The International Training Course will be organised through Symmetric s.r.o. ORPHA Strategy's Principal, David Schwicker, will be the Trainer
Lydia Makori | Business Development Specialist at Symmetric
T +421 222 200 543 | Email email@example.com
Symmetric s.r.o. | Mliekarenska 9 |82109 Bratislava | Slovak Republic | Office: +421 948 262 346
The existing pipeline of cell & gene therapies is expected to lead to 40-60 launches, with 15-30 launching within the next five years, and around 500,000 patients in the United States treated with 40-60 products by 2030 (source: FoCUS, an MIT NEWDIGS Project, October 2018). These therapies represent an unprecedented opportunity for patients and caregivers, potentially offering cures in numerous complex conditions that previously had inadequate treatments, were extremely debilitating and life-threatening. In addition to manufacturing requirements, the intense debate on how to value, price and pay for cures challenges the successful commercialisation of ATMPs. Reasons enough to invest time to investigate the entire value chain of gene & cell therapies.
This highly interactive training has been designed to teach participants specifically about autologous and allogeneic gene therapies, tissue engineered products and somatic cell therapies.
Heads of R&D, Global program leads, Regulatory executives, C-level executives, Senior business development strategists, Portfolio executives, Pricing and market access executives, Medical affairs executives, Marketing and Commercial executives, Finance executives, Patient advocates, Epidemiologists, Outcomes researchers, Health economists, Digital health specialists, Big data and RWE researchers.
This International Training is for professionals who want to take a deep dive into the opportunities and challenges of the expanding business of Advanced Therapy Medicinal Products (ATMPs).
It is also for those who want to learn more about the 6-7.000 rare and ultra-rare conditions, 80% of which have identified genetic origins, making them potential targets for gene therapies. The 50-60 million individuals living with rare conditions of genetic origin represent a huge unmet medical need, which is often life-threatening, highly debilitating and requires great urgency.
The cross-cutting theme of this masterclass is therefore “fast to patient”: the acceleration of discovery, development, authorisation, manufacturing and market access in order to ensure the timely access to potentially curative treatments, contributing to the survival and improved quality of life of individuals affected by severe and complex conditions.
Throughout the three days, delegates will discuss, develop and brainstorm strategies to address ATMP challenges based on the presentation of current case studies.
A deep-dive into Voretigene Neparvovec-rzyl (Luxturna) in RPE65 Mutation Associated Inherited Retinal Dystrophy (IRD) – adeno-associated viral vector (AAV) Gene Therapy: clinical development, regulatory strategy, market access and pricing in the US and Europe.*
* Delegates will receive selected pre-reading materials to familiarize themselves with these topics before the course. Note that all the information presented in the case examples and the deep dive workshop is based on publicly available scientific papers, articles and press releases.
We provide creative and analytically rigorous advice on strategic problems and devise effective solutions to drive our clients' programmes forward. We do this through collaboration, by channelling and merging our expertise with that of our clients. Our approach has been honed in more than 25 years of consulting experience and will flexibly adapt to specific and unique challenges. Please contact us today to start a strategic discussion about early market access strategy.
ORPHA Strategy Consulting
Phase IV Programs LLC
David Schwicker, Principal
4051 Basle, Switzerland
Tel: +43 676 362 9571