President Donald Trump’s budget sought a boost in funding for the FDA and now FDA Commissioner Scott Gottlieb is offering his own take on the initiatives and investments the agency plans to pursue.
Plans outlined by Gottlieb include the creation of two new centers related to drug compounding and digital health.
Promote Domestic Drug and Biologic Manufacturing
As pharmaceutical and biotech companies move toward advanced manufacturing technologies, such as continuous manufacturing, Gottlieb said vaccine supplies 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 US.
“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,” he added.
These “small-footprint, high-technology manufacturing platforms” could “return product manufacturing to domestic sites, helping to foster job creation,” Gottlieb said.
FDA is proposing to create, as it did for oncology, a “Center of Excellence on Compounding for Outsourcing Facilities,” with 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 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,” Gottlieb said.
Advance Medical Device Manufacturing and Quality
FDA is looking to establish a voluntary program for device manufacturers to receive certification for meeting objective manufacturing and product quality criteria.
“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,” Gottlieb said.
FDA already is working 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.
Advance the Use of Real-World Evidence
Gottlieb said 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.”
As part of such efforts, he said the FDA will establish the ability to conduct near-real-time evidence evaluation down to the level of individual electronic health records for at least 10 million people in a broad range of US 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,” he added.
Digital Health Technology
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 an expansion of the current pre-certification pilot for digital health, Gottlieb said.
In addition, the agency aims to 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,” he added.
Validating New Drug Development
As part of efforts to advance the science of drug development, Gottlieb said 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 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,” he said.
Medical Products Targeted to Rare Diseases
Gottlieb said the FDA also is looking to develop clinical trial networks to create an understanding of the natural history and clinical outcomes of rare diseases.
“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,” he said. “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
On the generic drug front, meanwhile, the FDA will create a new review platform that would modernize generic drug review “from a text-based to a data-based assessment with structured submissions and FDA assessments,” Gottlieb said.
“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,” he added.
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email firstname.lastname@example.org for more information.
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