Time: 14:00 - 15:30 UK Time Presenters: Kim Cocks (Adelphi Values), Andrew Thompson (EMA) and Christoph Gerlinger (Bayer)
Clinical Trials are increasingly featuring a wide variety of Patient Reported Outcomes (PROs). PROs are important endpoints to both regulatory and health technology bodies in their assessments and approvals. There are standard ways in which PRO instruments are developed and validated, as well as important concepts for designing and interpretation of PRO data in clinical trials such as assessing the Minimally Clinically Important Differences (MCIDs/MIDs) and the use of a Responder definition.
The intent of this webinar is to describe an overview of how PROs tools are developed and used in clinical trial settings and how results can be clinically and meaningfully interpreted. In addition, a regulatory viewpoint will be shared on the key considerations for PROs. The talks will also include examples and case-studies showing the derivation of a validated thresholds for treatment responder and interpretation of PROs in the context of regulatory approval.
Kim Cocks is a Director and Principal Statistician at Adelphi Values, a global healthcare consultancy. She has worked as a medical statistician and clinical trial methodologist for over 20 years across pharmaceutical, academic and consultancy environments. She specialises in PRO interpretation and analysis and is an active member of both the EORTC Quality of Life group and ISOQOL.
Abstract Overview of PROs and clinically meaningful interpretation
PROs are validated instruments designed to provide a direct report from the patient on aspects of their health including symptoms and functioning. This talk will provide a brief overview of how PROs are developed, how they provide a score for multi-dimensional aspects of a patient’s health and why these can be difficult to interpret. The variety of quantitative and qualitative methods available to aid interpretation will be introduced.
Andrew Thomson (EMA)
Andrew Thomson is a statistician at the EMA, in the Human Medicines Research & Development Support Division. Prior to joining the EMA in 2014, he spent 7 years at the MHRA, initially as a Statistical Assessor in the Licensing Division, and subsequently Head of Epidemiology in the Vigilance & Risk Management of Medicines Division.
Abstract Regulatory Considerations for PROs In this talk I will present some regulatory considerations on the use of PROs in regulatory assessment, and how they can be developed. These will be discussed alongside a recent case example that has been through the qualification procedure at EMA.
Christoph Gerlinger (Bayer)
Christoph Gerlinger is Bayer’s Expert Statistician for Health Technology Assessment and Women’s Health. He is the regulatory chair of the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and a work package leader for the IMI BigData@Heart project. Christoph worked as statistician over 25 years in the pharmaceutical industry and in his spare time he teaches at the University Medical School of Saarland.
Abstract Empirical derivation of the minimal important difference for PROs In this talk I will present some methods on how to derive the minimal important difference for PROs from a clinical trial. I will present a worked example where we used this empirical minimal important difference to formulate a responder definition that was then accepted by the FDA.
Time: 14:00 - 15:30 UK Time Presenters: Kim Cocks (Adelphi Values), Andrew Thompson (EMA) and Christoph Gerlinger (Bayer)
Clinical Trials are increasingly featuring a wide variety of Patient Reported Outcomes (PROs). PROs are important endpoints to both regulatory and health technology bodies in their assessments and approvals. There are standard ways in which PRO instruments are developed and validated, as well as important concepts for designing and interpretation of PRO data in clinical trials such as assessing the Minimally Clinically Important Differences (MCIDs/MIDs) and the use of a Responder definition.
The intent of this webinar is to describe an overview of how PROs tools are developed and used in clinical trial settings and how results can be clinically and meaningfully interpreted. In addition, a regulatory viewpoint will be shared on the key considerations for PROs. The talks will also include examples and case-studies showing the derivation of a validated thresholds for treatment responder and interpretation of PROs in the context of regulatory approval.
Kim Cocks is a Director and Principal Statistician at Adelphi Values, a global healthcare consultancy. She has worked as a medical statistician and clinical trial methodologist for over 20 years across pharmaceutical, academic and consultancy environments. She specialises in PRO interpretation and analysis and is an active member of both the EORTC Quality of Life group and ISOQOL.
Abstract Overview of PROs and clinically meaningful interpretation
PROs are validated instruments designed to provide a direct report from the patient on aspects of their health including symptoms and functioning. This talk will provide a brief overview of how PROs are developed, how they provide a score for multi-dimensional aspects of a patient’s health and why these can be difficult to interpret. The variety of quantitative and qualitative methods available to aid interpretation will be introduced.
Andrew Thomson (EMA)
Andrew Thomson is a statistician at the EMA, in the Human Medicines Research & Development Support Division. Prior to joining the EMA in 2014, he spent 7 years at the MHRA, initially as a Statistical Assessor in the Licensing Division, and subsequently Head of Epidemiology in the Vigilance & Risk Management of Medicines Division.
