3 day Masterclass

Orphan Drugs



The 3 Day Masterclass in Orphan Drugs is for professionals who want to know about the opportunities and details of entering the business of rare diseases and the orphan medicinal product (OMP) and the Advanced Therapy Medicinal Products (ATMP) and gene therapy markets. It is also for those who want to learn about new trends in medical therapy for the 1 in 17 persons who have one of 6.000 rare or ultra-rare diseases.

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develop new skills

Create an accelerated Orphan Drug development plan

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fully engage

Develop a “rapid market access” Orphan Drug Pricing and Reimbursement Strategy

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get practical

Write a business plan for developing a business based on new therapies for rare diseases


This interactive course has been designed to teach participants through practical exercises in six clearly structured modules and workshops that pertain to very important aspects of the orphan drug market: (1) orphan drug designation; (2) rare disease development strategies; (3) orphan drug regulatory and legislative strategy; (4) early market access; (5) unmet need, value, market access, pricing and reimbursement; and (6) how to write a business plan based on a rare disease therapy.

Given the often life-threatening and urgent nature of rare patient unmet needs, the overriding focus of the workshop is “fast to market”: i.e. the acceleration of development, authorisation and market access timelines in order to ensure the timely access to potentially transformative treatments, contributing to the survival and improved quality of life of patients affected by rare conditions.

Participants will be invited to share their specific and unique challenges with the workshop leader ahead of the masterclass, ensuring that the workshop content is tailored specifically to help them resolve their current programme challenges and devise effective solutions to drive their programme forward. 

In just 3 days you will:

  • Write an orphan drug designation for the Food and Drug Administration (FDA) or European Medicines Agency (EMA)
  • Create and implement an accelerated orphan drug and rare disease development plan with smaller and faster trials
  • Develop a “fast to market” orphan regulatory strategy for the US/FDA and Europe/EMA
  • Achieve early market access and revenues with a compassionate use strategy
  • Develop a “rapid market access” orphan pricing and reimbursement strategy
  • Write a business plan for an orphan drug involving advanced new therapies
  • And many facts, issues and examples of the rare disease market

Organisations that will benefit:

  • Pharmaceutical & Biotech Companies
  • Rare Disease Research Organisations
  • Suppliers
  • Investors

Who will attend:

  • C-Level Executives: CEO, CFO, COO
  • Business Development and Sales
  • Researchers

Course Agenda

  • Introductions
  • Delegates’ key interests and objectives for the masterclass
  • Focus on “fast to market” early access strategy
  • Programme overview
  • Introduction to Orphan and Advanced Therapy Medicinal Products
  • Orphan Designation and Incentives
  • Orphan designation application and maintenance
  • Case study - Acromegaly (somatostatin analogs/receptor ligands) major contribution to the patient care of the oral vs parenteral formulation
  • OMP Regulatory and Legislative Strategy (Morning)
  • Develop a “fast to market” OMP regulatory strategy
  • Early Market Access through Compassionate Use (Afternoon)
  • Write an early market access CUP strategy
  • Rapid Market Access Pricing & Reimbursement Strategy
  • Overview of Health Technology Assessment (Europe) o National agencies and competencies
  • Current HTA approaches to OMPs and ATMPs
  • “Rapid market access” orphan P&R strategy
  • Rare Disease Therapy Business Planning
  • Creating a biotech startup business plan
  • Write a rare disease orphan development business plan

For further information please call: +44 (0)207 092 1045

  • “The depth and knowledge of my trainer was extremely impressive. In 3 days I have had, without a doubt, a masterclass on the industry. Truly world class.”

    - Gordon Dowall Potter, Past Delegate

  • “Well worth 3 days of study!”

    - Syed Hamza, Past Delegate

  • “The training was excellent! I give it a 10 and will get my trainer's support for my next challenges.”

    - Head of Partnerships & Customer Loyalty at Avis Budget, Past Delegate

  • “Extremely constructive course with a very good dynamic and passionate trainer.”

    - Managing Director at BNP Paribas, Past Delegate

  • “Impressive and cooperative trainer who wanted to make sure the delegates understood.”

    - Budgeting, Planning & Reporting Manager, Past Delegate


We can come to you too! If you have a team of six or more, why not hold a private course? Our experts come to you and the course is tailored to your requirements.

Call +44 (0)20 7092 1045 (United Kingdom)
or emma-jane.dinan@terrapinn.com (Asia Pacific)
for more details


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