Select Page

Proof of Concept Studies

A Proof of Concept study is an early-stage, exploratory clinical trial that collects initial evidence regarding the efficacy of new chemical entities (NCEs) by measuring their pharmacological activity. A clinical Proof of concept study usually follows the acquisition of preclinical and early clinical safety data.

Even though proof of concept studies are multidimensional, they focus primarily on key attributes of NCEs, including their efficacy, safety, pharmaceutical properties, and regulatory issues. Proof of concept studies are usually early Phase II clinical trials and serve as a link between Phase I and more advanced Phase II clinical trials.

Significance of Proof of Concept for Clinical Drug Development

A Proof of concept study helps investigators make Go/No-Go decisions about the further development of NCEs in more advanced human clinical trials. In addition to providing initial data regarding the efficacy of NCEs, proof of concept studies also help identify the appropriate indications, primary outcome measures, and pharmacological dosages for later-stage clinical trials. Thus, an efficiently designed and conducted proof-of-concept studies may help reduce the failure rate of NCEs in more advanced stages of clinical drug development.

Did You Know?

The estimated pre-launch research and development (R&D) costs for a new drug reportedly range between $161 million & $4.54 billion. Identifying drug candidates with potential efficacy and an acceptable safety profile early on is one of the important strategies to reduce costs and streamline the drug development process. A Proof of concept study plays a crucial role in all of this.

Factors to Consider When Designing A Proof-of-Concept Study

Proof of concept studies should be planned to collect the maximal efficacy data in a relatively small number of patients while minimizing costs. They are usually designed as exploratory, randomized studies with a placebo or an active comparator.

Study Duration

The duration of a proof-of-concept study is generally limited, since it provides only initial data regarding the pharmacological activity and potential efficacy of NCEs. However, the study duration should be selected in a manner that both enables the detection of a pharmacological effect of the investigated NCE and minimizes costs.

Statistical Considerations

Generally, statistical analysis requirements are less stringent in proof-of-concept studies than in more advanced clinical trials due to their exploratory nature. In addition, Bayesian statistical models often play a role in the design of proof of concept studies. BioPharma Services’ team of biostatisticians is proficient in the creation of statistical analysis plans, as well as in data management and the conduct of detailed statistical analyses.

Integrating Clinical Safety Data and Preclinical Data

Carefully considering the results from Phase 1 safety trials and the toxicological, pharmacokinetic, and pharmacodynamic data from preclinical studies is vital for the successful design of proof of concept studies. At BioPharma Services, we work individually with our clients to design their proof of concept study in the most efficient manner possible, using their preclinical datasets and early clinical safety data.

Considering Regulatory Requirements

Designing a proof-of-concept study that complies with all regulatory requirements is a prerequisite for obtaining reliable data that can potentially be used in regulatory submissions. BioPharma Services involves its accomplished regulatory scientists in the design of early phase clinical trials, including proof of concept studies, to ensure they adhere to all relevant regulatory requirements.

Study Population

Proof of concept studies are typically the first stage of the clinical drug development process, including patients with the disease of interest. The exception is oncology clinical trials, in which patients are often already enrolled during Phase 1 clinical trail studies evaluating safety. BioPharma Services, Inc., a full-service clinical research organization (CRO), has extensive expertise in all aspects of participant recruitment and has established a comprehensive database of over 18,000 study volunteers that include both normal healthy volunteers (NHVs) and special populations.

Analytical Approaches & Their Role in Proof-of-Concept Studies 

Biomarker Incorporation

The incorporation of biomarkers may improve the efficiency of proof-of-concept studies. Biomarkers that indicate disease activity or burden are available only for a limited number of disorders, but should be implemented whenever possible. In addition, pharmacodynamic biomarkers indicating the effect of an NCE on its pharmacological target may aid Go/No-Go decisions based on proof of concept studies.

Implementing Advanced Imaging Techniques

Advanced imaging techniques, including positron emission tomography (PET), also have found important implications in proof of concept studies. BioPharma Services has established a successful collaboration with the University of Washington’s PET Center that enables us, if indicated, to employ PET imaging in the clinical trial programs of our clients.

Bioanalytical Methods

The detection and quantification of analytes in biological matrices is also a component of a proof of concept study. BioPharma has established an in-house bioanalytical laboratory that quantifies NCEs, their metabolites, and biomarkers in various biological matrices. The laboratory is equipped with a state-of-the-art liquid chromatography with tandem mass spectrometry (LC/MS/MS) and is operated by a skilled team of bioanalytical scientists.


The PK-PD modeling tool can be used to determine the dose-response relationship for NCEs and to select appropriate dosing regimens for proof-of-concept studies. BioPharma Services’ clinical pharmacologists have comprehensive experience applying pharmacokinetic-pharmacodynamic modeling for early-stage clinical trials.

Efficient, Full-service Solutions for Your Proof-of-Concept Studies

As a full-service CRO, BioPharma Services, Inc. offers its clients efficient and comprehensive solutions for the design and conduct of proof-of-concept studies. BioPharma’s multidisciplinary, award-winning team encompasses over 250 experts, including physicians, clinical pharmacologists, bioanalytical scientists, biostatisticians, and regulatory scientists. 

Furthermore, BioPharma’s staff involves principal investigators (PIs) with over 50 years of cumulative experience and at least 8 Ph.D. graduates. As a result of our multidisciplinary competence, we are able to design and perform all aspects of our clients’ early-stage trials, including proof of concept studies, promptly and efficiently. 

In addition to offering full-service solutions, our team also has niche know-how in several areas, including human abuse potential studies, pharmaceutical formulations, and working with NCEs with challenging routes of administration.

Have Other Questions?

Contact us so we can help.

Ready To Get Started?

Complete the Form below to schedule a Discovery Call with a member of our medical team to learn how BioPharma Services can be your trusted Phase 1 CRO partner!

Schedule a Discovery Call

You can unsubscribe at any time. For more details, please read our Privacy Policy.