'Take-home' messages

  • Biomarkers are increasingly used to streamline medicines development, and help tailor treatments for individual patients.
  • In medicines development, biomarkers are commonly used in early development studies (Phase I), proof of concept studies, and molecular profiling, and sometimes as surrogate endpoints.
  • As new method, the use of biomarkers in medicines development faces technical, regulatory and ethical challenges.
  • Companion Diagnostics.
Further reading (optional)

If you like to learn more about this topic, we recommend having a look at the the following resources (with the link):

Bai JPF, Barrett JS, Burckart GJ, Meibohm B, Sachs HC, Yao L. Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants. The AAPS Journal, 2013; 15(2):447-454.

Tsigkos S, Llinares J, Mariz S, Aarum S, Fregonese L, Dembowska-Baginska B et al. Use of biomarkers in the context of orphan medicines designation in the European Union. Orphanet Journal of Rare Diseases, 2014; 9:13. (open access article)

Evers M, Kulkarni S, Ma P, Moller M, Ostojic I. Managing for success in biomarker R&D: Challenges and opportunities. In Personalized Medicine The Path Forward. McKinsey and Company. 2013.

Industry Pharmacogenomics Working Group (I-PWG). Understanding the Intent, Scope, and Public Health Benefits of Exploratory Biomarker Research.  A Guide for IRBs/IECs and Investigational Site Staff.

Papp et al. Anti-IL-17 Receptor Antibody AMG827 Leads to Rapid Clinical Response in Subjects with Moderate to Severe Psoriasis: Results from a Phase I, Randomized, Placebo-Controlled Trial. J Inv Derm 2012 132, 2466–2469.


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