Optimization Models for Faster and Affordable Access to Rare Disease Therapeutics


There are 30 million patients who suffer from rare diseases in Europe. Medical drugs used in the treatment of these rare diseases are called orphan drugs. There are more than 7000 known rare diseases but treatments are available only for 300 of them. Improving the accessibility and affordability of these treatments is an important societal concern.

To address this concern, the European Medicines Agency introduced the expedited R&D program. The expedited R&D allows an orphan drug to enter the market while clinical trials are in-progress. The expedited R&D represents a new business environment that has not been analytically modelled and analysed from the joint perspective of pharmaceutical manufacturers and policy makers (i.e., government agencies). There is a strong need for optimizing the expedited R&D: Most pharmaceutical companies put significant effort on the underlying know-how of these drugs but ignore their unique manufacturing and supply chain dynamics. Government agencies call for a better understanding of the manufacturing and supply chain challenges to be able to provide the right incentives for innovation.

To address this societal problem, the proposed research will develop timely and effective optimization models to provide insights for pharmaceutical manufacturers and policy makers. For manufacturers, analytical models will be developed to optimize manufacturing capacities and timelines under failure risks, limited information on future clinical trials, and expensive resources. For policy makers, new rules and strategies will be developed to strike a balance between expediting R&D timelines and assuring the safety and quality of the drugs. The research outcomes will stimulate innovation, reduce costs, and lower lead times for orphan drugs.

A research foundation will be developed combining stochastic optimization, manufacturing systems engineering, and game theory. New knowledge obtained from this research will find future applications in other emerging fields such as nanotechnology and biotechnology.





Dr. T.G. Martagan

Verbonden aan

Technische Universiteit Eindhoven, Faculteit Industrial Engineering & Innovation Sciences, Operations, Planning, Accounting, and Control (OPAC)


Dr. T.G. Martagan


20/01/2018 tot 02/10/2021