A WORKSHOP SPONSORED BY Numerus Limited

20th September 2011
Restaurant "Museum"

Wilhelmstraße 3, 72074 Tübingen, Germany
Register

In association with
BioRegio STERN

Programme

The programme for BioS 2011 has been hand-picked to include a broad selection of presentations that are of relevance to personnel who conduct research and development in the biotech industry. Although limited to one day, the event will attempt to cover some of the statistical principles, concepts and methods that are used during pre-clinical and clinical development, as well as discussing the statistical challenges associated with regulatory submissions and reimbursement.

Is it for me?
Bios 2011 is designed to meet the needs of non-statisticians who are faced with the daily challenge of understanding statistical concepts within drug research and development. The following people will benefit from attending:

  • Biologists
  • Clinical scientists / directors
  • Clinical operations managers
  • Clinical monitors
  • Clinical study / programme managers
  • Data managers
  • Marketing directors
  • Medical writers
  • Pre-clinical scientists / directors
  • Technical directors

The workshop is set in the beautiful city of Tübingen, a quaint, picturesque city set on the Neckar river and renowned for its medical university. Visit www.tuebingen.de.

An outline of the day's activities is given below. The final programme will be available by mid July.

Time Event/Actvity
08.30 Registration open
Restaurant "Museum", Wilhelmstraße 3, 72074 Tübingen
First floor
09.00 - 09.30 Welcome and Opening Remarks
(tea, coffee, soft drinks and small snacks)
09.30 - 10.15 Dancing with Alice - Statistics and Drug Development
Dr. Richardus Vonk, Vize-Präsident der Deutschen Region der Internationalen Biometrischen Gesellschaft (IBS-DR)

Statistics is an integral part of the drug development process. Conclusions from experiments and (clinical) studies and the subsequent business decisions are often based on statistical reasoning. In this lecture, a high-level overview of the role of statistics in the drug discovery and development process is offered. Moreover, the presentation will elucidate how innovative statistical methodology facilitates early phases of the drug discovery and drug development process.

10.15 - 11.00 Dr. Marisa Bacchi, Head of Biostatistics, Actelion Pharma AG
11.00 - 11.30 Break
(tea , coffee, soft drinks)
11.30 - 12.15 Data Mining Techniques with Applications in the Pharmaceutical Industry
Dr. Denise Welsch, Director, Rey Analytical Research, Fachhochschule Koblenz

Data Mining is the process of extracting information from large datasets and making forecasts in the future. Typical techniques used in data mining are from the fields of Statistics, Machine Learning and Database Management. Data Mining, as opposed to classical statistical analysis, can uncover unknown and potential useful hypothesis in large unstructured data volumes by combining the human input with the support of highly automated algorithms.
Chemical, biological and clinical data can be merged to improve the quality of drug discovery, delivery methods and time-to-market. Various applications of data mining techniques in the pharmaceutical industry can be classified into preclinical applications which are mostly chemoinformatics or bioinformatics based or clinical applications as data mining for predicting individual drug response or data mining in pharmacovigilance. A brief overview of data mining applications in the pharmaceutical industry will be given.
Several technological aspects and algorithms of data mining will be presented in this talk. Moreover, the most important software tools used in data mining will be briefly presented.

12.30 - 13.30 Lunch
Restaurant "Museum", Wilhelmstraße 3, 72074 Tübingen
Ground floor
13.30 - 14.15 Statistical significance and clinical relevance - Regulators' expectations
Dr. Avril Mankel, Senior Manager Regulatory Affairs, NDA Regulatory Service GmbH

The presentation will focus on the interpretation of results from statistical analyses in clinical trials from a regulatory perspective. Special critical topics will be addressed, e.g. general choice of study design, safety monitoring, orphan drugs and particular considerations for specific indications. These topics frequently trigger discussions in Scientific Advice procedures and in the course of the review of the Marketing Authorization Application in EU. As a consequence the acceptance of the benefit/risk ratio by the Authorities could be at risk, which is the ultimate basis for Marketing Approval.

14.15 - 15.00 Statistical design and sample size
Dr. Sylvia Tamara Lenz, Director, Statistical Consulting

A good study requires good planning. From the statistical perspective this means choosing the statistical design or model appropriate for the medical questions that the study is investigating. Based on this design the statistician then has to choose which statistical analysis or tests are best for the data in question. Once the statistical test has been determined it is possible to consider the sample size that will be required to satisfy a multitude of criteria such as regulatory requirements, ethical considerations, study cost and most important of all to guaranteeing the scientific validity of the study.

15.00 - 15.30 Break
(tea, coffee, soft drinks and small snacks)
15.30 - 16.15 The new German law and evidence based medicine: A statistical challenge
Dr. Volker Hösel, Director, StatSciConsult

In December 2010 a new regulation, concerning reimbursement of newly marketed drugs, has been launched. It requires from the pharmaceutical companies dossiers addressing comparative effectiveness and clinical relevance of their drugs. We discuss statistical challenges for these two requirements.

  1. Comparative Effectiveness:
    In general, clinical studies compare a new treatment to an established therapy or placebo, but not to a competitor drug. Therefore, comparative effectiveness needs to be assessed with indirect methods. We discuss potentials and limitations of such methods which also involve Bayesian networks.
  2. Clinical Relevance:
    Here, the statistical significance of efficacy, shown in randomized clinical trials, is not enough. The new drug has to demonstrate, that there is a patient relevant benefit which significantly surpasses a given threshold of irrelevance. Agreement on clinical relevance for different medical scenarios seems feasible, but the irrelevance threshold is much disputed. IQWiG published a hierarchical guideline to determine this threshold.
16.15 - 16.45 Final word