Biomarker Tests for Molecularly Targeted Therapies

An integrated set of actions to address clinical practice, regulatory and reimbursement policy, and data challenges

A perspective article published on 7 July 2016 in The New England Journal of Medicine elaborates recommendations issued earlier this year by the Institute of Medicine (IOM) of the US National Academies of Sciences, Engineering, and Medicine on biomarkers for molecularly targeted therapies, in particular the regulatory, reimbursement, and clinical practice issues, in order to guide precision medicine.

In their NEJM article, Gary H. Lyman and Harold L. Moses wrote: “The processes of identifying and validating biomarker tests and of developing and evaluating targeted therapies are complex. Potentially useful tests have not been adopted into clinical practice rapidly, in part because we lack common evidentiary standards for regulatory, clinical, coverage, and reimbursement decisions. Furthermore, clinical implementation will require the consistent collection and sharing of data on biomarker tests, treatments, and patient outcomes.”

Over the past several years, the IOM has convened several expert committees to consider a range of issues related to biomarkers, their testing, genomics, and related issues. “These efforts have highlighted the need for a systematic examination of all the challenges and opportunities associated with biomarker assays for molecularly targeted therapies. Most recently, the IOM assembled a Committee on Policy Issues in the Clinical Development and Use of Biomarkers for Molecularly Targeted Therapies which recommended an integrated set of actions aimed to address clinical practice, regulatory and reimbursement policy, and data challenges through the framework of a rapid learning system.”

The committee identified ten goals to further advance the development and appropriate clinical use of biomarker tests for molecularly targeted therapies:

Goal 1: Establish common evidentiary standards of clinical utility—using evidence generated both within and outside the context of clinical trials—across all stakeholders.

Goal 2: Establish a more coordinated and transparent federal process for regulatory and reimbursement decisions for bio-marker tests for molecularly targeted therapies.

Goal 3: Enhance communication to patients and providers about the performance characteristics and evidence for use of specific biomarker tests for molecularly targeted therapies.

Goal 4: Update and strengthen the oversight and accreditation of laboratories providing biomarker tests for molecularly targeted therapies.

Goal 5: Ensure ongoing assessment of the clinical utility of biomarker tests for molecularly targeted therapies.

Goal 6: Ensure development and use of EHRs and related biomedical informatics tools and assessments that support the effective clinical use of biomarker tests for molecularly targeted therapies.

Goal 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.

Goal 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.

Goal 9: Enhance specimen handling and documentation to ensure patient safety and the accuracy of biomarker test results. 

Goal 10: Improve the processes for developing and updating clinical practice guidelines for the effective use of biomarker tests for molecularly targeted therapies.

Read the full report here

Reference

Lyman GH, Moses HL. Biomarker Tests for Molecularly Targeted Therapies — The Key to Unlocking Precision Medicine. N Engl J Med 2016; 375:4-6 DOI: 10.1056/NEJMp1604033