New York, 13-15 Nov 2019


3 day Masterclass

Gene Therapies

AUTOLOGOUS & ALLOGENIC GENE THERAPIES TISSUE ENGINEERED PRODUCTS SOMATIC CELL THERAPIES

EXPLORE THE WORLD OF ADVANCED THERAPY MEDICINAL PRODUCTS

There are around 1000 cell, gene and tissue engineered therapies in development. This 3 Day Masterclass 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 6-7000 rare and ultra-rare conditions, 80% of which have identified genetic origins, making them potential targets for gene therapies.

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address key challenges

Fully explore the genetic origins behind rare and ultra-rare conditions

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get practical

Discuss, develop and brainstorm how to tackle ATMP challenges through interactive case studies

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seize opportunities

Analyse the development & commercialisation of Advanced Therapies

from discovery to patient access: a deep dive into the world of advanced therapy medicinal products 

 

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.

Successful development and commercialisation of Advanced Therapies requires the integration of development, regulatory, manufacturing and market access strategies. The intersection point of these strategies is proposed as the joint vision of earliest patient access.

Participants will be invited to share their unique challenges with the workshop leader ahead of the training, ensuring that the workshop content is tailored specifically to help them devise effective solutions to drive their programmes forward.

 

 

book today saving USD $710

In just 3 days you will:

  • Explore the opportunities of digital and big data to accelerate discovery, development, regulatory approval and market access
  • Gain insight into the most recent methodologies for faster, smaller, smarter clinical trials, including Phase I/II studies that are robust enough for marketing application
  • Discover the newest regulatory approaches for gene therapies
  • Consider the crucial role of patient involvement throughout the lifecycle
  • Deliberate the CMC and GMP manufacturing challenges of ATMPs
  • Review the current market access challenges and the debate how to value and price curative “one and done” gene therapies
  • Examine next-generation market access strategies that integrate patient-centric real-world evidence
  • Discuss novel commercialisation models needed to meet the complex demands of these unique therapies as the science continues to rapidly advance
  • Develop a business plan and a biotech investor pitch for an ATMP startup

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:

  • Gene Therapy Industry Professionals
  • Large pharmaceutical industry
  • Biotech industry
  • University cell and gene therapy departments
  • Banks and Investors
  • Consultants and Advisors

Who will attend:

  • Industry Professionals who want to take a deep dive into the opportunities and challenges of the expanding business of Advanced Therapy Medicinal Products (ATMPs).
  • 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.

Course Agenda

  • Introductions
  • Delegates' key interests and objectives for the ATMP Masterclass
  • “Fast to patient” early access strategy as the cross-cutting theme
  • An Introduction to Advanced Therapy Medicinal Products (ATMPs)
  • Accelerated Development Strategy for ATMPs
  • Interactive Case Studies:
  • Zalmoxis (somatic cell therapy) historical controls from the EBMT registry
  • CAR T-cell therapy Kymriah cGMP manufacturing issues with clinical batches
  • Platform trials: ISPY-2, Beat AML and Precision Promise (targeted oncology)
  • Mepsevii (enzyme replacement therapy) N=1 “trial”, expanded access (eIND) in MPSVII
  • Strimvelis (ex-vivo autologous gene therapy) ultra-rare population registry (ADA-SCID)
  • Luxturna, a one-time therapy for Inherited Retinal Disease (IRD): the first randomized, controlled gene therapy trial for a genetic disease
  • Allogene Therapeutics - the next revolution in donor-derived cell therapy?
  • "Fast to Patient" ATMP Regulatory Strategy
  • Interactive Case Studies:
  • Two-year experience with PRIME: CAR T-cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), the first products approved through the scheme
  • Kymriah special protocol assessments, PIPs, regulatory interactions and timelines (FDA)
  • EMA/HTA parallel consultations –alignment vs disagreement of recommendations
  • Alofisel (darvadstrocel, human allogeneic mesenchymal adult stem cells), orphan designation and orphan exclusivity at marketing authorisation
  • Earliest Patient Access with Compassionate Use
  • Next-Generation Commercialisation Strategies for ATMPs
  • Interactive Case Studies:
  • Kymriah US pricing: expectations of payers, patients, the scientific community, analysts, investors and shareholders; indication-based pricing vs Yescarta
  • Kymriah and Yescarta: the first HTA assessments and pricing negotiations in Europe
  • Zalmoxis (somatic cell therapy) P&R timelines, AIFA’s new innovation appraisal
  • Alectinib vs Ceritinib comparative effectiveness study in ALK+ advanced NSCLC
  • NICE’s Highly Specialised Technology (HST) appraisal of Strimvelis (ex-vivo autologous gene therapy) in an ultra-rare population (ADA-SCID)
  • Performance-Based Risk-Sharing Arrangements (PBRSAs) in Italy, AIFA’s monitoring registries and “success fee” scheme
  • ATMP Business Planning
  • Key Takeaways and Wrap

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.
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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

IN-HOUSE

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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