Anticipating Changes in Drug Development for Children: Building on Paediatric Rheumatology

Using the field of rare paediatric rheumatic disorders as a paradigm, the first conference "Anticipating Changes in Drug Development for Children Building on Paediatric Rheumatology" will examine the medicines research and development process, from pre-clinical and early-phase clinical development, to biomarker validation, study design and management, data analysis, ethical review, regulatory submission and research governance. The conference is meant to further stimulate interaction among the three major stakeholders in the drug development process, namely, academia, industry and regulatory authorities.

One benchmarking deliverable of the conference will be a publishable consensus report examining in detail the critical issues driving the development of medicines for children with rheumatic diseases, highlighting as well the emerging means, tools and methodologies to facilitate and perform trials in children with rare disorders.

Researchers, clinicians, clinical pharmacologists, pre-clinical and methodological researchers, biostatisticians, and representatives of governmental/non-governmental drug authorities and pharma industries are invited to submit abstracts for selected oral presentations or poster display on the following topics:

·    Networking: pre-clinical and/or clinical results achieved through networking in rare, orphan diseases;

·    Methods: biometric and in-silico models for rare, orphan diseases;

·    Biomarkers in rheumatic diseases: definition and validation processes.

As many as 60 grants covering registration, hotel and travel expenses will be awarded on a competitive basis to the authors of selected abstracts.

PROGRAM HIGHLIGHTS

The Regulatory Framework

-    Regulations on medicinal products for paediatric use: the EU and US legislation compared

-    The Paediatric Investigation Plan in the EU: the scientific and regulatory viewpoints

-    Directives on GCP and clinical trials: specific issues concerning ethics of clinical research in children

-    Rare diseases, orphan legislation and how to develop an orphan drug: EU and US perspectives

-    Pre-clinical strategies to obtain regulatory approval for a first in children study

-    The need for pharmaceutical forms of medicinal products adapted to children

Clinical Trials: Methodology and Networking

-    Paediatric clinical trials:

-         Generalities

-         Managing safety of paediatric clinical trials

-         Maturational changes in organs critical for the disposition and efficacy of drugs

-    Clinical trials in small populations:

-         Level of evidence

-         Choice of endpoints and control groups

-         Methodological and statistical considerations

-    The European register of clinical trials on medicines for children (DEC-net)

-    Networks in Paediatric Rheumatology

Translational Medicine and Therapeutics for Paediatric Rheumatology

-    Biomarkers and surrogate endpoints:

-         The academic, regulatory, and statistical perspectives

-         Consideration of biomarkers in juvenile idiopathic arthritis

-         Manipulating B-cells in rheumatic diseases

-         Translational medicine in autoinflammatory diseases

-         Proteomics in rheumatology

-    Anticipating change in drug development: the emerging era of translational medicine and therapeutics - the perspectives of industry, academia and regulatory agencies

-    Funding schemes for translational medicine

Salvatore Albani (San Diego, US)

Arthur J. Atkinson (Chicago, US)

Maurizio Bonati (Milan, IT)

Daniel Brasseur (Brussels, BE)

Irun Cohen (Rehovot, IL)

Ornella Della Casa Alberghi (Genoa, IT)

Fergal Donnely (Bruxelles, BE)

Garret A. FitzGerald (Philadelphia, US)

Marco Gattorno (Genoa, IT)

Edward Giannini (Cincinnati, US)

Raphael Hirsch (Pittsburgh, US)

Wietse Kuis (Utrecht, NL)

Pekka Kurki (Helsinki, FI)

Lawrence J. Lesko (Rockville, US)

Dan Lovell (Cincinnati, US)

Barry Mangum (Durham, US)

Alberto Martini (Genoa, Italy)

Nello Martini (Rome, IT)

Dianne Murphy (Rockville, US)

Tan Nguyen (Rockville, US)

Tony Nunn (Liverpool, UK)

Chiara Pandolfini (Milan, IT)

Harsuk Parmar (Loughborough, UK)

Gerard Pons (Paris, FR)

Berent Prakken (Utrecht, NL)

Anders Rane (Stockholm, SE)

Giuseppe Remuzzi (Bergamo, IT)

Klaus Rose (Basel, CH)

Nicolino Ruperto (Genoa, IT)

Agnes Saint Raymond (London, UK)

Jeffrey Siegel (Rockville US)

Beatriz Silva-Lima (Lisbon, PT)

Elisabetta Traggiai (Genoa, IT)

Stefano Vella (Rome, IT)

Kerstin Westermark (Uppsala, SE)

Patricia Woo (London, UK)

Fred Zepp (Mainz, DE)

Participation: limited to 100 registrants.

Target audience: Researchers, clinicians, clinical pharmacologists, pre-clinical and methodological researchers, biostatisticians, and representatives of governmental/non-governmental drug authorities and pharma industries

Registration Fee: € 500.00

Registration includes:

• Conference tuition;
• Conference materials and proceedings (Course Handbook and CD);
• Meals, refreshments, social events:
  - 6 Coffee Breaks
  - 2 Working Lunches
  - 1 Cocktail Reception/Buffet Dinner
  - 2 Conference Dinners