Clinical trials

Clinical trials – lessons learned from neglected diseases’ drugs development

The level of investments for research and development of new medicines and their consequential costs have reached a level posing threats to the public health system. Today, the development costs for a new drug are estimated between CHF 1 and 2.5 billion. The development process is very lengthy and intricate, from molecular research, pre-clinical tests in vitro and in animals to three phases of clinical testing.

Among the reasons for the raising costs the increasing complexity of trial protocols, number of amendments and the significant attrition rate during the development process (over 90%) are mentioned. Clinical trials and drug development are also subject to extensive regulations. Managing clinical trials operations and quality assurance has consequently become a major task. The Swiss TPH Clinical Operations Unit has supported clinical trials conducted by pharmaceutical and biotech companies, not-for profit organizations called Product Development Partnerships (PDPs) and academia for the past twenty years. We have observed the increased complexity of regulations and their implementation, but also hypothesize that the organizational complexity, the risk perception and the resulting internal requirements for quality assurance and documentation, beyond the regulatory requirements, are an additional major cause of the increasing costs, particularly in large-scale companies.

Our teams’ main niche are neglected tropical diseases, where drugs do not have a profitable market and due to limited funding, alternative models of drug development have been successfully established. The PDPs are partnerships, which are usually organized as small think tanks, which perform the actual drug development activities through consortia tailored to their requirements. The development of a drug using such model costs about 20% of the expense incurred by conventional pharmaceutical drug development. Only 10% of research and development funding’s are dedicated to the 90% high burden diseases namely tropical and neglected diseases. Given their success, policymakers have started to discuss whether PDP-style drug development may be more generalizable to drug research, particularly for medicines against rare (orphan) diseases in the North. Lastly, academic research is contributing to knowledge through product optimization and development of new combinations with a strong focus on patient’s needs. Costs for academic clinical trials have also significantly increased but in comparison still conducted on a shoestring and hence this model merits investigation, too.

This sub-project will assess the time and resource efficiency of the conduct of clinical trials in various settings (industry, PDP, academia) through a comparison of clinical trial operations and processes. It is connected to the subproject 1.2, which investigates the history of drug development.