Commercialisation Resources

Market access for ATMPs

• Post-Authorisation scientific and regulatory guidance for all products types, European Medicines Agency (EMA)​​​​​​
• EU market access roadmaps for cell and gene therapies, Cell and Gene Therapy Catapult (CGT Catapult)
• The European regulatory framework and drug approval process, Lung Cancer Europe (LuCE)
• Commercialisation toolbox: guides, checklists, and self assessment, Canada’s Centre for Commercialization of Regenerative Medicine (CCRM)

Marketing authorization pathway  

Standard approval process: centralized procedure 

• Advanced therapies: marketing authorisation, European Medicines Agency (EMA)
• Obtaining an EU marketing authorisation, European Medicines Agency (EMA)
• Evaluating marketing authorisation applications, European Medicines Agency (EMA)

Marketing Authorisation Application file

• Marketing authorisation guidance documents, European Medicines Agency (EMA)
• Dossier requirements by EMA, European Medicines Agency (EMA)

Expediting access pathways

• Accelerating Patients’ Access to Advanced Therapies in the EU

Conditional MA

• Conditional MA, European Medicines Agency (EMA)
• 10-year report on the conditional marketing authorisation, European Medicines Agency (EMA)
• Pre-authorisation guidance: 'Could my application qualify for a conditional marketing authorisation?', European Medicines Agency (EMA)
  • see Q&A Question 1.9

MA under exceptional circumstances   

• Pre-authorisation guidance: 'Is my medicinal product eligible for approval under exceptional circumstances?', European Medicines Agency (EMA)
  • see Q&A Question 1.10

Accelerated Assessment 

• Accelerated assessment, European Medicines Agency (EMA) 
• ​​​​​Pre-authorisation guidance: ​​'Is my medicinal product eligible for an accelerated assessment? (Rev. October 2021)', European Medicines Agency (EMA) 
  • see Q&A Question 2.8

Biosimilars

• Biosimilar medicines: marketing authorisation, European Medicines Agency (EMA)
• information guide on biosimilars for healthcare professionals, European Medicines Agency (EMA) and European Commission (EC)
• information guide for patients on biosimilars medicinal products, European Commission (EC)

EMA regulatory support schemes 

PRIME 

• PRIME: priority medicines, European Medicines Agency (EMA)
• PRIME: Analysis of the first 5 years’ experience, European Medicines Agency (EMA) 
• Toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME and certain marketing authorisation applications targeting an unmet medical need, European Medicines Agency (EMA) 
  • New guidance for developers of medicines supported by EMA's PRIME scheme on the tools they can use to generate robust quality data packages for their MAA

Adaptive pathways   

Distribution

Good Distribution Practices

• Good Distribution Practices (GDP), European Medicines Agency (EMA)

Advertising

This section is in development.

Pharmacovigilance (medicines/ATMP)  

• Pharmacovigilance (general medicine), European Medicines Agency (EMA)
• Pharmacovigilance for ATMPs, European Medicines Agency (EMA)
• Guidance notes on the management of safety information and product complaints from digital activities, The Association of the British Pharmaceutical Industry (ABPI)
• Drug post-marketing follow up and pharmacovigilance, Lung Cancer Europe (LuCE)
• French pharmacovigilance: Missions, organization and perspectives

Good Pharmacovigilance Practices

• Good Pharmacovigilance Practices (GVP), European Medicines Agency (EMA)

Pricing & reimbursement

• Health technology assessment, European Commission (EC)
• HTA fundamentals, European Patients’ Academy on Therapeutic Innovation (EUPATI)
• Health Technology Assessment , European Patients' Forum (EPF)
• Health Technology Assessment (HTA) and drug pricing, Lung Cancer Europe (LuCE)
• Getting Ready for Advanced Therapy Medicinal Products (ATMPs) in Europe, The Alliance for Regenerative Medicine (ARM)
• Health Technology Assessment briefing and Guide, Northern Alliance Advanced Therapies Treatment Centre (NA-ATTC)
• ATMP market entry from an HTA perspective, ATMP Sweden
• Introduction on HTA. Provides overview, pricing, HTA methodology, pathways, stakeholder by each European country
• A mock HTA, The National Institute for Health and Care Excellence (NICE)
• OECD Pharmaceutical Pricing Policy project, Organisation for Economic Co-operation and Development (OECD)
• Establishing payment models that support timely access to ATMPs The Association of the British Pharmaceutical Industry (ABPI)
• Pricing medicines: theory and practice, challenges and opportunities
• The business case for cell and gene therapies
• To Seek or Not to Seek Parallel European Medicine Agency and Health Technology Assessment Scientific Advice?
• Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries
• An overview of critical decision-points in the medical product lifecycle: Where to include patient preference information in the decision-making process