5 Concluding Remarks
Ma and Lu ( 2011 ) suggest that the goal of personalized medicine is for physicians
to prescribe an appropriate medication to the right target of the disease at the right
dose for individual patients to achieve maximal therapeutic benefit with minimal,
tolerable adverse effects. The question we posed in this chapter is how well the
conventional methodology of economic evaluations responds to the rising chal-
lenge of personalized medicine. From the overview of the literature, we found that
the methodology cannot fully address the new requirements and the new paradigm,
although given the rapid growth in the area of personalized medicine, the methods
of economic evaluations will need to adjust to particular features of personalized
medicine products at a quicker pace. Institutions recognize that the regulatory
framework (of which economic evaluations are a part of, at least in many jurisdic-
tions in the EU) will need to help personalized medicine develop further and
accelerate the translation of research into medical products and treatments.
In this chapter, we touched upon some of the issue facing economic evaluation
and outcomes research in the context of personalized medicine and provided an
overview of the literature on the subject. It is clear that conducting economic
evaluations of personalized medicine products poses a challenge, for different
reasons. Some of the challenges discussed in this chapter are, however, not unique
to personalized medicine. Some challenges may be resolved within health econom-
ics, and some will likely require a broader interdisciplinary effort. It seems that a
supportive health care policy will be needed to address each of these issues as a
necessary step in defining how to allocate resources as to steer the development of
personalized medicine in the desired direction. There are many pending questions
that await resolution, although the current literature is expanding on proposals that
sponsors could use to deal with different challenges and provide strategies to help
guide the future codevelopment of drugs and diagnostics.^52
One of the emerging issues is certainly the lack of exploration regarding the
value of personalized medicine, which—given its particularities—lags behind the
developments in the value-based analysis of pharmaceuticals. To assess the value of
personalized medicine, we may need to move away from the current cost-based
valuation of tests and further the debate about how to best assess their value. Likely,
broader measures of health will be needed to better accommodate the type of
benefits that personalized medicine generates. In that sense, HTA and its essential
element—economic evaluation—may be the appropriate framework and strategy
for obtaining the value-for-money estimates because they assess not only the price
of a test but also the benefits it produces and places the two factors in adequate
comparisons.
This chapter also addressed the issue of the available evidence on clinical utility
of companion diagnostics, which is a vital aspect of understanding its added value.
Unlike the test-drug codevelopments, for stand-alone diagnostics, the link between
(^52) As discussed, for instance, by Fridlyand et al. ( 2013 ).
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