Patient and Caregiver
Investigator
Regulator
IRB
Tech, Device,
PayerTrial Design Stage
Published Date: April 29, 2025
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Helpful Terms and Definitions
Study Concept
It is critical for trial designers to deeply understand and accommodate the diversity of the patient population. A variety of site types may be necessary to reach a representative group.
If this is a new relationship, there will be a lot of relationship building, depending on timelines/budgets
Questions To Ask
- Was a risk to benefit analysis performed?
- What are the options for patients whose disease worsens during treatment?
- What is the long-term strategy - open label extension period?
- Why is the trial of interest to patients and the physician community?
- Are there any recruitment challenges anticipated?
- Where is the disease prevalent?
- What particular countries/cities/sites are of focus?
- What are common operational challenges? How can these be addressed before they happen?
Who makes up Investigators and Sites?
In protocol development, “investigators and sites” encompass more than the principal investigator alone. This group includes experienced investigators, site‑based research staff such as study coordinators and research nurses, and operational experts with feasibility or startup experience. Engaging these perspectives early helps identify feasibility challenges, operational burden, and potential sources of delay before they are embedded in the final protocol. Ensure there is diversity by having representation from community sites, large research centers, and international locations (if needed).
Where will you find them?
Tapping into existing investigator and site networks can help jump start the process, but also consider establishing 'new' connections for fresh perspectives.
Mapping exercise
- Look at the landscape in that disease area - where is it prevalent, where is it treated, by whom
- Explore related conferences
- Identify PIs/authors in the literature
Why involve Investigators and Sites now?
Early and active engagement of investigators and sites lays the foundation for successful study design and execution. When sponsors involve sites early in the development of the study concept, investigators can serve as key thought partners—contributing real‑world insights that strengthen feasibility, validate the treatment rationale, confirm that the study addresses an unmet need, and ensure alignment with clinical practice. Early engagement enables sites to better plan for patient enrollment and retention, support protocol compliance, resolve data queries efficiently, and improve overall study conduct. It also allows sponsors to build trust‑based relationships with the clinicians and operations teams who will carry the trial through to completion. By treating investigators and sites as early collaborators rather than downstream operators, sponsors can design more feasible, patient‑centric, and operationally sound trials.
Their insights help ensure the trial reflects clinical reality, not just scientific aspiration.
How do you find appropriate sites?
Note: This framework is fully transferrable to sponsors, CROs, academic centers, or nonprofit research groups.