PRE-CONGRESS WORKSHOPS

 

With 80+ speakers the multi streamed conference enables you to choose the sessions which are the most applicable to help your business plan for the future of orphan drug research, development and manufacture.

 

Morning Pre-congress Workshop Monday 13 November

Hosted by MME (Medical Marketing Economics) Hosted by MME (Medical Marketing Economics)
 
9.00- 12.30
Pricing, Reimbursement and Market Access Challenges for Advanced Therapy Medicinal Product / Orphan Drugs in the EU 
 
Major hurdles exist even in the EU health systems delaying and preventing patient access to Orphan Drugs (ODs) approved by the EMA. If possible, such hurdles are even greater for ODs that are also Advanced Therapy Medicinal Products (ATMPs) - Cell therapies, gene therapies and hybrid cell-gene therapies.
 
In this workshop we will review the actual market access (MA) status and timing in the main EU countries of ODs approved by the EMA in the recent past and will then discuss the additional issues for ATMP/ODs.
 
Real-world experience will be presented and discussed, with the testimony of a medium-size European Company with a specific focus on ODs and, in particular, ATMP/ODs.

Finally, potential solutions will be discussed, to address key pricing, reimbursement and MA challenges for ATMP/ODs in the EU context.
Gain from a unique combination of 1) manufacturer, customer, economic, and clinical perspectives combined with 2) solid academic theory from attending this workshop. 
 

Dr Renato Dellamano, President, MME Europe

Dr Renato Dellamano, President, MME Europe  
More industry co-speakers to be confirmed shortly

 
Expertise shared from MME: 
  • In addition to work in oncology and orphan markets, we have helped developers and marketers of >40 ultra-orphan drugs and several ATMPs to identify, frame, communicate and capture the value of their therapies. 
  • Unique combination of manufacturer and customer perspectives combined with solid academic theory 
  • Strategy development and tactical execution to support informed decision making 
  • Assessment and planning of opportunities and competitive situations at every stage of the product life cycle
  • Our methods are designed to develop an understanding of the value perceptions of all meaningful stakeholders 
 
What are the benefits of attending this workshop? 
  • The orphan markets have examples of products that have exceeded expectations as well as some that have yet to achieve initial forecasts: pricing and reimbursement is a factor that can work in either direction. 
  • During this workshop we will look at the general issues and challenges for ODs and at the additional particular challenges for ODs that are also ATMPs. 
  • In this particularly innovative field, understanding the current orphan drug environment and the potential changes in the near future include sustainability of orphan drug pricing plus the additional key issues related to pricing and reimbursement of cell and gene therapies. 
 
12.30 Networking Lunch followed by afternoon workshop
 

 

 

Afternoon Pre-congress Workshop Monday 13 November

Hosted by MAPI Group
Hosted by MAPI Group
 
Assuring success from Regulatory to Global Market Access of Rare Disease Treatments
 
13.30 – 17.00: 
Partnering with patients for clinical success
  • This interactive workshop will introduce participants to a 360° approach to obtaining patient insights and applying them for the successful design and execution of clinical studies. Through engaging activities, we will address how to get to the heart of the patient’s decision-making process, assess their physical/emotional/social needs, and identify their motivators, barriers and influencers as they relate to clinical study participation.
  • Participants will then learn how insights are analysed and applied in order to:
    • Design studies that appeal to the targeted patients
    • Determine effective and motivating recruitment methods
    • Engage and communicate with patients and caregivers
    • Identify retention methods that meet the needs of a specific patient population
    • Develop materials that resonate with patients and caregivers
 

Kelly Franchetti, Vice President Global Patient Insights and Engagement, Mapi Group

Kelly Franchetti, Vice President Global Patient Insights and Engagement, Mapi Group 
Patient representative to be announced

 
 
An introduction to the science of endpoint design in the current regulatory and HTA environment in the US and Europe
  • Regulatory strategies for early scientific advice: FDA & EMA scientific advice, PRIME & adaptive pathways link to scientific advice
  • The creation, categorization and hierarchy of endpoints and requirements of different stakeholder groups, including: regulatory agencies, clinicians, HTA bodies, payers, increasingly active patient groups
  • Real-life examples of endpoints used in selected therapeutic area & techniques for the development and validation of new endpoints
  • Highlighting guidance and publications that will be of use to newcomers to the area
  • Solving the non-alignment between different agencies and geographical  differences in approach
Rory Graham, Senior Director, EU Regulatory Services, Mapi Group

 

Rory Graham, Senior Director, EU Regulatory Services, Mapi Group
 
 
Martine Zimmermann, Global Head of Regulatory Affairs, Alexion pharma GmbH

Martine Zimmermann, Global Head of Regulatory Affairs, Alexion pharma GmbH

 
 
Methodologies in developing Real World Data: Engaging stakeholders in developing evidence strategies
  • How is expanded access used to provide treatment for patients with rare diseases?
  • Compassionate Use Programs in the window between submission and commercialization
  • What are the benefits of conducting clinical studies and registries for rare diseases?
  • What are the types of studies conducted during different phases of orphan drug development?
  • How are patient registries used to generate real-world data for rare diseases and orphan drugs?
  • Ethical Patient Warehousing – working with Advocacy to identify populations in advance of research
  • What are the unique challenges of conducting rare disease/orphan drug registries?
  • How can proactive study management strategies successfully address these challenges?
  • Potential risk mitigation strategies, and alternative registration strategies will be discussed.  
 

Dr Will Maier, Chief Scientific Officer and Head of Patient-Centered Sciences, Mapi Group

Dr Will Maier, Chief Scientific Officer and Head of Patient-Centered Sciences, Mapi Group 
More co-speakers to be confirmed shortly


17.00 End of Workshop Followed by Networking Drinks Hosted by Mapi Group