As the first stage of testing a new treatment in humans, the results from an early phase clinical trial provide critical data about safety and efficacy and help inform decision-making. To provide guidance to biopharma companies, this article shares some of the high-level considerations for planning an early phase clinical trial, spanning study design, patient recruitment and retention, site selection, and the role of a CRO partner.
Designing an Effective Study
Effective study design represents the foundation of any successful early phase clinical trial. To evaluate the safety and efficacy and identify therapeutic signals, sponsors must plan dose escalation, dose optimization, and dose expansion.
Dose escalation involves assessing the safety of the treatment at increasing dose levels. Typically, a small group of patients are enrolled to establish how they respond at each stage. A safety review committee then meets to evaluate the results before increasing the dose for the next cohort.
At this stage, it’s helpful to limit the number of sites, selecting those that can start quickly and sustain recruitment. Once researchers identify the optimal tolerated dose, the dose expansion phase involves wider enrollment across additional sites to gather more comprehensive data.
Patient-centric practices should also be considered in early phase development. When designing the study visit schedule, it’s important to closely align the study with the established standard of care (SOC), if possible. A trial aligned to the SOC can make the trial easier to run at sites, minimize the burden of participating, and attract more potential participants.
Recruiting Patients—and Holding Onto Them
Patient recruitment, followed by retention for the duration of the study, are critical components of any clinical trial, especially in early phase studies, where every data point is essential. A well-designed informed consent form helps ensure patients fully understand the trial and its requirements. Supporting materials, such as brochures, leaflets, videos, and a dedicated website, can also improve patient understanding and promote engagement.
To strengthen patient retention during the study’s follow-up period, patients may be more likely to remain engaged and motivated if offered options for flexible study visits, for example, with home nursing services or local sample collection. These patient-centric practices are especially valuable in pediatric trials to help meet the needs of families and caregivers, whose involvement represents a critical component of patient retention.
Finding the Right Investigators and Sites
Selecting the right investigators and sites is a pivotal step in the strategic planning and execution of early phase clinical trials. Drug development sponsors need to consider the strategic and regulatory fit of the countries and sites, as well as the expertise of the investigators. A thorough analysis of how the trial will operate is important for finding sites that can:
- Start quickly and have the appropriate resources in place
- Demonstrate a proven track record of strong enrollment
- Deliver quality data
To develop a comprehensive site selection strategy and facilitate investigator and site identification, many sponsors work with key opinion leaders and rely on the experience of a clinical research organization (CRO) partner.
Evaluating a Clinical Research Organization (CRO) Partner
Choosing a CRO is an important decision that can significantly impact a trial’s success. When evaluating CROs, sponsors should consider not only their corporate experience but also the experience of the proposed study team that will run the trial. Several additional factors should be evaluated to inform the decision-making process:
- Operational expertise: Determine how the CRO will drive an efficient and effective study.
- Range of services and support: Understand the services and support needed from a CRO to supplement a sponsor’s internal capabilities and advance their trial.
- CRO size: Sponsors should consider the CRO size in relation to their company. Smaller CROs can typically provide a more tailored and flexible service for small to mid-sized biopharma companies.
To help make a comprehensive evaluation of CROs, drug development sponsors can create a comprehensive Request for Proposal (RFP). The RFP gathers detailed information, helping sponsors understand the available options and create a standardized budget grid to compare CROs. After receiving the RFP, the bid defense process allows the sponsor to further evaluate each CRO’s capabilities, their proposed approaches to a clinical trial, and their overall understanding of the study before selecting a partner to support their product’s development.
Whether you need full-service, end-to-end support or a single service, our team at Caidya is here to help optimize your early phase development and bring your science to life. Learn more at: https://www.caidya.com/what-we-do/services/early-phase-development-and-clinical/