Pre-conference tutorials

Pre-conference tutorial topics:

Tutorial 1: Orphan Drug Development

Overview: How can a company or a developer navigate the process of developing an orphan drug in the most time-effective and successful way to end up with a real treatment that will deliver real outcomes for the patients and which is recognised as such by the healthcare systems?  Developing an orphan medicinal product can be even more complex and time-consuming than developing a treatment for a more common condition.  This is mainly due to the specificities of rare diseases, and the heterogeneity of the condition, but also to scarce medical expertise, limited models, small and geographically spread patient populations and the complexity of appropriate trial designs.  In addition, an orphan drug also has to follow the overall EU pharmaceutical legislative framework, which in itself can be a challenge.  This Tutorial will give experience from colleagues with more than 45 years of combined experience, to review how to make the best pathway through these elements to reach the optimal outcome – a treatment available for patients.

Tutorial Instructors: Henk SCHURING, Group Vice President Regulatory Affairs, Genzyme and An VAN ES JOHANSSON, Head of Clinical Development, Sobi

Tutorial 2: Orphan Drugs in the EU: From Designation to Marketing Authorisation

Overview: This tutorial will focus specifically on the criteria for orphan drug designation, current experience on designations and orphan drugs marketing authorisation. In addition, the tutorial will include in depth review and analyses of topics such as orphan market exclusivity, similarity, clinical superiority and significant benefit. Speakers will include the Head of Orphan Medicines at the European Medicines Agency, and you will also hear from an industry representative about the strategic aspects, experiences and learning’s with regard to regulatory framework for orphan drugs in the EU.

Tutorial Instructors: Jordi Llinares, EMA, UK and Catarina Edfjäll, Shire Human Genetic Therapies, Switzerland

  

Tutorial 3: Health Technology Assessment 101 for Rare Diseases

Overview: Health technology assessment is a multidisciplinary process that summarises information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased and robust manner. Its purpose is to guide policymakers to make decisions on the adoption of new and existing technologies. This course will introduce the participant to the principles of health technology assessment, describe the methods of economic evaluation, illustrate the unique circumstances surrounding rare disease evidence, provide an overview of the different approaches for assessing rare disease drugs across countries and discuss other factors which may contribute to funding decisions. At the conclusion of the course, participants will have a better understanding of HTA in order to contribute and engage more effectively in future assessments of medicines for rare diseases.

Tutorial Instructors: Francis Pang, Shire Human Genetic Therapies, Switzerland; Josie Godfrey, AGNSS, UK; Keith Tolley, TolleyHealthEconomics and SMC, UK; Ana Palma, Shire Human Genetic Therapies, Belgium

 

Tutorial 4: Registries

Overview: Getting to the Premier League of Data Sets, The most important objective is to understand the primary purpose of clinical data  - restricting registries to outcome-predictive drivers that might range from the hard outcome of survival or death to the softer outcomes of surrogate markers of disease severity: all need to be placed into a rank order (league table). For each, a points system needs to be established which then determines the cost benefit ratio for each driver to be included in the data set. We will explain how to do this and why it should be done:  as published in Early Human Development 2010: p723-728 : all will be exemplified in this tutorial.

Tutorial Instructors: Anil Mehta, University of Dundee and Christophe Bayrou