Overcoming Skepticism Regarding the Unmet Medical Need and Clinical Benefit of a New Treatment 

Situation

Mock advisory committees were skeptical about the unmet medical need in patients with transfusion-dependent anemia associated with a chronic blood disorder and expressed concern that the novel therapy had modest clinical benefit with short duration of response, might worsen quality of life, and could increase the risk of disease progression.

Strategy

To drive home the unmet need, we developed a visual patient journey to clearly establish where this new therapy fit in the treatment armamentarium and the limitations of existing therapies. We also superimposed the findings from the pivotal trial onto graphical presentations of historical data to visualize how the new therapy reduced transfusion rates over time without increasing the risk of disease progression.

To put the efficacy data into perspective, we highlighted data showing multiple episodes of response and cumulative duration of response to demonstrate meaningful clinical benefit over time.

We placed the patient-reported outcomes (PRO) data into the context of the disease and engaged PRO experts to clearly communicate the appropriate interpretation of the findings.

Results/Outcome

The key messages and materials created during AdCom preparation were used extensively during FDA interactions. After the late-cycle review meeting, the FDA decided to cancel the AdCom, and the product was subsequently approved. It has since become a new standard of care.