METHODS: Systematic review was conducted to identify new and on-going observational (Phase 4) studies in Multiple Myeloma. The inclusion criteria were Multiple Myeloma indication, Phase 4 status and study completion date of 2016 or after. The data field extracted were study title, intervention, sponsor, age subgroups, planned enrollment, study type, study design, completion date and outcome measures.

RESULTS: Overall, 10 observational studies with total planned enrollment of 18,479 patients were identified. Eight of the ten studies were interventional, while two were observational only. Six of the ten studies were with randomized subjects, two were non-randomized, 1 was cohort and 1 was case control study. Two of the largest studies with 8100 and 9500 patients were sponsored by Janssen and Genzyme, respectively. Five of the ten studies were ‘Active’, while others were ‘Active and recruiting’. Nine of the ten studies were enrolling senior adult patients, while one study was for pediatric and adult patients. The most commonly measured outcome was survival. Other measured outcomes were complete response, relapse and PFS, time to next need treatment, time to first skeletal related event and economic impact.

CONCLUSIONS: This study provides an overview of latest trends in design of observational studies for Multiple Myeloma.