Rare Diseases and Orphan Drug Development in Europe

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Training Schedule (3 & 4 July, 2024)

(3rd July, 2024)
14:00 - 18:30 PM UK time


(4th July, 2024)
14:00 - 18:30 PM UK time

Online

Join visionary speakers from leading organisations for crucial insights and expert opinions.

Dr. Klaus Rose,

Dr. Klaus Rose is a physician with decades of experience in clinical research, drug development and medical affairs in the life science industry. He was global head pediatrics Novartis 2001-2005; same position Genentech/Roche 2005-2009; CEO klausrose Consulting since 2011. He speaks on international conferences, publishs, co-edits and authors medical textbooks.

Training Schedule (3 & 4 July, 2024)

14:00 - 18:30 PM UK time

Introduction:

For most frequent diseases such as hypertension or dyslipidemia good treatment exists already.

Occasionally, new treatments emerge for old or new frequent challenges, such as vaccines against

COVID-19 or drugs against obesity. To stay competitive, in 2023 the EU anounced a fundamental

revision of its pharmaceutical legislation, including the duration of marketing exclusivity, unmet

medical needs, incentives, clinical trials for rare/orphan & pediatric diseases, and cell & gene

therapies. Also changes in health technology assessment (HTA) were anounced, including

requirements & procedures for innovative orphan medicinal products (OMPs) and advanced therapy

medicinal products" (ATMPs). Learn the key points and to what degree the EU anouncements are

realistic.

Your trainer

Dr. Klaus Rose is a physician with decades of experience in clinical research, drug development and

medical affairs in the life science industry. He was global head pediatrics Novartis 2001-2005; same

position Genentech/Roche 2005-2009; CEO klausrose Consulting since 2011. He speaks on

international conferences, publishs, co-edits and authors medical textbooks.

METHODOLOGY:

Based on the history of medicine and drug development, we go through current EU laws, the

literature, definitions, and trends in innovative orphan medicinal products (OMPs), advanced

therapy medicinal products" (ATMPs), and additional obligations such as pediatric investigation

plans (PIPs). We interactively discuss strengths & weaknesses and how companies can deal with

regulatory demands, obstacles, and rewards at the interface of science, regulations, and the market.

KEY OUTCOMES

Learn about

  • New proposed EU legislation
  • EU successes, strengths and weaknesses in dealing with OMPs and ATMPs
  • How to navigate in the tension between regulatory demands and scientific input

Who Should Attend? The course addresses in first line professionals, executives, and heads of department in the life science industry, including clinical research organisations (CROs), from

  • Research & development (R&D)
  • Clinical development, project managament, strategic planning
  • Medical affairs (MAs)
  • Regulatory affairs (RAs)
  • Drug safety
  • Marketing & commercial planning
  • Communication

Participants from academia will be welcome, too

Draft Schedule:

DAY 1:

14:00 - 15:00h Session 1: Introduction of lecturer and participants. Rare Diseases and Orphan Drug Development (ODD)

  • Drug development: past, present & the future
  • Definition of rare/orphan diseases and why ODD is facilitated
  • The worlds of clinical care, clinical research, advocacy groups, drug approval Conflicts of interest.

15:00 - 16:00h Session 2: Global drug development, European countries, EU & EMA

  • Why does the EU announce a revision of its pharmaceutical legislation?
  • Big pharma, patient associations, start-ups in ODD
  • Re-purposing of out-of-patent drugs vs. innovative research

15:00 - 15:15 Biobreak

Join visionary speakers from leading organisations for crucial insights and expert opinions.

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