Date: Wednesday 23rd March 2022
Time: 14:00-16:30 GMT
Speakers: Gerry Downey (Bluebird Bio), Shihua Wen (Novartis), and Chenxuan Zang (Duke University).
Who is this event intended for? Statisticans working in - or interested in - gene therapy treatments.
What is the benefit of attending? Attendees will have the opportunity to hear topics and case studies covered that relate to: Gene therapy, Estimands, Real world data and Trial design.
You can now register for this event. Registration fees are as follows:
- Members of PSI = Free of charge
- Non-Members of PSI = £20+VAT
To register for the session, please click here.
Research and clinical trials for gene therapies pose many specific challenges. These can be due to comparing potentially curative treatments to a standard of care with different modes of action or even no currently available treatments. There may also be small sample sizes due to rare or ultra-rare diseases. The talks in this webinar will cover challenges related to study design, including estimands, and use of real-world data and evidence to supplement clinical trial data. Case studies of such challenges will be presented.
|
Speaker |
Biography |
Abstract |
|
|
Gerry has over 20 years experience in biostatistics and began his statistical career as an industrial placement student with GlaxoWellcome in 1998, before moving from the UK to the United States to work in academic funded clinical research in HIV. Prior to joining bluebird bio in 2021, Gerry worked in gene therapy at Orchard Therapeutics and Amgen, most recently supporting the regulatory approval and reimbursement of gene therapy products including Libmeldy (@ Orchard) and Imlygic (@ Amgen).
In his current role at at bluebird bio, Gerry oversees biostatistics activities for real world evidence and reimbursement dossiers for bbb gene therapy programs. |
Gene Therapy, Real-World Data and Real-World Evidence |
|
|
Dr. Shihua Wen is currently a director of biostatistics in Novartis Pharmaceuticals Corp. (US). He joined Novartis in 2016 and started to work in gene therapy area since last year. Dr. Wen has rich experience in late phase clinical development across multiple therapeutic areas. At Novartis, he serves as the statistical lead for global clinical development programs in neurology area and successfully supported multiple regulatory submissions for health authorities’ approval. Prior to Novartis, he worked in Abbott Laboratory / AbbVie Inc. as a biostatistician as well with increasing responsibility. Dr. Wen received his doctoral degree in statistics at University of Maryland, Collage Park, in 2007. His research interests are drug development, benefit-risk assessment, innovative trial design, data fusion, etc. |
Estimands in gene therapy trials |
|
|
Chenxuan is a 2nd-year Master student, studying Biostatistics and Bioinformatics at Duke University School of Medicine. Her research interests include: clinical trial methodology, real-world data/real-world evidence, composite index, statistical genetics.
|
Statistical Considerations for Gene Therapy in Rare Diseases Clinical Trials |
Date: Wednesday 23rd March 2022
Time: 14:00-16:30 GMT
Speakers: Gerry Downey (Bluebird Bio), Shihua Wen (Novartis), and Chenxuan Zang (Duke University).
Who is this event intended for? Statisticans working in - or interested in - gene therapy treatments.
What is the benefit of attending? Attendees will have the opportunity to hear topics and case studies covered that relate to: Gene therapy, Estimands, Real world data and Trial design.
You can now register for this event. Registration fees are as follows:
- Members of PSI = Free of charge
- Non-Members of PSI = £20+VAT
To register for the session, please click here.
Research and clinical trials for gene therapies pose many specific challenges. These can be due to comparing potentially curative treatments to a standard of care with different modes of action or even no currently available treatments. There may also be small sample sizes due to rare or ultra-rare diseases. The talks in this webinar will cover challenges related to study design, including estimands, and use of real-world data and evidence to supplement clinical trial data. Case studies of such challenges will be presented.
|
Speaker |
Biography |
Abstract |
|
|
Gerry has over 20 years experience in biostatistics and began his statistical career as an industrial placement student with GlaxoWellcome in 1998, before moving from the UK to the United States to work in academic funded clinical research in HIV. Prior to joining bluebird bio in 2021, Gerry worked in gene therapy at Orchard Therapeutics and Amgen, most recently supporting the regulatory approval and reimbursement of gene therapy products including Libmeldy (@ Orchard) and Imlygic (@ Amgen).
