Amgen’s Final Summary Comments on ICER’s CAR-T Assessment

Pediatric R/R ALL patients have devastating prognosis with 90 days’ median survival. Amgen’s comments are to ensure patients are at the center of this assessment. 

First, these vulnerable patients need every possible option available including CAR-T therapy and BLINCYTO (blinatumomab). There is no ‘either-or’ in this time-critical struggle to save lives.  Clinicians highlighted the importance to have multiple treatments that attack the cancer cells differently. BLINCYTO is an immunotherapy working differently than chemotherapy including clofarabine and used at different points along the treatment continuum.

Question 1 implies there is an ‘either-or’ decision between BLINCYTO and CAR-T, and wrongfully implies that BLINCYTO has similar efficacy/safety evidence as chemotherapy. We ask that BLINCYTO be removed from this question.

Second, the revised report assigns a B+ clinical rating comparing CAR-T and BLINCYTO/clofarabine despite this being an invalid comparison given the highly uncertain evidence. The CAR-T studies are single-arm with small number of patients and without long-term data. The populations in the CAR-T and BLINCYTO studies significantly differ in key prognostic factors – BLINCYTO trial has twice as many refractory (sicker) patients.

Patients’ treatment options should not be narrowed based on uncertain evidence. We ask that BLINCYTO be removed from voting question 1. If not, then the response should be “insufficient evidence” instead of “B+”.

Finally, we ask that physician/patient decision-making is preserved with no mandated treatment barriers (e.g., step therapy, mandated treatment order).  We are encouraged to see ICER highlighted manufacturer’s outcomes-based agreement and encourage ICER to incorporate these critical considerations in future assessments.