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Regulatory Strategies for Orphan Drugs Training Course

This must-attend event will provide an essential overview of the opportunities and challenges presented by orphan drug regulation.

★★★★★ "I believe as a starting point into the world of ODD this was very comprehensive and informative. Ver... more"

30 Jan 2023
+ 2 more dates

Need help?  Enrol or reserve

  • Format: Live online
  • CPD: 6 hours for your records
  • Certificate of completion

Course overview

The market for orphan drug products continues to expand and developing new treatments for rare diseases is becoming an increasingly important growth area for many pharmaceutical companies. This interest in rare and orphan diseases is linked not only to the incentives offered by the different regulatory authorities but also to the recent development of advanced therapies.

The interactive course will cover all the key aspects of orphan drug legislation in both the US and EU and the regulatory processes designed to enable early access to innovative medicines. The programme will help you to assess the strategic considerations of when to apply for orphan designation and to which agency and look at the development of advanced therapies and using orphan drug designation with your ATMP strategy.

A case study session on how to prepare and manage an orphan drug submission will consolidate learning and ensure that you leave the event with practical skills and knowledge that can be put to use in the workplace. The course will allow you not only to learn from our expert trainer but also to share the experiences of other delegates.

Benefits of attending:
  • Gain an overview of the orphan medicinal product legislation in the EU and the US
  • Receive practical advice on how to prepare and manage orphan submissions
  • Assess strategic considerations of when to apply for orphan designation and to what agencies
  • Discuss the use of orphan drug designation with your ATMP strategy

Who should attend?

This event will be particularly relevant to regulatory affairs professionals who are involved in orphan drug submissions. It will also be of interest to those working in rare diseases who would benefit from an overview of orphan drug designation.

Enrol or reserve

The Regulatory Strategies for Orphan Drugs course will cover:

Orphan Medicinal Product Legislation

  • Overview of the legislation in the EU, US and Japan
  • What the Regulations cover and why, what they try to protect
  • Benefits of Orphan Drug Designation
  • Key Considerations
    • Country Submissions
    • Differences of Orphan versus non-orphan
    • Paediatric Conditions

Obtaining Orphan Drug Designation

  • Orphan drug designation in the EU
    • Application
    • Procedure
  • Similarities and differences with the US
    • Application, procedure and incentives
  • Global Issues around Rare Disease
  • Strategic Considerations
  • Case Study

Maintenance of Orphan Drug Designation

  • How to maintain
  • Maintenance during MAA and NDA
  • Assessment and Case Study

Global Environment

Enrol or reserve

26 Jun 2023
20 Oct 2023

Joanne Roza
Qdossier

Joanne Roza works as a senior regulatory affairs consultant at Qdossier. She is involved in regulatory affairs consultancy and has also expertise in regulatory operations for big- and small-sized pharmaceutical companies. Joanne holds a bachelor degree in Health Sciences from Maastricht University and a master degree in Health Economics, Policy and Law from the Erasmus University in Rotterdam. She started her career at Qdossier in 2016 and has been involved in a variety of regulatory operations and affairs projects. Joanne’s areas of expertise are: regulatory affairs (strategy and planning), preparation/creation and dispatch of different types of electronic submission formats (e.g. US FDA, CA, EMA, CH), lean/structured authoring and the creation of baseline (CMC) submissions.

More details

30 Jan 2023
20 Oct 2023

Marloes van der Geer
Qdossier

Marloes van der Geer works as senior regulatory affairs scientist at Qdossier. Marloes holds a master in Life Sciences (Drug Innovation) from the University of Utrecht, the Netherlands. For nine years she held several positions at Hoffmann-la Roche in Switzerland, including the role of regulatory intelligence manager and regulatory policy lead for the EMEA region. Marloes has been actively involved in several industry associations like EFPIA and IFPMA. Also, she contributed to the ongoing IMI PREFER project in Europe, focused on the generation and use of patient preference data. In 2019, Marloes joined Qdossier. Marloes’ areas of expertise are: eCTD, lean authoring, publishing, biopharmaceuticals (incl. biosimilars), clinical development, regulatory convergence & harmonization and facilitated registration pathways (incl. orphan drugs), local RA activities (e.g. product information management).

