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Compliance In Focus
Posted by Michael Marotta on Wed, Aug 13, 2014

Personalized Medicine, the FDA, and Health Care Professionals

Health Care ProfessionalsRecently we have reviewed the role of direct-to-consumer genetic testing  within the sphere of Personalized Medicine, the FDA, and Health Care Professionalspersonalized medicine.  Personalized medicine can refer to tailoring disease treatment, therapy, or prevention to a particular individual, often utilizing some of the genomic signatures that make each of us unique.  Today we will look at a recent FDA news release which looks at personalized medicine from the perspective of the health care professional in respect to two areas: in vitro diagnostic (IVD) companion devices and laboratory developed tests (LDT).

IVD Companion Devices

As defined by the FDA, an IVD companion device is an “in vitro diagnostic device that provides information that is essential for the safe and effective use of a corresponding therapeutic product.”  These devices have been used to aid and tailor treatment for certain gene-based cancers (i.e. ERBB2 positive breast cancer). The FDA recently issued a guidance on August 6, 2014 to reflect the increase in both scientific knowledge and technology which has allowed for a greater individualization of medical therapy.  Additionally it was to encourage upfront communication between sponsors and the FDA so that potential joint development of both therapeutic product and diagnostic devices may take place.  As an example, the FDA illustrated the case of ERBB2/Her-2 positive breast cancer. In this subtype of breast cancer, the effectiveness of the therapeutic product (i.e. Herceptin or trastuzumab therapy) is tightly linked to an effective diagnosis of the subtype.  This is where a patient may greatly benefit from the use of a companion diagnostic device.  To further sub-define an IVD companion diagnostic device, the FDA provided four additional definitions:

  • Identify patients who are most likely to benefit from the therapeutic product.
  • Identify patients likely to be at increased risk for serious adverse reactions as a result of treatment with the therapeutic product.
  • Monitor response to treatment with the therapeutic product for the purpose of adjusting treatment (e.g., schedule, dose, discontinuation) to achieve improved safety or effectiveness.
  • Identify patients in the population for whom the therapeutic product has been adequately studied, and found safe and effective, i.e., there is insufficient information about the safety and effectiveness of the therapeutic product in any other population.

The application and use of an IVD companion device will be viewed within the framework of the associated therapy and potential use. Although it is possible that both a therapeutic product and IVD companion diagnostic device would be developed concurrently, the FDA recognizes a number of scenarios where this might not be possible.  Furthermore, the FDA also envisions possibilities where approval of a therapeutic product may precede that of a companion diagnostic device.  From a study development perspective, it is possible for both a diagnostic device and therapeutic product to be wrapped into the same investigational study.

Again, the FDA proposes this guidance as an attempt “to stimulate early collaborations that will result in faster access to promising new treatments for patients living with serious and life-threatening diseases”.  The FDA references taking into consideration public comments from the previous draft guidance of 2011; it can be speculated that additional public comments and further increases in scientific understanding of the human genome at all levels (i.e. DNA, RNA, methylation status) will continue to shape the development and oversight of companion diagnostic devices.

Laboratory Developed Tests

Laboratory developed tests can also be used to help tailor an individual’s medical therapy.  These tests are often “designed, manufactured, and used within a single laboratory” and can potentially complement or compete with FDA-approved tests without the support of clinical trials.  These tests are used by a health care professional and do not reflect direct-to-consumer LDTs, many of which already fall  under the oversight of the FDA and have been discussed previously.  The FDA news release serves as a 60-day notification to Congress, as required by the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA), and as such, the details of what may be included in the upcoming draft guidance are still unclear.    However in the news release it was discussed that oversight enforcement of an LDT may be dependent upon the risk associated with the test (i.e. a low-risk LDT for a rare disease may not trigger FDA enforcement whereas a medium to high-risk would).  Therefore keep your eyes peeled on what is issued in the guidance within the next few months.

As we enter an era of personalized medicine, it may be wise to be cognizant of new knowledge, new technology, new companies, new regulations, and new ways of thinking.  All of these revolve around an individual and how all of this “new-ness” may better prevent, treat, or diagnose disease. What are your thoughts? Is the pace too fast, too slow, or just right? And what role do you feel that advancements in personalized medicine may place in your health?

Photo Credit: euthman

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Topics: Personalized Medicine, IVD, Laboratory Developed Tests, FDA


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