intend to engage in the debate about the definition or the boundaries of the term
“personalized medicine”; here we focus on a particular way of “personalizing”
medicine. We adhere to personalized medicine as an approach that uses molecular
and companion diagnostic technologies to look at genetic profiles and biomarkers
of diseases to match each patient with the treatment that will provide her with the
best health outcome, i.e., the “biomarker-based stratification,” as referred to by
Towse and Garrison ( 2013 ). Similarly, the Personalized Medicine Special Interest
Group of the Institutional Council of the International Society for
Pharmacoeconomics and Outcomes Research (ISPOR) defines personalized medi-
cine as the use of genetic or other biomarker information to improve the safety,
effectiveness, and health outcomes of patients via more efficiently targeted risk
stratification, prevention, and tailored treatment and treatment-management
approaches.^6 This emphasis on the genomic tests and genetic profile of diseases,
with a heavy focus on companion diagnostics, is one of the key factors for the new
personalized approach in science and medical care. According to the US Food and
Drugs Administration (FDA),^7 “a companion diagnostic is an in vitro diagnostic or
an imaging tool that provides information that is essential for the safe and effective
use of a corresponding therapeutic product.” Using the information on genotypes
obtained from companion diagnostic technology enables physicians to administer
(newer, more expensive) drugs only to patients who are most likely to benefit from
them while withholding the (expensive) treatments from patients who are not likely
to benefit but who may—instead—experience harmful side effects. The question
this chapter poses is how well the conventional methodology of economic evalu-
ations and related outcomes research responds to the challenge of personalized
medicine, especially to the challenge of evaluating new diagnostic technologies
(i.e., companion diagnostics) instead of, or alongside, pharmaceuticals. This is a
timely debate since the field is relatively rapidly developing and the new insights of
personalized medicine do not yet seem well embedded into the current health care
systems—in fact, scientists, payers, and manufacturers are currently debating on
some of the fundamental issues related to personalized medicine.^8 Some of the
emerging issues relate to the process of HTA, the new demands personalized
products may have on traditional health economic and outcomes research methods,
and the implications of personalizing medicine for reimbursement decisions and
market access. The question of conducting economic evaluations in the area of
increasingly personalized technologies is very important at this relatively early
stage of technology development because the potential benefits of personalized
(^6) Examples of the debates revolving around the definition of personalized medicine available in
Faulkner et al. ( 2012 ), pp. 1162–1171.
(^7) The definition available at FDA website, http://www.fda.gov/MedicalDevices/
ProductsandMedicalProcedures/InVitroDiagnostics/ucm301431.htm.
(^8) For further insight into the debate, see, for instance, US Food and Drugs Administration (FDA),
2014 Policy paper titled “Paving the Way for Personalized Medicine – FDA’s Role in a New Era of
Medical Product Development,” available athttp://www.fda.gov/downloads/ScienceResearch/
SpecialTopics/PersonalizedMedicine/UCM372421.pdf.
110 A. Bobinac and M. Vehovec