By: Stephen M. Hahn, M.D., Commissioner of Food and Drugs, and Anand Shah, M.D., Deputy Commissioner for Medical and Scientific Affairs
Americans may be surprised to learn that many 21st century medical products are still being manufactured using technologies commonly employed since the middle of the last century. These manufacturing platforms are not dynamic and can increase the risk of shortages, limit flexibility during an emergency, and contribute to the high cost of medical products. For the past several years, the U.S. Food and Drug Administration has sought to encourage and facilitate the adoption of advanced manufacturing, which refers to new and emerging approaches for the production of medical technologies.
Advanced manufacturing approaches are applicable to different medical product areas. For example, process intensification methods, such as continuous manufacturing, can simplify and centralize the production of many essential medicines. Likewise, techniques such as 3D printing can help produce patient-specific medical devices. Furthermore, digital and smart design and manufacturing processes also promise to increase efficiency and reduce uncertainty.
The potential public health value of advanced manufacturing is even greater in the context of the ongoing COVID-19 pandemic, which has highlighted the strain on supply chains and the need for adaptive manufacturing systems to accelerate the production of medical countermeasures. The FDA has established a strong regulatory foundation to support the uptake of advanced manufacturing, and COVID-19 provides the unique impetus to spur further advancement of medical manufacturing.
FDA regulations cover both sides of the innovation equation: development (whether the product meets the appropriate statutory standard) and manufacturing (whether quality products can be produced for widespread use). Many manufacturers continue to use the same production techniques that were developed more than 50 years ago. Typical manufacturing processes that use long shipping lines or outsourced supply chains render U.S. manufacturing vulnerable to delays, disruptions, and quality control issues. These existing supply chain vulnerabilities have been exacerbated during the COVID-19 pandemic. Additionally, batch manufacturing lacks the flexibility needed to sustainably produce therapies for the personalized medicine era; this is a significant concern given that the FDA anticipates approving approximately 40 gene therapies in the next few years.
Advanced manufacturing often enables innovation, increases in efficiency, and improved supply chain resiliency for medical products that provide wide-ranging public health benefits. Over the past decade, the FDA made strategic, forward-looking investments in personnel, policies, and processes to create a clear regulatory pathway for innovators across the three medical product areas of drugs, biologics, and devices.
First, the agency recognized that innovators seeking to adopt advanced manufacturing technologies may be concerned about the technical and regulatory challenges associated with transitioning away from their existing platforms. To this end, the FDAs Center for Drug Evaluation and Research (CDER) created the Emerging Technology Program, which has a dedicated team available to provide pre-submission support on issues such as the development of process control measures for continuous manufacturing of drugs. To provide focused expertise for advanced manufacturing of biological products, the FDAs Center for Biologics Evaluation and Research (CBER) established the Advanced Technologies Team, which works with prospective developers on issues such as technical considerations for platform technologies in gene therapy.
Second, the FDA recognizes that policy must keep pace with innovation. To expedite the development of newer technologies, the agency developed a series of leapfrog guidance documents, which the FDA uses to share initial thoughts regarding emerging technologies that are likely to be of public health importance. Such leapfrog guidance documents include the FDAs Center for Devices and Radiological Healths (CDRH) 2017 guidance on Technical Considerations for Additive Manufactured Medical Devices, which encompasses many technologies including 3D printing. The agency has provided further regulatory clarity as technologies mature and are commercialized; for example CDER issued guidance in 2019 on Quality Considerations for Continuous Manufacturing. The FDAs engagement in public dialogue supports the proactive identification and resolution of potential barriers for the transition to advanced manufacturing.
Third, as a science-based agency, the FDA supports research and partnerships that expand the knowledge base for advanced manufacturing. For example, the FDA has used authorities in the 21st Century Cures Act to award research grants to support investigators exploring key questions around monitoring and control techniques for advanced manufacturing platforms. To foster collaborations across the public, private, and non-profit sector, the FDAs Office of the Chief Scientist (OCS) launched a new program for advancing regulatory science in public health and formed partnerships with stakeholders such as America Makes, BioFab USA, the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and a number of industry organizations and clinical societies. OCS has also spearheaded multiple intramural research programs to help develop the regulatory expertise required to evaluate advanced manufacturing technologies. These initiatives will enable the FDA to identify and address cross-cutting scientific, technical, and regulatory challenges and opportunities for advanced manufacturing.
