Academicians, payers, and manufacturers alike are struggling to embed the new insights that personalized medicine promises.  But, fundamental issues exist such as:  application in early drug development, implications for health technology assessment, new demands on traditional health economic and outcomes research methods, and implications for reimbursement and access.  Inevitably, practitioners are increasingly compelled to find practical solutions to the challenges and opportunities presented by the evolving face of personalized medicine today.

Accordingly, the first paper in the Special Issue seeks to bring together various threads in definition and nomenclature to advance a basic framework with which to support the subsequent discussion, and which thereby may also be of service to the broader practitioner community seeking some level of consensus upon which to engage in increment health systems adaptation.

The Special Issue also addresses emerging methodological issues focused in three broad (sometimes overlapping) areas:  applications in comparative effectiveness research (CER), drug development, and economic evaluation.  With respect to CER, it becomes clear to even the casual observer that variation in patient population’s impact outcome and thus can have profound implications for evidence generation, its utilization and the overlay of economic considerations.

Personalized medicine has presented a brand new set of challenges and opportunities in the drug development process, and manufacturers have faced a classic ‘institutional lag’ in which standard operating procedures and organizational boundaries are having to give way to new processes and procedures through with to identify, understand and embed personalized medicine insights.

In a similar fashion, personalized medicine offers the promise, as evinced in emerging economic evaluations studies, to point the way to greater efficiency and effectiveness in healthcare.  A number of leading thinkers in this area, at both the ‘macro’ and ‘micro’ economic policy levels (some of whom have joined this Special Issue), have showcased how personalized medicine can help target appropriate patients, but more important, how this can yield positive net benefit to manufacturers, payers and governments as a whole.

In the US payer environment, arguably the most active current reimbursement space for personalized medicine, emerging issues and use cases abound and the author felt it important to illuminate and contrast the way in which US payers have opportunistically at times embraced personalized medicine approaches, against current regulatory barriers and practice inertial.

With these key issues helping to characterize the current ‘state of the state’, we conclude this Special Issue with a look ahead at emerging trends and the need for future research.  A broad set of opportunities are addressed, with emphasis on evidentiary needs ex ante and ex post stratification, regulatory gaps and evidence of clinical utility in actual practice.

The following papers comprising this Special Issue offer applied perspectives targeted to professionals and policy makers confronted with developing, assessing, implementing and reimbursing personalized medicine approaches.

Click on the title of each article to access the full text. You may also access this issue at: using your member ID.


ISPOR Tools for Health Care Decision-makers