martes, 12 de diciembre de 2017

Bringing Early Feasibility Studies for Medical Devices Back to the United States

Posted on December 12, 2017 by FDA Voice

By: Maureen L. Dreher, Ph.D., Andrew Farb, M.D., and Owen Faris, Ph.D.

Ten years ago, when medical device manufacturers wanted to gain early clinical experience with their new devices they often went overseas to conduct first-in-human or small clinical studies. Moving a device from bench to bedside for use in patients is a critical step along the development path. However, going overseas delayed access to potentially beneficial devices for American physicians and patients.

The Early Feasibility Studies (EFS) Program is supporting medical device innovation and enhancing early patient access to new technologies.

Today, FDA’s Center for Devices and Radiological Health (CDRH) has an Early Feasibility Studies Program (EFS) that provides a route for innovators, sponsors, FDA review teams, and clinicians to work together to facilitate the early clinical evaluation of medical devices in the United States under the investigational device exemption (IDE) regulations.

An EFS is a limited clinical study on a device early in its development, typically before the device design has been finalized, for a specific indication. It may be used to evaluate the device design concept with respect to initial clinical safety and device functionality. The EFS Program includes enhanced opportunities for collaboration, increased regulatory flexibility, and consideration of benefit-risk principles, while maintaining appropriate patient protection measures.

Maureen L. Dreher, Ph.D., Research Biomedical Engineer in the Office of Science & Engineering Laboratories and Early Feasibility Study Program Representative in FDA’s Center for Devices and Radiological Health

Since the release of our EFS guidance document in October 2013, and our promotion of the EFS Program as part of the 2014-2015 Strategic Priorities, we’ve seen more early clinical studies on devices inside the United States. Specifically, the number IDEs submitted for EFS has more than doubled with 26 submitted in the first year after the EFS guidance document was finalized as compared to 57 submitted in fiscal year 2017.

More importantly, most of these studies have received timely FDA approval. During the last two years, 75% of the IDE submissions which are required to conduct any clinical study on an investigational device, have been either approved or approved with conditions by FDA within one 30-day review cycle.

There are many examples of how the EFS Program is supporting device innovation and enhancing early patient access to new technologies. One is Mitralign, Inc., which is developing a novel treatment for tricuspid regurgitation (TR). TR occurs when the heart’s tricuspid valve does not open and close properly. Many TR patients are not candidates for open heart surgery due to high risk comorbidities. But the Mitralign valve repair system may expand treatment options for these patients through the use of minimally-invasive transcatheter technology. Mitralign’s IDE approval in only 28 days enabled them to conduct a U.S.-based EFS that saved time, money, and the human resources to conduct the study:

Andrew Farb, M.D., Medical Officer in the Division of Cardiovascular Devices, Office of Device Evaluation in FDA’s Center for Devices and Radiological Health

“The EFS program has given Mitralign the opportunity to advance our Tricuspid program clinically, technologically and also from a regulatory pathway perspective. It is a clear example of the FDA striking the right balance between the need to advance healthcare technology for the US population and the need to protect the patients who may one day benefit from that advancement.” —Rick Geoffrion, President & CEO, Mitralign.

Enabling medical device innovation with EFS has a direct and positive impact on all medical device developers, and may be particularly important for small manufacturers. About half of the EFS IDEs are submitted by medical device manufacturers, most of which are small companies for whom early clinical experience is crucial for obtaining financial resources. This was true for Enspire DBS, a startup medical device company, which used the EFS Program to begin a first-in-human study of their Deep Brain Stimulation technology in combination with physical rehabilitation to restore function in stroke patients:

“The FDA was engaged and quite interested in working with us to find a mutually acceptable way to move forward with our early feasibility study. We were happy with the interactive review and having an approved IDE significantly helped the company in securing funding.” —Enspire DBS.

Owen Faris, Ph.D., Director of the Clinical Trials Program, Office of Device Evaluation in FDA’s Center for Devices and Radiological Health

An important lesson learned from the EFS Program so far is that the enhanced opportunities for collaboration between the sponsor and FDA’s review team are crucial for success. Martha Murray, M.D., at Boston Children’s Hospital found this to be a significant advantage of the EFS Program when she approached FDA about translating her novel bridging scaffold, invented at an academic medical center, to treat anterior cruciate ligament injuries:

“Participating in the Early Feasibility Studies Program opened up the door for us to see how collaborative working with the team at CDRH could be. The program gave us access to a team of experts as we tested our device in the preclinical studies. Knowing we had that depth of expertise behind us made us more confident we had done as much as possible to ensure the safety of the subjects in our First-in-Human trial.” —Martha Murray, M.D., Orthopedic Surgeon, Boston Children’s Hospital.

The success of the EFS Program requires stakeholder collaboration beyond FDA. The Medical Device Innovation Consortium (MDIC) is a non-profit, public-private partnership whose mission of advancing medical device regulatory science for patient benefit is aligned with CDRH’s EFS program. MDIC EFS working groups are identifying opportunities to improve the EFS ecosystem by increasing administrative and clinical efficiencies and developing resources for device innovators, such as tools that increase efficiencies in contracting between developers and clinical sites.

We invite manufactures to visit our new webpage devoted to Early Feasibility Studies. With the availability of this webpage, we hope to expand awareness of this important pathway and provide the medical device community with a one-stop shop for EFS resources.

The program represents a critical component of CDRH’s larger efforts to streamline the clinical trial initiative, to increase access for patients in the U.S. to high-quality, safe and effective medical devices as quickly as possible.

Maureen L. Dreher, Ph.D., is a Research Biomedical Engineer in the Office of Science & Engineering Laboratories and Early Feasibility Study Program Representative in FDA’s Center for Devices and Radiological Health

Andrew Farb, M.D., is a Medical Officer in the Division of Cardiovascular Devices, Office of Device Evaluation in FDA’s Center for Devices and Radiological Health

Owen Faris, Ph.D., is the Director of the Clinical Trials Program, Office of Device Evaluation in FDA’s Center for Devices and Radiological Health