Washington, DC - New scientific opportunities, as well as advances in manufacturing and commerce, give the U.S. Food and Drug Administration new ways to advance our mission to protect and promote public health. Leveraging these opportunities requires us to make investments in regulatory science that can reduce uncertainty for innovators, spur investment in new industries and provide principles for the safe and effective development of new technologies. These same advances also give us new ways to support greater availability and use of generic drugs as a way to promote price competition and patient access.
Toward these goals, the Administration’s newly released budget request provides the FDA with the resources to continue to fund our current programs at consistent levels. The request will allow the agency to continue to support our core public health mission, including protecting the safety of the foods we eat. It also includes about $400 million in additional funding to advance planned initiatives to further promote innovation and competition, and advance the health and safety of American families.
These initiatives are aimed at supporting new and ongoing efforts to foster more investment and innovation in the development of therapeutics and diagnostics that target unmet medical needs; advance drug and device competition; stand up new domestic industries – such as pharmacy outsourcing facilities; and create more modern, domestically-based manufacturing, including continuous manufacturing of drugs and biological products, including vaccines. These manufacturing platforms can bring more businesses back to the U.S., help lower drug and device development costs and reduce the risk of shortages.
Investing in these initiatives will help the FDA advance goals that we all share: improved treatment and diagnostic options for patients; lower healthcare costs; the development of new industries that will lead to U.S.-based jobs; and manufacturing advances that are more reliable, lower cost and high quality.
Here's a closer look at some of the initiatives and investments that we plan to pursue:
Promote Domestic Manufacturing: Advancing Modern Drug and Biological Product Manufacturing Technologies, Through the Development of Efficient Regulatory Pathways
The FDA recognizes that the U.S. pharmaceutical and biotechnology industries are moving toward advanced manufacturing technologies, such as continuous manufacturing, for both small-molecule drugs and biological products (including cell therapies and vaccines) to improve the agility, flexibility, cost and robustness of manufacturing processes. These technologies have great potential to accelerate new, more targeted therapies, enhance product quality and bolster stability in the U.S. drug supply to meet domestic and global needs. These new manufacturing platforms may be especially important in the development of personalized medicines and novel technologies. This includes innovations such as cell- and gene-based therapies, and vaccines. With continuous manufacturing platforms, vaccine supply can be more easily ramped up on short notice, and vaccines themselves adapted over a shorter time period to address infectious diseases, such as the flu. The application of this kind of enabling technology to vaccine production has long been a strategic priority for the U.S. Armed with a robust scientific understanding of the requirements and the impact of these advanced manufacturing technologies, the FDA can help industry make investments in these new technologies and grow these opportunities. By developing a science-based framework that includes the regulatory tools and guidance for how products developed in these systems will be evaluated, and by funding research, development and testing of the enabling technologies, the agency can help reduce the cost and uncertainty of adopting these new manufacturing platforms. The FDA would lead stakeholders in the development of clear scientific standards, policy and guidance to support the effective and efficient adoption of these new manufacturing platforms, including the new inspectional methods they’ll require.
As an additional benefit, these small-footprint, high-technology manufacturing platforms are more likely to be domiciled in the U.S. Their adoption could return product manufacturing to domestic sites, helping to foster job creation. Specific regulatory support for small molecule drugs, as well as biological products, would promote innovation in these manufacturing platforms and support the development of this advanced domestic technology to improve patient care and facilitate access to new therapies.
Advance a New Domestic Drug Industry and Promote Access by Establishing the Outsourcing Facility Sector as a Robust and Reliable Source of Compounded Products
The FDA proposes the creation of a “Center of Excellence on Compounding for Outsourcing Facilities” and expanded FDA engagement with outsourcing facilities and states to help the pharmacy outsourcing industry grow to meet its intended function and adhere to higher quality standards to protect patient health. The wider availability of reliable compounded drugs that meet standards for Good Manufacturing Practices could promote access to compounded drugs for patients who need them. These new outsourcing facilities would represent a largely domestic industry.
