What is this event about?
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Rare diseases have been largely neglected until very recently. Lack of research and lack of interest from the pharmaceutical industry has meant that very few of the 6000+ rare diseases had any treatment available.
The Regulation (EC) 141/2000 of the European Parliament of 16 December 1999 was introduced to try to change this situation. This legislation introduced a new set of regulations which, if properly understood and followed, can provide significant incentives to companies to develop treatments for rare diseases.
The development of such treatments has many unique challenges and this Masterclass aims to help delegates understand what differentiates orphan drugs from other drugs and to approach the design of development programs for rare diseases.
Our Masterclass will be delivered through a combination of diversely skilled speakers who will give presentations, case studies and encourage interaction and discussion. This is designed to help inspire delegates with an understanding of and a synergistic approach for successful orphan drug development.
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Where is the event?
This Masterclass will take place in central London, UK
Who is leading this event?
Tony Hall, Chief Medical Officer,PSR Group, The Netherlands
Tony Hall graduated in London with first class honours in physiology & pharmacology from King's College then as a doctor at the Royal Free Hospital. He specialised in Emergency Medicine and joined the pharmaceutical industry in 1994. At Boehringer Ingelheim he was responsible for the strategic development of many clinical programmes and for two global products. He worked at Yamanouchi before starting his own business.
Now as Chief Medical Officer of PSR, Tony has an in-depth knowledge of the orphan drug market, applicable regulations and procedures. He provides advice and guidance on development plans for orphan drug designation, protocol assistance and clinical programmes. He has built trusted relationships with patient groups to help ensure their voice is heard during PSR’s clinical trial involvement.
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In association with
PSR is a leading expert in Orphan drug development. We provide the essential ingredients necessary to help our clients through the logistical and regulatory hurdles of clinical study programs for these products.
PSR has established links with physician networks, key opinion leaders, government bodies and patient groups; we can provide regulatory expertise and access to scientific (protocol) advice at the EMEA level, all of which helps to ensure the best design of the clinical program.
PSR has the experience necessary to set up and run complex clinical trials in rare diseases, where access to patients and overcoming the logistical challenges of sparsely distributed patients are the keys to success. Our approach is tailored to enable us to perform efficient and successful orphan drug studies, which often require a small number of sites per country spread over several countries together with novel approaches to ensure protocol compliance.
By attending this event you will be better able to:
Understand the background of orphan drugs and the related legislation.
Learn how to write an orphan drug application for Europe and the US.
Appreciate the key aspects of designing clinical trials for orphan drugs.
Evaluate the importance of patient registries for rare diseases.
Gain insight into the perspective of the rare disease patient.
Observe the effect of reimbursement issues on patient access to orphan drugs.
KEY TOPICS INCLUDE:
Masterclass agenda
Welcome & Introduction to Orphan Drugs • Background
• Regulations
• Definitions
• Incentives
• Relevant regulatory bodies Guide to writing an orphan drug application (EU&US) • Procedure
• Sections of application
• Sub-setting Designing clinical trials for orphan indications • Choice of endpoint
• Ethical issues
• Protocol assistance
• Predictors of success or failure
• Surrogates
• Guidance documents
• Case studies Registries for rare diseases Rare diseases from the patient’s perspective • Problems with diagnosis
• Isolation
• Lack of medical expertise
• Roles & achievements of EURORDIS
• NORD & individual patient groups
Market access and reimbursement for orphan drugs • Value of orphan drugs
• Variations in patient access between countries Conclusions and end of masterclass
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