In his current role at at bluebird bio, Gerry oversees biostatistics activities for real world evidence and reimbursement dossiers for bbb gene therapy programs. |
Gene Therapy, Real-World Data and Real-World Evidence |
|
|
Dr. Shihua Wen is currently a director of biostatistics in Novartis Pharmaceuticals Corp. (US). He joined Novartis in 2016 and started to work in gene therapy area since last year. Dr. Wen has rich experience in late phase clinical development across multiple therapeutic areas. At Novartis, he serves as the statistical lead for global clinical development programs in neurology area and successfully supported multiple regulatory submissions for health authorities’ approval. Prior to Novartis, he worked in Abbott Laboratory / AbbVie Inc. as a biostatistician as well with increasing responsibility. Dr. Wen received his doctoral degree in statistics at University of Maryland, Collage Park, in 2007. His research interests are drug development, benefit-risk assessment, innovative trial design, data fusion, etc. |
Estimands in gene therapy trials |
|
|
Chenxuan is a 2nd-year Master student, studying Biostatistics and Bioinformatics at Duke University School of Medicine. Her research interests include: clinical trial methodology, real-world data/real-world evidence, composite index, statistical genetics.
|
Statistical Considerations for Gene Therapy in Rare Diseases Clinical Trials |
Date: Wednesday 23rd March 2022
Time: 14:00-16:30 GMT
Speakers: Gerry Downey (Bluebird Bio), Shihua Wen (Novartis), and Chenxuan Zang (Duke University).
Who is this event intended for? Statisticans working in - or interested in - gene therapy treatments.
What is the benefit of attending? Attendees will have the opportunity to hear topics and case studies covered that relate to: Gene therapy, Estimands, Real world data and Trial design.
You can now register for this event. Registration fees are as follows:
- Members of PSI = Free of charge
- Non-Members of PSI = £20+VAT
To register for the session, please click here.
Research and clinical trials for gene therapies pose many specific challenges. These can be due to comparing potentially curative treatments to a standard of care with different modes of action or even no currently available treatments. There may also be small sample sizes due to rare or ultra-rare diseases. The talks in this webinar will cover challenges related to study design, including estimands, and use of real-world data and evidence to supplement clinical trial data. Case studies of such challenges will be presented.
|
Speaker |
Biography |
Abstract |
|
|
Gerry has over 20 years experience in biostatistics and began his statistical career as an industrial placement student with GlaxoWellcome in 1998, before moving from the UK to the United States to work in academic funded clinical research in HIV. Prior to joining bluebird bio in 2021, Gerry worked in gene therapy at Orchard Therapeutics and Amgen, most recently supporting the regulatory approval and reimbursement of gene therapy products including Libmeldy (@ Orchard) and Imlygic (@ Amgen).
In his current role at at bluebird bio, Gerry oversees biostatistics activities for real world evidence and reimbursement dossiers for bbb gene therapy programs. |
Gene Therapy, Real-World Data and Real-World Evidence |
|
|
Dr. Shihua Wen is currently a director of biostatistics in Novartis Pharmaceuticals Corp. (US). He joined Novartis in 2016 and started to work in gene therapy area since last year. Dr. Wen has rich experience in late phase clinical development across multiple therapeutic areas. At Novartis, he serves as the statistical lead for global clinical development programs in neurology area and successfully supported multiple regulatory submissions for health authorities’ approval. Prior to Novartis, he worked in Abbott Laboratory / AbbVie Inc. as a biostatistician as well with increasing responsibility. Dr. Wen received his doctoral degree in statistics at University of Maryland, Collage Park, in 2007. His research interests are drug development, benefit-risk assessment, innovative trial design, data fusion, etc. |
Estimands in gene therapy trials |
|
|
Chenxuan is a 2nd-year Master student, studying Biostatistics and Bioinformatics at Duke University School of Medicine. Her research interests include: clinical trial methodology, real-world data/real-world evidence, composite index, statistical genetics.
|
Statistical Considerations for Gene Therapy in Rare Diseases Clinical Trials |
Date: Wednesday 23rd March 2022
Time: 14:00-16:30 GMT
Speakers: Gerry Downey (Bluebird Bio), Shihua Wen (Novartis), and Chenxuan Zang (Duke University).
