Invited Sessions
The President’s invited speaker for the occasion of convening the Society’s 30th annual conference will be Marc Buyse (International Institute for Drug Development, Louvain-la-Neuve and University of Hasselt, Belgium). He will address the audience with his talk entitled: "On trial endpoints that combine clinical relevance and statistical efficiency: ‘Analyze the data, the whole data, and nothing but the data’ ".
ABSTRACT: Recent developments in biostatistics and bioinformatics are reshaping the landscape of clinical research. With the number of promising new molecules available for clinical testing, trials need to detect a drug’s benefit and harm as fast as possible. There are three major ways in which the clinical development process can potentially be accelerated and/or improved. First, by making use of biomarkers and surrogate endpoints, if any are available. Second, by designing trials with provisions for interim analyses and adaptations whenever appropriate. Third, by focusing on endpoints that combine clinical relevance and statistical sensitivity. Most endpoints currently recommended for regulatory approval make use of a surprisingly small fraction of the available data, for instance, whether a subject is in a given state of disease or experiences certain symptoms at a fixed time point, without regard to repeated measurements of the disease state or symptoms over time. In situations where the patient may experience a number of untoward events, many trials analyse the time to the first event and ignore all subsequent events. Throwing away some of the available data may result in a loss of statistical power, which could be viewed as a minor price to pay in order to define an endpoint that is “clinically relevant”. The problem becomes more serious, however, when assumptions are made for patients in whom endpoints thus defined are unobserved (or unobservable), for these assumptions may lead to seriously biased inferences, whether data are imputed using sophisticated techniques or silly ones (such as the infamous “last observation carried forward”). This presentation will show ways to avoid all of these problems and to use “the data, the whole data, and nothing but the data”, using actual examples of clinical trials in ophthalmology and oncology.
Invited sessions (with organisers):
Indirect Comparisons of Health Care Interventions (Opening plenary session)
Organisers:
Andrew Briggs (University of Glasgow, Scotland, U.K.)
Rolf Holle (Institute of Health Economics, Oberschleißheim, Germany)
Speakers:
Neil Hawkins (University of York & Oxford Outcomes Ltd, U.K.): "You can run, but you cannot hide: decision-making and indirect comparisons in a resource limited healthcare system"
Tony Ades (University of Bristol, U.K.): Pairwise meta-analysis and indirect comparisons: equally biased, or equally meaningless?/
Discussant:
Theo Stijnen (Leiden University, the Netherlands)
Joint Modelling of Longitudinal and Survival Data
Organisers:
Robin Henderson (Newcastle University, U.K.)
Hélène Jacqmin-Gadda (INSERM, Université Victor Segale, France)
Speakers:
Jane-Ling Wang (UC Davis, CA, U.S.A.): Challenges in modelling longitudinal and event-time data
Daniel Farewell (Cardiff University, U.K.): Generalized estimating equations for censored data
Cécile Proust-Lima (University of Bordeaux, France): Joint modeling of cognitive decline and time-to-dementia: a latent variable approach
Regulatory Affairs
Organisers:
Robert T O’Neill (Food and Drug Administration, VA, U.S.A.)
Speakers:
Frank Rockhold (Glaxo SmithKline, U.S.A.): Some perspectives on the impact of the International Conference on Harmonization's E9 'Statistical Principles for Clinical Trials' - 10 years of experience
Joachim Rohmel (Consultant, U.S.A.): Recent methodological advances contributing to clinical trials and regulatory statistics
Robert T O’Neill (Food and Drug Administration, VA, U.S.A.): A perspective on the challenges and opportunities for statistics in the evolution of product development and regulation
Incomplete Data
Organisers:
Geert Molenberghs (Universiteit Hasselt and Katholieke Universiteit Leuven, Belgium)
Speakers:
James Carpenter (London School of Hygiene & Tropical Medicine, U.K.): Statistical methods for clinical studies with missing data: what's hot, what's cool and what's useful?
Craig Mallinckrodt (Eli Lilly & Company, Indianapolis, Indiana, U.S.A.): Conceptual considerations regarding endpoints, hypotheses, and analyses for incomplete longitudinal clinical trial data
Robert T O’Neill (Food and Drug Administration, VA): title TBA
Statistical Methods in HIV/AIDS Research
Organisers:
Benoit Masse (Fred Hutchinson Cancer Research Centre, Seattle, WA, U.S.A.)
Michal Kulich (Charles University in Prague, Czech Republic)
Speakers:
Nicholas Jewell (UC Berkeley, CA, U.S.A.): Alternatives to intention to treat: direct effects of HIV prevention methods in the MIRA study
Victor deGruttola (Harvard University, Boston, MA, U.S.A.): Statistical issues in models investigating the possibility of control of the HIV epidemic
Alex Welte (presenter) and Tom McWalter (University of the Witwatersrand, Johannesburg, South Africa): Inferring rates from instantaneous state variables: Application to HIV incidence estimation