Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Factor VIII Concentrates and the AMNOG Early Benefit Assessment - Case Study Turoctocog alfa and Simoctocog alfa ***

Dr. Olga Neumüller (Abschlußjahr: 2015)

Summary
Language: English
Health technology assessment (HTA) has become increasingly important in the pricing and reimbursement process regarding medicinal products across much of Europe and beyond. In particular, the early benefit assessment (EBA) pursuant to AMNOG has been introduced to cut costs and to illustrate the additional medical benefit for each newly-authorised reimbursable medicinal product entering the German market. Coagulation factor VIII concentrates indicated for the prophylaxis and treatment of haemophilia A, which is a rare bleeding disorder, are not exempted from this process.
With the marketing authorisation of NovoEight^® (INN: turoctocog alfa) and Nuwiq^® (INN: simoctocog alfa), for the first time two recombinant factor VIII concentrates underwent the EBA procedure and were appraised by the G-BA in terms of their additional medical benefit over a pre-selected appropriate comparative treatment.
Using these products as examples, this case study aims to analyse the procedure and the requirements of the early benefit assessment pursuant to AMNOG in Germany with regard to factor VIII concentrates. In addition, the EBA requirements are compared with the regulatory requirements for the clinical development and marketing authorisation of these products in the EU.
Finally, the applicability to and plausibility of those requirements and procedures with regard to factor VIII concentrates and haemophilia A, considering that it is a rare disease, are discussed. Ultimately, the conclusion is drawn that alternative strategies, practical solutions, and early extensive dialogue among stakeholders are needed to overcome the situation in which pharmaceutical manufacturers are unable to reconcile and meet the different requirements as set by the regulatory and HTA authorities in their elaborate research and development programmes.
Pages: 47
Annexes: 1, Pages: 7