Abstract Regulatory Considerations for PROs In this talk I will present some regulatory considerations on the use of PROs in regulatory assessment, and how they can be developed. These will be discussed alongside a recent case example that has been through the qualification procedure at EMA.
Christoph Gerlinger (Bayer)
Christoph Gerlinger is Bayer’s Expert Statistician for Health Technology Assessment and Women’s Health. He is the regulatory chair of the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and a work package leader for the IMI BigData@Heart project. Christoph worked as statistician over 25 years in the pharmaceutical industry and in his spare time he teaches at the University Medical School of Saarland.
Abstract Empirical derivation of the minimal important difference for PROs In this talk I will present some methods on how to derive the minimal important difference for PROs from a clinical trial. I will present a worked example where we used this empirical minimal important difference to formulate a responder definition that was then accepted by the FDA.
Time: 14:00 - 15:30 UK Time Presenters: Kim Cocks (Adelphi Values), Andrew Thompson (EMA) and Christoph Gerlinger (Bayer)
Clinical Trials are increasingly featuring a wide variety of Patient Reported Outcomes (PROs). PROs are important endpoints to both regulatory and health technology bodies in their assessments and approvals. There are standard ways in which PRO instruments are developed and validated, as well as important concepts for designing and interpretation of PRO data in clinical trials such as assessing the Minimally Clinically Important Differences (MCIDs/MIDs) and the use of a Responder definition.
The intent of this webinar is to describe an overview of how PROs tools are developed and used in clinical trial settings and how results can be clinically and meaningfully interpreted. In addition, a regulatory viewpoint will be shared on the key considerations for PROs. The talks will also include examples and case-studies showing the derivation of a validated thresholds for treatment responder and interpretation of PROs in the context of regulatory approval.
Kim Cocks is a Director and Principal Statistician at Adelphi Values, a global healthcare consultancy. She has worked as a medical statistician and clinical trial methodologist for over 20 years across pharmaceutical, academic and consultancy environments. She specialises in PRO interpretation and analysis and is an active member of both the EORTC Quality of Life group and ISOQOL.
Abstract Overview of PROs and clinically meaningful interpretation
PROs are validated instruments designed to provide a direct report from the patient on aspects of their health including symptoms and functioning. This talk will provide a brief overview of how PROs are developed, how they provide a score for multi-dimensional aspects of a patient’s health and why these can be difficult to interpret. The variety of quantitative and qualitative methods available to aid interpretation will be introduced.
Andrew Thomson (EMA)
Andrew Thomson is a statistician at the EMA, in the Human Medicines Research & Development Support Division. Prior to joining the EMA in 2014, he spent 7 years at the MHRA, initially as a Statistical Assessor in the Licensing Division, and subsequently Head of Epidemiology in the Vigilance & Risk Management of Medicines Division.
Abstract Regulatory Considerations for PROs In this talk I will present some regulatory considerations on the use of PROs in regulatory assessment, and how they can be developed. These will be discussed alongside a recent case example that has been through the qualification procedure at EMA.
Christoph Gerlinger (Bayer)
Christoph Gerlinger is Bayer’s Expert Statistician for Health Technology Assessment and Women’s Health. He is the regulatory chair of the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and a work package leader for the IMI BigData@Heart project. Christoph worked as statistician over 25 years in the pharmaceutical industry and in his spare time he teaches at the University Medical School of Saarland.
Abstract Empirical derivation of the minimal important difference for PROs In this talk I will present some methods on how to derive the minimal important difference for PROs from a clinical trial. I will present a worked example where we used this empirical minimal important difference to formulate a responder definition that was then accepted by the FDA.
Time: 14:00 - 15:30 UK Time Presenters: Kim Cocks (Adelphi Values), Andrew Thompson (EMA) and Christoph Gerlinger (Bayer)
Clinical Trials are increasingly featuring a wide variety of Patient Reported Outcomes (PROs). PROs are important endpoints to both regulatory and health technology bodies in their assessments and approvals. There are standard ways in which PRO instruments are developed and validated, as well as important concepts for designing and interpretation of PRO data in clinical trials such as assessing the Minimally Clinically Important Differences (MCIDs/MIDs) and the use of a Responder definition.
The intent of this webinar is to describe an overview of how PROs tools are developed and used in clinical trial settings and how results can be clinically and meaningfully interpreted. In addition, a regulatory viewpoint will be shared on the key considerations for PROs. The talks will also include examples and case-studies showing the derivation of a validated thresholds for treatment responder and interpretation of PROs in the context of regulatory approval.
