More research needed to develop cures for rare diseases

The US Food and Drug Administration’s drug approval process is the fastest in the world, which means Americans typically have first access to new drugs when they are demonstrated to be safe and effective. But drug discovery and development is not keeping pace for many diseases, according to Robert Califf, the FDA’s Deputy Commissioner for Medical Products and Tobacco.

In the FDA Voice blog, Dr Califf says that in many cases, what’s holding back progress is a lack of understanding of the biology of disease. He cites an FDA report, Targeted Drug Development: Why Are Many Diseases Lagging Behind?, that compares diseases where there is a robust pipeline of new therapies with certain diseases that have few known treatments or cures. For example, when it comes to cancer, HIV/AIDS, and other viral infections, on-going research has led to the discovery of biomarkers, which are indicators of normal biological processes, pathogenic processes or response to a therapeutic intervention. Some types of biomarkers give insight on the genetic and metabolic characteristics that alter patients’ responsiveness to particular drugs, and others give insight into whether drugs in development are likely to work. This knowledge has resulted in important breakthroughs, rapid drug development and speedy FDA approvals.

More research needed