Who is this event intended for? Statisticans working in - or interested in - gene therapy treatments.
What is the benefit of attending? Attendees will have the opportunity to hear topics and case studies covered that relate to: Gene therapy, Estimands, Real world data and Trial design.
You can now register for this event. Registration fees are as follows:
- Members of PSI = Free of charge
- Non-Members of PSI = £20+VAT
To register for the session, please click here.
Research and clinical trials for gene therapies pose many specific challenges. These can be due to comparing potentially curative treatments to a standard of care with different modes of action or even no currently available treatments. There may also be small sample sizes due to rare or ultra-rare diseases. The talks in this webinar will cover challenges related to study design, including estimands, and use of real-world data and evidence to supplement clinical trial data. Case studies of such challenges will be presented.
|
Speaker |
Biography |
Abstract |
|
|
Gerry has over 20 years experience in biostatistics and began his statistical career as an industrial placement student with GlaxoWellcome in 1998, before moving from the UK to the United States to work in academic funded clinical research in HIV. Prior to joining bluebird bio in 2021, Gerry worked in gene therapy at Orchard Therapeutics and Amgen, most recently supporting the regulatory approval and reimbursement of gene therapy products including Libmeldy (@ Orchard) and Imlygic (@ Amgen).
In his current role at at bluebird bio, Gerry oversees biostatistics activities for real world evidence and reimbursement dossiers for bbb gene therapy programs. |
Gene Therapy, Real-World Data and Real-World Evidence |
|
|
Dr. Shihua Wen is currently a director of biostatistics in Novartis Pharmaceuticals Corp. (US). He joined Novartis in 2016 and started to work in gene therapy area since last year. Dr. Wen has rich experience in late phase clinical development across multiple therapeutic areas. At Novartis, he serves as the statistical lead for global clinical development programs in neurology area and successfully supported multiple regulatory submissions for health authorities’ approval. Prior to Novartis, he worked in Abbott Laboratory / AbbVie Inc. as a biostatistician as well with increasing responsibility. Dr. Wen received his doctoral degree in statistics at University of Maryland, Collage Park, in 2007. His research interests are drug development, benefit-risk assessment, innovative trial design, data fusion, etc. |
Estimands in gene therapy trials |
|
|
Chenxuan is a 2nd-year Master student, studying Biostatistics and Bioinformatics at Duke University School of Medicine. Her research interests include: clinical trial methodology, real-world data/real-world evidence, composite index, statistical genetics.
|
Statistical Considerations for Gene Therapy in Rare Diseases Clinical Trials |
Date: Wednesday 23rd March 2022
Time: 14:00-16:30 GMT
Speakers: Gerry Downey (Bluebird Bio), Shihua Wen (Novartis), and Chenxuan Zang (Duke University).
Who is this event intended for? Statisticans working in - or interested in - gene therapy treatments.
What is the benefit of attending? Attendees will have the opportunity to hear topics and case studies covered that relate to: Gene therapy, Estimands, Real world data and Trial design.
You can now register for this event. Registration fees are as follows:
- Members of PSI = Free of charge
- Non-Members of PSI = £20+VAT
To register for the session, please click here.
