Innovation Pathway at FDA
HHS Secretary Sebelius on FDA's Innovation Pathway
Patients deserve faster access to safe and effective medical devices that address unmet public health needs. The FDA's Innovation Pathway is a new way of doing business within our existing regulatory framework that could yield significant benefits to patients in the U.S. by giving them first-in-the-world access to medical devices, including those with breakthrough technology.
The Innovation Pathway is an evolving system designed to help safe, breakthrough medical products reach patients in a timely manner. The Pathway ultimately aims to shorten the overall time and cost it takes for the development, assessment and review of medical devices, and to improve how FDA staff and innovators work together.
By engaging with innovators much earlier, more collaboratively, and in new ways, we believe we can reduce the time and cost of the entire process of bringing safe and effective technologies to patients more quickly.
On April 9, 2012, the FDA's Center for Devices and Radiological Health (CDRH) launched its second version of the Innovation Pathway, called "Innovation Pathway 2.0."
Innovation Pathway 2.0 offers new and modified tools and methods to deepen collaboration between the FDA and innovators early in the process, prior to pre-market submission, with the goal of making the regulatory process more efficient and timely.
The Pathway also serves as a living laboratory to test new tools and methods for breakthrough devices that we may also apply to other technologies to enhance all of our device pre-market programs.
Goal #1: Shorten Time to Develop Safe & Effective Products
Innovation Pathway 2.0 jump starts collaboration with innovators much earlier, which we think will help patients receive safe and effective devices sooner, and reduce the time and cost of the entire process.
Innovation Pathway 2.0 looks at the time it takes to get to patients from product development — or, from the first point of engagement with the FDA, which could be much earlier in a product's life, all the way to the final approval decision on the marketing application.
This longer view is not only more relevant to innovators and the patients who await new technologies, but also gives review teams greater opportunity to find ways to influence and shorten the whole timeframe.
Our experience with earlier collaboration under the Innovation Pathway 2.0 will feed more data into this process, and will build on our ideas to shorten the time period for innovative, breakthrough medical devices to reach patients safely.
A Stronger Balance of Benefit and Risk
The FDA's approval decisions hinge on our analysis of a medical device's benefit and risk, and Innovation Pathway 2.0 incorporates new methods for agency reviewers to analyze the potential benefit and risk of a devices, including a decision support tool and the application of a Center process for assessing patient perspectives.
Decision Support Tool
Innovation Pathway 2.0 is testing a small-scale decision support tool for FDA reviewers to use in evaluating first-in-human (FIH) clinical trials.
A decision support tool is a semi-quantitative way to streamline decision-making by focusing on the most important issues. While we believe the tool can be useful at many points during the device review process, we apply it first to FIH decisions, which are particularly important, as they influence later outcomes.
For example, the decision to allow FIH clinical trials is critical to the development of new medical technology, yet the decision to allow a device to be tested in humans for the first time — with relatively little information and much uncertainty — is one of the toughest the agency makes.
This tool seeks to provide clarity to the question of whether the device can proceed to early feasibility studies or whether further preclinical testing is needed to ensure safety and effectiveness. The tool’s framework is based on the draft guidance, "Investigational Device Exemptions (IDE) for Early Feasibility Medical Device Clinical Studies, Including Certain First in Human (FIH) Studies."
In addition, the tool helps reviewers explicitly consider the balance between allowing the development of the device to proceed, and its potential benefit to the relevant general patient population upon market release, while considering the safety of the test patient population.
Innovators and FDA staff begin to create a framework that captures how they made a decision on a FIH trial — this can then be applied to other decisions.
As we find success with the decision support tool in FIH trials, we will be able to expand its application to other key decision areas, such as pivotal trials necessary for product approval.
Clinical data is and always will be the foundation for determining safety and effectiveness of medical devices. However, safety and effectiveness data alone may not provide a complete picture of the benefits and risks of some devices.
