Takeda launches 'first-of-its-kind' global observational study
In order to learn more about the study, which Takeda says is the largest pharmaceutical company-sponsored global observational study of its kind in multiple myeloma, Outsourcing-Pharma.com talked with Liviu Niculescu, VP, Global and U.S. Oncology Medical Affairs, Takeda Oncology.
Outsourcing-Pharma.com: What are the challenges of running such a large study?
Liviu Niculescu: With a minimum of 5,000 patients to be enrolled globally across more than 150 study sites, it will take the combined efforts of Takeda, a 24-member Steering Committee of multiple myeloma experts and partners across the study locations to collect and analyze data from participants over the course of at least five years.
One of the main challenges we anticipate is recruitment, which is never easy in a large study of a relatively rare disease. However, we believe patients will be motivated to participate in the INSIGHT-MM study, as it gives them the opportunity to help increase global understanding of how multiple myeloma and its treatment affect people in the real world.
Takeda has invested years of planning to begin this study in order to gather important insights with the aim of advancing care for multiple myeloma patients. We have designed the trial to account for a large patient population, and we are confident that it will be executed properly, regardless of size.
Outsourcing-Pharma.com: How will you work with a CRO and other partners to organize and run the study?
Niculescu: The collaborative nature of the INSIGHT-MM study is one of the qualities that makes it unique. We are working with a CRO partner to help conduct the INSIGHT-MM study, under the direction of a Steering Committee of global multiple myeloma experts who will review all data and study results. Together, we will enroll patients with newly diagnosed and relapsed/refractory multiple myeloma aged 18 years or older and gather data via routine office visits, medical records and patient self-reported outcomes.
In addition, the study design takes into account feedback from patients with multiple myeloma whose experience of living with multiple myeloma and undergoing treatment will be captured by validated patient reported outcomes (PRO) questionnaires.
The INSIGHT-MM study aims to collaborate rather than compete with other registries around the world, and it remains open for the multiple myeloma community to propose analysis and request data that has been collected during the study.
Outsourcing-Pharma.com: What will the study look like? What do you hope to achieve?
Niculescu: INSIGHT-MM is a global non-interventional, observational study. Patients will be enrolled over three years, and each included patient will be evaluated for a follow-up period of at least five years, until death or the end of the study, whichever comes first.
Information regarding disease presentation, patient characteristics and previous treatments will be recorded based on review of hospital or clinic records. Multiple myeloma management data and safety data will be obtained as part of routine office visits.
Patients will complete health-related quality of life and patient self-reported outcomes surveys at inclusion and at predefined intervals throughout the study, and this information will be collected using a secure electronic data collection system (technical devices and/or paper survey forms will be provided for patients who cannot access the internet).
By gathering data on this scale, we hope to uncover best practices across the globe, identify knowledge gaps and gather important insights with the aim of advancing care for patients with multiple myeloma.
Outsourcing-Pharma.com: Why is gathering real-world data so important?
Niculescu: The treatment approaches to multiple myeloma are changing rapidly, with the introduction of new treatments that have demonstrated an impact on survival. However, there remains a need for real-world data on treatment patterns and patient outcomes.
The recently approved drugs bring hope for patients, but also bring complexity. Multiple myeloma is treated with combinations of drugs and is a disease characterized by multiple relapses. In order to identify the best combinations and the best sequencing for the right patients, we need large trials such as INSIGHT-MM.
Making the results available to the multiple myeloma community will allow researchers to generate hypotheses of what drug combinations are best for certain groups of patients, for example, and then conduct interventional trials to test those hypotheses. It is the only way to cut through the veil of complexity and find best options for patients.
Outsourcing-Pharma.com: What makes this study unique?
Niculescu: Along with an emphasis on collaboration, the openness of the INSIGHT-MM study differentiates this effort from many other pharmaceutical company-sponsored trials.
As we gather data from the study, this information will initially be available through a portal to Steering Committee members, participating sites and Takeda. After an embargo period, there will be a process in place to make the data available to the larger multiple myeloma community.
The multiple myeloma community will also have the opportunity to submit requests for the data and analyses. Requests will be reviewed by the Steering Committee, and if approved, access to the relevant data and analyses will be granted. In addition, patient-focused newsletters will be distributed that will include study progress updates and data summaries as appropriate.
