The Statisticians in the Pharmaceutical Industry (PSI) held their annual conference in Amsterdam in June 2024. Four ICON biostatisticians attended and in this article we share their insights. They also showcased their expertise in an individual presentation and during a panel session.
During the three-day conference there were talks on a multitude of relevant topics of interest. The conference provided an ideal opportunity to expand our knowledge of current methodological considerations and trends within the clinical trials industry. ICON participants attended sessions on:
- digital biomarkers
- advanced methods in vaccine trials
- patient focused drug development
- Bayesian borrowing
- adaptive designs
- estimands for non-inferiority trials
- use of R programming
- decision making
- European Union (EU) Health Technology Assessment (HTA) in 2025
What the COVID-19 pandemic taught statisticians
The opening keynote set the tone for the conference by putting statistical theories into a real world context. Geert Molenberghs, Director of I-Biostat, Professor at Universiteit Hasselt KU Leuven explored the lessons learned from the COVID-19 pandemic. Under the title “Biostatistics in a post-pandemic era”, he looked at ways of presenting and interpreting COVID-19 incident rates. He illustrated his talk with examples from Belgium and covered the implications for research, public health, drug and vaccine development, policy advice and communication.
Index-based ranking, R and Machine Learning
Samdhan Ghubade, Senior Biostatistician II, ICON, presented a paper in the decision making stream. He spoke about the detriment index-based ranking technique for painkillers for noncommunicable diseases (NCDs).
The ICON attendees found the use of R programming stream particularly useful. Guiyan Lei’s paper shared how Roche handled the use of R for primary analysis and submission. Both Lei’s paper and one from Janina Linnik of Novartis touched on using open source code.
Another emerging trend is the use of machine learning (ML) for data driven biomarker identification. Bayer’s Karl Kochert, Nils Ternes of Sanofi and Mairzia Scelsi of Roche spoke about ML use for digital endpoint validation and analysis methods.
Digital health technology and challenging conventional thinking
Dr Mia Tackney, MRC Biostatistics Unit at Cambridge University, joined Rosemary Abbott, Associate Director, Biostatistics, ICON and Susanne Schaefer, Senior Director Biostatics, ICON, for their one and a half hour session. Karen Smith, Director, Biostatistics, ICON, chaired the session on “Advancements in digital health technology (DHT): Opportunities, challenges and solutions”. Their presentations covered decentralised clinical trials, digital endpoints, and statistical challenges.
The presentations took an industry-wide perspective, outlining considerations and challenges for researchers contemplating adopting DHTs in clinical trials. As well as the broader picture, the session covered more particular challenges such as handling missing data. It was emphasized that missing data was most likely not to be missing at random. Approximately 200 people attended the presentation and the link between academia and industry was considered noteworthy. The audience had many questions which contributed to an interesting discussion. Some commented that the session had challenged their usual thinking, especially in relation to missing data.
Another talk that challenged conventional thinking was the day two keynote speech from Theo Smith, founder of Neurodiversity World and Neuromine. His thought-provoking speech “Neurodiversity at work: Unleashing potential in every mind”, initiated a lively panel discussion.
As well as learning through the formal sessions, we enjoyed the informal learning opportunities that arise during networking sessions and breaks. We left the conference with important insights on the latest trends. We were happy to share ICON’s experience of using DHT in clinical trials with a wider audience of our industry colleagues. We look forward to seeing them again at next year’s conference and seeing how the trends develop in the meantime.
