FDA REGULATION OF DIAGNOSTICS ENSURES PATIENT SAFETY AND PROMOTES PRECISION CANCER MEDICINE
This is the age of precision medicine in cancer. Clinicians can identify mutations in a patient’s tumor and tailor an individual’s treatment to specifically target them. These targeted therapies are not only personalized for each patient but are also more effective and less toxic than traditional chemotherapies. A critical aspect of targeting mutations is the use of diagnostic tests that can detect the mutations in a patient’s tumor. These diagnostic tests are so crucial for the success of targeted therapies that the U.S. Food and Drug Administration (FDA) approves these therapies only in conjunction with a test called a companion diagnostic to ensure that the right patient gets the right treatment at the right time. Therefore, it is essential to ensure that the tests used to make important treatment decisions are reliable and accurate.
Watch the recorded presentation by Curtis Hanson, MD, Mayo Clinic Cancer Center, which was given at the AACR Annual Meeting 2015 in Philadelphia, Pennsylvania.
AACR Policy Statement on LDTs
The AACR believes that FDA regulation of molecular diagnostic tests, especially tests used to make high-risk treatment decisions, will ensure patient safety while promoting medical product innovation. Read the AACR Policy Statement, in which we make the following recommendations:
- Ensuring the safety, reliability, and accuracy of diagnostic tests is vital to safeguard patients, advanced personalized medicine, and promote innovation.
- Implementing a single, strict regulatory pathway through the FDA will help reassure clinicians, patients, and the public that the tests used to make treatment decisions are safe, accurate, and effective.
- Having a predictable and reliable regulatory environment is important to encourage an innovative biomedical ecosystem in the United States.
In addition to FDA regulated companion diagnostics, patients and physicians can avail themselves of tests known as Laboratory Developed Tests or LDTs. LDTs are subject to oversight under the Clinical Laboratory Improvement Amendments (CLIA). CLIA ensures that labs follow proper practices and it is important to note that
CLIA oversight is not equivalent to FDA regulation in that it does not:
- regulate the safety and efficacy of individual LDTs;
- require a regulatory review process for LDTs before they are offered to patients and physicians; or
- require independent review of clinical claims made by LDTs.
Testimony Before Congress
On Sept. 9, 2014, former AACR President Charles Sawyers, MD, testified before the House Energy and Commerce committee, reiterating the AACR’s position on the regulation of laboratory developed tests.
Read Sawyers’s written testimony.- Read the transcript of Sawyers’s oral testimony.
- Visit the House Energy and Commerce committee’s webpage, including complete video and transcripts from the hearing.
LDT Webinar with the FDA
On Dec. 17, 2014, the AACR hosted a webinar presented by the FDA, in which it gave an overview of the proposed LDT guidelines and took questions from the audience.
FDA Public Workshop
On Jan. 8-9, 2015, the FDA held a Public Workshop at the National Institutes of Health, in which it invited public comments to the proposed guidance and held panel discussions for many of the key issues to be addressed. The AACR was invited to participate and was represented on the third panel discussion on Day 1 entitled “Categories for Continued Enforcement Discretion.”
- View the video from Day 1.
- View the video from Day 2.
- View the workshop transcript and PowerPoint slides.
AACR Public Comments Regarding LDTs
On Dec. 9, 2014, the 21st Century Cures initiative requested feedback regarding A Modernized Framework for Innovative Diagnostic Tests. Read the AACR’s comments.
On Feb. 2, 2015, the AACR submitted comments to the FDA regarding its draft guidance. These comments are intended as the beginning of a dialogue between the AACR and the FDA as the guidelines are revised and finalized.