Other Regulatory Issues

Companion Diagnostics

The term “companion diagnostic” was defined in a guidance document issued by the FDA in 2014 which stated that a companion diagnostic assay is an in vitro diagnostic (IVD) device that provides information that is essential for the safe and effective use of a corresponding therapeutic product.  Regulatory agencies are continuing to assess the challenges associated with co-development and co-approval of a therapeutic product and its companion diagnostic (CDx).

Clinical Trials

New clinical trial designs are also emerging that may be more compatible with genomic testing and precision medicine including basket, umbrella and adaptive trials (Love-Koh et al. 2018, Renfro et al. 2017). These are particularly being used in cancer studies where there is greater understanding of biomarker and genetically defined cancer sub populations. 

There are also regulatory considerations to acknowledge the difficulties of developing medicines for rare genetic diseases. As an example, in 2017 the European Medicines Agency and the Food and Drug Administration released a joint proposal entitled "Paediatric Gaucher disease - A strategic collaborative approach from EMA and FDA". This is a document focused on drug development for Gaucher disease, but its underlying principles may be extended to other areas of drug development in rare diseases. The approach recognises that rare diseases typically have only a limited number of patients available to take part in trials. Therefore, the proposal deals with general considerations for study population, and practicalities in the design and execution of pediatric trials of drugs for rare diseases, and the use of extrapolation of efficacy data for these diseases.

Privacy and sharing health and genomic data