents/regimens must be active.

•Further long-term treatment options are needed that are efficacious across patient subgroups, including those with high-risk disease¹⁷, for whom longer-term treatment is a particular requirement to achieve sustained disease control.

•Additional options are also needed that have demonstrated real-world feasibility and effectiveness, with no impact on feasibility or efficacy of subsequent treatment at relapse.

•Given the heterogeneity of MM, long-term treatments incorporating multiple drugs with differing mechanisms of action may be required for prolonged disease control in specific patient subgroups⁹.

Tolerability/safety

•Must be able to be tolerated for a prolonged period with little-to-no cumulative or chronic toxicity or substantive adverse impact on patients’ QoL.

Minimal treatment burden

•Minimal treatment burden through convenience of administration is important, highlighting the preference for all-oral treatment options that avoid the patient and caregiver burden associated with repeat parenteral administration.

•Indeed, patient preference for all-oral vs. injectable proteasome inhibitor-based treatment has been reported in the relapsed/refractory setting¹⁴.

•All-oral regimens have been shown to have lower economic burden of illness, less activity impairment, lower productivity loss, and a trend towards greater convenience than injectable regimens in the frontline setting^15,16.

•A minimal treatment and toxicity burden is also important in the context of patients potentially otherwise preferring a treatment-free interval.

QoL quality of life.