by Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, PMC

The Personalized Medicine Coalition’s most recent white paper, Personalized Medicine and Value Assessment Frameworks: Context, Considerations, and Next Steps, contends that to improve clinical outcomes and facilitate more cost-effective health care, value assessment frameworks (VAFs) must incorporate the principles of personalized medicine. The report examines the characteristics of U.S.-centered VAFs and identifies how each integrates or, as in most cases, fails to integrate personalized medicine.

Health insurance companies have been conducting health technology assessments for years. As part of these processes they may or may not have considered value assessment reports from independent organizations.  Recent indications from both public and private payers, however, have suggested a greater interest in the use of VAFs for coverage and payment decision-making.  Health care delivery organizations and physician groups have also taken a greater interest in using frameworks to help quantify the value of various treatments and interventions. This has increased the likelihood that VAFs will impact access to care.

Several frameworks are already in use by different end-users. ICER’s framework, the Drug Abacus developed by Memorial Sloan Kettering, and frameworks developed by the American Society for Clinical Oncology (ASCO) and the National Comprehensive Cancer Network focus primarily on value to payers and providers at a health system level, while newer VAFs, including FasterCures’ Patient Perspective Value Framework and the Innovation and Value Initiative, were developed with patients in mind.

To facilitate access to treatment options of highest value at both the individual patient and health care system levels, however, VAFs must consider all of these perspectives. PMC’s report argues that to assess the value of new treatments in a way that meets the needs of patients and innovators as well as payers and providers, VAFs must integrate personalized medicine.

By identifying which medical treatments and procedures will work best for each patient, personalized medicine improves efficiency at both the individual and health system levels. But as they are currently designed, most VAFs involve static comparisons based on population health, and therefore neglect to adequately consider patient-level efficiencies learned through ongoing clinical practice. PMC’s report highlights five personalized medicine considerations — namely diagnostic testing, heterogeneity of treatment effects, treatment efficiency, individual values and circumstances, and emerging or evolving value elements, which, if accounted for in VAF methodologies, would help ensure that value is considered at both the health system and patient levels.

In a panel discussion to launch the report, Kristen Migliaccio-Walle, who was the report’s lead author from Xcenda, Dan Leonard, President of the National Pharmaceutical Council, Alan Balch, CEO of the National Patient Advocate Foundation, and Dana Wollins, Director of Health Policy at ASCO, agreed that alignment of VAFs with the goals and benefits of personalized medicine is critical to ensure informed decision making.

Other VAF developers concur. ICER’s President, Steven Pearson, for example, recently noted in an email to PMC that he is “convinced that we share the same vision: a health care system in which insurers and drug makers can continue the progress toward even more [personalized] tests and treatments while making them more affordable and more accessible for the patients who need them.”

The report concludes with five strategic recommendations for developers of VAFs:

1. Consider diagnostic testing as an explicit and integral part of the value assessment of treatment options where efficacy and/or safety information can be obtained;
2. Include a formal mechanism for consideration of heterogeneity of treatment response appropriately balanced with population-based considerations;
3. Develop methods for the consideration of emerging or evolving elements of value over time to fully account for emergent benefits at the health system and individual patient levels;
4. Ensure appropriate awareness and education about the use of value assessment frameworks in personalized medicine to reduce the risk of inappropriate restriction of reimbursement and/or access to individualized care; and
5. Consider the perspectives of all stakeholders, especially patients.

As VAFs have gained prominence and become a focus of discussion by decision-makers within the health care community, it has become more important than ever to realize that patient-level efficiencies convey real value at the health system level. Currently, the value assessment viewpoints of payers and providers are often not aligned with those of patients. Following the recommendations in PMC’s report will help bridge that gap.
Personalized Medicine and Value Assessment Frameworks: Context, Considerations, and Next Steps