Improving Access to Medical Devices: FDA Uses Existing Clinical Data to Reduce Premarket Data Needs

At the FDA, we recognize the value of encouraging medical device innovation. We recently have acted to reduce the time and cost of clinical trials while maintaining patient protections. By doing so, FDA is helping to ensure that manufacturers will be more likely to conduct their clinical studies in the U.S., and patients in this country will have earlier access to innovation.
One way the FDA can reduce the time and cost of a clinical trial is to determine if publicly available clinical data for medical devices with which we have considerable experience can be leveraged to develop a less burdensome clinical trial design. For example, take the case of global endometrial ablation (GEA) devices, used to treat heavy menstrual bleeding by applying heat or extreme cold to the inner lining of the uterus.

Since 1997, the FDA has approved five GEA devices based on the results of randomized clinical trials (RCTs) of 250-350 women in each trial. The participants were assigned to a group that received treatment with a new GEA investigational device or to a control group treated with rollerball ablation, an older, well-known technology for treating heavy menstrual bleeding.

Each of the RCTs shared similar study and control populations, study design, and endpoints. Those characteristics, combined with the consistent performance of the roller ball ablation device (the control device) across the RCTs, prompted the FDA to assess whether we could leverage the RCT data to help support a less burdensome clinical trial design for future premarket approval applications for GEA devices.
medicaldevices77: With input from industry and members of the FDA Obstetrics and Gynecology Devices Advisory Panel, the FDA was able to apply a statistical analysis model, called an objective performance criterion (OPC), to determine the minimum acceptable success rate for demonstrating device effectiveness. The FDA will post detailed information on how we developed this OPC on our website soon. 

The FDA’s development of an OPC means that less burdensome clinical trial designs without a control group may be appropriate for clinical studies of GEA devices, resulting in studies that require fewer subjects, thereby reducing the length and cost of such clinical trials compared with RCTs.

In addition, development of an OPC may help encourage subjects to enroll in these clinical trials since all study subjects would undergo treatment with the investigational device.

The FDA has established a strategic priority of strengthening the clinical trial enterprise. This includes finding ways to streamline clinical trials so that fewer resources are required to bring a new device to the market.

Through strengthening the clinical trial enterprise, we hope to encourage manufacturers to study new and important medical devices in the U.S., helping us fulfill our vision of providing patients with high-quality, safe and effective medical devices of public health importance first in the world.

Ben Fisher, Ph.D., is FDA’s Director, Division of Reproductive, Gastro-Renal, and Urological Devices, in the Office of Device Evaluation at the Center for Devices and Radiological Health

Advancing precision medicine by enabling a collaborative informatics community (fda)

medicaldevices77: FDA plays an integral role in President Obama’s Precision Medicine Initiative, which foresees the day when an individual’s medical care will be tailored in part based on their unique characteristics and genetic make-up. Yet while more than 80 million genetic variants have been found in the human genome, we don’t understand the role that most of these variants play in health or disease. Achieving the President’s vision requires working collaboratively to ensure the accuracy of genetic tests in detecting and interpreting genetic variants. We are working towards that goal by developing an informatics community and supporting platform we call precisionFDA.

Sophisticated, relatively inexpensive technology known as next generation sequencing (NGS) already exists to sequence a person’s genome quickly. Developers and users of NGS tests must then comb these sequences to look for segments that suggest potentially meaningful differences and determine whether those differences provide useful and actionable information about the state of a person’s health, and their future risk of disease, behavior, or treatment choices.

Special features of this technology pose novel regulatory issues for FDA. Most diagnostic tests follow a one test-one disease paradigm that readily fits FDA’s current device review approaches for evaluating a test’s accuracy and clinical interpretation. Because NGS tests may be used in many ways in the clinic and can produce an unprecedented amount of data about a patient, we are working to evaluate whether a better option might simply be requiring each NGS test developer to show that the test meets certain standards for quality. Similarly, to demonstrate a test’s clinical value, we are assessing whether it may be more efficient for developers to refer to evidence in well-curated, validated, and shared databases of mutations instead of independently generating data to support a mutation-disease association.

To begin to realize this new vision, precisionFDA is designed as a crowd-sourced, cloud-based platform to advance the science needed to develop the necessary standards. PrecisionFDA will supply an environment where the community can test, pilot, and validate new approaches. For example, NGS test developers, researchers, and other members of the community can share and cross-validate their tests or results against crowd-sourced reference material in precisionFDA.

Planned for beta release (work in progress) in December 2015, precisionFDA will offer community members access to secure and independent work areas where, at their discretion, their software code or data can either be kept private, or shared with the owner’s choice of collaborators, FDA, or the public. Initially, precisionFDA’s public space will offer a wiki and a set of open source or open access reference genomic data models and analysis tools developed and vetted by standards bodies, such as the National Institute of Standards and Technology (e.g., Genome in a Bottle). We believe precisionFDA will help us advance the science around the accuracy and reproducibility of NGS-based tests, and in doing so, will advance consumer safety. We look forward to continuing to update the community on the development of these new tools.

Taha A. Kass-Hout, M.D., M.S., is FDA’s Chief Health Informatics Officer and Director of FDA’s Office of Health Informatics.