LATEST BIOPHARMACEUTICAL REPORT
Previous Webinar
The distance learning committee is excited to announce the upcoming event:
Real-World Evidence HHSU01 FDA grantees series Part 1
Date and Time: March 8 2024 10.00am – 11.00 am Eastern Time
Speakers:
Marie Bradley, FDA
Tianxi Cai, Harvard
Ashita Batavia & Benjamin Ackerman, Janssen
Registration link:
https://asabiop.ticketleap.com/real-world-evidence-nihu01-fda-grantees-series-part-1/
Below are the titles and abstracts of the talks by Marie, Tianxi, and Ashita/Ben, respectively.
Title: Overview of CDER’s Real-World Evidence Demonstration Projects
Abstract: Aligned with the U.S. 21st Century Cures Act, FDA established a program to evaluate the potential use of real-world evidence (RWE) in regulatory decision-making. The program is multifaceted and supports activities such as demonstration (research) projects, guidance development, internal Agency processes, external stakeholder engagement, and the Advancing Real-World Evidence initiative. This talk will present an overview of several RWE demonstration projects and will describe, for select projects, how learnings directly or indirectly serve to support FDA regulatory decision-making in evaluating the effectiveness and safety of medical products.
Title: Deriving reliable Real world evidence with electronic health records data
Abstract: Real-world clinical data hold tremendous potential to advance our understanding on the efficacy and safety of therapeutic interventions in broader populations, including disease modifying therapies for chronic diseases. However, these data remain underutilized due to methodological constraints and the inability to efficiently link and integrate data sources across study types and healthcare settings. This talk will discuss opportunities and challenges in leveraging electronic health records data to derive reliable real world evidence.
Title: Novel methods for aligning real-world progression-free survival (rwPFS) and clinical trial PFS endpoints in Multiple Myeloma
Abstract: Randomized clinical trials remain the gold standard for evaluating treatment efficacy because of their rigorous design and data collection parameters, which reduce bias and allow for valid inference of causal relationships. In Multiple Myeloma (MM), the brisk pace of drug development has seen twelve new therapies approved in the past decade - many of these were accelerated approvals based on single arm trials. Robust Real World Evidence (RWE) can enhance the interpretation of single arm studies, however comparisons between real world and clinical trial endpoints are limited by measurement bias.
J&J Innovative Medicine has established a consortium that includes leading academic RWE methodology experts, MM clinician scientists and Flatiron Health to develop novel treatment-agnostic methods for aligning rwPFS and clinical trial PFS. We will discuss our research approach, inclusion of under-represented minorities, and potential future applications of this work in this ASA Biopharma webinar.
Previous Webinar
Open-Source Software for Regulatory Submissions/Environments & Introducing openstatsware BIOP working group
Date and Time: February 23 2024 11.00am – 12.00 pm Eastern Time
Speakers:
Paul Scheutte, FDA
Ya Wang, Gilead
Registration link:
https://www.eventbrite.com/e/webinar-open-source-software-tickets-802097293597
Title: Open-Source Software for Regulatory Submissions and Regulatory Environments
Abstracts: 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.
Title: Introducing openstatsware: Who we are and what we build together
Abstracts: In this talk, we would like to introduce openstatsware, an official working group of the American Statistical Association (ASA) Biopharmaceutical Section. The working group has a primary objective to engineer R packages that implement important statistical methods, and a secondary objective to develop and disseminate best practices for engineering high-quality open-source statistical software. We will talk about what R packages we have been developing and what we have done to disseminate the best practices, as well as our long-term perspective and next steps.
We would also like to give an overview of our three active workstreams. The MMRM R package development workstream aims to develop a comprehensive R package for mixed models for repeated measures (MMRM) that is robust, well documented, and thoroughly tested. The Bayesian MMRM R package development workstream aims to develop an R package for Bayesian MMRM to support robust analysis of longitudinal clinical data. The HTA-R workstream aims to develop open-source R tools of good quality to support crucial analytic topics in Health Technology Assessment (HTA) dossier submission across various countries.
LATEST PODCAST
Episode 107: SPAIG Committee
Date: 2024-02-09
Description: Scott Clark, Satrajit Roychoudhury, and Michelle Shardell discuss the Statistical Partnerships Among Academe, Industry & Government (SPAIG) Committee. https://community.amstat.org/spaig/home
A message for 2021 ASA Fellow Candidates
This message is intended for BIOP members who plan to submit their ASA Fellows dossiers in 2021. Effective October 1, 2020, as approved by the Biopharmaceutical Section Executive Committee, an anonymous BIOP section working panel of dossier reviewers has been formed. The reviewers serving on the panel are experienced statisticians including former and present members of the ASA Committee on Fellows. Nominators from BIOP are encouraged to send completed dossiers for review to a current chair of the BIOP Fellows Committee (Ilya Lipkovich, ilya.lipkovich@lilly.com). All dossiers will be treated in strictest confidence. Nominators may anonymize the dossier before sending. The reviewing committee chair will send it to one of the available reviewers and provide feedback regarding potential gaps and improvements within approximately two weeks. Please submit for review well in advance of the ASA submission deadline, in order to receive feedback in time to make adjustments to the dossier. The committee will not share any personalized information about the reviewed dossiers with the BIOP Section Executive Committee, or any other committee beyond the reviewers.
You can see more details at BIOP page
We encourage you to take advantage of this service offered to the members of BIOP, and give our members the best opportunity to achieve the honor of ASA Fellow.