Kim Cocks is a Director and Principal Statistician at Adelphi Values, a global healthcare consultancy. She has worked as a medical statistician and clinical trial methodologist for over 20 years across pharmaceutical, academic and consultancy environments. She specialises in PRO interpretation and analysis and is an active member of both the EORTC Quality of Life group and ISOQOL.
Abstract Overview of PROs and clinically meaningful interpretation
PROs are validated instruments designed to provide a direct report from the patient on aspects of their health including symptoms and functioning. This talk will provide a brief overview of how PROs are developed, how they provide a score for multi-dimensional aspects of a patient’s health and why these can be difficult to interpret. The variety of quantitative and qualitative methods available to aid interpretation will be introduced.
Andrew Thomson (EMA)
Andrew Thomson is a statistician at the EMA, in the Human Medicines Research & Development Support Division. Prior to joining the EMA in 2014, he spent 7 years at the MHRA, initially as a Statistical Assessor in the Licensing Division, and subsequently Head of Epidemiology in the Vigilance & Risk Management of Medicines Division.
Abstract Regulatory Considerations for PROs In this talk I will present some regulatory considerations on the use of PROs in regulatory assessment, and how they can be developed. These will be discussed alongside a recent case example that has been through the qualification procedure at EMA.
Christoph Gerlinger (Bayer)
Christoph Gerlinger is Bayer’s Expert Statistician for Health Technology Assessment and Women’s Health. He is the regulatory chair of the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and a work package leader for the IMI BigData@Heart project. Christoph worked as statistician over 25 years in the pharmaceutical industry and in his spare time he teaches at the University Medical School of Saarland.
Abstract Empirical derivation of the minimal important difference for PROs In this talk I will present some methods on how to derive the minimal important difference for PROs from a clinical trial. I will present a worked example where we used this empirical minimal important difference to formulate a responder definition that was then accepted by the FDA.
Time: 14:00 - 15:30 UK Time Presenters: Kim Cocks (Adelphi Values), Andrew Thompson (EMA) and Christoph Gerlinger (Bayer)
Clinical Trials are increasingly featuring a wide variety of Patient Reported Outcomes (PROs). PROs are important endpoints to both regulatory and health technology bodies in their assessments and approvals. There are standard ways in which PRO instruments are developed and validated, as well as important concepts for designing and interpretation of PRO data in clinical trials such as assessing the Minimally Clinically Important Differences (MCIDs/MIDs) and the use of a Responder definition.
The intent of this webinar is to describe an overview of how PROs tools are developed and used in clinical trial settings and how results can be clinically and meaningfully interpreted. In addition, a regulatory viewpoint will be shared on the key considerations for PROs. The talks will also include examples and case-studies showing the derivation of a validated thresholds for treatment responder and interpretation of PROs in the context of regulatory approval.
Kim Cocks is a Director and Principal Statistician at Adelphi Values, a global healthcare consultancy. She has worked as a medical statistician and clinical trial methodologist for over 20 years across pharmaceutical, academic and consultancy environments. She specialises in PRO interpretation and analysis and is an active member of both the EORTC Quality of Life group and ISOQOL.
Abstract Overview of PROs and clinically meaningful interpretation
PROs are validated instruments designed to provide a direct report from the patient on aspects of their health including symptoms and functioning. This talk will provide a brief overview of how PROs are developed, how they provide a score for multi-dimensional aspects of a patient’s health and why these can be difficult to interpret. The variety of quantitative and qualitative methods available to aid interpretation will be introduced.
Andrew Thomson (EMA)
Andrew Thomson is a statistician at the EMA, in the Human Medicines Research & Development Support Division. Prior to joining the EMA in 2014, he spent 7 years at the MHRA, initially as a Statistical Assessor in the Licensing Division, and subsequently Head of Epidemiology in the Vigilance & Risk Management of Medicines Division.
Abstract Regulatory Considerations for PROs In this talk I will present some regulatory considerations on the use of PROs in regulatory assessment, and how they can be developed. These will be discussed alongside a recent case example that has been through the qualification procedure at EMA.
Christoph Gerlinger (Bayer)
Christoph Gerlinger is Bayer’s Expert Statistician for Health Technology Assessment and Women’s Health. He is the regulatory chair of the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and a work package leader for the IMI BigData@Heart project. Christoph worked as statistician over 25 years in the pharmaceutical industry and in his spare time he teaches at the University Medical School of Saarland.
Abstract Empirical derivation of the minimal important difference for PROs In this talk I will present some methods on how to derive the minimal important difference for PROs from a clinical trial. I will present a worked example where we used this empirical minimal important difference to formulate a responder definition that was then accepted by the FDA.
