Investigator and Site : Study Concept

Trial Design Stage

Published Date: April 29, 2025

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Helpful Terms and Definitions

ClinicalTrials.gov Glossary Terms

NIH Glossary of Common Terms

FDA Drug Development and Review Definitions

Resources*

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Study Concept

Who makes up investigators and sites?

Investigators and sites include more than the principal investigator. This group may include:

Investigators and sub-investigators
Study coordinators and research nurses
Site operations and startup personnel
Feasibility and operational experts

Engaging a diverse mix of perspectives (community sites, academic centers, and global locations when applicable) helps identify feasibility challenges, operational burden, and potential delays early in design.

Where will you find them?

Leverage both existing networks and new connections to ensure broad input.

Approaches include:

Mapping disease landscapes (prevalence, treatment settings, provider types)
Identifying investigators through publications and conference participation
Engaging professional societies and research networks
Exploring new site types for broader representation

Why involve investigators and sites now?

Early engagement helps ensure that study design reflects real-world clinical practice and site capabilities.

Investigators and site teams can:

Validate study feasibility and protocol realism
Identify operational burdens that may affect enrollment
Highlight workflow constraints and competing priorities
Provide insight into local patient populations and recruitment drivers
Improve alignment with standard of care

Early collaboration also supports stronger relationships, better planning, and more predictable study execution.

Recommended Methods of Engagement

What aspects of the study concept should they inform?

Background and scientific rationale
Natural history of the condition
Most bothersome symptoms or side effects
Standard of care and clinical workflow realities
Target population and unmet need
Study design, endpoints, and comparators
Trial design approaches (e.g., pragmatic, hybrid, decentralized)
Eligibility criteria (feasibility and appropriateness)
Ethical considerations and human subjects protections

Site Identification and Feasibility Framework

Step 1: Conduct feasibility assessment

Evaluate sites using consistent, structured criteria:

Key domains:

Patient access: Availability of target population
Operational capacity: Staffing, workload, competing studies
Experience and performance: Enrollment history, data quality, timelines
Infrastructure: Technology, lab/imaging, storage, monitoring capabilities
Diversity reach: Access to underrepresented populations
Quality and compliance: SOPs, audit history, QA processes

Step 2: Build site identification strategy

Sources of site candidates:

ClinicalTrials.gov
Professional societies (American Society of Clinical Oncology, American Society of Neurology, etc.)
Disease foundations and advocacy networks
Academic medical centers and community sites
Prior sponsor or CRO relationships
Publications, conference abstracts, and investigator networks

Mapping approach:

Align sites to disease prevalence and care settings
Identify active investigators in the field
Prioritize geographies with strong patient access

Step 3: Integrate early investigator input

Early investigator engagement strengthens site selection by:

Validating protocol feasibility
Identifying burdensome design elements
Highlighting operational constraints
Providing insight into patient populations
Offering candid feedback on site fit

Best practices:

Engage before eligibility criteria are finalized
Share draft concepts early and iterate
Use advisory boards for meaningful input (not just validation)
Discuss diversity and community considerations
Clearly communicate expectations and study intent

Step 4: Evaluate and prioritize sites

Step 5: Finalize site network

Suggested tools for feasibility assessment

Note

These approaches are intended as general guidance and may be adapted based on study design, therapeutic area, and organizational strategy.

To reach a representative patient population, a diverse mix of site types is often required. This may include academic centers, community sites, and international locations. If relationships are new, additional time, resources, and coordination will be needed to establish trust and ensure successful collaboration.

Questions To Ask

Was a risk–benefit assessment performed and is it aligned with clinical practice?
What options are available if a patient’s condition worsens during treatment?
Is there a long-term strategy (e.g., open-label extension)?
Why will this trial be of interest to both patients and clinicians?
What recruitment challenges are anticipated?
Where is the disease most prevalent and treated?
Which countries, regions, or site types should be prioritized?
What common operational challenges may arise, and how can they be mitigated early?

Investigator and Site Resources

Regulatory & Quality Standards (Foundational)

- ICH E6(R3) Good Clinical Practice (GCP): The latest international standard for clinical trial design, conduct, and data reporting, emphasizing risk-based approaches.
- ICH E8(R1) General Considerations for Clinical Studies: Essential for your QbD framework; specifically defines Critical-to-Quality (CTQ) factors and study design elements across the lifecycle.

Clinical Trial Feasibility & Risk Assessment

- Clinical Trial Risk Tool (Understanding Feasibility & Cost): An AI-based resource for assessing the risk of a protocol ending without delivering informative results, useful for pre-submission design reviews.
- NIDCR Site Assessment Questionnaire: A practical template for evaluating site readiness, facilities, and personnel experience—highly applicable for operationalizing site selection in your CTQ process.

Site Engagement & Operational Relationships

- Site Engagement for Multi-Site Clinical Trials (PMC7358177): Scientific analysis of how early site engagement influences enrollment and trial success.
- Investigator Engagement: Solving the Enrollment Crisis: Industry perspectives on improving the investigator experience to boost trial retention and performance.
- The Art of Investigational Site Relationships (ACRP): Tactical guidance on building productive, collaborative partnerships between sponsors and trial sites.
- Strengthening Investigator-Sponsor Relationships (ASCO): Targeted research on aligning incentives and communications in oncology trials.
- Evaluating Site-Sponsor Collaborative Quality (ScienceDirect): Academic research on the drivers of effective multi-stakeholder trial execution.