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. 

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