Statistics & Data Science Community

Thursday, September 12 at 11:00 AM EST

Please join the Good Clinical Practices & Quality Assurance Community for a dicussion with Arthur Allignol, HTA Statistician at Daaichi Sankyo. Arthur will be discussing the impact that EU HTA will have on statisticians across the industry

Register in Advance for this Meeting

https://diaglobal.zoom.us/meeting/register/tJIlfu2grj0sH9V7XeJJTHAVsYUoUYGfkGOw#/registration

What is EU HTA? In 2021, the European Commission passed a new regulation [1] on Health Technology Assessment (HTA) which will require manufacturers to submit clinical evidence as part of a joint clinical assessment (JCA), addressing the needs of all member states in terms of data and analyses. EU HTA is getting closer (applies as of January 2025 for certain oncology medicines) and will have a major impact on the work of statisticians across the industry.

Final versions of the Implementing Act describing the high-level submission process, and methodological guidelines, that provide applicants and assessors guidance on which statistical methods are appropriate in given situations, have recently been published [2]. Further guidelines, in particular related to processes, are yet to be finalized.

The PICO (Patient, Intervention, Comparator, Outcome) framework is a key topic in EU HTA. This framework will be the standard format in which HTA bodies will specify the research questions that define the assessment scope. Meanwhile, estimands are becoming standard for describing "the treatment effect reflecting the clinical question posed by a given clinical trial objective" in clinical studies.  While the PICO and estimands frameworks overlap, the latter further defines the population-level summary and intercurrent event strategy.

We review some of the challenges we can foresee, such as the potential need for a number of different analyses to satisfy the needs of various EU member states, as multiple PICOs may require multiple analyses strategies. Statisticians must also be prepared to handle tight timelines in case of label changes as the joint clinical assessment is due before the CHMP opinion. We will also go through the many unknows surrounding this new process.

Reference

---------

[1] [Regulation on Health Technology Assessment (europa.eu)](https://health.ec.europa.eu/health-technology-assessment/regulation-health-technology-assessment_en#stakeholder-consultation) last accessed 15th August 2024

[2] https://health.ec.europa.eu/health-technology-assessment/key-documents_en?f%5B0%5D=topic_topic%3A225&f%5B1%5D=topic_topic%3A226&f%5B2%5D=topic_topic%3A227&f%5B3%5D=topic_topic%3A228 (last accessed 15th August 2024)

Bio

===

Arthur Allignol is currently HTA statistician at Daiichi Sankyo and is involved in the preparation work for the new EU-HTA regulation. Arthur is an active member of the PSI/EFSPI HTA Special Interest Group.

Before joining Daiichi Sankyo, he worked as Real-World Data Scientist at Merck

Healthcare KGaA and in academia, specializing in observational studies and complex event-history analysis. Arthur Allignol obtained his PhD in biostatistics at the university of Freiburg in 2013.

The DIA Statistics and Data Science Community are pleased to announce a new webinar "AI Redefining Medical and Safety Review: A Paradigm Shift" which will be held on 05 Sep 2024, 11:00am - 12:30pm EST. 

You will be able to access the seminar using the following meeting details

Topic: AI Redefining Medical and Safety Review: A Paradigm Shift

Time: Sep 5, 2024 08:00 AM Pacific Time (US and Canada)

Join Zoom Meeting

https://diaglobal.zoom.us/j/96494820146?pwd=kgeO24aNBkD12FAPaFcAtaanpnY0AC.1

Meeting ID: 964 9482 0146

Passcode: 057813

Hi Everyone,

The DIA Statistics and Data Science Community is pleased to announce a new webinar which will be held on Thursday 9th May, 2024. The "Open Source Software for Clinical Trial Reporting and Regulatory Submissions" webinar will feature presentations from industry and regulatory experts. Further details are given below. Registration for this free webinar is now open and is accessible via this web address: https://diaglobal.zoom.us/meeting/register/tJcqd-GurjkrGtwVlzPSc67AfVe58a9domMW

Webinar Abstract: Validated proprietary software systems and packages have traditionally been the norm for statistical analyses underlying regulatory submissions for approval of new therapeutic products. While academic institutions and other industries have embraced open-source software, the pharmaceutical industry has been slower to adopt the open-source counterpart. This webinar brings in several industry and regulatory experts to share their experiences and perspectives on using open source software for clinical trial reporting and regulatory submissions.

Webinar presentations and abstracts:

1. Eric Nantz (Director, Eli Lilly and Company): Highlights of the Successful R-Consortium Pilot Submissions

Abstract: Within the life sciences industry, the usage of open-source statistical software has made tremendous gains across the lifecycle of clinical studies in many organizations. One particular area that open source has encountered challenges historically is the clinical submission process. The R Consortium R Submissions working group, with representatives from industry sponsors and regulatory agencies, was created to assess the feasibility of using the R statistical programming language to create clinical submission deliverables while adhering to current guidelines used in submissions. This presentation will highlight the successful pilot submissions that cover a wide spectrum of using R to produce traditional clinical outputs, sophisticated Shiny applications, and analytical data sets. Additionally, we will highlight the ongoing efforts that aim to push the envelope even further with the use of web-assembly and container technology to distribute a Shiny application in a future pilot.

2. Ning Leng (People and Product Leader (Director), Genentech): Embracing open source language and modernized technologies - Roche's journey in clinical trial reporting

Abstract: In 2024, Roche embarks on a transformative journey aimed at modernizing our clinical studies by embracing cloud-based system, container and modernized version control solutions. This transition also empowers individuals to utilize the R language for generating SDTM, ADaM, and TLG outputs. This presentation will delve into Roche's transformational path, highlighting our inaugural milestone of leveraging end-to-end R solutions for pivotal readouts and filings. Additionally, we will discuss our dedication to the open-source community and our endeavors to enhance data science efficiency through the adoption of LLM.

3. Regulatory Expert: Open-Source Software for Regulatory Submissions and Regulatory Environments

Abstract: Regulatory submissions and regulatory computing environments have traditionally been associated with the use of proprietary software packages. While academic institutions have embraced open-source software, both industry and government have been slower to adopt open-source alternatives. I will discuss some of the challenges and issues with using open-source software in a regulatory environment, followed by some of the lessons learned in the ongoing R Consortium R Submission Pilot, as well as emerging issues