Designing a cost-effective Proof-of-Concept (PoC) clinical trial is a delicate balance between statistical rigor, clinical feasibility, and financial constraints. Achieving this requires strategic collaboration between the statistical team, principal investigators (PIs), and clinic heads. Here’s how to streamline your PoC protocol while ensuring it remains effective and aligned with industry expectations.
What to Eliminate
Before approaching clinical sites with your synopsis draft, remove unnecessary elements that do not contribute to the trial’s core objectives. Key areas to consider:
Unnecessary Endpoints: Focus only on endpoints that are commonly accepted by regulatory bodies and potential strategic partners. Exploratory trials should prioritize safety and identification of the responder population, rather than introducing endpoints unlikely to be considered in later phases.
“Interesting” but Unvalidated Additions: Avoid including endpoints purely for academic curiosity. These can add complexity and costs without clear benefits.
Overly Complex Study Designs: While adaptive and cross-over designs may seem innovative, simplicity is key for both execution and external validation. A straightforward study design is easier for potential big pharma partners to understand and accept.
Expensive and Unnecessary Technologies: Any additional device or technology—such as wearable monitors—is not a good idea. You will need full validation study to even consider it as approvable end point measurement.
Redundant Devices and Procedures: Keep the study design lean by eliminating any additional hardware or diagnostic tools that do not add direct value to the proof-of-concept objectives.
Key Steps in Drafting a Cost-Effective PoC Protocol
1. Understand Your Patient Population
Ensure that the target patient population is well-defined.
Evaluate historical enrollment rates for similar trials to assess feasibility based on the anticipated enrollment criteria.
Adjust inclusion/exclusion criteria to optimize recruitment without compromising study integrity.
2. Facilitate Collaboration Between Clinical and Statistical Teams
Work closely with statisticians and clinicians to fine-tune enrollment criteria.
Aim for the smallest patient sample size necessary to achieve clinically meaningful and statistically significant results.
3. Select the Right Statistical Model
The choice of statistical model can significantly impact required sample sizes.
Example: A client initially planned a study using a Poisson distribution, requiring 140 patients, but switching to a binomial model reduced the requirement to just 100 patients. Your statistical group has to be top-notch: this group needs to research which model this specific population fits better.
4. Drive Efficient Team Coordination
Act as the bridge between statistical and clinical teams.
This requires deep expertise in statistics, clinical trial design, therapeutic areas (TA), competitive trial landscape, and mechanism of action (MOA).
5. Optimize for Budget Constraints
If resources are limited (e.g., budget for only 100 patients), adjust enrollment criteria to ensure statistical and clinical relevance within these constraints.
Final Thoughts
Creating a cost-effective PoC protocol requires a pragmatic approach that balances clinical necessity with financial viability. By stripping away unnecessary complexities and fostering collaboration between statistical and clinical experts, sponsors can achieve efficient, trial designs that attract both investors and potential strategic partners.
At Phase Bridge, we specialize in designing streamlined, cost-effective clinical trials deliver actionable proof-of-concept data. Connect with us to ensure your study is effective and investment-ready.
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