FDA Outlines its Approach to Personalized Medicine in Report

A new report issued by the U.S. Food and Drug Administration (FDA) outlines the steps the agency is taking to foster the development of personalized medicine.

The report, “Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development,” describes how the FDA is guiding the direction of personalized medicine by providing clarity and guidance to industry to encourage the development of new products, and working with university scientists, standards organizations, practicing physicians and patients to validate new diagnostics and therapeutics. The agency also hpoes to address the pipeline problem for drugs and devices by identifying ways to streamline regulatory processes and drive innovation. It defines personalize medicine in general terms as “the use of two medical products – typically, a dianostic device and a therapeutic product – to improve patient outcomes.”
In the report’s introduction, FDA Commissioner Margaret Hamburg pledges to remain committed to personalized medicine. “I have made it a priority to continue to evolve FDA’s regulatory processes in response to—and in anticipation of—scientific developments that are critical for the development of personalized therapeutics and diagnostics,” she said.

The move toward personalized medicine has gained steam as a way to tailor medical treatments to the characteristics, needs, and preference of individual patients. Hamburg wrote in a blog post earier this fall that cancer drugs increasingly are partnered with diagnostic devices to ascertain whether a patient will respond to a treatment based on a tumor’s genetic characteristics. She noted that clinicians can use medical imaging to identify the best implantable device to treat a patient with clogged coronary arteries. “Given these trends, the future of medicine is rapidly approaching the promising level of care and cure once imagined by Hollywood in futuristic dramas like ‘Star Trek,’ ” she wrote.

The report lists several examples of personalized medical devices, including a tinnitus masker, which custom tailors audio signals to suit the hearing requirements of patients, pedicle screw spinal systems, an endovascular graft, and an artificial pancreas system that monitors glucose levels and delivers insulin.

While there is great promise in personalized medicine, challenges remain, including a limited understanding of the intrinsic biology of diseases; the reluctance of some clinicians to use the new diagnostics; and a field in which the returns on investment are perceived as low.

The report touches on several substantive issues of particular interest to the medical device industry, including:

• Challenges in regulating whole genome sequencing. The FDA notes in the report that whole genome sequencing (WGS) will present regulatory challenges: the platforms are “still evolving rapidly,” approaches to analytically assess WGS test performance have not been agreed on, and the “exceedingly rare” variants to be analyzed make patient recruitment difficult. According to the report, the agency is considering possibilities for identifying a representative set of markers that could be assessed in order to validate a platform’s performance.

• 3-D printing of personalized devices. FDA describes how physicians are using computed tomography imaging, computer-aided design, and 3-D printing to create personalized device implants tailored to a specific patient’s anatomy. The FDA’s emergency-use regulations were applied in one example highlighted in the report, but FDA’s efforts to regulate 3-D printing at the point-of-care could prove challenging.

• Potential regulation of laboratory developed tests (LDTs). FDA announced in June 2010 that it intends to begin regulating LDTs as medical devices, and it has been widely reported that FDA plans to do so in a series of guidance documents. In the report, FDA reiterates that “FDA has been developing a risk-based framework for regulatory oversight of LDTs.” The report does not provide a release date for the guidance documents, and CDRH did not list them in its proposed guidance development list for fiscal year 2014.
• Drug-diagnostic co-development. The CDRH had previously stated that it would release draft guidance on drug-diagnostic co-development in September, but it did not do so. According to the report, agency staff in the relevant centers “are working together to develop guidance that outlines principles for the co-development process,” as well as developing recommendations for how to coordinate the review of co-development programs. The timeline for this guidance now is unclear; for example, CDRH did not include the co-development guidance in the recently-released list of guidance documents that it intends to publish in FY 2014. The report discusses the two draft guidance documents on companion diagnostics and clinical trials enrichment strategies as providing insight into the logistical challenges inherent in product co-development, including timing and alignment of development pathways for two products and technical and ethical issues in clinical trial design.