The Personalized Medicine Coalition (PMC) has released two new reports designed to advance the field of personalized (precision) medicine in 2021 and beyond so that many more patients will benefit from individually targeted therapies based on genomic testing. Both reports emphasize that barriers continue to keep the promise of tailored precision medicine from reaching as many patients in clinical practice as it should.
Barriers may include limited access to predictive biomarker tests, challenges in the interpretation of test results, limited access to targeted therapies, a lack of familiarity with molecular diagnostic testing, and inconsistent reimbursement.
The first report, "Understanding and Delivering the Clinical and Economic Benefits of Personalized Medicine," proposes a comprehensive three-part research program which covers evaluating progress in health care, assessing clinical and economic value, and examining clinical integration strategies. This report contains nine PMC-initiated research projects (some of which are already under way), and calls on "organizations in multiple sectors of the health care ecosystem" to provide support for these new initiatives in PMC's expanding research portfolio. Several of these research studies are specific to oncology.
The second report, "Expanding the Frontiers of Personalized Medicine through Education, Advocacy and Evidence Development: A Strategic Plan for Advancing the Field in 2021," sets forth a blueprint for bringing the promise of personalized medicine to more patients through specific efforts and initiatives.
"Although the number of personalized drugs with biomarker strategies on their label in the United States has grown from five in 2008 to 286 today, complicated policy challenges and clinical adoption obstacles continue to make it difficult for health care systems around the world to capitalize on groundbreaking science and technology that point to a new era in the history of medicine that for the first time promises to put the individual at the center of care," noted PMC President Edward Abrahams, PhD, in an introduction to the new strategic plan. "In fact, amidst the tumult of COVID-19 and a turbulent political landscape, most Americans have never heard the words 'personalized medicine.'"
In their joint introduction to the report on the comprehensive PMC research program, Abrahams and PMC Chairman of the board Jay G. Wohlgemuth, MD, state that, unfortunately, "Personalized medicine-linking therapies to molecular diagnostic tests to ensure that the right treatments are targeted to the right patients-is still more aspiration than reality.
"In brief, we need more evidence that personalized medicine works-that it can improve clinical outcomes while making health care more efficient and therefore less costly," they noted. "We also need to understand the opportunities and challenges shaping the pace of our progress toward integrating personalized medicine into clinical workstreams."
As previously reported by Oncology Times, in 2020 the PMC released a patient-centered research agenda called "Moving Beyond Population Averages: A Patient-Centered Research Agenda Advancing Personalized Medicine." That report defined 45 research questions that need to be addressed across nine areas to expand the field. In the information, oncology is cited as the model for personalized medicine, recognizing the progress that has been made in targeted cancer treatments tailored to individual patients.
But that report, along with a previous PMC summary on genomic testing, also identified barriers to integrating personalized medicine into clinical practice-including a lack of awareness about personalized medicine treatments and strategies.
PMC's new strategic plan contains the following resources:
* a package of programs designed to educate decision makers about the significance of personalized medicine;
* a patient-focused educational campaign about personalized medicine and its benefits;
* a series of advocacy priorities designed to "cultivate a friendlier landscape" for advancing the field; and
* a research program aimed at building the evidence base for the clinical and economic benefits of personalized medicine (set forth specifically in the strategic plan's companion report).
Advocacy Priorities
As a new federal administration and a new Congress take over in Washington, PMC's advocacy priorities include the following:
* Guiding an agenda for the Congressional Personalized Medicine Caucus. This group was launched on Capitol Hill last year, and the PMC plans to play a leading role in its agenda and in growing its membership in 2021.
* Increasing funding for critical federal health agencies. Through Congressional testimonies, public statements, and meetings with members of Congress, PMC plans to advocate for increases in the fiscal year (FY) 2022 budgets of the National Institutes of Health and the FDA. Noting that the FDA is "the gateway for many personalized medicine products entering the market," PMC also will support activities at the FDA to advance personalized medicine in the context of the Prescription Drug and Medical Device User Fee Act reauthorizations.
* Modernizing regulatory policies. PMC will actively support legislation, emerging guidance documents, policies, and initiatives at the FDA, which ensure personalized therapies and products reach patients in a timely and efficient manner.
* Modernizing coverage and payment policies. To ensure that patients have access to personalized therapies, PMC will continue to work with Congress and the Centers for Medicare and Medicaid Services to ensure adequate payment rates and flexible coverage policies for personalized medicine products and therapies.
* Modernizing value assessment methodologies. The PMC notes that, by influencing decisions made by patients, providers, payers, and policymakers, value-based payment frameworks "could accelerate or undermine progress in personalized medicine."
Genomics Research Planned
The PMC strategic plan calls for health technology assessments and value-based reimbursement that fully account for the value of personalized medicine.
Among the studies cited in PMC's new research report is "Defining the Clinical Utility of Genomic Testing in Cancer Care," which is now in progress. The report states, "The consistent use of biomarker testing faces implementation challenges related to insufficient awareness and education; a lack of recognition by payers and providers of the clinical value of testing; and outdated practices, policies, and procedures."
PMC will develop recommendations to broaden the definition of the clinical utility of multiplex genomic testing in patient care. PMC notes that many organizations have emphasized the need to better understand the clinical utility of genomic profiling in cancer care, and "this project is PMC's answer to that call." This study will be done in two phases; Phase I will include a stakeholder roundtable event with discussions and case studies.
A second study cited in the PMC's new research report is "Addressing Practice Gaps in the Implementation of Personalized Medicine in Cancer Care." This study focuses on improving the integration of biomarker testing into clinical cancer care. A previous PMC report found that, although more than 30 percent of patients with non-small cell lung cancer (NSCLC) have tumors linked to specific driver mutations, only 65-75 percent of NSCLC patients with an actionable mutation-as determined by genomic testing-actually receive targeted therapies based upon testing results. And, as cited in the new PMC research report, a recent analysis by the firm Diaceutics, a diagnostics data analytics and implementation services company, estimated that 30 percent of U.S. cancer patients never receive targeted therapies that might benefit them.
This practice gap is due, in part, to "lagging awareness of the rapidly evolving field of personalized medicine," according to the new PMC research report. The PMC study on the practice gap in oncology practice will use the Diaceutics Global Diagnostic Index (GDI) database to examine clinical practice gaps associated specifically with biomarker testing in the care of NSCLC patients. Based on results from studying datasets in the GDI database, which contains approximately 260,000 NSCLC patients, PMC will make recommendations on closing practice gaps in biomarker testing and targeted therapy of NSCLC patients in clinical practice.
Other planned studies cited in the new PMC research report include the following: Evaluating the Clinical and Economic Value of Sequencing-Based Diagnostic Tests for Patients with Rare and Undiagnosed Diseases (in progress), Improvements in Clinical Care Associated with Personalized Medicine, Addressing Disparities and Improving Health Equity in Research Advancing Personalized Medicine, and Addressing Challenges in Data Management to Advance Personalized Medicine
Peggy Eastman is a contributing writer.