In order to bring the voice of the patient into future research on personalized (precision) medicine, the Personalized Medicine Coalition (PMC) has released a new report, "Moving Beyond Population Averages: A Patient-Centered Research Agenda Advancing Personalized Medicine." The report is the result of 2 years of work with more than 120 contributors composed primarily of patients and including caregivers, patient advocacy communities, health care professionals, and researchers.
Overall, the report concludes that "PMC's interactions with patients over the past 2 years revealed that many patients and caregivers simply do not know what to ask, when, and to whom during the diagnosis and treatment decision-making process in order to receive personalized medicine." The report also concludes that "the health care community must shift away from a one-size-fits-all paradigm to a system accounting for a patient's unique needs and priorities."
Research Elements
The new PMC report defines 45 research questions that need to be addressed across nine areas that patients themselves defined as important: patient-provider communication; patient education; caregiver, pediatric, and family considerations; provider education, resources, and collaboration; access, affordability, and utilization; coverage and reimbursement; clinical trials; new technologies and data management; and outcomes research.
In this report, oncology is cited as the model for personalized medicine, recognizing the progress that has been made in targeted cancer treatments tailored to individual patients. The report states that the patient-centered research agenda for the future "should consider how to use oncology as a prototype for patient and provider education in disease areas beyond oncology."
Cancers addressed in the report's writing include breast, lung, multiple myeloma, colorectal, lymphoma, and rare blood cancers, along with genetic risk for cancer. Other diseases considered in setting the patient-centered research agenda include Alzheimer's disease, COPD, cystic fibrosis, food allergy, HIV, kidney disease, and rare diseases.
PMC President Edward Abrahams, PhD, said that studying the 45 research questions contained in the report "will bring us one step closer to an era of personalized medicine by revealing how health care decision-makers can work even more closely with patients to develop prevention and treatment strategies that are tailored to patients' biological characteristics, preferences, values, and circumstances." The report was funded through an award from the Patient-Centered Outcomes Research Institute.
"This research agenda will go a long way toward putting patients with lung cancers and other devastating diseases at the center of efforts to advance personalized medicine-which is exactly where they belong," said Bonnie J. Addario, Chairman of the Board and Co-Founder of the GO2 Foundation for Lung Cancer and a PMC board member.
Improving the Patient Experience
In a roundtable on improving the patient experience with personalized medicine, patients shared their experiences with stage IV breast cancer, cystic fibrosis, a rare genetic epilepsy, caregiving, and health disparities. A common thread was that, although most patients are treated in community settings, in some cases personalized medicine may only be available in academic settings. As a result, community-based physicians may not have conversations with patients about personalized medicine "due to their lack of awareness about personalized medicine treatments and strategies." Even in academic settings, physicians may not be fully educated about personalized medicine options, the new PMC report notes.
So, roundtable participants concluded that "the potential benefits of a personalized medicine approach may not be realized unless the patient is aware that this option is available and is fortunate enough to have a caregiver and provider who will advocate for them," in the words of the report. But, it states, "Good fortune, however, should not play a central role in one's ability to access personalized medicine. Broad public educational programs to increase health literacy around personalized medicine, as well as provider-focused education programs, can help take luck out of the equation."
The report notes that educational materials on personalized medicine should reach non-physician specialists, patient ambassadors, peer educators, and patient navigators, as well as patients and physicians, and that telemedicine can play a role in such education. The report stresses the need for multidisciplinary teams to ensure comprehensive care, support, and education for patients who have personalized medicine treatments, especially those with genetic mutations.
Barriers to Personalized Care
In a roundtable session on improving the delivery of personalized medicine in the clinic, participants noted several barriers. The first barrier is the uncertainty of some genetic testing-assuming it is available to the patient.
The report notes that "many new targeted agents are very specific to small populations of people with a specific disease." Consequently, patients need to be divided into small subgroups through genetic testing. But, the PMC report states, "some genetic testing results are indeterminate, in that while they identify a probability of a therapy being effective for a patient, the clinical implication of that test result remains uncertain." Without effective counseling, some patients may mistake a test for genetic risk with a test for a treatment option.