Research and clinical trials for gene therapies pose many specific challenges. These can be due to comparing potentially curative treatments to a standard of care with different modes of action or even no currently available treatments. There may also be small sample sizes due to rare or ultra-rare diseases. The talks in this webinar will cover challenges related to study design, including estimands, and use of real-world data and evidence to supplement clinical trial data. Case studies of such challenges will be presented.
|
Speaker |
Biography |
Abstract |
|
|
Gerry has over 20 years experience in biostatistics and began his statistical career as an industrial placement student with GlaxoWellcome in 1998, before moving from the UK to the United States to work in academic funded clinical research in HIV. Prior to joining bluebird bio in 2021, Gerry worked in gene therapy at Orchard Therapeutics and Amgen, most recently supporting the regulatory approval and reimbursement of gene therapy products including Libmeldy (@ Orchard) and Imlygic (@ Amgen).
In his current role at at bluebird bio, Gerry oversees biostatistics activities for real world evidence and reimbursement dossiers for bbb gene therapy programs. |
Gene Therapy, Real-World Data and Real-World Evidence |
|
|
Dr. Shihua Wen is currently a director of biostatistics in Novartis Pharmaceuticals Corp. (US). He joined Novartis in 2016 and started to work in gene therapy area since last year. Dr. Wen has rich experience in late phase clinical development across multiple therapeutic areas. At Novartis, he serves as the statistical lead for global clinical development programs in neurology area and successfully supported multiple regulatory submissions for health authorities’ approval. Prior to Novartis, he worked in Abbott Laboratory / AbbVie Inc. as a biostatistician as well with increasing responsibility. Dr. Wen received his doctoral degree in statistics at University of Maryland, Collage Park, in 2007. His research interests are drug development, benefit-risk assessment, innovative trial design, data fusion, etc. |
Estimands in gene therapy trials |
|
|
Chenxuan is a 2nd-year Master student, studying Biostatistics and Bioinformatics at Duke University School of Medicine. Her research interests include: clinical trial methodology, real-world data/real-world evidence, composite index, statistical genetics.
|
Statistical Considerations for Gene Therapy in Rare Diseases Clinical Trials |
Date: Wednesday 23rd March 2022
Time: 14:00-16:30 GMT
Speakers: Gerry Downey (Bluebird Bio), Shihua Wen (Novartis), and Chenxuan Zang (Duke University).
Who is this event intended for? Statisticans working in - or interested in - gene therapy treatments.
What is the benefit of attending? Attendees will have the opportunity to hear topics and case studies covered that relate to: Gene therapy, Estimands, Real world data and Trial design.
You can now register for this event. Registration fees are as follows:
- Members of PSI = Free of charge
- Non-Members of PSI = £20+VAT
To register for the session, please click here.
Research and clinical trials for gene therapies pose many specific challenges. These can be due to comparing potentially curative treatments to a standard of care with different modes of action or even no currently available treatments. There may also be small sample sizes due to rare or ultra-rare diseases. The talks in this webinar will cover challenges related to study design, including estimands, and use of real-world data and evidence to supplement clinical trial data. Case studies of such challenges will be presented.
|
Speaker |
Biography |
Abstract |
|
|
Gerry has over 20 years experience in biostatistics and began his statistical career as an industrial placement student with GlaxoWellcome in 1998, before moving from the UK to the United States to work in academic funded clinical research in HIV. Prior to joining bluebird bio in 2021, Gerry worked in gene therapy at Orchard Therapeutics and Amgen, most recently supporting the regulatory approval and reimbursement of gene therapy products including Libmeldy (@ Orchard) and Imlygic (@ Amgen).
In his current role at at bluebird bio, Gerry oversees biostatistics activities for real world evidence and reimbursement dossiers for bbb gene therapy programs. |
Gene Therapy, Real-World Data and Real-World Evidence |
|
|
Dr. Shihua Wen is currently a director of biostatistics in Novartis Pharmaceuticals Corp. (US). He joined Novartis in 2016 and started to work in gene therapy area since last year. Dr. Wen has rich experience in late phase clinical development across multiple therapeutic areas. At Novartis, he serves as the statistical lead for global clinical development programs in neurology area and successfully supported multiple regulatory submissions for health authorities’ approval. Prior to Novartis, he worked in Abbott Laboratory / AbbVie Inc. as a biostatistician as well with increasing responsibility. Dr. Wen received his doctoral degree in statistics at University of Maryland, Collage Park, in 2007. His research interests are drug development, benefit-risk assessment, innovative trial design, data fusion, etc. |
Estimands in gene therapy trials |
|
|
Chenxuan is a 2nd-year Master student, studying Biostatistics and Bioinformatics at Duke University School of Medicine. Her research interests include: clinical trial methodology, real-world data/real-world evidence, composite index, statistical genetics.
