Amid Widely Variable Effects of COVID-19, New Developments Point to Maturing Field of Personalized Medicine
by Edward Abrahams
Since its inception in 2004, the Personalized Medicine Coalition has been tracking the evolution of the public policies and clinical integration efforts influencing the pace at which scientific progress in personalized medicine is translated into improved care for patients. As the widely variable effects of the SARS-CoV-2 virus in individual patients now foster an increased appreciation for the immutable principles of human heterogeneity that underpin personalized medicine, recent developments in regulation, reimbursement, and clinical adoption have put advocates for personalized medicine in position to highlight the opportunities and challenges shaping a field that is maturing steadily, if incrementally.
Expanded approvals coming from the U.S. Food and Drug Administration (FDA) point to a growing footprint for personalized medicine in cancer care. The agency has broadened the approved uses for Merck’s Keytruda (pembrolizumab), for example, to include the
treatment of adults and children with cancerous tumors anywhere in the body that are “tumor mutational burden high” (TMB-H), meaning that the tumors’ cells express at least 10 mutated genes.1 The approval has accelerated the trend toward the use of multi-gene biomarkers prevalent across tumor types, nudging the field beyond its early stages when personalized cancer drugs could only be used to target single genes known to drive cancers originating in narrowly specified locations in the body. Despite the challenges of the pandemic, FDA has expanded or approved more than 20 new indications for personalized cancer drugs so far this year, by some counts eclipsing its final total for all of 2019.2
On the reimbursement front, payers have begun to adopt innovative solutions to ensure that patients have access to emerging personalized cancer treatments as well as those developed for patients with rare diseases. In May, the U.S. Centers for Medicare and Medicaid Services (CMS) proposed a permanent mechanism to more appropriately reimburse hospitals for the costs of administering chimeric antigen receptor (CAR) T-cell therapies, which genetically re-engineer a patient’s own immune cells to combat cancer.3 And after years of debate about how to determine a payment rate for Trikafta (elexacaftor/tezacaftor/ivacaftor), a personalized medicine that delivers unprecedented benefits to 90 percent of patients with cystic fibrosis by addressing the root cause of the disease, the United Kingdom and Vertex Pharmaceuticals have signed a reimbursement agreement.4
These emerging coverage and payment policies will likely encourage the continued development of innovative personalized treatments by demonstrating that governments around the world are willing and able to establish creative reimbursement approaches to help pay for them.
Finally, new research has sharpened our understanding of the clinical integration challenges that are inhibiting patient access to the genomic testing necessary to target personalized therapies to the right patients.
In partnership with the Blue Cross Blue Shield Association, Concert Genetics, and Illumina, the Personalized Medicine Coalition recently published a report titled Understanding Genomic Testing Utilization and Coverage in the US.5 Comparing real-world health insurance claims for genomic testing with applicable coverage policies in the United States, the report shows that while wide variation and a lack of clarity in payer coverage policies may present barriers to the utilization of genomic testing, such testing is inconsistently utilized even among states where favorable coverage policies exist (see Table 1). The document emphasizes that to improve utilization rates, proponents of personalized medicine must continue to develop and disseminate evidence supporting the clinical and economic utility of genomic testing. The report’s findings may direct additional attention to the complete range of administrative, educational and technical challenges complicating efforts to integrate genomic testing into clinical work streams, leading to an improved environment for clinical adoption.
The shifting landscape points to an expanded role for personalized medicine — and this point is not lost on key decision-makers.
During a virtual educational briefing6 organized in June by the co-chairs of the Congressional Personalized Medicine Caucus, Eric Swalwell (D-CA) noted that as COVID-19 challenges health systems around the world, “we are seeing that a one-size-fits-all approach is no better for COVID than it is for any other illness.” Sen. Tim Scott (R-SC) agreed.
“Both in our response to COVID-19 and in our continued efforts to facilitate continued innovation across the board, personalized medicine has a vital role to play,” Scott said.
- Melillo, (2020, June 17). “FDA Approves Second Biomarker- Based Indication for Pembrolizumab.” American Journal of Managed Care: Newsroom. Retrieved from https://www.ajmc.com/newsroom/fda-approves-second-biomarkerbased-indication-for-pembrolizumab.
- Ray, (2020, July 21). “FDA Approves Record Number of Precision Oncology Drugs in H1 2020.” GenomeWeb. Retrieved from https://www.precisiononcologynews.com/cancer/fda-approves-record-number-precision-oncology-drugs-h1-2020#. XxmCoPhKiu5.
- Florko, (2020, May 12). “Trump Administration Proposes a Dramatic Payment Boost for CAR-T Treatments Under Medicare.” STAT. Retrieved from https://www.statnews.com/2020/05/12/medicare-car-t-boost/.
- Silverman, (2020, June 30). “After Years of Feuds, Vertex and the U.K. Government Reach a Deal Over Cystic Fibrosis Medicines.” STAT. Retrieved from https://www.statnews.com/pharmalot/2020/06/30/vertex-uk-cystic-fibrosis-drug-prices- cost-effective/.
- Personalized Medicine Coalition/Blue Cross Blue Shield Association/ Concert Genetics/Illumina. (2020, July 28). Understanding Genomic Testing Utilization and Coverage in the US. Retrieved from http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/PMC_Understanding_Genomic_Testing_Utilization_and_Coverage_in_the_US1.pdf.
- Personalized Medicine (2020, June 30). The Personalized Medicine Community’s Response to COVID-19. Retrieved from https://www.youtube.com/watch?v=f9TDRXvogn8&feature=youtu.be
Edward Abrahams is president of the Personalized Medicine Coalition, a nonprofit education and advocacy organization in Washington, DC.