Cultivating Innovation in Precision Medicine Through Regulatory Flexibility at the FDA
Medical researchers perpetually aspire to a future where an individual’s genes will chart the way for the identification and prescription of the right lifesaving drug. The drug will be tailored to a patient’s specific genetic profile, assuring that the drug will work safely and effectively for that patient. During the course of care, a physician will extract a biological sample from the patient, run it through a diagnostic test, and the test results, along with the patient’s medical history, will guide the medical decision on which drug to prescribe. Such an integrative process eliminates the potential for that patient to experience devastating adverse drug events because the drug is not safe or because the treatment is ineffective (and assuredly expensive). Such a future provides benefits not only to the patient but also to countless entities and actors within the public health and medical enterprise.
This scenario may be coming closer to fruition as research initiatives and public support for the field of precision medicine gain momentum. The National Institutes of Health (NIH) describes precision medicine as "an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person."1 President Obama initially channeled $215 million into the Precision Medicine Initiative (PMI), with funds to the NIH and the Food and Drug Administration (FDA) “to accelerate biomedical discoveries and provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patients.”2 In remarks at the White House East Room, Obama touted precision medicine as "delivering the right treatments at the right time, every time to the right person.”3 The 21st Century Cures Act, signed into law on December 13, 2016, further supports the PMI and related research with a $4.8 billion pledge over the next ten years.4
