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.
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
Your Trainer
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).
Course Participants
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
How Does It Work
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.
Next available dates: December 10-11, 2024
Register for your exclusive training now!
Discounted course offer: £2,598
Related Courses
5Training
Contact
International House, 64 Nile Street, London N1 7SR
+44 (0) 781 628 5327