Recently IQVIA’s Erin Finot’s recently discussed the importance of site selection for clinical trials involving advanced therapies (ATs). Represented by gene and cell therapies, this movement pushes the scientific and technological envelope in the quest to improve quality of life and combat disease.
Traditional small molecule-based biotech therapies focus on addressing symptoms of disease while AT’s according to Finot’s recent piece in Contract Pharma “restore or establish normal function through the modification of human cells (cell therapy), or of DNA or RNA (gene therapy).
Growing Number of ATs
Ms. Finot, helming IQVIA Biotech’s Immuno-Oncology division, notes 17 ATs have thus far been approved by the FDA. Although ATs are applicable to multiple therapeutic areas most are presently focused on oncology where there is great unmet need. A recent MIT-based study indicates that there are 628 U.S.-based clinical trials programs for gene and cellular therapies making up 211 indications, 335 preclinical studies, 91 phase I studies, 174 phase 2 studies and 19 phase III studies. The study’s authors projected between 40 and 50 AT product launches by 2030 and approximately 12 within the next five years.
AT’s Introduce Site Selection Challenges
With complex requirements and considerations typically greater than traditional studies, sponsor clinical operational strategies may morph factoring in the need for new expertise involved with the science, regulatory elements and logistics of managing the AT trial—including the manufacturing process and biospecimen handling.
Site identification, qualification and selection in the world of ATs represents a competitive differentiation for sponsors and undoubtedly in many cases will seek advice and counsel from experts in the field such as Ms. Finot. She notes that sites that have the wherewithal and sophistication to manage the required coordination and integration of the specialized activities represents a fundamental prerequisite. Often sponsors have aggressive timelines but Finot cautions that additional time during standard qualification and initiation visits must be committed in the study start up plan.
The author suggests sponsors consider institutional accreditation from the Foundation for the Accreditation of Cellular Therapies (FACT) as one indicator of quality and readiness for AT site qualification. Why? Because this certification represents at least one accepted measure of readiness for advanced therapy standards. She suggests sponsors can also probe CRO or third-party databases or subscription services to study a particular site’s past performance. Importantly, sponsor and CRO relationships in the field are important—tapping into specialized knowledge networks—involving investigator relationships, connectivity to thought-leaders and other relevant networks of investigators for recommendations. Many sponsors have outsourced much of their clinical site qualification activity hence they can hire the expertise such as the kind possessed by Ms. Finot.
TrialSite News currently is surveying all sites involved with the existing approved ATPs and will share results as and when they are available.
MS. Finot discusses other challenges with ATs including the careful navigation and maneuvering through regulatory requirements, coordination of logistics and how CROs can help sponsors handle some of the complexities. With the transformation from volume to value in the drug development sphere many more emerging biopharmaceutical companies are raising funds and developing ATs. They too will need specialized support and assistance from third parties as it is difficult to manage the entire AT R&D and clinical trials process in house.
Call to Action: TrialSite News is undertaking a survey of sites involved with AT approved therapies—we will share this when it is complete—available for all. ATs require specialized knowledge for conducting site selection and if that knowledge isn’t in house we recommend reaching out to experts such as Ms. Finot.