2 Day Masterclass Orphan Drugs

Accelerate discovery, development, regulatory approval and market access in rare diseases. A deep dive into orphan medicinal products and cell and gene therapies.

Objectives | Your Trainer | Course Participants | How It Works

Course Objectives

• Understand the specific requirements of rare conditions, OMPs and ATMPs

• Gain insight into the most recent methodologies for faster, smaller, smarter clinical trials, including early-phase studies that are robust enough for marketing application

• Discuss strategies and novel tools to limit the number of patients and the placebo exposure of participants in pivotal clinical trials in rare and ultra-rare conditions

• Explore the opportunities of RWE to accelerate discovery, development, regulatory approval and market access in rare diseases

• Consider the crucial role of patient engagement throughout the OMP and ATMP lifecycle

• Engage in early dialogues and leveraging Joint Scientific Consultations (JSCs)

• Discover the sweeping regulatory revisions and their impact on innovative medicines

• Develop actionable recommendations to futureproof clinical development and regulatory strategies to address these comprehensive regulatory changes

• Analyse the current market access challenges for OMPs and ATMPs as well as innovative strategies that integrate patient-centric RWE

• Propose next-generation value evidence for market access and pricing success in the increasingly challenging rare disease area

Mr. David Schwicker is a sought-after expert for accelerating time to approval, launch and patient access for orphan medicinal products (OMPs) and cell and gene therapies (Advanced Therapy Medicinal Products, ATMPs).

David’s experience includes rare oncology and haematology (ALL, AML, MM, CD30+ HL/NHL, CTCL, ALK+/EGFR+ NSCLC, breast cancer), metabolic and endocrine diseases (MPS, Graves’ disease, acromegaly), rare CNS disorders (CIDP, RTT, MG, narcolepsy, Friedrich’s ataxia), inherited retinal dystrophies (RA, LCA, USHERs), hepatic conditions (PSC), rare cardiopulmonary diseases (IPF, PAH, PH-ILD), dermatology (PN, EB, XLHED), and rare immunology (AMR, GvHD).

This unique training focuses on leveraging patient engagement, innovative rare disease clinical trial design, analytical tools and disease-specific endpoints tailored to small, heterogenous populations, regulatory incentives, and real-world evidence (RWE) to accelerate the clinical development, regulatory approval, and patient access of novel orphan medicinal products and cell and gene therapies.

The highly interactive course will discuss and co-create concrete, actionable strategic recommendations to support the successful development, regulatory approval, and commercialisation of innovative therapies in rare diseases.

• Rare disease, orphan, paediatric and advanced therapy medicinal product leads

• Clinical development leaders and program directors

• Regulatory affairs executives

• Value, pricing and market access executives

• Marketing and commercial leaders

• Medical affairs executives

• Evidence Generation, Real-world Evidence executives

• Patient Centricity/Engagement and COA/PRO leadership

• The 2 Day Masterclass is delivered live online.

• Course reminders, access passwords, digital course materials and slides will be sent out before the course.

• Course Timings: 12 - 4 pm (UK GMT/BST). Timings can be adjusted to suit your time zone.

• A Certificate of Participation will be given to each attendee.