References & Further Reading

Regulatory guidelines, clinical guidelines, key studies and reference sources supporting DEFRATAJ website content.


Selected references, guidelines and literature sources. Verify clinical statements against current local prescribing information and peer-reviewed literature.

Regulatory Guidelines #

  • ICH Q1A(R2) Stability Testing
  • ICH Q2(R1) Analytical Procedures Validation
  • ICH Q3B(R2) Impurities
  • ICH Q8 / Q9 / Q10 Development, Risk, Quality Systems
  • WHO GMP Guidelines (WHO Technical Report Series)
  • PIC/S PE 009 GMP Guide
  • ISO 9001:2015 QMS Requirements

Clinical Guidelines #

  • Thalassaemia International Federation (TIF) Guidelines
  • British Society for Haematology - Iron Chelation
  • NCCN Guidelines for MDS (current edition)
  • American Society of Hematology - SCD Guidelines

Representative Clinical Studies #

  • Cappellini MD et al. - deferasirox pivotal programme
  • EPIC study programme
  • Vichinsky E et al. - deferasirox in SCD
  • List AF et al. - deferasirox in lower-risk MDS
  • Pennell DJ et al. - cardiac iron mobilisation

Precise citations should be verified from original publications.

Regulatory Information Sources #

  • EMA EPAR (deferasirox innovator)
  • US FDA approved labelling
  • CDSCO India prescribing information
  • WHO Model List of Essential Medicines

Pharmacovigilance #

  • CIOMS Working Group reports
  • VigiAccess (WHO UMC)
  • National PV authorities

Full Reference List #

Full citation lists supporting pharmacology, clinical and quality content are in the CTD Dossier Module 2, shared under CDA.