Regulatory

PSI Chair: Anna Berglind        EFSPI Chair: Christoph Gerlinger

The committee members are: 
Anna Berglind, AstraZeneca, Sweden
Olivier Collignon, GSK, UK
Erika Daly, ICON, Ireland
Anne Danniau, UCB, Belgium
Daniel Evans, Pfizer, UK
Chrissie Fletcher, Amgen, UK
Christoph Gerlinger, Bayer, Germany
Kerry Gordon, IQVIA, UK
Jürgen Hummel, PPD, UK
Teppo Huttunen, 4Pharma, Finland
Melanie Jones, Covance, UK
Mette Krog Josiassen, Lundbeck, Denmark
Murray Lowe, Phastar, UK
Frances Lynn, Orchard Therapeutics, UK
Roland Marion-Gallois, Cellgene, Switzerland
Sireesha Pamulapati, Biogen, UK
Carol Reid, Roche, UK 
Véronique Robust, Servier, France
Kaspar Rufibach, Roche, Switzerland
Antony Sabin, AstraZeneca, UK
Florian Voss, Boehringer-Ingelheim, Germany
Heike Wöhling, Sandoz Pharmaceupt, Germany

Objectives

The role of the regulatory committee is to provide the PSI/EFSPI viewpoint on regulatory issues. This includes promoting best practice, reviewing regulatory policy and guidelines, driving debate on future guidance, and engaging with statisticians in European regulatory agencies.

 

2019

Meeting with Regulators 

On Wednesday 13 November 2019 the PSI/EFSPI Regulatory Committee met with the MHRA statisticians in London for an informal exchange of statistical topics important for both regulators and industry. There was great discussion on topics such as estimands, subgroup analyses and predefined Quality Tolerance Limits as well as quality attributes, model-based dose escalation studies, real world evidence, use of historical controls and the lack of statisticians in many ethics’ committees in England.

The planned meeting with EMA’s biostatistics working party was postponed to 2020 due to EMA’s business continuation plan for Brexit.

 

The 4th Regulatory Workshop 

The 4th Regulatory Statistics Workshop took place in Basel, Switzerland on the 23rd and 24th September 2019. Approximately 230 delegates attended the workshop with great discussion between regulators, academics, and industry on real-world data, analysis of safety in clinical trials, estimands, modern approaches for rare disease and contributed short topics.

 

Webinars 

On July 4th, 2019, the PSI/EFSPI Regulatory Committee organized the Webinar: ‘Adaptive design: updated draft FDA guidance and its implications’. In the webinar Jürgen Hummel (PPD) shared an overview of the FDA’s updated guidance on adaptive design for clinical trials of drugs and biologics, and discussed implications for industry. This was followed by the introduction of an open-source statistical software for adaptive designs, RPACT (an R package available on CRAN that enables the design and analysis of confirmatory adaptive clinical trials) led by Kaspar Rufibach (Roche) introduced. Kit Roes (Working Group Methodology of the Dutch Medicines Evaluation Board and member of the EMA Biostatistics Working Party) closed the webinar by commenting on the guidance from a European Regulatory perspective.

 

 

Review of Guidelines
The regulatory committee collated comments on the following documents during the year:

Europe

  • Draft questions and answers on Data Monitoring Committees issues
  • Qualification opinion of clinically interpretable treatment effect measures based on recurrent event endpoints that allow for efficient statistical analyses 

USA:

  • Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products.  Comments are due by 23rd December 2019. 
  • Patient-Focused Drug Development: Methods to Identify What Is Important to Patients. Comments are due by 30th December 2019. No comments
  • Draft guidance on Preparedness of Medicines' Clinical Trials in Paediatrics.
  • Adjusting for Covariates in Randomized Clinical Trials for Drugs and Biologics with Continuous Outcomes 

China:

  • The Center for Drug Evaluation in the NMPA (National Medical Product Administration) in China have released two draft guidance on Guideline on Clinical Trial Data Monitoring Committees and Guideline on Non-inferiority Clinical Trials. 

Upcoming Events

PSI New Starters Half-Day Networking Event

An opportunity to meet statisticians from across the pharmaceutical industry in a relaxed and informal setting. An exciting program of events and a chance to work in small groups on a data analysis challenge. Lunch provided.

A Non-PSI Event - Protecting confidentiality and privacy in clinical trial and medical data sets

We are increasingly living in a data driven world. Data are collected in many different ways for a variety of purposes. As such, concerns around protecting the privacy of individuals have increased in recent times.

A PSI Training Course - Practical Approaches to Designing Adaptive Clinical Trials

This hands-on course will provide a deep dive into 4 software packages used to design adaptive clinical trials. The course will start by providing a general overview of adaptive designs, explaining the different type of adaptations possible and the benefits of each design. Following this, participants will be given the opportunity to have a go at designing trials in R (using RPACTS), EAST, FACTS, and nQuery.

PSI Training Course - Bayesian Practical Course using R and SAS

This practical training course will give a deep dive into performing Bayesian analyses in R and SAS. It is aimed at statisticians who need to be able to conduct Bayesian analyses as part of their day to day work. By the end of the course participants will be able to conduct their own analyses.

Medical Statistics Careers Event 2020

Our annual PSI Medical Statistics Careers Event, will be held at the De Montfort University in Leicester on Wednesday 4th March 2020.

PSI Webinar - Statistical Operations Research

This webinar will address operational issues of paramount importance within the healthcare industry with a view to using statistics for the benefit of patients. In attending this webinar, you'll hear more about work being conducted to address some operational issues we face in the health care industrys e.g. patient rectuitment, drug supply and meeting NHS 18 week targets.

PSI Toxicology SIG Workshop

PSI Toxicology SIG workshop – 16th and 17th March 2020 The Toxicology SIG provides a forum for statisticians working in regulatory/investigative toxicology, as well as most other pre-clinical areas, to discuss issues and interact with one another. This 1.5-day workshop will involve approximately 20 statisticians, focusing on discussions around “best practice” in the statistical analysis of various data types. The afternoon of Day 1 will include a 4.5 hour Bayesian training course focused towards applications in toxicology/pre-clinical, provided by Prof. Dr. Katja Ickstadt and is included in the workshop fee. The cost will be £270 including VAT per delegate, inclusive of food and one night’s accommodation (and the training course). The workshop is being held at the Crowne Plaza Hotel, Heathrow. The agenda and topics that will be discussed are yet to be finalised, but please get in touch with gareth.thomas@covance.com if you have suggestions. Full details will be circulated in the coming weeks.

A PSI Training Course - R for SAS Users

This course is aimed at Statisticians and Programmers experienced in SAS, but little or no experience with R. An Introduction to R studio and the R language, statistical graphics, programming statistical models, simulations and more…

Non-proportional hazards and applications in immuno-oncology

Designs of clinical trials with time to event primary endpoints usually rely on hazards being constant over time. A major challenge in immuno-oncology is the delayed onset of benefit with such therapies and the presence of non-proportional hazards. The impact of this needs to be accounted for in sample size calculations, analysis methodology and reporting. At this meeting, we will examine possible strategies to handle such features, which may not be fully known when the trial is initiated.

Introduction To Industry Training Course 2020

The ITIT course will take 25 delegates new to the industry on a complete drug development experience from discovery to marketing. They will visit 6 companies from October 2020 to July 2021 to learn about 6 topics from experts in their field. The ITIT course will have 6 sessions in continental Europe and 3 - 4 sessions in the UK. It promises to be a truly memorable course.

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