In this section
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Digital Disruption
- AI and clinical trials
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Clinical trial data anonymisation and data sharing
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Clinical Trial Tokenisation
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Closing the evidence gap: The value of digital health technologies in supporting drug reimbursement decisions
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Digital disruption in biopharma
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Disruptive Innovation
- Remote Patient Monitoring
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Personalising Digital Health
- Real World Data
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The triad of trust: Navigating real-world healthcare data integration
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Patient Centricity
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Agile Clinical Monitoring
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Capturing the voice of the patient in clinical trials
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Charting the Managed Access Program Landscape
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Developing Nurse-Centric Medical Communications
- Diversity and inclusion in clinical trials
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Exploring the patient perspective from different angles
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Patient safety and pharmacovigilance
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A guide to safety data migrations
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Taking safety reporting to the next level with automation
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Outsourced Pharmacovigilance Affiliate Solution
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The evolution of the Pharmacovigilance System Master File: Benefits, challenges, and opportunities
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Sponsor and CRO pharmacovigilance and safety alliances
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Understanding the Periodic Benefit-Risk Evaluation Report
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A guide to safety data migrations
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Patient voice survey
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Patient Voice Survey - Decentralised and Hybrid Trials
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Reimagining Patient-Centricity with the Internet of Medical Things (IoMT)
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Using longitudinal qualitative research to capture the patient voice
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Agile Clinical Monitoring
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Regulatory Intelligence
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An innovative approach to rare disease clinical development
- EU Clinical Trials Regulation
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Using innovative tools and lean writing processes to accelerate regulatory document writing
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Current overview of data sharing within clinical trial transparency
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Global Agency Meetings: A collaborative approach to drug development
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Keeping the end in mind: key considerations for creating plain language summaries
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Navigating orphan drug development from early phase to marketing authorisation
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Procedural and regulatory know-how for China biotechs in the EU
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RACE for Children Act
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Early engagement and regulatory considerations for biotech
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Regulatory Intelligence Newsletter
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Requirements & strategy considerations within clinical trial transparency
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Spotlight on regulatory reforms in China
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Transfer of marketing authorisation
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An innovative approach to rare disease clinical development
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Therapeutics insights
- Endocrine and Metabolic Disorders
- Cardiovascular
- Cell and Gene Therapies
- Central Nervous System
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Glycomics
- Infectious Diseases
- NASH
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Oncology
- Paediatrics
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Respiratory
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Rare and orphan diseases
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Advanced therapies for rare diseases
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Cross-border enrollment of rare disease patients
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Crossing the finish line: Why effective participation support strategy is critical to trial efficiency and success in rare diseases
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Diversity, equity and inclusion in rare disease clinical trials
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Identify and mitigate risks to rare disease clinical programmes
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Leveraging historical data for use in rare disease trials
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Natural history studies to improve drug development in rare diseases
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Patient Centricity in Orphan Drug Development
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The key to remarkable rare disease registries
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Advanced therapies for rare diseases
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Transforming Trials
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Accelerating biotech innovation from discovery to commercialisation
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Ensuring the validity of clinical outcomes assessment (COA) data: The value of rater training
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Linguistic validation of Clinical Outcomes Assessments
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Optimising biotech funding
- Adaptive clinical trials
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Best practices to increase engagement with medical and scientific poster content
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Decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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Decentralised and Hybrid clinical trials
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Practical considerations in transitioning to hybrid or decentralised clinical trials
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Navigating the regulatory labyrinth of technology in decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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eCOA implementation
- Flexible delivery models
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Implications of COVID-19 on statistical design and analyses of clinical studies
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Improving pharma R&D efficiency
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Increasing Complexity and Declining ROI in Drug Development
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Innovation in Clinical Trial Methodologies
- Partnership insights
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Risk Based Quality Management
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Transforming the R&D Model to Sustain Growth
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Accelerating biotech innovation from discovery to commercialisation
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Value Based Healthcare
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Strategies for commercialising oncology treatments for young adults
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US payers and PROs
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Accelerated early clinical manufacturing
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Cardiovascular Medical Devices
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CMS Part D Price Negotiations: Is your drug on the list?
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COVID-19 navigating global market access
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Ensuring scientific rigor in external control arms
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Evidence Synthesis: A solution to sparse evidence, heterogeneous studies, and disconnected networks
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Global Outcomes Benchmarking
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Health technology assessment
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Perspectives from US payers
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ICER’s impact on payer decision making
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Making Sense of the Biosimilars Market
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Medical communications in early phase product development
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Navigating the Challenges and Opportunities of Value Based Healthcare
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Payer Reliance on ICER and Perceptions on Value Based Pricing
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Payers Perspectives on Digital Therapeutics
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Precision Medicine
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RWE Generation Cross Sectional Studies and Medical Chart Review
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Survey results: How to engage healthcare decision-makers
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The affordability hurdle for gene therapies
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The Role of ICER as an HTA Organisation
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Strategies for commercialising oncology treatments for young adults
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