The second identified barrier, as stated in the new report, is "the lack of receptivity among some health care professionals to listen carefully to patients and consider, for example, what the patient has experienced as helpful in treating their condition. These challenges make the dialogue between provider and patient both increasingly important but also increasingly fraught."
The third identified barrier to using personalized medicine in the clinic concerns reimbursement for genetic testing. The new report states, "While genetic testing done in a research setting may not require reimbursement by health insurance, once the test becomes available in clinical settings, issues arise not only in navigating criteria for reimbursement, but in selecting one from a number of competing tests available from different laboratories and/or companies."
The report notes that strategies to reduce the costs of genetic testing are advisable: "For example, a test that assesses multiple genes may provide much more useful information at a cost that is the same as one that tests for a single gene."
A previous report from the PMC, "Understanding Genomic Testing and Coverage in the U.S.," found that genomic testing is inconsistent even among states with favorable coverage policies. That report found that payer genomic testing coverage policies vary considerably among states, favorable coverage policies do not always correlate with higher use across states, and inconsistent coverage and reimbursement policies remain barriers to genomic testing access, but do not entirely explain the inconsistent use of such testing.
Abrahams commented on the findings of this previous PMC report that the United States needs to address "a wide range of clinical adoption issues," since genomic tests are the essential underpinning of personalized medicine.
These findings are similar to those of other organizations. As previously reported in Oncology Times, the American Cancer Society Cancer Action Network found that many cancer patients are not getting the guideline-indicated biomarker testing of their tumors that is integral to identifying those who may benefit from targeted, personalized medicine (2020; doi: 10.1097/01.COT.0000697464.89061.36).
A previous PMC report, "The Evolution of Biomarker Use in Clinical Trials for Cancer Treatments: Key Findings and Implications," underlines the importance of aligning patients, policymakers, and representatives from all sectors of the health care ecosystem on reimbursement policies that will ensure sustainable access to biomarker-based cancer therapies.
Future Research Needed
Among the 45 research questions in the new PMC report suggested to inform a future patient-centered research agenda for personalized medicine are important topics relating to clinical trials, including the following:
* What changes are needed in the design of clinical trials (including outreach, recruitment, and funding) to ensure representation of all populations affected by a disease, including minority populations and women?
* How can health care providers build trust with patients/caregivers and patient communities from diverse backgrounds to address their concerns and fear of mistreatment, including misuse of their health information, when deciding on personalized medicine treatment options, including whether or not to enroll in a clinical trial?
There are also a number of patient-centered research questions in the report on coverage and reimbursement, especially as they relate to genomic tests. These include the following:
* How can personalized medicine help employers and payers design benefit structures or create utilization management strategies to allocate resources that are more streamlined, patient appropriate, and based more on patients' heterogeneity?
* How can the use of billing codes and other payment incentives, including those in the Medicare and Medicaid programs, be improved to encourage additional education and training of patients, caregivers, and family members about genetic testing, the meaning of the results, and the relevance of potential treatment options based on that information?
* How would coverage and reimbursement for genetic consultations with trained genetic counselors and/or pharmacists about personalized treatment options and strategies improve the utilization of personalized medicine?
* What clinical utility studies, cost-effectiveness studies, and budget impact studies are needed to inform payers about the value of covering the cost of a genetic test or genomic panels?
* What data are needed to establish consistent reimbursement policies for genetic testing and to guide the selection of the most appropriate genetic test for a patient?
* What evidence is needed, and where is there already adequate evidence, to support payers and other members of the health care community in collaborating to develop value-based contracts for personalized treatments that may have higher up-front costs?
* How can patient/caregiver perspectives, including their preferences regarding personalized medicine treatment options, decision-making criteria, and successful adherence to treatment, factor into the "value" defined in value-based contracts?
Peggy Eastman is a contributing writer.