London, 11-13 Dec 2019

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. 


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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

Meet your instructor

David Schwicker has biopharmaceutical consulting expertise spanning more than 25 years. He is the founder of ORPHA Strategy Consulting, based in Basle, Switzerland, and focused on the timely patient access to orphan and advanced therapy medicinal products.

This dedication to client consulting has enabled David to develop a capacity for rapid complex problem solving allowing him to provide creative and analytically rigorous advice on strategic challenges and to devise effective solutions to drive his clients' programs forward. David has longstanding scientific experience and expert contacts in rare diseases, has authored and co- authored peer-reviewed publications and is a speaker at international meetings. He is an accomplished trainer in rare diseases, orphan, gene and cell therapy medicinal products, and leads Masterclasses as well as customised in-house training workshops.

As Vice President with PAREXEL International in the US, David was responsible for registries, outcomes research, market access, and expanded access programs. Since returning to Europe, David has designed and implemented real-world studies including more than 200,000 patients.

From 2010, David's capabilities have expanded to rare diseases, including acute myelogenous leukaemia (AML), multiple myeloma (MM), neuroendocrine tumours, pulmonary arterial hypertension (PAH), Niemann Pick disease, acromegaly, graft versus host disease (GvHD), primary sclerosing cholangitis (PSC), Mucopolysaccharidosis (MPS), Myasthenia Gravis (MG), Graves Orbitopathy (GO), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Antibody- Mediated Organ Rejection (AMR), Narcolepsy, and Friedrich’s ataxia. David is currently working in rare autoantibody-mediated diseases, oncology/haematology, metabolic, CNS and GI indications.

David has developed a unique understanding of accelerating marketing authorization and market access in the hyper-dynamic European environment. Based on this, David supports clients in the attainment of “fast to market" objectives by integrating rare disease development, regulatory and market access strategies. This involves indication prioritisation, evidence generation planning (RCTs and RWE), patient engagement and PROs, orphan designation, compassionate use programs, product differentiation, and early value demonstration with fewer data. A crucial component of early access strategy is the application of innovative regulatory, health technology assessment, pricing and reimbursement pathways that leverage the use of real-world evidence (RWE).

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 earlier you book, the more you save…

Additionally, VAT may be applicable depending on course location.
Before 1 Nov 2019Before 22 Nov 2019After 22 Nov 2019
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  • “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 (Asia Pacific)
for more details


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