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Statistical Consultancy Team

Statistical Consultancy Team

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FDA Guidance for Human Gene Therapy for Hemophilia A & B

Statistical Consultancy Team

Having seen an increasing number of gene therapy approvals, the FDA has issued draft guidance1 to help the developers of human gene therapy (GT) products for the treatment of hemophilia A & B.  In this article we will be focusing our attention on what guidance has been provided about the design of human gene therapy clinical trials for hemophilia A & B, including what is needed to support an accelerated approval approach. 

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Topics: rare diseases, Clinical Trials, FDA, hemophilia, Statistical Consultancy, Phase I Studies, Phase 2 Studies, Phase 3 Studies, Phase 4 Studies, Regulatory Requirements, Clinical Study Design, Additional Monitoring

A Guide to Phase 1 Clinical Trial Designs

Statistical Consultancy Team

The primary aims of Phase 1 Clinical Trials are to determine the safety, tolerability and pharmacokinetics (PK) of a compound. Trials have historically been conducted in the logical sequence of single ascending dose, multiple ascending dose, examination of preliminary effect of food on exposure, and potential drug-drug interaction, with assessments to determine the effect of gender, age, bioavailability and bioequivalence performed as necessary.

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Topics: Pharmacokinetics and Pharmacodynamic, Phase I Studies, Phase I Study Design, Clinical Trial Phases, Bioequivalence, Drug-drug Interaction, Bioavailability, Phase 3 Studies, Peadiatric Assessments

Statistical Methods in Risk Based Monitoring

Statistical Consultancy Team

This blog explores the statistical methods used in Risk Based Monitoring (RBM) and how the result of such statistical methods enables improved data integrity across a clinical trial.

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Topics: Statistical Consultancy, Laboratory Datasets, ICHGCP E6, Risk Based Monitoring, Statistics, Centralized Monitoring

[Published Journal] Using Observational Studies to Investigate the Relationship Between Fatigue and Work Disability

Statistical Consultancy Team

Our Statistical Consultancy Team recently collaborated on a study to investigate the long-term relationship between fatigue and work disability in patients initiating treatment with etanercept for rheumatoid arthritis (RA) or ankylosing spondylitis (AS); and this work has now been published. (https://arthritis-research.biomedcentral.com/articles/10.1186/s13075-018-1598-8)

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Topics: Statistical Consultancy, Observation Longitudinal Database, Observational Studies, Inflammatory Rheumatic Diseases, Rheumatoid Arthritis (RA)

Nutraceutical trials: demand, design and challenges

Statistical Consultancy Team

Food ingredient and nutraceutical manufacturers are increasingly looking to market their products by substantiating their unique health benefits.

There are many generic, widely used claims - in the EU, Article 13.1 of the European Food Safety Authority’s (EFSA) EC Regulation permits manufacturers to use some 4,6371 that pertain to vitamin and mineral content.

This proliferation is driving manufacturers to develop unique claims to use in marketing efforts, which in turn has led to a significant growth in demand for nutraceutical trials.

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Topics: Nutraceutical Trials, Biostatistics Consulting, Statistics, Clinical Study Design, Regulatory Requirements

Bayesian approaches to use historical data in the analysis of clinical trials

Statistical Consultancy Team

The BAYES 2018 Bayesian Biostatistics workshop took place from the 20th to the 22nd of June, and was an extraordinary chance to discuss how Bayesian methodology can be used within the pharma industry.

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Topics: Bayesian Study Design, Bayesian Methods, Bayesian Statistics, Historical Data, Statistical Consultancy, Statistics, Biostatistics Consulting, Clinical Study Design

[Video] Bayesian Methodology - How to Analyse Multiple Endpoints

Statistical Consultancy Team

In this Statistical Knowledge Share Video our Principal Statisticians, Sonia, presents an example of Bayesian Methodology using simulated data and examines how to analyze Multiple End Points in Clinical Trials.

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Topics: Statistics, Statistical Knowledge Share, Multiple Endpoints, Bayesian Methods, Bayesian Statistics, SAS Programming

[Video] Tipping Point Analysis in Multiple Imputation for Binary Missing Data

Statistical Consultancy Team

In this Statistical Knowledge Share Video our Senior Statisticians, Niccolo, presents an example on simulated data of Tipping Point Analysis in Multiple Imputation for Missing Data.

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Topics: Tipping Points Analysis, Multiple Imputation, Missing Data, Statistics, Statistical Knowledge Share

Risk-Based Monitoring and the Need for Programmers and Statisticians

Statistical Consultancy Team


The ICH E6 on Good Clinical Practice was updated on 9 November 2016, the first addendum for 20 years. Sections 5.0 on Risk and 5.18.3 on Extent and Nature of Monitoring in particular created an increased need for Risk-Based Monitoring (RBM) and Centralized Monitoring (CM). The details of how this may be covered and the increasing evolution of a risk in the industry have been noted. The required changes involved create a number of different team roles. These include opportunities for biostatisticians and statistical programmers - players who may not have been so directly involved in onsite monitoring, a more traditionally clinical domain.

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Topics: Risk Based Monitoring, International Conference on Harmonisation (ICH), ICHGCP E6, Centralized Monitoring, Fraud Detection, Source Data Verification (SDV), On-Site Monitoring, Visualization

Patient Reported Outcomes (PRO) to Support Medical Product Labeling Claims

Statistical Consultancy Team

With encouragement from the U.S. Food and Drug Administration (FDA), using Patient Reported Outcomes (PRO) data to claim labeling became more and more popular. Well-defined and reliable PRO can be used to support a claim in medical product labeling. [1] It is found that there are an increasing number of regulatory submissions for new drugs to provide PRO data to support claims. DeMuro et al. (2013) [2] have reviewed drug approvals by both FDA and EMA for the years 2006–2010. They found that out of 75 drugs approved by the EMA, 35 (47%) had at least one PRO related claim approved by the EMA compared to 14 (19%) for the FDA.

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Topics: Patient Reported Outcomes, medical labeling, Biostatistics Consulting, PRO Instrument, FDA, European Medicines Agency, Electronica Patient Reported Outcome (ePRO), Statistical Analysis Plan

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