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Orphan Drug Designation in the USA and Europe
Course Summary
Medicines for the prevention, diagnosis, or treatment of rare diseases have become known as ‘orphan drugs’ because of their commercial unattractiveness. Development of such products is successfully encouraged through incentives offered by regulatory authorities. To qualify for important incentives, the sponsor of a drug must gain ‘orphan designation’ for its use in an indication. This module describes the requirements for orphan designation and how to apply for it in the USA and the European Economic Area.
Purchasing Information
When you have completed your course order, Zenosis will finalise the setup of your course materials and contact you on the email address that you provide in your order. You can expect this process to be completed within one business day (using the UK business calendar) of completing your payment.
It is therefore essential that you use your real email address for your order, or indicate in the purchase notes the email address to be used for the course set-up, and check that any messages from Zenosis or grapl are not lost in your junk or spam folder.
You will have access to the course module(s) for a period of 180 days after your purchase is complete.
Detailed Course Information
Learning Objectives
• Explain why and how governments encourage the development of medicines for rare human diseases, and identify important sources of information.
• Specify incentives offered for the development of medicines for rare diseases in the USA and in Europe.
• State the criteria for orphan drug designation in the USA and in Europe.
• List the contents of an application for orphan designation in the USA and in Europe, describe how to make an application in each case, and outline the process of review by the regulatory authority.
• Outline the sponsor’s obligations and options after orphan designation in the USA and in Europe.
Who will benefit from this course
This module is intended primarily for regulatory affairs professionals. Staff inexperienced in regulatory affairs and compliance will find the module an invaluable introductory training course; more-experienced personnel will find it a useful reference tool. More generally, it will be of interest to all those involved in the development and registration of medicinal products.
Module Outline
• Module overview
– An outline of the module’s scope and objectives, and notes on terminology.
• Rare diseases and orphan drugs
– Development of medicines for prevention, diagnosis, or treatment of rare diseases is commercially unattractive, so governments offer incentives to encourage it. In this session, we introduce the concept of orphan drug designation, discuss how it fits within a product development strategy, and identify some important sources of information on rare diseases and orphan drugs.
• US designation
– Legislation to encourage research and development of drugs for rare diseases was introduced first in the USA. In this session we describe the US legal framework for orphan drug designation and specify the incentives offered. We set out the criteria for orphan designation and how they should be satisfied. We list the contents of an application for designation and outline how to apply. Finally we identify the sponsor’s obligations and options after designation.
• European designation
– In this session we describe the European Union’s legal framework for orphan medicine designation and specify the incentives offered. We set out the criteria for orphan designation and how they should be satisfied. We specify the contents of an application for designation, describe how to apply, discuss the procedures for validation and evaluation of the application, and outline the provisions for appeal against refusal of designation. Finally we identify the sponsor’s obligations and options after designation.
• Assessment
– Multiple-choice mastery assessment.
Roles
Regulatory
CPD Points
1.5
Region
Level
Foundation, Introductory
Course Study Time
Approx 1.5 hours
USA, Europe