Regulatory benefit – risk assessment; different perspectives

This thesis shows that a medicine’s safety can be preserved if innovative adaptive procedures to medicine regulation are applied. In addition, it describes how different stakeholders in diabetes medication development make similar benefit-risk assessments of new medicines. Regulators of medicine development are pressured to balance a desire for a rapid market access of new medicines with the need for proper knowledge of a product’s benefits and – especially – risks. This thesis evaluates whether an appropriate balance is struck between the appraisal of benefit and risk in market authorization procedures. We found that high levels of innovation and relatively limited clinical testing did not increase the incidence of serious post-approval safety issues. Also, patients with type 2 diabetes and their doctors make similar benefit-risk assessments of oral anti-diabetes medicines as regulators. This research indicates that acceptance of greater uncertainty about a medicine’s risks at the time of approval seems acceptable for pharmaceuticals addressing unmet medical needs. Whether adaptive procedures to medicine regulation should be implemented for other types of medicines as well should be studied further.

Results

• Arnardottir AH, Haaijer-Ruskamp FM, Straus SM, Eichler HG, De Graeff PA, Mol PG. Additional safety risk to exceptionally approved medicines in Europe? In: Br J Clin Pharmacol, 2011; 72(3): 490-9. (journal publication)
• Arnardottir AH, Haaijer-Ruskamp FM, Straus SM, De Graeff PA, Mol PG. Effect of safety issues with HIV medicines on the approval process of other medicines in the same class: an analysis of European Public Assessment Reports. In: Medicine Saf, 2011; 34(11): 1101-14. (journal publication)
• Regulatory benefit-risk assessment (thesis)