The Center of Excellence would identify and propose solutions to market barriers to lower the cost for pharmacies to become outsourcing facilities. The Center would provide much-needed education and training to improve product quality, safety and purchaser confidence, and help the FDA adjust its regulatory oversight to better match the scope of production of an individual compounding pharmacy. The FDA would work with industry to improve manufacturing practices, create new programs relating to requested review of method design and stability study protocols, and work with state partners to reduce challenges associated with state regulatory diversity and support state-based oversight of pharmacies.
Bring MedTech Manufacturing Home: Advance Medical Device Manufacturing and Quality
The FDA would establish a voluntary program for device manufacturers to receive certification for meeting objective manufacturing and product quality criteria. This would make the process for introducing innovations in how medical devices are manufactured more efficient and predictable. In turn, this program would encourage device manufacturers to make investments to re-tool their manufacturing processes in ways that can facilitate manufacturing innovation, encourage investment in new production methods and materials, and lead to better medical products.
This more modern and nimble framework would make it more efficient for device developers to innovate manufacturing processes in ways that can allow devices to better meet the needs of patients and the expectations of providers – such as through intelligent, automated processes that monitor and record manufacturing quality metrics, incorporating features and technological characteristics that can contribute to better options and higher quality that achieves their clinical purpose. The agency is already working collaboratively with industry, patients, providers and payers through the Medical Device Innovation Consortium to develop the parameters of the program. As part of this approach, the FDA would recognize third-party certifiers and offer regulatory incentives for those manufacturers who receive certification demonstrating their quality capability. These actions would increase manufacturing innovation, accelerate availability of high-quality devices to patients and foster a competitive marketplace around device quality similar to other industries, such as automotive and aerospace, that would advance device innovations, reduce manufacturing costs and improve the quality and safety of medical devices. As medical devices become more complex ‒ and given the frequent modifications made to devices -- spurring advanced manufacturing and creating a competitive marketplace for device quality is critical for both driving technological innovations and assuring patient safety.
Create a New Medical Data Enterprise: Advance the Use of Real-World Evidence to Improve Human and Animal Health and Support Pre-Market Evaluation and Post-Market Safety
The FDA will advance the use of real-world experience to better inform patient care and provide more efficient, robust and potentially lower-cost ways to develop clinical data that can inform product review and promote innovation. The FDA will establish a new capability, including the development of data and analytical tools, to conduct near-real-time evidence evaluation down to the level of individual electronic health records for at least 10 million individuals in a broad range of U.S. healthcare settings.
Toward these ends, an expanded use of natural language processing for the assessment of information submitted to the agency would be developed in an effort to markedly speed recognition and remediation of emerging safety concerns. The effort would cover a broad range of medical products, including drugs, biologics and medical devices. The healthcare settings would be carefully selected to cover data gaps in the Sentinel and National Evaluation System for health Technology (NEST) systems for FDA-regulated products not currently easily assessed with existing systems.
Expanding the FDA’s capacity to utilize real-world evidence to evaluate the pre- and post-market safety and effectiveness of medical products would generate processes that could improve the efficiency of the regulatory process, better inform patients and providers about pre-and post-market safety, reduce some of the burdens that drive up the time and cost required to bring beneficial innovations to the market and address barriers that can make certain important safety and effectiveness information around the real-world use of products hard to collect and evaluate. The agency has already leveraged the use of real-world data to reduce the time and cost of clinical evidence development resulting in more timely and informative post-market data collection and more timely and efficient approvals of new devices and expanded indications of already marketed drugs and devices, including for drug-eluting stents, pacing leads, companion diagnostics, a spinal cord stimulator and a pediatric ventricular assist device. In the case of transcatheter heart valves, leveraging real-world evidence has already resulted in a greater than 400 percent cost savings for industry, improved post-market surveillance and moved the United States from 42nd to, in some cases, first-in-the-world approvals for life-saving technologies.
Facilitate Growth and Spur Transformation of the Digital Health Technology Industry by Shifting Regulation to an Efficient and Novel Framework for Reliable Post-Market Oversight
The FDA is working collaboratively with industry, patients and providers to establish a new paradigm for digital health technologies under which a company could market lower-risk products without FDA premarket review and market higher-risk products following a streamlined FDA premarket review if the company receives a prior third-party certification for engaging in high-quality software design and testing (validation) and ongoing maintenance. This regulatory model would be fully proven and expanded from its current pilot status to a broader program. For low-risk products, rather than evaluate each individual digital health product before the product comes to market, the FDA would instead focus its resources on validating the quality of a firm’s software design and the firm’s methods for certifying the quality and reliability of its underlying software performance. The agency would further reduce the time and cost of market entry of digital health technologies while assuring appropriate patient safeguards by relying on post-market collection of real-world data to support new and evolving product functions.