More details

Book Regulatory Strategies for Orphan Drugs Live online training

NEW higher discounts for multiple bookings - bring your colleagues to make your training budget go further:

  • 30% off the 2nd delegate*
  • 40% off the 3rd delegate*
  • 50% off the 4th delegate*

Please contact us for pricing if you are interested in booking 5 or more delegates

30 Jan 2023

Live online

09:00-17:00 UK (London) (UTC+00)
10:00-18:00 Paris (UTC+01)
04:00-12:00 New York (UTC-05)
Course code 12509

  • GBP 549 649
  • EUR 789 929
  • USD 893 1,049

Until 26 Dec

View basket 

 
Not ready to book yet?

for 7 days, no obligation

26 Jun 2023

Live online

09:00-17:00 UK (London) (UTC+01)
10:00-18:00 Paris (UTC+02)
04:00-12:00 New York (UTC-04)
Course code 12729

  • GBP 549 649
  • EUR 789 929
  • USD 893 1,049

Until 22 May

View basket 

 
Not ready to book yet?

for 7 days, no obligation

20 Oct 2023

Live online

Course code 12337

  • GBP 549 649
  • EUR 789 929
  • USD 893 1,049

Until 15 Sep

View basket 

 
Not ready to book yet?

for 7 days, no obligation

* Early booking discounts may not be combined with other discounts or offers. As such, the discounts for 2nd/3rd/4th delegates are based on the full price.

Reviews of IPI's Regulatory Strategies for Orphan Drugs training course


I believe as a starting point into the world of ODD this was very comprehensive and informative. Very clear and concise.

Sep 26 2022

Khayyam Darr
Senior Regulatory Affairs Executive, Chiesi Ltd

Jun 20 2022

Superb, excellent delivery and very knowledgeable [...] the best training course I attended in a log time!

John McIntyre
Senior Consultant, Parexel Ireland USD

Mar 16 2022

Excellent would attend other similar webinar/trainings

Cheryl Dicks
Sr. Mgr. Regulatory, CANbridge Pharmaceuticals

Sep 26 2022

Very instructive training with a very nice speaker. The speaker knew her subject very well and she was able to share some of her experiences with ODD which made the training more interactive.

Laetitia Misslin
Regulatory Affairs Director, Laboratoires DELBERT

Sep 26 2022

Everything was OK

Sarmite Liberte
Head of Regulatory Affairs, AS Kalceks

Sep 26 2022

Very instructive training with a very nice speaker. Speaker knew her subject very well and she was able to share some of her experiences with ODD which made the training more interactive.

Laetitia Misslin
Regulatory Affairs Director, Laboratoires DELBERT

Mar 10 2021

I liked the webinar very much, because it provided a comprehensive and detailed overview of the ODD process in the EU and other countries. Andrew presented himself as an excellent speaker who answered all questions of the participants in a very detailed and knowledgeable manner.

Burkhard Siefert

Mar 10 2021

Very dynamic and interesting speaker, interactive webinar.

Anna Sarao
Regulatory Affairs Project Manager, Servier

Mar 10 2021

Very dynamic and interesting speaker, interactive webinar.

Anna Sarao
Regulatory Affairs Project Manager, Servier

United Kingdom

  • Blue Reg Pharma Consulting
  • Brightwater Research
  • Chiesi Ltd
  • Fujifilm Kyowa Kirin Biologics
  • Galderma (UK) Ltd
  • JensonR+ Ltd
  • Kyowa Kirin
  • NDA Regulatory Science Ltd
  • Orphan Reach Ltd
  • Vertex Pharmaceuticals (Europe) Limited

France

  • Bluereg
  • IPSEN
  • Laboratoires DELBERT
  • Servier

Italy

  • Angelini Pharma
  • Angelini Pharma s.p.a.
  • Sifi S.p.A.

Ireland

  • Parexel
  • Parexel Ireland USD

Spain

  • FAES FARMA SA
  • GAT Therapeutics

Belgium

  • Imcyse

Germany

  • CureVac AG

Latvia

  • AS Kalceks

Switzerland

  • Takeda

United States of America

  • CANbridge Pharmaceuticals

Enrol or reserve

Run Regulatory Strategies for Orphan Drugs Live online for your team

1 day

Typical duration

Pricing from:

  • GBP 450
  • Per attendee
  • Course tailored to your requirements
  • At your choice of location, or online

 

We can customise this course to your requirements and deliver it on an in-house basis for any number of your staff or colleagues.

Contact our in-house training experts Aleksandra Beer and Yesim Nurko to discuss your requirements:

Multiple colleagues? See above for details of our discounts for 2, 3, or 4 delegates. For more, talk to one of our training experts to discuss how to:

Run this course conveniently and cost-effectively in-house for your staff and colleagues

Aleksandra BEER
Training expert

Yesim NURKO
Training expert

+44 (0)20 7749 4749

inhouse@ipi.academy