Transport
yourself 10 to 15 years from now and try to imagine what the future of
personalized medicine will look like.
The vision of every drug prescription decisions being driven by a test
aimed at tailoring the treatment to a particular individual is probably
utopian. Rather, I would argue that the
realm of personalized medicine will still be limited to the treatment of severe
and/or life-threatening diseases that require expensive medications. Under this premise, what are the forces that
will shape the future of personalized medicine?
In my
mind, this question can be addressed by considering the field from a supply and
demand perspective. On the supply end, the
pharmaceutical and diagnostics industries will remain the main forces driving the
future of personalized medicine. The imperative
of improving the return on investment in drug development will dominate the
future of the pharmaceutical industry. With
the era of relying mainly on “one-size-fit-all” drugs fading away, the focus will
shift towards precision/personalized medicine where drugs are designed to
address the need of smaller targeted patient populations. Hence, the need to develop the tools that
will identify the right patient population (for efficacy and/or safety reasons)
will constitute a major theme in drug development. This does not exclude the continuing effort
of the pharmaceutical industry to develop and commercialize broadly applicable
drugs for the management and/or treatment of conditions for which a
personalized approach is not warranted (for cost-benefit and/or clinical
utility reasons). Still on the supply
end but with an eye on the demand side, the regulatory authorities will
continue to play a major role in the harmonization of the biomarker and
companion diagnostic development process. Beyond the current regulatory framework
governing the regulatory approval of drugs and companion diagnostics, the
regulators have been working on developing a new process for an integrated
development of biomarkers intended to become companion diagnostics (see earlier
posts: Harmonization
of Biomarker Qualification Regulatory Submissions; Companion
In Vitro Diagnostics (IVD) Development).
Probably
the most significant force that will shape the future of personalized medicine
will be on the demand side, represented by the patients, the medical
practitioners, and most importantly the health insurance/payers. For all three entities, the adoption of a new
companion diagnostic will require proof of clinical utility (Ref1,
Ref2,
& Ref3). In a nutshell, clinical utility for a molecular
diagnostics is the third level of a three-tiered evaluation framework that
includes “analytical validity”, “clinical validity/qualification”, and
“clinical utility” (Ref2).
Hence, clinical utility encompasses the overall
medical impact of a diagnostic. A
diagnostic is considered clinically useful if it provides a real and
substantial advantage to the patients, positively alters the practice of
medicine, and/or improves the cost / benefit equation for a given
treatment. Although clinical utility is
a distinct concept from analytical and clinical validity, it cannot be
established without first establishing the latter. The reciprocal is however not true:
establishing analytical and clinical validity does not imply proof of clinical
utility.
While the
pharmaceutical industry and the regulators are currently focusing most of their
efforts on defining and implementing the rules of diagnostics analytical and
clinical validation, I would argue that the next decade will be dedicated to the
third part of the equation: defining and implementing the rules of diagnostics clinical
utility evaluation.
Thierry
Sornasse for Integrated Biomarker Strategy
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