Also, the agency would create a Center of Excellence on Digital Health to establish the regulatory paradigm, build new capacity to evaluate and recognize third-party certifiers, and support a cybersecurity unit to complement the advances in software-based devices. To modernize and expand its existing capabilities to ensure the cybersecurity of medical devices, the FDA would enhance its ability to coordinate device-specific responses to cybersecurity vulnerabilities and incidents. The FDA also would implement the modern, agile information technology systems necessary to support this new regulatory paradigm, foster and review breakthrough device innovations, and leverage real-world evidence. Implementing these regulatory innovations and information technology improvements are essential for advancing software-based technologies to improve the health and quality of life of patients while assuring critical safeguards as the current regulatory framework is not well-suited for driving the development of safer, more effective software-based devices, including the use of machine learning and artificial intelligence.
Create a New Platform for How the Agency More Efficiently Develops and Validates Modern Science-Based Principles for New Drug Development and Shares this Information with Innovators
In order to better keep pace with rapidly advancing science in drug development, the FDA would build a knowledge management system and portal to existing and developing information on drug development and previous regulatory decisions. This content management platform would greatly improve workflow and review program efficiency and foster greater collaboration. It will enable the FDA to build on evolving information and decisions and identify gaps in regulatory policies and pathways enabling rapid, consistent responses to regulatory questions and preventing delays in response to innovations in drug development.
As part of this platform, the agency also would expand its capability to quickly evaluate new regulatory questions, using laboratory research or other appropriate methods. With the additional funding, the Oncology Center of Excellence also would stand up a new model for team-based product review that fosters collaboration across our medical product centers, improves review efficiency and expedites the development of novel science that can improve the lives of patients with cancer.
Stimulate Investment In, and Innovation of, Medical Products Targeted to Rare Diseases
To foster investment and innovation in, and medical product development for, rare diseases, the FDA would develop clinical trial networks to create an understanding of the natural history (such as individual patient experiences and progression of symptoms) and clinical outcomes of rare diseases. The FDA would leverage this novel framework when promising medical products have been identified for patients.
The initial focus would be on rare and ultra-rare diseases, where product development can be challenging because of the difficulty of recruiting clinical trials. The FDA has already invested, on occasion, in the development of natural history models for a small subset of rare diseases and has expertise in this area. The FDA would stimulate medical product development for rare diseases by expanding and enhancing the understanding of rare diseases and the research and drug development processes in this space.
Modernize Generic Drug Development and Review to Enable Increased Competition, Promote Generic Drug Substitution and Provide Affordable Options for American Patients
The FDA will create a new review platform that would significantly modernize generic drug review from a text-based to a data-based assessment with structured submissions and FDA assessments. This more automated system would help to support timely development and review of generic drugs by improving clarity for generic sponsors, making initial reviews more efficient and decreasing the risk of refuse-to-file letters, increasing the rate of first-cycle approvals and greatly increasing overall efficiency.
This investment would also support efforts to update generic drug labeling, with an initial focus on oncology products, as part of the agency’s efforts to ensure that patients and their providers have access to up-to-date information to inform clinical decisions. If more generic drugs had up-to-date product labels reflecting the latest treatment information, it would encourage wider adoption of generic medicines.
Conclusion
As I’ve previously noted, our work at the FDA is taking place during an inflection point in both science and policy. There’s perhaps never been a better moment in the history to be engaged in public health, and to be leveraging the capabilities of the FDA to support new investment and product innovation.
The U.S. life sciences sector represents one of our nation’s great modern achievements. It’s a source of rich intellectual property, high-paying jobs and products that are improving the lives of people around the world. We have more opportunity to deliver on the promises of science than at any time before and use new tools and medical advances to alter the trajectory of disease. We are grateful for the Administration’s support of these initiatives and believe these new investments in our agency’s mission will ultimately lead both to better health outcomes for American families and to greater U.S. economic development.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.