The CCTS supports translational science pilot projects that align with the CCTS mission of accelerating the translation of research into improvements for human health and healthcare delivery. Projects must be focused on translational science – the generation of generalizable innovations that are applicable to a broad range of diseases and conditions and address translational barriers, such as those that*:

• Lack of patient/community engagement in the development and implementation of health interventions
• Ineffective clinical trial recruitment
• Failure/inability to retain participants
• Lack of novel clinical trial design (e.g. adaptive designs)
• Lack of novel endpoints for clinical studies/trials (e.g. behavioral endpoints)
• Complexity of study protocols
• Complexity in management of multi-site studies
• Clinical trials not completed on time or budget
• Translation of effective health interventions between patient populations (e.g. adult to pediatric)
• Lack of rigor, transparency, and reproducibility (e.g. clinical to real-world settings)
• Lack of data interoperability and transparency
• Challenges to data acquisition, integrity, and analysis
• Failure to utilize existing data for research (e.g. electronic health records, national cohorts)
• Challenges in testing new therapeutic modalities and drug repurposing
• Failure to correctly predict drug toxicology or efficacy
• Failure to technically execute complex mechanistic studies in human or animal models
• Lack of access to biospecimens
• Failure in translation from animal models to human trials
• Lack of common solutions across research on a range of diseases and conditions
• Lack of incentivization for collaboration
• Lack of training research teams
• Lengthy regulatory approval processes (e.g. participant consent content/process)
• Failure to disseminate health practice or policy updates
• Failure to implement health practice or policy updates

* Not listed in order of priority.

What is a Pilot Project?

• Pilot projects are small projects often done to assist in the preparation of a larger, more comprehensive study.
• Study aims focus on assessing feasibility (of recruitment, consent, randomization, data collection), scalability, acceptability, fidelity, or estimating parameters needed for a larger study.
• While pilot projects consist of planned methods to collect and analyze data, descriptive statistics are generally utilized (e.g. mean, range, standard deviation, frequency).
• References and Suggestions:
  • David Redden, PhD, Research Methodologist, discusses “Designing & Conducting Pilots”
  • NIH NCCIH “Pilots: Common Uses and Misuses”
    • Spoiler: Pilot projects are intended to answer, “Can I do this?”, not “Does the intervention work?”.
  • What are NOT pilot projects?
    • A pilot project is NOT a repackaged, underpowered version of a larger study.
    • A pilot project is NOT intended to test a hypothesis. Therefore, a pilot project is NOT designed to evaluate the effect (sizes) of an intervention on the participants (see Clinical Trial Decision Tool Question 3) and inferential statistics are NOT appropriate (e.g. T-tests, ANOVA, Chi-Square, Regression).
    • A pilot project should NOT be the basis of a sample size calculation for a main trial, as the minimum important difference (MID) should be based primarily on clinical judgement rather than statistics (doi:10.1136/bmj.i5239).

What should every pilot proposal address?

• How will the proposed work inform the next study?

Translational Barrier: Engagement of affected individuals in the development and implementation of health interventions

Development of health interventions often overlooks the very individuals the interventions are intended to help, leading to unforeseen ‘fatal flaws’ in the study design. To address this gap, a CCTS pilot study systematically assessed the patient and their caregivers’ knowledge and needs to inform the development of a home-based health program. The information gathered during this pilot, such descriptive statistics charactering participants, inform targeted recruitment for future mixed methods research studies focused on identifying perceived barriers and facilitators to participation and adherence of a home-based program intended to improve frailty and post-operative outcomes.

Translational Barrier: Patient Recruitment

Clinical trial failure is frequently attributed to lack of patient recruitment. A CCTS pilot project sought to assess the feasibility of a novel recruitment method – adolescent peer recruitment. The study team evaluated if peer recruiters could engage adolescents of similar age, background, and experiences. This work will inform the development and understanding of novel recruitment methods, which are desperately needed as part of clinicians providing evidence-based health interventions in populations most negatively impacted.

Translational Barrier: Patient Recruitment

Clinical studies and trials aim for participants to be representative of the population affected by disease. However, study participation is frequently a reflection of availability, rather than clinical presentation. To overcome this misalignment, a CCTS pilot project assessed the feasibility of engaging individuals inadequately represented in a clinical program and identify drivers towards their participation to inform future programmatic outreach.

Translational Barrier: Fidelity of Remote Data Collection

Remotely delivered health interventions represent a relatively new era of clinical research. However, remote measurement of health represents a barrier to larger utilization and participation, particularly for individuals with transportation issues. A CCTS pilot project aimed to validate a method to remotely measure health outcomes, which is expected to increase the enrollment and retention of healthy and physically disabled participants

Translational Barrier: Novel Clinical Trial Design

In several clinical settings, physicians lack evidenced-based methods to predict health outcomes, especially at the point of care, which represents a major translational barrier towards examining the efficacy of health interventions (i.e. clinical trial design). To address this barrier, a CCTS pilot project seeks to identify clinical predictors of poor health outcomes. Using sepsis survivors as the initial use-case, the study team will conduct an observational, prospective study to assess if physical function recovery trajectory and rehospitalization events can inform the development of post-intensive care unit recovery subgroups. Establishment of these subgroups could inform a novel approach to clinical trial design within and beyond the context of sepsis.

With modest pilot support, investigators can systematically and proactively address barriers in research design, thereby developing more predictive and successful health interventions.