THE MOST COMPREHENSIVE RARE DISEASE EVENT GLOBALLY

 

 

 

 

 

 

 

 

 

WHO ATTENDS

Pharma, biotechs

  • VP Clinical Development
  • Chief Medical Officer 
  • Chief Scientific Officer 
  • Regulatory Affairs 
  • President, Heads of Rare Diseases

     

TOPIC AREAS

  • how to set up and run expanded access programs 
  • how to generate evidence and use it in regulatory review 
  • how to use machine learning and discovery platforms for drug repurposing 
  • how to reduce patient burden through siteless data capture and evidence generation from virtual trials 
  • how to use machine learning algorithms to search for undiagnosed patients with rare diseases

     

 

WHO ATTENDS

Pharma, biotechs

  • VP Market Access 
  • Pricing and Reimbursement 
  • Chief Commercial Officer
  • International Business

Regulators/ Government

  • Ministry of Health
  • Policy makers

TOPIC AREAS

  • how to navigate the regulatory, pricing and reimbursement landscape of foreign orphan drug markets 
  • how to negotiate pricing and reimbursement with foreign payers 
  • how to choose between licensing and setting a commercial footprint on foreign markets 
  • how to stay compliant with evolving orphan drug regulation in other markets 
  • how to collaborate with patient advocacy groups to ensure and improve access to orphan drugs

 

WHO ATTENDS

Pharma, biotechs

  • VP Market Access 
  • Pricing and Reimbursement 
  • Chief Commercial Officer 
  • International Business

Venture Capital Firms
Grant Agencies (Public & Private)
Licensing pharma

TOPIC AREAS

  • how to attract VC funding at early stage development 
  • how to apply for new public grants for advanced therapies 
  • how to benefit from licensing models 
  • how to assess M&A opportunities in a mature orphan drug market 
  • how to assess barriers to entry including therapeutic modality and IP



     

 

WHO ATTENDS

Pharma, biotechs

  • VP Cell Therapy 
  • Heads of Oncology 
  • Heads of Immunotherapy 
  • Chief Medical Officer




     

TOPIC AREAS

  • how to define clinical endpoints in rare oncology trial design
  • how to accelerate diagnosis and improve its accuracy 
  • how to produce epidemiology data and studies for rare cancers 
  • how to overcome logistical challenges for rare cancers clinical trials


     

 

WHO ATTENDS

Pharma, biotechs

  • Chief Commercial Officer
  • VP Commercial Operations
  • VP of Marketing
  • VP Medical Affairs
  • Pricing & Reimbursement

TOPIC AREAS

  • how to build the patient forecast based on available epidemiological information
  • how to leverage pre-approval initiatives, forecasting and epidemiology for commercial success
  • how to leverage social media, machine learning and data for post-market patient identification
  • how to propose value based frameworks for pricing and reimbursement of orphan drugs
     

 

WHO ATTENDS

Pharma, biotechs

  • Chief Executive Officers
  • VPs, Heads of Market Access
  • VP, Head of Pricing and Reimbursement
  • VP of Health Economics/HEOR
  • VP of HTA
  • Head of Value

Payers (public & private)
Government Agencies

TOPIC AREAS

  • how to develop common tools to support HTA and reimbursement decisions
  • how to ensure the patients voice in reimbursement for rare diseases
  • hot to generate evidence for optimal price and access of orphan drugs

 

WHO ATTENDS

Pharma, biotechs

  • VP Gene Therapy
  • Chief Medical Officers
  • Chief Manufacturing Officers
  • VP Manufacturing & Technical Operations

TOPIC AREAS

  • how to overcome challenges in early stage trials for gene editing
  • how to advance next generation therapy programs such as RNA t
  • how to run a smooth logistics operation for cell and gene therapies
  • how to educate the physician community about advanced therapies

 

WHO ATTENDS

Pharma, biotechs

  • Chief Operating Officer
  • Chief Manufacturing & Technology Officer
  • VP Technical Operations
  • VP of CMC
  • VP Manufacturing
  • VP of Process Development
  • VP of Quality Control

TOPIC AREAS

  • how to scale up process development and manufacturing efficiently
  • how to assess in-housing vs outsourcing manufacturing capabilities
  • how to improve batch efficiency for viral vectors and bring the cost of manufacture down
 

 

 

WHO ATTENDS

Pharma, biotechs

  • Chief Patient Officer
  • VP Patient Advocacy
  • Medical Affairs
  • Patient Engagement

Patient Advocacy Groups
 

TOPIC AREAS

  • how to collaborate with industry to advance rare disease research 
  • how to leverage funding to support rare disease research efforts 
  • how to educate the patient community about newly approved and advanced therapies 
  • how to shorten the diagnostic discovery and connect patients to research and treatments 
  • how to continue encouraging research and investment in rare disease after approval