7th EFSPI Workshop on Regulatory Statistics 2022

Recordings

Materials

Opening remarks

Introductory Keynotes: Regulator on “What happened in the last two years in regulatory and HTA landscape”

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

Postbaseline subpopulation analyses: Known to be improper, but frequently done. Can we fix them?

Relevant scientific questions for drug development

Relevant scientific questions for HTA

Regulators and HTA bodies for new designs in Europe – how to deal with different priorities, especially for new design types?

Neos pediatric trial of Kesimpta and Mayzent in multiple sclerosis

Regulatory view on complex innovative designs

HTA’s view on complex innovative designs

Generalizability and external validity: How to generate evidence about a treatment effect?

Clinical trials areabout comparability, not generalizability

Regulatory view on generalizability

Let them in or build a wall? Transporting inferences across borders

Role of statistics / quantitative science in regulatory decision-making

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

Posters

Estimands in Oncology

Is medical affairs the wild west of statistics?

6th EFSPI Workshop on Regulatory Statistics 2021

Materials

Day 1

Opening remarks

Introductory session: What is on European regulator’s mind? Where are we with the workplan 2021? What guidelines are planned to be updated? What is industry constantly missing in submissions?

Decentralised trials: discussion topics across EMA activities

FDA Guidance on Decentralized Clinical Trials

Concept for evaluation of remote endpoint assessment by integration of an orthogonal crossover equivalence substudy within longitudinal parallel trial designs

alpha-T: a pre-pandemic decentralized trial in oncology

DCT, the new kid on the block. Here to stay?

Day 2

Welcome and scene setting

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

Welcome and scene setting

Opportunities and Challenges of RWE to Support Regulatory Decision-Making

Meeting the expectation of patients with rare diseases: examples of high quality medicines and timely approval

Pragmatic trials in the real world: DaRe2THINK – a novel approach to healthcare-embedded clinical trials

Use of RWD to contextualize post-hoc analysis to support regulatory submission: an example from the ORATORIO Trial and the Long-Term MSBase Registry

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

RWD – PMDA’s view on it

5th EFSPI Workshop on Regulatory Statistics 2020

Materials

Day 1

Welcome and scene setting

The new EMA Q&A document on data monitoring committees

An example of a difficult interim decision – what should be the role of the iDMC in ensuring trial integrity?

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

Panel discussion

Q&A

Day 2

Welcome and scene setting

Regulatory update: learnings, planned guideline updates, and recommendations & asks for industry

Feedback from EFSPI / EFPIA estimand implementation working group

Using a mix of strategies in handling intercurrent events and missing values for studies impacted by the COVID-19 pandemic

Impact of COVID-19 and risk mitigation in a global cardiovascular outcomes trial

Q&A

4th EFSPI Workshop on Regulatory Statistics 2019

Materials

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

Development of a smartphone based monitoring tool for people with Multiple Sclerosis Challenges and Opportunities

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

Matching up

How statisticians deal with the difference between efficacy and safety reporting

How the estimand framework becomes standard practice in applications, and where we still need to learn

Treatment policy and hypothetical strategies for intercurrent events in chronic pain and Parkinson’s disease

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

Masking of open label studies

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

3rd EFSPI Workshop on Regulatory Statistics 2018

Materials

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

Uncertainty in Benefit-Risk Assessment

2nd EFSPI Workshop on Regulatory Statistics 2017

Materials

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

Delayed start design in neurodegenerative diseases

1st EFSPI Workshop on Regulatory Statistics 2016

Materials

The Per Protocol principle

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

Quality issues in biosimilars

Short Topics: Quantitative Safety and Epidemiology