Characteristics of patients outside typical RCTs:
“The overall incidence and median age were both higher than in most previous studies, indicating a more complete coverage of older patients. Myeloma survival in Sweden is comparable to other large registry studies, and responses and survival improved during the study period.”
Cecilie Hveding Blimark, Ingemar Turesson, Anna Genell, Lucia Ahlberg, Bo Björkstrand, Kristina Carlson, Karin Forsberg, Gunnar Juliusson, Olle Linder, Ulf-Henrik Mellqvist, Hareth Nahi, Sigurdur Y. Kristinsson, for the Swedish Myeloma Registry. Outcome and survival of myeloma patients diagnosed 2008–2015. Real-world data on 4904 patients from the Swedish Myeloma Registry. Haematologica 2018;103(3):506-513; https://doi.org/10.3324/haematol.2017.178103.
Finding sub-groups with unmet need:
“National registries that include all MM patients are an important source of knowledge on epidemiology, treatment and outcome with implications for the planning of MM care. Despite the introduction of new and better treatments, rapidly implemented in our countries, our registries uncover subgroups of patients that still have inferior outcome. Our RWE can help to identify important research questions to be studied in further clinical trials also in patients currently not included in RCTs.”
Blimark, Cecilie Hveding, Annette Juul Vangsted, Tobias W. Klausen, Henrik Gregersen, Agoston Gyula Szabo, Emil Hermansen, Göran Wålinder, et al. “Outcome Data from >10 000 Multiple Myeloma Patients in the Danish and Swedish National Registries.” European Journal of Haematology 108, no. 2 (2022): 99–108. https://doi.org/10.1111/ejh.13707.
Demonstrate the role of comorbidities:
"This large study shows that over 50% of multiple myeloma patients have a comorbidity at diagnosis and survival decreased with increasing numbers of comorbidities. This emphasizes the importance of comorbidities when evaluating patients and deciding on treatment strategies for individuals with multiple myeloma."
Sverrisdóttir, Ingigerður S., Sölvi Rögnvaldsson, Sigrún Thorsteinsdottir, Gauti K. Gíslason, Thor Aspelund, Ingemar Turesson, Magnus Björkholm, et al. “Comorbidities in Multiple Myeloma and Implications on Survival: A Population-Based Study.” European Journal of Haematology 106, no. 6 (June 2021): 774–82. https://doi.org/10.1111/ejh.13597.
Evaluate the impact of different treatment paradigms within a country:
“We observed a superior survival in region A for patients receiving ASCT. Explanations may be higher usage of modern initial treatment or regional residual”
Wålinder, Göran, Anna Genell, Gunnar Juliusson, Ronald Svensson, Antonio Izarra Santamaria, Jacob Crafoord, Kristina Carlson, et al. “Regional Differences in Treatment and Outcome for Myeloma Patients in Sweden: A Population Based Swedish Myeloma Register Study.” Cancer Reports n/a, no. n/a (n.d.): e1614. https://doi.org/10.1002/cnr2.1614.
It is time that we learn how to use real world data to make better informed decisions.