Innovation Pathway 2.0 will incorporate its Benefit-Risk guidance for including patient perspective in decision-making, such as patient tolerance for risk, how the disease the device is intended to treat or diagnose affects the patient that have it, the ability to mitigate risk to the patient, availability of alternative treatments, and the value the patient places on treatment.
Future versions of the Innovation Pathway will develop other techniques and tools to help broaden our understanding of patient perspective.
Goal #2: Collaboration is Key
Innovation Pathway 2.0 ultimately aims to shorten the overall time and cost it takes for the development, assessment and review of medical devices, including those with breakthrough technology, and to improve how FDA staff and innovators work together.
In order to meet those goals, we must improve our collaboration.
Innovation Pathway 2.0 incorporates a "Collaboration Phase" — a loosely structured timeframe where innovators and FDA staff map out the future regulatory pathway for a product.
While we have always worked interactively with sponsors, the Innovation Pathway amps up that collaboration and increases the level of interaction throughout the device's development.
The "Collaboration Phase Playbook" is a document that provides guiding principles and case examples that illustrates how we can strengthen the working relationship. The guiding principles include creating a shared understanding of product success, applying best practices in framing benefits and risks, and striving for greater transparency by quickly delivering guidance to industry.
During the Collaboration Phase innovators and review staff discuss the device technology, benefits, and risks and are encouraged to experiment with new ideas and seek opportunities for experiential learning.
The result of the Collaboration Phase is map, which outlines a customized regulatory pathway specific to that device.
Each device team of reviewers and sponsors on the innovation pathway will be assigned an FDA Case Manager who will:
- assure that the team applies the principles and behaviors outlined in the Collaboration Playbook;
- assure that the Collaboration Phase results in a memo outlining the regulatory path forward, and that the sponsor and the FDA agree to key decisions;
- coordinate activities between the review team and innovators; and
- find appropriate internal and external experts to the project to resolve scientific issues or remove obstacles.
Map of Regulatory Pathway
No device on the Innovation Pathway is guaranteed FDA approval, and sponsors seeking to market a medical device must comply with the requirements of the PMA, de novo, or 510(k) programs. Part of Innovation Pathway 2.0 is that the FDA will produce a memo within 120 days of engagement that identifies key scientific and regulatory requirements, outlines a shared understanding of success and maps out a customized and more predictable regulatory pathway.
The memo will also include specific commitments by CDRH, including re-engagement in the Collaboration Phase at future points.
IT Tools for Collaboration
The FDA has set up a secure online conference center for each device team on the Innovation Pathway. This will allow the FDA and device sponsors to interact freely, regardless of location or time. Features include an e-workspace for FDA review team and device sponsors to engage in private collaboration, take part in embedded threaded discussions, and for storing and sharing documents; and a meeting center for one-on-one meetings.
Network of Experts
One source of expertise available to teams on the Innovation Pathway is the Network of Experts. The Network of Experts is a vetted network of outside scientists, clinicians and engineers who will provide the Center for Devices and Radiological Health (CDRH) staff with rapid access to scientific, engineering, and medical expertise when it is needed to supplement existing knowledge and expertise within the CDRH. This program is designed to broaden CDRH exposure to scientific viewpoints, but not to provide external policy advice or opinions.
CDRH has a tremendous internal cadre of scientific expertise, including over 800 scientists, engineers, and clinicians. Despite this internal resource, it is unrealistic to expect CDRH staff to encompass all of the applicable expertise and experience necessary to fulfill our mission, given the rapidly growing variety and complexity of medical devices. This is particularly true when it comes to new and emerging fields of science and pioneering technologies. In these areas, it is often necessary for our experts to gain further scientific understanding from sources outside of the federal government. The Network of Experts will facilitate this exchange.
Innovation Pathway: Collaboration Phase Playbook Driving Biomedical Innovation: Initiatives for Improving Products for Patients Medical Device Innovation Initiative White Paper Public Workshop: Identifying Unmet Public Health Needs and Facilitating Innovation in Medical Device Development, June 24, 2010