Keys to Unlocking Precision Medicine's Potential: IOM Report

Megan Brooks

March 07, 2016

The field of precision medicine is evolving rapidly, with new molecularly targeted therapies and associated biomarker tests entering the drug development pipeline and being approved for use. But how can healthcare providers, regulators, payers, and test developers ensure that patients have timely access to biomarker tests that can accurately direct targeted treatments, while at the same time protect them from potential harm caused by the use of poorly validated or inappropriate tests?

That question is addressed in a new report from the Institute of Medicine (IOM) titled Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine.

Biomarker tests for targeted therapies are not being adopted appropriately into clinical practice because of a lack of evidence-based standards needed for regulatory, reimbursement, and treatment decisions, the IOM says.

Rapid Learning System

"The timely development of biomarker tests and associated therapies is critical to realizing the full potential of 'precision medicine,'" Harold L. Moses, MD, chair of the committee that wrote the report, said in an IOM news release. "Our report lays out a strategy to ensure that patients have access to effective tests and treatments that are based on solid evidence of their ability to improve health outcomes," added Dr Moses, chair of the department of cancer biology at Vanderbilt University, Nashville, Tennessee.

The report calls for the creation of a "rapid learning" system that would integrate research on biomarker tests and associated treatments with clinical practice. The report outlines 10 goals to advance the development and appropriate clinical use of biomarker tests for molecularly targeted therapies:

1. Establish common evidentiary standards of clinical utility of biomarker tests using evidence generated both within and outside clinical trials, across all stakeholders. "Currently, lack of such standards is a significant limiting factor for patients, health care providers, test developers, regulators, and payers," the report says.

2. Establish a more coordinated and transparent federal process for regulatory and reimbursement decisions for biomarker tests for molecularly targeted therapies. "Processes for making regulatory and reimbursement decisions regarding biomarker tests used in clinical care are misaligned, creating inefficiencies. The Food and Drug Administration and the Centers for Medicare & Medicaid Services (CMS) should work closely together to enable effective coordination of these decision-making processes," the report says.

3. Enhance communication to patients and providers about the performance characteristics and evidence for use of specific biomarker tests for molecularly targeted therapies. "Health care providers and patients both lack adequate information about biomarker tests for molecularly targeted therapies. New patient- and provider-friendly labeling information, including evidence to support test use in specific clinical scenarios, would increase transparency and empower patients and their health care providers to make more informed treatment decisions," the report notes.

4. Update and strengthen oversight and accreditation of laboratories providing biomarker tests for molecularly targeted therapies. "Currently, all clinical laboratories are regulated by CMS through the Clinical Laboratory Improvement Amendments (CLIA). However, regulatory oversight under CLIA is widely viewed as insufficient for laboratories providing increasingly complex biomarker tests," the report says.

5. Ensure ongoing assessment of the clinical utility of biomarker tests for molecularly targeted therapies. "The generation of evidence of the clinical utility of any biomarker test should be viewed as a continuous process. CMS and other payers should develop payment models to support ongoing data collection to further assess the clinical utility of promising biomarker tests," the report advises.

6. Ensure development and use of electronic health records (EHRs) and related biomedical informatics tools and assessments that support the effective clinical use of biomarker tests for molecularly targeted therapies. "EHR and laboratory information systems developers need to facilitate the collection of real-time patient data that includes information about tests, treatments, and outcomes. These data should be structured to provide support for health care providers in making decisions about test ordering and treatment, as well as to facilitate data transfer to a national data repository," the report says.

7. Develop and maintain a sustainable national database for biomarker tests for molecularly targeted therapies through biomedical informatics technology to promote rapid learning for the improvement of patient care. "Currently, a tremendous learning opportunity is lost because biomarker test data are maintained in separate siloes at individual institutions. A national repository for such data needs to be developed, and incentives should be used to encourage all health care systems and providers to submit their data to the repository, which should have appropriate de-identification, security, and patient consent measures," the report notes.

8. Promote equity in access to biomarker tests for molecularly targeted therapies and the expertise for effective use of the results in clinical decision-making. "Patients of particular economic, ethnic, and cultural backgrounds and geographic locations may face challenges in obtaining access to biomarker tests and associated therapies. Research should identify existing barriers to equitable access and develop approaches to address them. Areas of focus include the potential for telemedicine to expand access to relevant clinical expertise. Licensing and specialty boards should ensure provider competency in communicating about these tests and therapies to patients," the report says.

9. Enhance specimen handling and documentation to ensure patient safety and the accuracy of biomarker test results. "The reliability of biomarker test results depends on the quality of the patient specimens. Professional organizations and health care institutions should develop and implement standards for obtaining adequate specimens," the report says.

10. Improve the processes for developing and updating clinical practice guidelines for the effective use of biomarker tests for molecularly targeted therapies. "Increasingly, a broader base of interdisciplinary expertise is needed to generate trustworthy clinical practice guidelines for biomarker tests. Such guidelines should consider the evolving nature of evidence for biomarker tests for molecularly targeted therapies," the report says.

Important Step Forward

The report was sponsored by the American Society for Radiation Oncology, American Society of Clinical Oncology, Breast Cancer Research Foundation, Centers for Disease Control and Prevention, College of American Pathologists, Gilead Sciences, Janssen Diagnostics, National Cancer Institute, Novartis, Pfizer, Quest Diagnostics, and Susan G. Komen.

In a statement, Julie M. Vose, MD, president of the American Society of Clinical Oncology (ASCO) says this report is "an important step toward realizing the promise of precision medicine." She notes that several ASCO initiatives are helping reach a number of the goals spelled out in the IOM report.

ASCO's CancerLinQ, for example, will "fulfill IOM's recommendation for the implementation of a rapid learning system that will track long-term patient outcomes related to the use of biomarker tests and targeted therapies," Dr Vose says. "However, the current lack of interoperability among clinical databases and with genomic data sets is a major roadblock to achieving the promise of precision medicine. In response to this critical need, ASCO has called on Congress to take outlined steps to ensure the interoperability of electronic health records, with the ultimate goal of improving the care of people with cancer."

Another example is ASCO's clinical trial — the Targeted Agent and Profiling Utilization Registry (TAPUR) Study, which will use genomic biomarker testing to match patients with molecularly targeted therapies. "The findings of this precision medicine trial will yield new knowledge about new uses for these therapies outside of current indications approved by the U.S. Food and Drug Administration and provide information about how oncologists use tumor genomic tests in clinical practice," Dr Vose predicts.

"We commend the IOM for this thorough and forward-thinking report, and we welcome the opportunity to work with the cancer community to fulfill these recommendations in the best interests of patients," she adds in the release.


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