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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

Why do a 3rd of Submissions fail the Technical Rejection Criteria?

Clinical Programming Team

At the July 25th PhUSE Webinar Wednesday, Ethan Chen (Director, Division of Data Management Services and Solutions Office of Business Informatics, FDA CDER) gave us a glimpse of the FDA’s view on Technical Rejection Criteria for Study Data. Those that were at the US Connect Conference earlier in the year may have seen this already.

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Topics: Define.xml, ADaM Datasets, CDISC Standards, SDTM, SAS Programming, FDA, CDISC, SDTM Domains, CDISC SDTM, CDISC Guidelines

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

The Trend in Biosimilar Development and Recent FDA Guidance

Statistical Consultancy Team

At the present time, the regulatory and drug development communities are adapting to a rising trend in biosimilar development in a number of therapeutic areas.

The regulatory framework for biosimilars in the US is still evolving; the number of biosimilars approved by the FDA in 2015 was 1, this rose to 4 in 2016 and is set to increase in 2017. The FDA is developing and consulting on draft guidance documents that will shape future trials, and at this early stage there are a number of legal issues to be agreed around licencing conditions, such as the period of exclusivity and the applicability of the biosimilar to all approved indications of the reference product. To put this in context with the European landscape, biosimilars have been approved and used in the EU for over a decade without highlighting any major safety concerns. As of April 2017, there were 28 approved biosimilars in the EU on 11 different biologics. However, there are aspects of the emerging FDA guidance that will almost certainly be reflected in the evolution of trial designs in the future, for products aimed at the US market.

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Topics: PK Analysis, Biosimilars, Clinical Study Design, FDA, Regulatory Requirements, European Medicines Agency, Pharmacokinetics and Pharmacodynamic, Bioequivalence, Demonstrating Biosimilarity

The History of Pharmacovigilance Infographic

Pharmacovigilance Team
Where and when did Pharmacovigilance begin? Medical remedies have been recognized by mankind for thousands of years, and so have their potential dangers, side effects, and benefits. During the 20th century there were some serious adverse events associated with medical products and drugs that resulted in pressure on governments, businesses to produce legalization and guidance, as well as the evolution of regulatory bodies to protect the safety of patients. This infographic identifies those significant milestones across the 20th century and into the modern era.
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Topics: Pharmacovigilance, New Pharmacovigilance Legislation, Adverse Events (AEs), FDA, Black Triangle, European Medicines Agency, Good Pharmacovigilance Practices, Patient Narratives

ICHGCP E6 Addendum R2 - What do you need to know?

Gavin Boodoo



The ICH Guidelines for Good Clinical Practice (E6) has morphed into the international standard for clinical trial conduct since its inception twenty years ago. However, within that period, clinical research has changed dramatically and it is a credit to it’s authors that ICHGCP remains relevant today, with only one previous notable update seen in response to electronic data capture (EDC) technologies being widely utilized by the industry. 

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Topics: FDA, Quality Assurance, Data Transparency, Remote Monitoring, Centralized Monitoring, Risk Based Monitoring, Additional Monitoring, Technology Trends, Good Clinical Practice (GCP), ICHGCP E6

The Evolution of Risk Based & Remote Monitoring

Gavin Boodoo

With a growing trend towards the use of risk based monitoring, and with new technologies available to support this alternative to traditional onsite monitoring, there remains inertia to implement this approach. Having discussed remote monitoring capabilities with industry peers, we have identified the root cause of this reluctance as an intrinsic fear of flouting regulatory compliance within the conduct of a clinical trial.

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Topics: FDA, Remote Monitoring, On-Site Monitoring, Risk Based Monitoring, Centralized Monitoring, Regulatory Requirements, Clinical Trials, Remote Data Capture, Source Data Verification (SDV)

Integrated Summaries of Safety & Efficacy for Regulatory Submissions

Statistical Consultancy Team

Far from being simply a summary of individual results, a comprehensive and detailed summary allows companies to make informed decisions. Producing comprehensive integrated summaries of safety and efficacy is a critical stage of the submission life cycle. These need to be designed and planned carefully in advance to ensure informed decision-making and effectiveness at the regulatory interface. A focus on the approval and whole lifecycle of the product, and not just the submission, will influence the quality and direction of the content. Traceability is key in all respects of the creation of information, from data that leads to knowledgeable decisions and the ultimate wisdom that forms the label of a product.

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Topics: ISS/ISE, Integrated Summaries, Phase 2 Studies, Phase 3 Studies, Regulatory Requirements, FDA, Medical Writing, Statistics, Biostatistics Consulting

mHealth Apps and Wearables in Clinical Trials to Consider

Nick Burch

Today, mHealth, Mobile Health, Mobile Apps and Wearables are hot topics with consumers and regulators alike. We're also starting to see consumer-grade wearables and mHealth technologies creeping into clinical trials. We've written this blog for those looking to use this area more.

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Topics: mHealth, Wearables, Wearables in Clinical Trials, Technology Trends, Mobile Device, European Medicines Agency, Quality Assurance, FDA, Regulatory Requirements

Is Lack of Data the Biggest Challenge for Early Phase Pharmacovigilance?

Pharmacovigilance Team

We recently hosted a webinar on the challenges of Pharmacovigilance in early phase clinical trials. During the registration process we had the chance to survey members of the industry, 70% of the respondents were from pharmaceutical, biotechnology or medical device companies. We wanted to present the data from the 150 respondents in this blog.

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Topics: Phase I Studies, Pharmacovigilance, Clinical Trial Phases, Oncology, Serious Adverse Events (SAEs), Adverse Events (AEs), Regulatory Requirements, FDA

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