Q&A and discussion of morning session
Postbaseline subpopulation analyses: Known to be improper, but frequently done. Can we fix them?
Generalizability and external validity: How to generate evidence about a treatment effect?
Role of statistics / quantitative science in regulatory decision-making
Guidances / restructuring / new ways to interact with stakeholders
What happened in the last two years in industry?
Selection of “HTA hot topics” that need discussion in coming months
Relevant scientific questions for drug development
Relevant scientific questions for HTA
Neos pediatric trial of Kesimpta and Mayzent in multiple sclerosis
Regulatory view on complex innovative designs
HTA’s view on complex innovative designs
Clinical trials areabout comparability, not generalizability
Regulatory view on generalizability
Let them in or build a wall? Transporting inferences across borders
Generating Actionable Insights Using RWD during COVID-19 pandemic
What role should statistics play in regulatory decision-making?
Role of biostatisticians in regulatory decision making: the current discussion in Japan
Experience with the project SignifiCanT
Day 1
Decentralised trials: discussion topics across EMA activities
FDA Guidance on Decentralized Clinical Trials
alpha-T: a pre-pandemic decentralized trial in oncology
DCT, the new kid on the block. Here to stay?
Day 2
Two estimands: double trouble or just the same old same old?
Is there something like “too much innovation”?
MHRA experience with CIDs and recommendations derived from it
Label-enabling dynamic borrowing for OS in DLBCL – FDA CID pilot example
Designing the EPAD (European Prevention of Alzheimer’s Dementia) Platform trial: Key issues
Day 3
Opportunities and Challenges of RWE to Support Regulatory Decision-Making
The use of external controls: To what extent can it currently be recommended?
Evolving strategies in generating medication safety data in pregnancy – an industry perspective
Day 1
The new EMA Q&A document on data monitoring committees
Use of futility interim boundaries to inform production or portfolio decisions
Role of iDMC for complex clinical trials, incorporating portfolio decisions
Navigating between sponsor, iDMC, and regulators
iDMC in the context of adaptive designs
Day 2
Regulatory update: learnings, planned guideline updates, and recommendations & asks for industry
Feedback from EFSPI / EFPIA estimand implementation working group
Impact of COVID-19 and risk mitigation in a global cardiovascular outcomes trial
Real World Data, Real World Evidence, Big Data
BBS spring seminar on synthetic controls: summary & follow-up
Development of a digital endpoint in Multiple Sclerosis – challenges and opportunities
A look on Best Practices in Pragmatic Trials
One single arm study plus a historic comparator equals two historic regulatory approvals
Using Real-World Data to Extrapolate Evidence from Randomized Controlled Trials
Use of RWD in gene – therapy approvals
Use of historical data to support gene-therapy approval: example from Kymriah
How far can we trust “Real World Data”?
Comparison of statistical methods to analyse safety data
Pooling and harmonizing of safety data for a robust statistical analysis
A shiny app to explore clinical data
How statisticians deal with the difference between efficacy and safety reporting
Estimand framework: opportunity to rethink some old (and new) problems in Oncology trials?
Regulatory experience with the Estimand framework
Observational vs. randomized analysesof digoxin-mortality in the DIG trial
Goal Attainment Scaling: Validation & use for rare disease
A regulator’s view on rare cancer drug development: Histology independent indications
To blind or not to blind? FDA guidance on placebos and blinding in oncology clinical trials
Underpowered RCTs and external data
EMA (2018) Q&A on Mahalanobisdistance (MD) to assess drug dissolution profiles
ICH E9(R1): Status of addendum and comments
Efficacy independent of relapse HA interactions before ICH E9 R1
Need for Treatment strategy as an estimand attribute
Trimmed mean to handle missing/meaningless outcomes – a recommendation from FDA
Estimands for Recurrent Event data
Efficiency comparisons of recurrent event and time-to-first event analysis
Group Sequential designs for recurrent events
Regulator’s view on estimands for time-to-event data
Master protocols: MHRA experience
European regulators’ view on platform trials
Master Protocols – FDA Oncology Experience
When a Threshold Crossing approach may and may not be appropriate
Complementing evidence from a small scale RCT by registry data in a rare disease setting
Regulatory considerations when supplementing confirmatory RCTs with non-randomised external data
HTA considerations when supplementing RCT with non-randomized data
Model-based extrapolation between dosing regimens
Testing treatment effect when hazards are non-proportional
Is adherence to placebo control sometimes doing a disservice to both current and future patients?
Use of historical data in Phase II dose-finding and confirmatory phase III trials
Regulatory experience with regard to submissions that include modelling and simulation
FDA’s Draft Guidance on Multiple Endpoints: Overview, Reactions and Next Steps
Regulatory Issues with Multiplicity in Drug Approval and Current Controversies
Translation of the estimand framework into regulatory guidance: what’s next?
How the ICH E9 addendum around estimands may impact our clinical trials
A journey towards estimand specification in pain: motivation and challenges
Construction of an Estimand in a Clinical Trial on Progressive Multiple Sclerosis
Facilitating the use of biomarkers in early development: the role of regulators
How to Gamble if You Must: Early Clinical Statistics in Decision Processes
Case study: Cosentyx in psoriasis – we need both, exploratory and confirmatory
Predictive Biomarkers in Drug Development
Drug-device co-development in the era of precision medicine: approval of Tafinlar
and Mekinist combination therapy and next generation sequencing companion
diagnostic in non-small cell lung cancer
Opportunities and risk related to companion diagnostics: The MET biomarker story
Short intro into biomarker and big data
Basket and platform protocols in full development in Oncology
Estimands in drug approval: the way forward
Estimands: are we estimating what we intend to estimate?
Regulatory considerations on estimands in specific indications
Estimands for time-to-event endpoints in oncology and beyond
Incorporating estimands in the clinical trial protocol
Defining estimands in a pain study – (short case study)
On the road to clinical extrapolation
Supporting a paediatric investigation plan in liver transplantation – a pharmacometric approach
Practical Benefit Risk assessment
What’s going on in benefit-risk and what is our role as a statistician?
Benefit-Risk assessment via case studies: key considerations and lessons learned
The role of statistics in ensuring Quality in pharmaceutical manufacturing
Assessment of analytical biosimilarity: the objective, the challenges and the opportunities
EFSPI Secretariat
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Tel: +44(0)1625 664549
Email: admin@efspi.org