FDA | Biologics Effectiveness and Safety (BEST)

The CBER BEST Initiative Seminar Series will have Dr. Katsiaryna Bykov on June 14, 2023, 11:00-12:00 PM ET. Dr. Bykov will discuss “Avoidable and bias-inflicting methodological pitfalls in real-world studies of medication safety and effectiveness,” see our flyer. Anyone can register and join for free.

Real-world data offer great potential to estimate treatment effectiveness and safety, when RCTs are not available, but come with limitations. Many RWE studies, however, incur substantial bias not from data limitations or lack of randomization, but from avoidable, bias-inducing study design choices. In her talk, Dr. Bykov will present major sources of avoidable bias in RWE studies of medication safety and effectiveness and discuss potential solutions and current efforts to improve the quality of RWE studies.

On May 3, 2023, Ms. Xintong Li and Dr. Dani Prieto-Alhambra discussed “Leveraging real-world data for better health in Europe through collaborations between regulators & academia.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On April 12, 2023, Dr. Kaat Bollaerts discussed ““Quantifying bias due to disease- and exposure misclassification in studies of vaccine effectiveness.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On March 22, 2023, Dr. Martijn Schuemie discussed “Negative controls and p-value calibration in RWE generation.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On February 8, 2023, Dr. Fan Bu discussed “Bayesian Safety Surveillance with Adaptive Bias Correction.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On November 2, 2022, Dr. Timothy Lash and Dr. Lindsay Collin discussed “Bayesian adaptive validation design and extensions to vaccine surveillance.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On October 19, 2022, Dr. Marc Lipsitch discussed the “Everything keeps changing: What COVID-19 has taught us about surveillance.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On September 7, 2022, Dr. Matthew P. Fox discussed “Quantitative Bias Analysis Methods to Improve Inferences.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On April 27, 2022, Dr. Eric Tchetgen Tchetgen discussed “Addressing Selection and Confounding Bias in Test-Negative Study Designs for Flu and COVID-19 Monitoring.”

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On February 23, 2022, Dr. Nicole Pratt discussed the “Evaluating Use of Methods for Adverse Event Under Surveillance (for vaccines) (EUMEAUS)” task force and its findings on the comparative performance of different analytical methods for the assessment of comparative vaccine safety.

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On December 1, 2021, Dr. Heather Whitaker introduced the self-controlled case series (SCCS) method in the context of vaccine safety evaluation, with examples.

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On October 20, 2021, Nicola P. Klein, MD, PhD reviewed statistical methods used in observational studies of the safety and effectiveness of COVID-19 vaccines.

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

The general registration for the 2021 Global OHDSI Symposium, which will be held virtually in Microsoft Teams, September 12-15, 2021, is open! Register here.

On September 8, 2021, Robert Platt, PhD discussed the Canadian Network for Observational Drug Effect Studies (CNODES), which is mandated by Health Canada to conduct studies on drug utilization, safety, and effectiveness.

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On July 28, 2021, Jessica Gronsbell, PhD discussed methods that bridge classical statistical theory and modern machine learning tools in an effort to efficiently and reliably extract insight from EHR data.

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On June 16, 2021, Bruce H. Fireman, MA presented on statistical methods used in observational studies of the safety and effectiveness of COVID-19 vaccines.

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On May 5, 2021, Dr. Ben Goldstein, PhD, MD presented on informed presence in electronic health records data.

Seminar Recording Coming Soon

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

The CBER BEST Initiative Seminar Series is designed to share and discuss recent research of relevance to ongoing and future surveillance activities of CBER regulated products, namely biologics. The series focuses on safety and effectiveness of biologics including vaccines, blood components, blood-derived products, tissues and advanced therapies. The seminars will provide information on characteristics of biologics, required infrastructure, study designs, and analytic methods utilized for pharmacovigilance and pharmacoepidemiologic studies of biologics. On Wednesday, March 24, 2021, Dr. Dan Salmon presented on “Vaccine Safety Surveillance Systems for Routine and Pandemic Immunization Programs.”

Seminar Recording: https://vimeo.com/528468920/0527eab12a

Disclaimer: This presentation reflects the views of the author and should not be construed to represent FDA’s views or policies.

On Tuesday, November 17, 2020 from 2:00-3:30 pm ET, the BEST Initiative panel is discussing The Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) Biologics Effectiveness and Safety (BEST) Initiative: How Informatics Can Assist the Secondary Use of Electronic Health Records to Inform Regulatory Decisions. The panel presents an overview of the BEST Initiative and informatics considerations for the secondary use of EHRs. Using case studies in biologics, the panel will discuss ascertainment of exposures and outcomes in EHRs, development of semi-automated tools for EHR-based chart review and adverse event reporting, and interoperability and portability of computable phenotypes across EHRs. This interactive session will engage participants to understand the goals and challenges of biologics surveillance using EHRs. For more information and registration, please see AMIA Annual Virtual Symposium 2020 (November 14-18, 2020).

Registration is now open for the 2020 OHDSI Symposium, which will be held virtually October 18-21, 2020. The 6th Annual OHDSI Symposium will feature everything that makes the event an annual highlight for so many community members, including plenary sessions, tutorials and the Collaborator Showcase, as well as a two-day study-a-thon, to bring its global community together to collaboratively generate real-world evidence on an important healthcare issue. This year there will be no fees to attend any of our four-day, virtual events.

The Twelfth Annual FDA Sentinel Initiative Public Workshop is scheduled for Wednesday, October 14, 2020 from 1-4 PM EST. The online workshop provides an opportunity to discuss recent achievements and engage with the broader community of patients, consumers, and scientific stakeholders. For more information and registration, please see 12th Annual Sentinel Initiative Public Workshop webpage

Access an on-demand oral presentation by Dr. Keran Moll (IBM Watson Health) on Validating Claims-based Algorithms Identifying Pregnancy Outcomes at the International Society for Pharmacoepidemiology, ICPE All Access, September 2020