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 SHOULD SPONSOR

  • Contract Research Organizations (CROs)
  • Contract Development and Manufacturing Organizations (CDMOs)
  • Clinical Trials Services
  • Home Clinical Trials
  • Patient Recruitment & Identification
  • Regulatory Consulting
  • Real World Evidence

 

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 SHOULD SPONSOR

  • Multinational and foreign Contract Research Organizations (CROs)
  • Market Access Consulting
  • Pricing & Reimbursement Consulting
  • Licensing
  • Government agencies
  • Distributors

 

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 SHOULD SPONSOR

  • Investment Banks
  • Pharma & Biotechs
  • Venture Capital Firms
  • Grant Agencies (Public and Private)
  • Contract Research Organizations (CROs)

 

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 SHOULD SPONSOR

  • Immunoncology pharma and biotechs
  • Contract Manufacturing Organizations (CMOs)
  • Contract Research Organizations (CROs)
  • Genomic/DNA sequencing

 

 

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 postmarket patient identification
  • How to propose value based frameworks for pricing and reimbursement of orphan drugs
     

WHO SHOULD SPONSOR

  • Specialty Pharmacy
  • Logistics, Supply Chain, 3PL
  • Strategic Consulting
  • Real World Evidence
  • Forecasting and Epidemiology
  • Marketing, Communications Agency
  • Market Research
  • Data analytics platforms

 

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

WHO SHOULD SPONSOR

  • Pharma & biotechs
  • Research Institutions
  • Patient Registries
  • Patient Engagement Platforms
  • Genetic Testing providers
  • Genomic Sequencing Platforms
  • Diagnostics

 

WHO ATTENDS

Pharma, biotechs

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

 

TOPIC AREAS

  • drugs how to scale up process development and manufacturing efficiently 
  • how to assess in-housing vs outsourcing manufacturing capabilities 
  • how to run a smooth logistics operation for cell and gene therapies 
  • how to educate the physician community about advanced therapies



     

WHO SHOULD SPONSOR

  • Contract Research Organizations (CROs)
  • Contract Manufacturing Organizations (CMOs)
  • Viral Vector Development and Manufacturing
  • Supply Chain & Logistics (3PL)
  • Providers
  • GMP, nonGMP process development