|
Statistical Considerations for Gene Therapy in Rare Diseases Clinical Trials |
Our monthly webinar explores examples of innovative data visualisations relevant to our day to day work. Each month a new dataset is provided from a clinical trial or other relevant example, and participants are invited to submit a graphic that communicates interesting and relevant characteristics of the data.
Develop your non-technical skills by reading The Art of Explanation by Ros Atkins and joining the Sept-Dec 2025 book club. You will be invited to join facilitated discussions of the concepts and ideas and apply skills from the book in-between sessions.
Our monthly webinar will allow attendees to gain practical knowledge and skills in Open-Source coding and tools, with a focus on applications in the pharmaceutical industry. The sessions will provide starting points in a number of areas, correct any common misconceptions and provide valuable resources for further learning.
Date: Tuesday 9th September 2025 Join us to hear Sofia Weigle and Weng-Kee Wong present their recent work.
This course is aimed at biostatisticians with no or some pediatric drug development experience who are interested to further their understanding. We will give you an introduction to the pediatric drug development landscape. This will include identifying the key regulations and processes governing pediatric development, a discussion on the needs and challenges when conducting pediatric research and a focus on the ways to overcome these challenges from a statistical perspective.
This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.
AI agents are large language models equipped with tools that can autonomously tackle challenging tasks. This talk will explore how generative AI agents can enable biomedical discovery.
The webinar is targeted at statisticians working in the pharmaceutical industry, and the objective is to 1) provide a basic understanding of IV methodology including how it relates to causal inference, and 2) present two inspirational pharma-relevant applications.
The Pre-Clinical Special Interest Group (SIG) Workshop 2025 will take place over two half-days on 7 - 8 October in Verona, Italy, bringing together experts from industry, academia, and regulatory institutions to discuss key challenges and innovations in pre-clinical research.
Four sessions will include ML foundation (including an introduction, data exploration for ML and dimensionality reduction and feature selection), Supervised learning (including support vector machines and model evaluation and interpretation), model optimization and unsupervised learning (including clustering) and advanced topics (including neural networks, deep learning and large language models).
The program will feature insightful sessions led by distinguished invited speakers, alongside a poster session showcasing the latest advancements in the field. Further details will be provided.
Date: Wednesday 5th November 2025 A careers talk about medical statistics and how it plays a crucial role in developing new medicines
Date: 19 November 2025 This event is aimed at students with an interest in the field of Medical Statistics, for example within pharmaceuticals, healthcare and/or medical research.
This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.
As an Associate Director Biostatistics Early Development, you will be a key member of our biostatistics group, you will play a crucial role in the design, analysis, and interpretation of clinical trials for early development programs.
If you are passionate about biostatistics and real-world data, and are looking for an exciting opportunity to contribute to groundbreaking research, we encourage you to apply.
Are you passionate about making a difference in the world of healthcare? Novartis is seeking a dynamic and experienced professional to join our team in London at The Westworks.
As the Director of HTA Biostatistics & Medical Affairs, you will play a pivotal role in shaping the future of healthcare by providing strategic biostatistical leadership and expertise.
An exciting opportunity has arisen for a permanent Senior Medical Statistician & Statistical Programmer to join the UKCRC fully registered Derby Clinical Trials Support Unit (Derby CTSU).
As a Senior Principal Biostatistician, you will be responsible and accountable for all statistical work, both scientific and operational, for one or more assigned clinical trials
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