Over the last decade, the development of blockbuster drugs has given way to the development of personalized medicine—an efficient, effective way of using specific therapies in a targeted patient population while avoiding toxicity in a broader group of patients who may not be ideal candidates.
The process of genetic testing grew out of a shortcut taken in 1950 when Peter Norell broke protocol by washing a white blood cell sample in Philadelphia tap water, resulting in enlarged chromosomes. His discovery, the Philadelphia chromosome, has expanded into modern genetic testing, biomarker analysis, and companion diagnostics. The goal of companion diagnostics is to provide essential information for the safe and effective use of a corresponding treatment.
Personalized medicine is good for patients because they can be treated precisely. Good for doctors because they can choose a therapy with a strong possibility for success. Good for payors because outcome measures have been established and a therapy is given only to those who will benefit. Good for pharmaceutical and diagnostic companies because targeted therapies, although they are sold to a targeted population, yield high profits.
The science is there. The technology is there. Personalized medicine using companion diagnostics is the clear combination of these two and has proved successful, with several agents already on the market. The means by which to achieve this companionship, however, is anything but clear.
Companion diagnostics or pharmacodiagnostics or theranostics or…
To use personalized medicine to its fullest, there is a need for companion diagnostics to be developed in tandem with biologics to identify the patients who will benefit most. Much like the lexicon surrounding these diagnostic tools (let’s refer to them as companion diagnostics here), there is the potential for misalignment throughout the development process.
Stakeholders might include pharmaceutical companies, companion diagnostic manufacturers, patients, physicians, payors, pathologists, and the FDA. Development of a companion diagnostic may not begin until Phase II of drug development. And, what happens when both the diagnostic and the biologic are not approved? Or when they are approved at different times? How to get them to market at the same time? And what of the different approval processes for diagnostics vs biologics? Licensing? Decision-making? Clinical trials?
Roll over Beethoven
The alignment of multiple stakeholders who have varying priorities and timelines represents a sea change in the drug discovery and development process. Instead of pharmaceutical companies leading the way, there is a need for early and clear communication among all stakeholders.
Once we establish a clear path forward, personalized medicine using companion diagnostics will alter the treatment paradigm.