Scientific dialogue and stakeholder interaction
Studies on marketing authorization of innovative drugs and scientific advice in the European regulatory system
This thesis demonstrates the import of development plans on marketing authorization of innovative (orphan) drugs in the EU and underlines the significance of scientific advice. The current regulatory system for marketing authorization of medicinal products has been criticized for its demanding evidence requirements and high non-approval rates. Insight in the system is needed to optimize its efficiency.
We conducted analyses of new (orphan) drug submissions to identify determinants of marketing approval and studied scientific advice. While clinical outcomes seem to contribute most significantly to current non-approval rates, the quality of drug development plans is also associated with marketing approval of both orphan and non-orphan drugs. This is especially so for exploratory studies, even though scientific advice questions were most often asked about Phase III studies. Pharmaceutical developers are encouraged to invest in the design and conduct of comparative Phase III studies for orphan and non-orphan drugs, and especially of exploratory Phase I and II studies. Scientific advice should be aimed at discussing drug development plans in a continuous and prospective way.
Results
- Putzeist M, Heemstra HE, Garcia JL, Mantel-Teeuwisse AK, Gispen-De Wied CC, Hoes AW, et al. Determinants for successful marketing authorisation of orphan medicinal products in the EU. In: Medicine Discov Today, 2012; 17(7-8): 352-8. (journal publication)
- Putzeist M, Mantel-Teeuwisse AK, Aronsson B, Rowland M, Gispen-de Wied CC, Vamvakas S, Hoes AW, Leufkens HG, Eichler HG. Factors influencing non-approval of new medicines in Europe. In: Nat Rev Medicine Discov, 2012; 11(12): 903-4. (journal publication)
- Putzeist M, Mantel-Teeuwisse AK, Gispen-De Wied CC, Hoes AW, Leufkens HG. Regulatory scientific advice in medicine development: does company size make a difference? In: Eur J Clin Pharmacol, 2011; 67(2): 157-64. (journal publication)