Set of Papers in Value In Health Address Methods for Moving Evaluation of Precision Medicine into Practice and Policy

A key challenge for the appropriate implementation of precision medicine is the need to evaluate its costs, benefits, and risks. However, several issues arise when measuring the value of precision medicine, particularly new genomic tests that measure multiple rather than single genes, often using next-generation sequencing (NGS) technologies. In the last few years, there has been an emerging consensus that conventional methods to assess utility and value are not well suited for evaluating the value of genomic testing.

To address these issues, we published six papers in a special theme section in Value in Health specifically focused on evaluation methods for moving precision medicine into practice and policy. This special theme section features five papers from the Global Economics and Evaluation of Clinical Genomics Sequencing Working Group (GEECS) and one paper by the ISPOR Special Interest Group on Precision Medicine and Advanced Therapies. The work was chaired by Kathryn A. Phillips, PhD, and her introduction lays out the context and findings of the papers.

The papers form a common theme of describing methods of evaluation of sequencing technologies using different approaches and perspectives to facilitate the adoption of the technology when appropriate. They incorporate a wide range of perspectives and topics and use simulation models, medical record reviews, systematic reviews, interviews, payer coverage policy analysis, and case examples. The findings and recommendations are intended not only for health economic researchers but also for other stakeholders including health technology assessment organizations, payers, clinical researchers, and the biotechnology and pharmaceutical industries. Key takeaways from these articles are:

• Much progress has been made in developing and applying methods to evaluate precision medicine. However, new tests such as minimally invasive liquid biopsies and emerging approaches such as artificial intelligence and machine learning platforms will continue to require the development and adaptation of methods used to assess the value of precision medicine, which are discussed in this set of papers. (Phillips) 
• It is important to define the core factors that should be considered in a value assessment framework for precision medicine with multiple perspectives obtained by building consensus among stakeholders for robust procedures and measures of value aspects. (Faulkner et al) 
• Improving the use of Real World Evidence for payer decision-making involves multiple strategies to overcome the data, methodologic and policy challenges, which can be advanced by coordinating with stakeholders to develop robust RWE that can be incorporated into assessments of NGS value. (Deverka et al)
• The use of temporal analyses to assess payer coverage policy for Next-Generation Tumor Sequencing (NGTS) by private U.S. payers reveals important trends in coverage decision and informs economic and affordability assessments. (Trosman et al)
• Different methods for identifying healthcare utilization attributable to unanticipated genomic results from newborn genomic sequencing are needed to assess follow-up care (MacKay et al)  
• The use of simulation modeling to provide potential solutions and approaches to dealing with complexities of the economic evaluation of precision medicine. (Marshall et al)

About Global Economics and Evaluation of Clinical Genomics Sequencing Working Group (GEECS)

The Global Economics and Evaluation of Clinical Genomics Sequencing Working Group (GEECS) is made up of an esteemed group of health economists and policy researchers from major institutions across the globe who have been at the forefront of the incorporation of genomics into clinical care. The working group is chaired by Kathryn A. Phillips, PhD, Director of the Center for Translational and Policy Research on Personalized Medicine (TRANSPERS) at UCSF. Members include:

• James Buchanan, DPhil, University of Oxford, Oxford, United Kingdom
• Kurt Christensen, PhD, Brigham and Women’s Hospital and Harvard Medical School, Boston, MA, USA
• Patricia Deverka, MD, MS, Innovation & Value Initiative, Alexandria, VA, USA
• Deborah Marshall, PhD, University of Calgary, Calgary, Alberta, Canada
• Kathryn A. Phillips, PhD, University of California San Francisco, San Francisco, CA, USA
• Dean Regier, PhD, University of British Columbia, Vancouver, British Columbia, Canada
• Sarah Wordsworth, PhD, University of Oxford, Oxford, United Kingdom

For more information or contact Kathryn Phillips.