Time: 14:00 - 15:30 UK Time Presenters: Kim Cocks (Adelphi Values), Andrew Thompson (EMA) and Christoph Gerlinger (Bayer)
Clinical Trials are increasingly featuring a wide variety of Patient Reported Outcomes (PROs). PROs are important endpoints to both regulatory and health technology bodies in their assessments and approvals. There are standard ways in which PRO instruments are developed and validated, as well as important concepts for designing and interpretation of PRO data in clinical trials such as assessing the Minimally Clinically Important Differences (MCIDs/MIDs) and the use of a Responder definition.
The intent of this webinar is to describe an overview of how PROs tools are developed and used in clinical trial settings and how results can be clinically and meaningfully interpreted. In addition, a regulatory viewpoint will be shared on the key considerations for PROs. The talks will also include examples and case-studies showing the derivation of a validated thresholds for treatment responder and interpretation of PROs in the context of regulatory approval.
Kim Cocks is a Director and Principal Statistician at Adelphi Values, a global healthcare consultancy. She has worked as a medical statistician and clinical trial methodologist for over 20 years across pharmaceutical, academic and consultancy environments. She specialises in PRO interpretation and analysis and is an active member of both the EORTC Quality of Life group and ISOQOL.
Abstract Overview of PROs and clinically meaningful interpretation
PROs are validated instruments designed to provide a direct report from the patient on aspects of their health including symptoms and functioning. This talk will provide a brief overview of how PROs are developed, how they provide a score for multi-dimensional aspects of a patient’s health and why these can be difficult to interpret. The variety of quantitative and qualitative methods available to aid interpretation will be introduced.
Andrew Thomson (EMA)
Andrew Thomson is a statistician at the EMA, in the Human Medicines Research & Development Support Division. Prior to joining the EMA in 2014, he spent 7 years at the MHRA, initially as a Statistical Assessor in the Licensing Division, and subsequently Head of Epidemiology in the Vigilance & Risk Management of Medicines Division.
Abstract Regulatory Considerations for PROs In this talk I will present some regulatory considerations on the use of PROs in regulatory assessment, and how they can be developed. These will be discussed alongside a recent case example that has been through the qualification procedure at EMA.
Christoph Gerlinger (Bayer)
Christoph Gerlinger is Bayer’s Expert Statistician for Health Technology Assessment and Women’s Health. He is the regulatory chair of the European Federation of Statisticians in the Pharmaceutical Industry (EFSPI) and a work package leader for the IMI BigData@Heart project. Christoph worked as statistician over 25 years in the pharmaceutical industry and in his spare time he teaches at the University Medical School of Saarland.
Abstract Empirical derivation of the minimal important difference for PROs In this talk I will present some methods on how to derive the minimal important difference for PROs from a clinical trial. I will present a worked example where we used this empirical minimal important difference to formulate a responder definition that was then accepted by the FDA.
Joint PSI/EFSPI Visualisation SIG 'Wonderful Wednesday' Webinars
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.
The event will open with an overview on drug development in women’s health from a clinician perspective. This talk is followed by talks about statistical challenges when planning IVF studies and analysing the menstrual cycles.
This webinar will provide an overview of surrogacy for licensing and reimbursement. In turn, the need of extensions of the SPIRIT and CONSORT statement will be defined and outlined, with case studies to support.
Joint PSI/EFSPI Pre-Clinical SIG Webinar: Virtual Control Groups in Toxicity Studies
Lea Vaas will present how replacement of concurrent control animals by Virtual Control Groups (VCGs) in systemic toxicity studies may help in contributing to the 3R's principle of animal experimentation: Reduce, Refine, Replace.
Joint PSI/EFSPI Data Science SIG Webinar: Developing Digital Measures (Digital Biomarkers) in Drug Development – insights from Mobilise D consortium
We will share a brief overview of what Mobilise D is and why it is an important step stone in the development of digital biomarkers, and how Mobilise D outputs can be relevant for you.
This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.
PSI Webinar: Development of Gene Therapies: Strategic, Scientific, Regulatory and Access Considerations
This webinar will cover the history of cell/gene therapy, major regulatory advances, the role of quantitative scientists in drug development of these novel therapeutics, and discuss opportunities for innovation and product advancement.
This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.
PSI Introduction to Industry Training (ITIT) Course - 2024/2025
An introductory course giving an overview of the pharmaceutical industry and the drug development process as a whole, aimed at those with 1-3 years' experience. It comprises of six 2-day sessions covering a range of topics including Research and Development, Toxicology, Data Management and the Role of a CRO, Clinical Trials, Reimbursement, and Marketing.
This networking event is aimed at statisticians that are new to the pharmaceutical industry who wish to meet colleagues from different companies and backgrounds.
Statisticians in the Pharmaceutical Industry Executive Office: c/o MCI UK Ltd | Unit 24/22 South | Building 4000 | Langstone Park| Langstone Road | Havant | PO9 1SA | UK