The COVID-19 pandemic has shown how conventional manufacturing practices and predominantly international supply chains may be a liability for Americas emergency response efforts. In addition to the policy and programmatic foundations described above, the FDA has also taken a number of actions to shore up manufacturing capacity specifically for public health preparedness. For example, CDER entered a multi-year partnership with BARDA, the Biomedical Advanced Research and Development Authority, to explore how continuous manufacturing techniques could improve Americas capacity to rapidly manufacture medical countermeasures during emergency situations. During COVID-19 specifically, OCS and CDRH helped develop a Memorandum of Understanding between the FDA, the National Institutes of Health, and the Department of Veterans Affairs to facilitate information sharing on 3D printing to help support manufacturing of essential medical supplies such as personal protective equipment and medical device parts.
Advances in regulatory science are not an end in themselves. True public health preparedness requires incentives and investments in the technologies that the agency has been promoting for years. Increasing emphasis on domestic manufacturing strengthens our response capability, yet it is not enough to bring supply chains back home. We must also ensure that the renewed focus on the importance of domestic manufacturing capacity is paired with a recognition of the capital requirements and scientific expertise needed to adopt more resilient and efficient platforms.
The FDA is committed to doing its part to foster the adoption of advanced manufacturing technologies. To reduce the burden on innovators, the agency is actively working to ensure international concordance on guidelines for continuous manufacturing as part of the International Council for Harmonisations Q13 proposal. To ensure that best practices are informed by the latest research, the agency is committed to monitoring ongoing grant programs, with the intent of fostering initiatives that can demonstrate tangible improvements in safety, quality, and efficiency. To promote communication about the adoption of best practices and innovative ideas between stakeholders, the FDA will continue to proactively provide forums for scientific discussion, participate in national and international workshops, and collaboratively engage stakeholders.
Because pandemics by nature are unpredictable, our approach to manufacturing must be adaptable. Advanced manufacturing provides an approach for protecting our supply chain and improving our response capacity during crisis situations. By establishing the regulatory foundation, the FDA has created a pathway for industry to continue adopting the needed improvements in manufacturing technology for the benefit of public health.
See the rest here:
- Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks - September 2nd, 2020
- Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and... - September 2nd, 2020
- First Gene Therapy Products Approved in Brazil - Lexology - September 2nd, 2020
- Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - The News Brok - September 2nd, 2020
- Prevail Therapeutics to Present at Upcoming Investor Conferences - Yahoo Finance - September 2nd, 2020
- Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - BioSpace - September 2nd, 2020
- The Safety and Clinical Activity of Gene-engineered T-cell Therapy Targeting HPV-16 E7 in Epithelial Cancers - Pharmacy Times - September 2nd, 2020
- AGTC to Host Management Update on September 9 at 8:00 am ET to Discuss Planned Phase 2/3 XLRP Trial, Share Additional XLRP Data and Report Fourth... - September 2nd, 2020
- Qualitative Analysis and Competitive Industry, CCOVID-19 Scenario of Personalized Gene Therapy Treatment Market - The News Brok - September 2nd, 2020
- Sorrento Announces the Closing of Its Acquisition of SmartPharm to Build Next Generation G-MAB-Encoded Plasmid DNA For Cost-Efficient and In Vivo... - September 2nd, 2020
- In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience:' report - FierceBiotech - August 10th, 2020
- Gene Therapy Promising in BCG-Unresponsive Bladder Cancer - Medscape - August 10th, 2020
- Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 - PRNewswire - August 10th, 2020
- How Gene Therapy Helped Conner Run : Short Wave - NPR - August 10th, 2020
- Preparing for an influx of cell and gene therapy approvals - - pharmaphorum - August 10th, 2020
- Catalent's Harmans Site Approved To Manufacture AveXis' Gene Therapy - Contract Pharma - August 10th, 2020
- Sangamo and Novartis partner on gene therapies for autism - BioPharma-Reporter.com - August 10th, 2020
- Global Gene Therapy in Oncology Market 2020 Growth Statistics, New Opportunities, Competitive Outloo - PharmiWeb.com - August 10th, 2020
- Research shows promising results for LHON gene therapy - Ophthalmology Times - August 10th, 2020
- The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Yahoo Finance - August 10th, 2020
