Role of Disease Progression Models in Drug Development

Pharm Res. 2022 Aug;39(8):1803-1815. doi: 10.1007/s11095-022-03257-3. Epub 2022 Apr 11.


The use of Disease progression models (DPMs) in Drug Development has been widely adopted across therapeutic areas as a method for integrating previously obtained disease knowledge to elucidate the impact of novel therapeutics or vaccines on disease course, thus quantifying the potential clinical benefit at different stages of drug development programs. This paper provides a brief overview of DPMs and the evolution in data types, analytic methods, and applications that have occurred in their use by Quantitive Clinical Pharmacologists. It also provides examples of how these models have informed decisions and clinical trial design across several therapeutic areas and at various stages of development. It briefly describes potential new applications of DPMs utilizing emerging data sources, and utilizing new analytic techniques, and discuss new challenges faced such as requiring description of multiple endpoints, rapid model development, application of machine learning-based analytics, and use of high dimensional and real-world data. Considerations for the continued evolution future of DPMs to serve as community-maintained expert systems are also provided.

Keywords: MIDD; decision-making; disease progression model; drug development.

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic
  • Disease Progression*
  • Drug Development*
  • Humans
  • Research Design