SEPTEMBER - 2023LIFE SCIENCES REVIEW8By Nelly Marmy Conus, DCT Manager, Decentralized Solutions, Syneos Health SETTING UP ORGANIZATIONS FOR LONG-TERM FOLLOW-UP STUDIES eporting on the long-term safety of patients who receive cell and gene therapies remains vitally important to regulators. The European Union, for example, mandates continued follow-up for the remainder of the patient's life while the United States mandates a follow-up period of up to 15 years. Similarly, the need to maintain contact with patients across a range of therapy areas including oncology, rare diseases, and chronic conditions to understand long-term survival, safety, quality of life, and other parameters is essential. This, however, poses challenges for investigators, sites, patients, and sponsors. Limited budgets, a drain of site resources, and difficulties with engaging patients for extended periods make long-term follow-up (LTFU) a challenge. Given the importance of minimizing patient attrition in long-term survival studies and the difficulties posed by traditional data collection methods, the process is ripe for an overhaul. By applying new pragmatic approaches, and making full use of the latest technologies, it is possible to better engage patients and reduce the resources required in these types of studies.Centralize for EfficiencyPatient data lost to follow-up can impact trial findings. One solution to relieve the burden on sites is to work through a "megasite" or international coordinating center--a facility responsible for centralized medical oversight and follow-up with patients, regardless of how many sites were involved in the pivotal trial. A centralized approach can ensure that safety signals are easily aggregated and flagged from multiple sources. In situations where the number of LTFU patients can be very high (e.g., a cardiovascular outcomes study), remote contact with patients can be maintained through a call center approach, with any clinical follow-up required being passed through to a clinical team from the megasite.To ensure a smooth and efficient process of centralizing LTFU, key initiatives can be implemented, including early engagement with sites, patient advocacy groups, and regulatory authorities to confirm the acceptability of a megasite model and anticipate any issues or delays. Medical release approval of patients in the informed consent, appropriate system of data capture and clearly outlined data ownership will also be key for success.Streamline Data Collection Another challenge facing LTFU studies is the time frame in which the data is collected. Traditionally, sponsors have collected data on patients in these types of studies in an unstructured way as patients complete scheduled visits. There is, however, no requirement that data be collected in real-time.It is far more efficient to have the megasite collect and enter data from all treating physicians (or patients) at selected points in time. For example, the megasite could ask all local treating hospitals for patient data during March. The data would then be processed all at once, which has many advantages. It streamlines data monitoring and allows better planning of interim analysis, reporting, and medical communication. Such efficiency in monitoring and analysis can also deliver significant savings in operational costs.rNelly Marmy ConusIN MY OPINION
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