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10 BioPharm International ® Partnerships for Outsourcing eBook May 2023 www.biopharminternational.com Early DE vElopmEnt Another aspect that poses problems for the ATMP market is the competition for sites, points out Ching Tian, chief innovation officer, Emmes. To come out on top of potentially competing clients, it is important for a trial to be viewed by the site as the most streamlined and productive to work on, she continues. "So, in the trial operations design stage, sponsors and CROs need to ask themselves are they making the pro- cess as efficient as possible for sites to spend their time on taking care of the patients, not getting bogged down by the disjointed tools and other operational challenges?" Tian says. "For cell and gene therapies there are more tri- als than researchers able to handle them, which means sites are under great pressure—and they want to make the maximum use of the resources they have." A further hurdle to overcome in ATMP development is the complexity of the clinical trials, Tian emphasizes. "In 2020, FDA issued guidance on Long Term Follow-Up After Administration of Human Gene Therapy Products (4). Study participants may be monitored for up to 15 years in the long-term follow-up period of a clinical study to understand and mitigate the risk of any delayed adverse events," she notes. "This [scenario] is where decentralised trials can help greatly as it is very difficult to predict where a patient might be over such long period of time." Therefore, Tian continues, it is necessary for a CRO to have the correct tools and design expertise to be able to look ahead and plan appropriately for what may be re- quired, "balancing the data and integrity needed against the patient and researcher burden," she says. "For exam- ple, one possible approach is to leverage electronic health records to pull in patients' data to ensure the full extent of the follow-up, and each patient is then less encum- bered with post therapy follow up." Shortage of resources Issues surrounding the shortage of manufacturing capacity and skilled personnel for ATMP development have been well documented and exacerbated by the recent COVID-19 pandemic. However, for Raja Sharif, CEO Hataali—a technical solution provider for the per- sonalized medicine sector—an under reported issue is that of logistics for ATMPs, which need to be improved also. "Poor logistics and supply chain systems are both endangering patients—sample security—and slowing the number of trials and patients that sites can handle. The back-end processes for most advanced therapies have simply not yet caught up with the need," he says. "Sponsors, CROs, and sites could greatly reduce their resource burden if they look to bring in new technol- ogies," Sharif affirms. "For example, the logistics of a therapy's journey throughout the trial is very complex. In response, bespoke systems have been built for the approved advanced therapies and each one is unique. This [individualized approach] is putting tremendous pressure on the clinical sites in particular as they are having learn how to use multiple systems—which were often not designed for advanced therapy supply chains—and as every new trial is added the situation gets further compounded." P rov id i ng a prac t ica l e x a mple, Sh a r i f poi nt s to the fact that it is common for multiple chain of iden- tity (COI) identifiers to be used, with different COIs being used by each stakeholder, meaning that the tracking of information can be rather complicated. A solution, re- cently launched by Hataali—the Hataali 2.0—allows for the use of multiple COIs, removing the disparity of conventional systems and having a singular tracking solution for monitoring real-time location and condi- tions of an individual therapy, Sharif emphasizes. Contract research organizations need to understand the workforce and what is important to attract and retain talent. —Rhonda Henry, Emmes BioPharma This [year] will be the 'make or break' year for the supply chain—it's either going to fail and trials will stop or slow, or we will move as an industry to platforms that all providers can use. —Raja Sharif, Hataali