1. Eastman, Peggy

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The Personalized Medicine Coalition (PMC) released a new white paper on how integrated health data can be used to its best advantage in helping patients with cancer and other serious diseases. The report, Using Health Data to Advance Personalized Medicine: Challenges and a Path Forward, was developed for the PMC by Innovation Horizons; the issues in it were identified by PMC's Health Data Working Group. The new white paper addresses the following three top priorities:

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1. streamlining the development and implementation of coordinated standards for the collection and interoperability of data from electronic health records (EHRs);


2. ensuring new legislative efforts at both federal and state levels recognize the balance between respecting and protecting the interests of patients, empowering patients with access to their own data, honoring patient choice, fortifying privacy protections, and supporting the public interest while using datasets for research to improve the quality of care; and


3. accelerating the development and adoption of practices and standards for datasets created from real-world data to enable the best use by researchers, clinicians, patients, regulators, and other stakeholders that helps lead to insights for improved decisions, equity, and outcomes in health care.



In addition to providing a comprehensive look at how the use of integrated health data is evolving to move the field of personalized medicine forward, the new PMC white paper provides recommendations on the best ways to realize the promise of integrated health data for patients (see sidebar below).


In a webinar to introduce the new white paper, Edward Abrahams, PhD, President of PMD, said the promise of personalized medicine "rests on our ability to aggregate and analyze data." The research shows that the COVID-19 pandemic focused public attention on the need for "faster health innovation to meet new challenges, better data for public health monitoring, and the urgency of finding treatments that work well for diverse patient populations."


Abrahams emphasized that these pandemic-spotlighted needs are "issues that are not going to go away." He also noted that the white paper addresses obstacles that must be overcome to realize "the extraordinary promise of personalized medicine for patients and health systems."


Personalized (precision) medicine is critically important in oncology. A second PMC report released shortly after the new white paper, the seventh annual edition of PMC's Personalized Medicine at FDA, shows that in 4 of the last 5 years, personalized medicines accounted for more than one-third of newly approved drugs in the U.S.


In oncology, the new report spotlights seven newly approved drugs, two newly approved cell-based therapies, and 20 newly approved indications for existing drugs. These advances will extend personalized medicine to more cancer patients, including those whose tumors harbor KRAS mutations once considered "undruggable." The report on the FDA states that there will be continued progress toward an era of so-called tumor-agnostic treatments, in which treatment decisions are based on key biomarkers, as opposed to where a cancerous tumor is located in the body.


To illustrate the promise of using data to enhance personalized medicine, Gregory Downing, DO, PhD, chief author of the new white paper and founder of Innovation Horizons, presented the case study of a women with an advanced gastric adenoma who had a very poor prognosis. During the webinar, he explained that the patient launched her own hunt to find a customized molecular therapy and is cancer-free today.


Downing, who stressed the need to turn health data into "actionable knowledge," pointed out that almost every day physicians are faced with new protocols and new decision points for patients like the one in his case study. He noted that "the pace of progress today is sometimes just dizzying" and can cause stress, but that dizzying pace also brings new hope to patients.


Agreeing was Michael Vasconcelles, MD, Chief Medical Officer at Flatiron Health and co-chair of the PMC Health Data Working Group. He predicted that in 5 years' time people will look back on 2022 as "a kind of antiquated time" in making the most of integrated health data. Referring to President Biden's newly ignited Cancer Moonshot goal of reducing cancer deaths by 50 percent over the next 25 years, Vasconcelles noted, "I'm a lot more optimistic that we're going to pull it off." He cited the value of the 21st Century Cures Act in enhancing the convergence of clinical care and research.


In the future, personalized medicine should become an expectation for all patients and not just those being treated in major medical centers, said Kris Joshi, PhD, Executive Vice President and President of Network Solutions at Change Healthcare, and a co-chair of the PMC Health Data Working Group. Joshi predicted that in the future consumer data from personal devices will be used more frequently, as will real-world data. In addition, there will be more clinical trials conducted in the community.


The new PMC white paper surveys the mind-boggling, diverse sources of health data today, including EHRs, genome databases, population health, disease registries, wearables/remote monitoring, clinical trials, mobile lifestyle apps, social determinants of health, social media, administrative claims, laboratory tests, pharmacy prescriptions, and behavioral health. There is a need for a learning system that creates a continuous loop of knowledge generation, leading to effective clinical decision-making, states the paper. Experts refer to this as the "fly-wheel effect," whereby the system overcomes its initial inertia to create momentum for knowledge development and acceleration of its application in health care.


While this is the ideal, the new PMC white paper also discusses how the pandemic revealed "shortcomings in public health data information systems," along with public distrust of accurate data and scientific evidence. The white paper states: "There is no doubt from this experience that robust, reliable, and secure data systems are a crucial element to the future of personalized medicine and vital to our future national security."


The research cites a number of federal laws enacted to overcome barriers to the effective access to and use of data. It also probes in detail ways that EHR data could be used more effectively to enhance personalized medicine, while managing the "documentation burden on clinicians." The paper states that EHR could complement and augment clinical trial data, and contribute to "more timely and robust research."


Peggy Eastman is a contributing writer.


Personalized Medicine Recommendations

The Personalized Medicine Coalition report, Using Health Data to Advance Personalized Medicine: Challenges and a Path Forward, offers the following recommendations to use integrated health data to enhance personalized medicine.


* Support the concepts and patient-centered priorities that are the cornerstone of the Cures 2.0 Act. As previously reported by Oncology Times, this legislation-a successor to the 21st Century Cures Act-contains provisions to speed the delivery of new and potentially life-saving therapies to patients.


* Support policies that expand the United States Core Data for Interoperability initiative of the Office of the National Coordinator for Health Information to align data elements and standardize digital exchanges across the U.S. information technology systems.


* Encourage the National Institutes of Health to deploy resources for research and training to create an initiative in artificial intelligence and machine learning, and develop clear guidance for avoiding bias in developing and using these tools.


* Encourage federal agencies, health care delivery systems, community public health agencies, and telecommunications companies to work together to enable interoperability and promote 5G broadband communications.


* Advocate for the development of ethical and practical integrated data systems that will improve access to personalized medicine.


* Advocate for Congress to develop policies that ensure privacy of consumer health information across all data collection sources.


* Charge federal agencies to work with the Federal Trade Commission, Congress, and the White House to create a new regulatory framework for protecting non-HIPAA covered health data sources.


* Develop a federal clearinghouse on state, federal, and international consumer health privacy protections.


* Encourage the Department of Health and Human Services and other health-related federal agencies to expand policy research on the ethical, legal, and social aspects of the use of non-clinical data for health care research and practice improvements.


* Support research into the application of real-world data in clinical trials, and support the expansion of translational research programs and data standards development that will expedite the integration of real-world data with clinical and biological data to enhance individualized approaches to therapy.


* Encourage payers of health care services to adopt payment practices to enhance the use of decision support tools that will accelerate the adoption of data-driven personalized medicine.


* Encourage FDA and other regulatory agencies to help prioritize the field of data science in real-world data. The FDA has taken a lead role in helping to guide the use of real-world data in scientific studies and defining its use in regulatory approval. The recent development of an FDA Digital Health Center of Excellence should heighten the importance of regulatory underpinnings for automated technologies to guide clinicians' decision-making and aid personalized medicine.