From start to finish, every stage of a clinical trial’s life cycle is critically reliant on the involvement of biostatisticians. These professionals perform various advanced statistical techniques to influence different aspects of a study, namely trial design, protocol development, data management, data analysis, and reporting. Examples of key statistical techniques applied in clinical trials include sample size determination, descriptive and inferential statistics, modeling approaches, and Bayesian methods. Although biostatistics are not typically in the public spotlight where clinical trials are concerned, these methods are the backbone of evidence-based decision-making in medicine.

Many contract research organizations (CROs) will offer in-house biostatistics services, although there are also niche companies which specialize in this field. Their goal is to ensure all clinical trials are conducted with scientific integrity and able to provide consistently reliable results. This article will explore several different types of biostatistics services that CROs like TFS HealthScience and others provide pharmaceutical, biotech, and medical device sponsors globally.

 

1. Study Design and Protocol Development Services

These biostatistics services first involve defining clear objectives and hypotheses for the new study. Then sample size calculations are carried out to determine an optimal statistical power; various mathematical formulas and considerations to determine the appropriate number of patients needed to ensure the trial’s validity and reliability. Biostatistics services are also used to select suitable randomization methods which have minimal bias, based on whether a study is open-label or blinded, using a placebo or an active comparator arm, or how many patients are needed in each treatment arm to reach the desired statistical power.

Other tasks related to robust study and protocol design include helping sponsors decide on the right primary and secondary endpoints (i.e., tangible measures used to meet the study’s objectives and hypotheses), as well as developing strategies to account for confounding factors. The overall goal of these biostatistics services is to lay a strong foundation for a new clinical trial such that its methods can effectively answer the research questions of interest.

 

2. Statistical Analysis Planning Services

All clinical trials require what is called a statistical analysis plan (SAP), which provides a comprehensive overview of specific statistical methods to be carried out during data analysis and how the results will be reported. An SAP also contains contingency strategies for handling missing data, as well as detailed plans for interim analyses where applicable.

When a CRO develops a SAP, it will typically address the following eight key components, as described in this article:

1. Introduction: Summarizes the trial’s objectives, study design, and key statistical considerations.
2. Study population: Outlines the desired participant inclusion and exclusion criteria, and any relevant subgroups or stratifications, if needed.
3. Study endpoints: Summarizes the primary and secondary endpoints of the study, including how each will be measured and analyzed.
4. Statistical methods: Describes in depth the statistical techniques used for hypothesis testing, sample size determination, and any expected adjustments.
5. Data handling and management: Outlines how the data will be collected, stored, and cleaned throughout the trial, as well as the plan to handle missing data.
6. Safety and efficacy analyses: Describes in depth the statistical methods used to analyze adverse event data, including any planned interim or subgroup analyses.
7. Results reporting: Provides guidance on how the data will be presented and in what formats.
8. Compliance with regulatory standards: Ensures the entire SAP adheres to relevant regulatory submission requirements.

Biostatistics services involving statistical analysis planning are crucial for guiding data management, analysis, and reporting throughout the entire clinical trial, as well as during regulatory submission and decision-making.

 

3. Data Management and Quality Control Services

Because statistical analysis is only as valid as the data it reviews, biostatistics services often overlap with data management and quality control services. For example, biostatisticians can offer their expertise to sponsors by helping them develop the right data capture forms (e.g., in an electronic data capture [EDC] system), implement data validation checks to promptly flag errors or inconsistencies, and suggest strategies to address data discrepancies quickly. These services can also involve providing support for regular data quality assessments, especially leading up to a database lock for interim analysis.

Most importantly, CROs with statistical expertise must possess detailed knowledge of local and international data standards and regulations, which ensure study conclusions are drawn based on reliable findings. Examples of common industry data standards include CDISC (Clinical Data Interchange Standards Consortium), FHIR^® (Fast Healthcare Interoperability Resources^®) and the OMOP (Observational Medical Outcomes Partnership) CDM (Common Data Model). The purpose of these data standards is to improve data quality and maintain consistency between clinical trials, allowing for more inter-study analyses.

Discover how TFS CRO’s Strategic Resourcing Solutions (SRS) can find expert data managers who deliver cost-effective solutions for your data management needs. Learn more here.

 

4. Advanced Statistical Modeling and Analysis

A 2024 machine learning (ML) analysis of over 16,000 randomized studies determined that clinical trial designs are increasingly becoming more complex. From decentralized clinical trials to ones with adaptive designs, biostatistics services have had to keep up with the changing study landscape in clinical research to offer more advanced statistical modeling and analysis.

