Only 50 percent of new NME (New Molecular Entity) applications are approved on the first submission to the US Food and Drug Administration (FDA) and there is a median delay of 435 days to approval following the first unsuccessful submission. Additionally, with the increased use of the JumpStart data fitness review by the FDA, there are an increasing number of Information Requests (IRs) made by the FDA to resolve data issues during the submission process. IRs are likely to increase further with the introduction of a risk-based approach to monitoring and this may result in an increase in data fitness issues.

Failures occur for a variety of reasons relating to data conformance, data quality, and statistical approaches affecting conclusions on efficacy and safety.

Answering IRs and resubmitting failed applications take time and money, delaying market approval of vital new drugs to patients.

With an awareness of common reasons for submission failure and independent review of data and statistical analyses, it is possible to improve the chances of first-time submission approval. In this free webinar, the featured speakers will discuss three strategies to ensure data fitness in regulatory submissions.

1) Study Data Conformance

All FDA submissions must be Clinical Data Interchange Standards Consortium (CDISC) and electronic Common Technical Document (eCTD) compliant. A large proportion of studies are not fulfilling these criteria. The FDA are growing increasingly frustrated by this and future submissions may fail if they are not compliant. Therefore, a pre-submission expert CDISC and eCTD review will highlight where submission is strong, where non-compliance should be explained and where changes should be made.

2) Data Quality Oversight

Submissions can often fail due to missing data or lack of data integrity. Since the ICH E6 (R2) addendum, the FDA has recommended new monitoring techniques such as Centralized Statistical Monitoring (CSM) for identifying and managing issues affecting data integrity and fitness. Data Quality Oversight (DQO) is the performance of running the statistical analytics within CSM and generating reports to identify outliers and anomalies and improve the quality and integrity of clinical trial data. Any potential data issues can then be investigated and explained in the submission where relevant.

3) Statistical Approaches and Inferential Conclusions

One of the fundamental principles of ICH E6 (R2) is to ensure that study data and conclusions are of high quality, have integrity and are fit-for-purpose. Sensitivity analyses on discovered data fitness issues will determine the data’s impact of the conclusions of the study. The robustness of the submission package will be enhanced by including results from such sensitivity analyses.

This webinar aims to provide guidance and support for those preparing a submission of their Investigational New Drug (IND) to regulatory bodies by exploring DQO combined with an independent statistical review of common reasons for submission failure and a thorough CDISC and eCTD review. Risk-based approaches to monitoring are likely to result in a higher incidence of issues in data fitness in a submission, and these issues should be addressed. Uncovering data fitness issues and investigating their impact on submission conclusions through appropriate sensitivity analyses allow the sponsor to be proactive and explain these findings in their submission package, thereby increasing both the chances of a first-time approval and reducing time to approval. Join this free webinar to explore each of these strategies in-depth and learn what makes a regulatory submission successful the first time.

Keywords:

Speakers

Karen Ooms, Executive Vice President, Head of Statistics, Quanticate

Karen Ooms oversees the Strategic Delivery Business Unit (SDBU) at Quanticate. This includes the management of clinical data management, biostatistics, programming, medical writing and pharmacovigilance departments. Karen is a Chartered Fellow of the Royal Statistical Society and has a background in biostatistics spanning over 25 years. Prior to joining Quanticate in 1999 (Statwood), Karen was a senior statistician at Unilever.

Message Presenter

Paula Finch, Senior Vice President, Project Delivery, Quanticate

Paula Finch has been in the industry for over 20 years as both a statistician and a SAS programmer, working for pharma and contract research organizations, after graduating with a Bachelor of Science in Mathematics and Master of Science in Applied Statistics. She joined Quanticate at the beginning of 2019, previously serving as head of Phase I programming at Eisai. Paula oversees the project delivery for statistics and programming in the fixed fee department at Quanticate.

Message Presenter

Santosh Tymms, Statistician II, Quanticate

Santosh Tymms provides high-quality statistical support to clients, contributes to process improvement and knowledge sharing within the department and researches data quality oversight. Santosh has worked in medical statistics for four years following a Master of Science with Distinction from Lancaster University. Prior to joining Quanticate in 2015, Santosh was a teacher of English as a foreign language in several countries where he utilized his Master of Arts in English Language and Linguistics.

Message Presenter

Laura Grey, Principal Statistician, Quanticate

Laura is a Principal Statistician at Quanticate, with an MSc in Medical Statistics and PhD in Actuarial Mathematics and Statistics. Prior to joining Quanticate Laura worked in research for 10 years investigating occupational and environmental health.

Message Presenter

Who Should Attend?

This is an ideal webinar for anybody dealing with IND/NME submissions to regulatory bodies addressing solutions and methodologies for successful first-time approval.

Clinical Operation (Directors, Head of, Executives, Managers, Leads, President,Vice President, Global)
Regulatory Affairs
Head of Biometrics
Biostatisticians (Directors, Head of, Executives, Managers, Leads, President, Vice President, Global)
Programmers (Directors, Head of, Executives, Managers, Leads, President, Vice President, Global)

What You Will Learn

Attendees will gain insights into:

Common reasons for failed first-time submissions to the FDA
Compliance with CDISC and eCTD
Strategies for data quality oversight including CSM
Sensitivity analyses and other statistical approaches to assess data quality
Other data fitness issues and how to overcome them

Xtalks Partner

Quanticate

Quanticate, headquartered in the UK and USA, is a leading global Clinical Research Organization (CRO) primarily focused on the management, analysis and reporting of data from clinical trials and post-marketing surveillance. As The Clinical Data Experts our team provides high quality, efficient outsourcing solutions for companies who need additional capacity or who want to outsource certain activities in their entirety.

Our clinical and post-marketing services include scalable on-site and off-site clinical data management, biostatistics, clinical programming, PK/PD analysis, medical writing, pharmacovigilance and consultancy. Quanticate was announced a five-category winner in the annual CRO Leadership Awards for Quality, Reliability, Productivity, Regulatory and Innovation. Quanticate was the first CRO to introduce the Centralized Service Provision (CSP) approach to outsourcing supported by its data centralization and visualization tool for both single study and cross-study data analysis. Please visit the website at www.quanticate.com for further information and access to white papers.