Real World Data and Its Impact on the Management of Plasma Cell Dyscrasias

The field of plasma cell dyscrasias has undergone dramatic evolution in recent yeast due to rapid therapeutic advances. While exciting, the growth of novel therapeutic strategies has created significant evidence gaps which cannot all be feasibly addressed with dedicated prospective trials. The role of real-world data (RWD) and real-world evidence (RWE) in informing management has thus also expanded. We aim to review the use of RWD and RWE in PCD management.

RWD has been used significantly to assess short- and long-term toxicities associated with rapidly expanding treatment modalities such as cellular therapies that were not explored in RCTs. Other RWE has shown that clinical efficacy of newer treatment options is maintained in patient populations that would be excluded from RCT participation. RWE has also lent greater support for and generated new ideas for optimal therapeutic sequencing in PCD. Furthermore, RWE has allowed for the development of treatment ideas for less common PCD that have not been studied in RCTs. The use of RWD and RWE in the field of PCDs has effectively supplemented the use of RCTs in expanding the treatment options available to combat PCDs. As this field continues to expand at a rapid pace, RWD and RWE will continue to be needed as a source of data to support new treatments.