Examples of advanced statistical methods include the following:

• Multivariate analysis is a statistical approach that considers many confounding or contributing factors that may influence the outcomes of a model to minimize bias in the results.
• A meta-analysis is a quantitative, epidemiological study design used to systematically assess the results of previous research, usually randomized clinical trials. This approach is able to mathematically combine and analyze outcomes from multiple studies to provide statistically valid insights across a research area. Meta-analyses often accompany systematic literature reviews.
• Bayesian statistical analyses involve complex statistical methods grounded in the science of probability and mathematically draw patient outcome insights from previous accumulated data in a study at the point of interim analysis. Based on the results, Bayesian analyses can inform planned mid-trial methodology changes, such as dropping an ineffective treatment arm, changing the sample size of a treatment arm, or stopping the study altogether if critical safety signals are detected.

 

5. Interim Analysis and Data Monitoring Services

Data monitoring and interim analysis support are among the top biostatistics services needed in clinical research when conducting a study. Interim analysis refers to an analysis of data that is conducted before data collection has been completed. The reason for this is that patient recruitment is an ongoing process that outputs staggered data outcomes as each patient enters the study. Services that support an interim analysis are related to continuous data monitoring and measuring performance indicators to ultimately minimize financial losses if a trial’s initial findings are not promising.

By helping sponsors with these tasks, they gain valuable insights at interim analysis which can identify safety signals early, indicate whether there is a need for sample size calculation adjustments, and demonstrate whether a clinical trial is progressing successfully. In the case of adaptive clinical trials, interim analysis is also the point at which the accumulated data to date are analyzed to determine whether planned protocol changes can take place in response to the findings. Ultimately, the goal of these specific services is to ensure patient safety by continuously monitoring incoming data and potentially stopping the trial early for futility.

 

6. Regulatory Support and Submission

CROs provide regulatory support in various ways, but within their biostatistics services, sponsors receive guidance on ensuring statistical compliance with regulatory guidelines. This starts with biostatisticians preparing a detailed SAP and carefully composing the relevant statistical analysis sections of regulatory documents, such as study protocols or drug approval applications. As with broader clinical trial regulations, standards that govern data management and analysis are continuously evolving, requiring sponsors to stay informed with the latest updates at all times with the help of these biostatistics services.

In the case of a New Drug Application (NDA), statistical experts help prepare the Integrated Summaries of Safety and Efficacy (ISS & ISE) form, which summarizes all relevant safety data for an investigational drug. The ISS & ISE form is a cornerstone of an NDA and ensuring it is filled out properly not only streamlines regulatory approval but also minimizes lengthy queries from the reviewing agencies. With the help of regulatory support from an experienced CRO with biostatistics services, sponsors can create a coherent, compelling narrative with their clinical trial data in compliance with strict regulatory requirements.

 

7. Data Reporting and Visualization Services

Lastly, CRO biostatistics services will provide sponsors with the appropriate reporting and visualization of data following analysis. These could involve creating clear, informative statistical reports, developing easily digestible data visualizations and infographics, preparing presentations of trial results for key stakeholders, assisting with manuscripts for publication, and even creating interactive dashboards that summarize important data highlights in a straightforward format. After the rigorous methods used to collect and analyze large volumes of data from a clinical trial, these biostatistics services in reporting and visualization serve as the final stretch that helps disseminate the findings to the scientific community and other broader audiences. The goal is to format the data in an accurate, transparent way, while still being accessible and impactful to clinicians, stakeholders, and laypeople.

 

Conclusion

In conclusion, we explored the top seven biostatistics services which are essential for conducting high-quality, scientifically rigorous clinical trials. From study design to regulatory submission, these services provide the statistical backbone that supports evidence-based medicine and drives innovation in healthcare. As the regulatory and technological landscape of clinical research evolves, biostatisticians will continue to stay informed of the latest changes to ensure the most advanced and appropriate statistical methods are available for application in clinical trials.

Interested in learning more about the role of biostatistics in clinical trials? Explore this resource here.

 

TFS CRO: Your Industry Partner in Data Management

At TFS HealthScience, we understand that strong data management is the foundation of a successful clinical trial. Our Data Management & Biostatistics team combine cutting-edge technology with seasoned expertise to deliver unparalleled accuracy and efficiency in clinical trials for clients worldwide. From study design to database lock, you can expect data integrity, compliance with regulatory standards, and seamless integration across all study phases. Our adaptive approach allows us to tailor our services to your unique needs, ensuring that your data not only meets but exceeds industry standards.

Ready to transform your clinical data management and biostatistics experience? Contact a TFS representative today or visit our website to learn what solutions we can offer for your next clinical trial!