PhD ceremony Ms. A.H. Arnardóttir: Regulatory benefit - risk assessment. Different perspectives

PhD ceremony: Ms. A.H. Arnardóttir, 12.45 uur, Academiegebouw, Broerstraat 5, Groningen

Dissertation: Regulatory benefit - risk assessment. Different perspectives

Promotor(s): prof. F.M. Haaijer-Ruskamp, prof. P.A. de Graeff

Faculty: Medical Sciences

The strict regulations of the pharmaceutical industry are aimed at ensuring that only safe, effective and high quality drugs reach the market. Regulators are pressured to balance the desire for new drugs to rapidly enter the market, with the need for an acceptable knowledge of benefits and risks at the time of approval. Such knowledge is limited, especially with regards to safety, as pre-approval clinical trials are limited in trial size, patient selection, and are focused on establishing efficacy.

This thesis aims to evaluate whether an appropriate balance is struck in the ascertainment of benefit and risk of new drugs at the time of market approval. It focuses on two issues; whether knowledge about safety issues during market authorisation is appropriately taken up, and whether regulators agree with patients and doctors when weighing benefits and risks of drugs. We found that class-related safety issues are integrated in the assessment of new drugs. Highly innovative drugs, or drugs registered with limited clinical data, were not more likely to be subject to serious post-approval safety issues. We concluded that accepting a greater uncertainty in the knowledge on risks at the time of approval, appears acceptable for drugs addressing unmet medical need. When comparing the preferences of regulators to those of patients with type 2 diabetes and their doctors regarding drug effects, no significant differences appeared. We concluded that regulators value drug effects of oral anti-diabetes drugs similar to doctors and patients when a trade-off between benefits and risks is made from a patient perspective.