- Detailed Information on Gene Therapy Market 2020 | Covid-19 Impact Analysis | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio,... - August 10th, 2020
- Global Cell & Gene Therapy Market Outlook and Forecast 2020-2025 - GlobeNewswire - August 9th, 2020
- Coronavirus Impact Editon of Gene Therapy for Mucopolysaccharidosis Market Research Study 2020 Future Development, Top Manufacturers, Technological... - August 9th, 2020
- The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Press Release - Digital... - August 9th, 2020
- Orgenesis Second Quarter 2020 Revenue Increases 55% Reflecting Progress of POCare Platform - GlobeNewswire - August 9th, 2020
- Regulatory Focus, July issue: Cell and gene therapy - Regulatory Focus - August 8th, 2020
- G-CON PODs Selected for Expression Therapeutics' Gene Therapy Manufacturing Facility - PR Web - August 8th, 2020
- CRISPR co-discoverer on the gene editor's pandemic push - Axios - August 8th, 2020
- Voyager Therapeutics Provides Update on AbbVie Vectorized Antibody Collaborations - GlobeNewswire - August 8th, 2020
- Gene therapy - Wikipedia - August 8th, 2020
- Gene therapy - Mayo Clinic - August 8th, 2020
- gene therapy | Description, Uses, Examples, & Safety ... - August 8th, 2020
- What Is Gene Therapy? How Does It Work? | FDA - August 8th, 2020
- 112M Fund Launched to Commercialize UCLs Gene Therapy... - Labiotech.eu - August 8th, 2020
- UK gene therapy pioneer tilts at $125m NASDAQ IPO - Business Weekly - August 7th, 2020
- Of mice and memories: Gene therapy study returns function in Alzheimer's mice - ABC News - August 7th, 2020
- Tessa Therapeutics Announces Results from Two Independent Phase 1/2 Trials of Autologous CD30 CAR-T Cell Therapy in Patients with Relapsed or... - August 7th, 2020
- Cancer Gene Therapy Market Latest Treatment Methodology 2020 to 2025 - Owned - August 7th, 2020
- Kriya Therapeutics announces the establishment of its internal manufacturing facility for process development and scalable cGMP production of gene... - August 7th, 2020
- Regenxbio announces positive one-year data on wet AMD gene therapy - Seeking Alpha - August 7th, 2020
- Covid-19 Forces Gene Therapy Companies to Shift... - Labiotech.eu - August 5th, 2020
- Cell and gene therapies: For biotechs, collaboration is key to successful innovation - BioPharma Dive - August 5th, 2020
- The Time for DMD Gene Therapy is Now: A Chat with the MDA - BioSpace - August 5th, 2020
- Gene Therapy Beats the Blood-Brain Barrier To Cure Cat Disease - Technology Networks - August 5th, 2020
- Analysis of COVID-19 Gene Therapy Market - Market Research Posts - August 5th, 2020
- World-first gene therapy reverses Alzheimer's memory loss in mice - New Atlas - August 5th, 2020
- Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 - Market Research Posts - August 5th, 2020
- Spark Therapeutics Recognized for Fourth-straight Year as One of Philadelphia's Best Places to Work by the Philadelphia Business Journal - BioSpace - August 5th, 2020
- AGTC to Present at the Wedbush PacGrow Virtual Healthcare Conference on August 11, 2020 - BioSpace - August 5th, 2020
- Global Hemophilia Gene Therapy Market is estimated to Experience a Notable Rise in the coming era by Spark Therapeutics, Ultragenyx, Shire PLC,... - August 5th, 2020
- MEDIPAL HOLDINGS Partners with CRYOPORT to Bring Temperature-Controlled Supply Chain Solutions to the Japanese Market - PRNewswire - August 5th, 2020
- Can progenitor cells go further than gene therapy in retinal disorders? A biotech's answer shines in PhIIb - Endpoints News - August 4th, 2020
- Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD - GlobeNewswire - August 4th, 2020
- Cell and Gene Therapy Market 2020 | Enormous Growth with Recent Trends & Demand By Top Vendors JW CreaGene, Vericel, Tego Sciences, CHIESI... - August 4th, 2020
- Comprehensive Report on Gene Therapy for Ovarian Cancer Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | Takara Bio, VBL... - August 4th, 2020
- Gene therapy reverses memory loss from Alzheimer's in mice - BioNews - August 3rd, 2020
- Hemophilia Gene Therapy Industry Market Analysis by Size, Share, Growth, Application, Segmentation and Forecast to 2025 - Express Journal - August 3rd, 2020
- Dyne Therapeutics Appoints Susanna High as Chief Operating Officer - Business Wire - August 3rd, 2020
- Cell and Gene Therapy Industry Market with manufacturers, Application, regions and SWOT Analysis 2025 - CueReport - August 3rd, 2020
- Genetic Studies Could Pave the Way to New Pain Treatments - Pain News Network - August 3rd, 2020
- Oxford Biomedica Signs Development, Manufacture & License Agreement with Beam Therapeutics Inc., for LentiVector Platform for Next Generation... - August 3rd, 2020
- AZ partners with Daiichi on ADC therapy - BioPharma-Reporter.com - August 3rd, 2020
- Getting gene therapy to the brain | Penn Today - Penn Today - August 2nd, 2020
- Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative... - August 2nd, 2020
- Axovant Signs Three-Year Clinical Supply Agreement With Oxford Biomedica for Manufacturing and Supply of AXO-Lenti-PD - BioSpace - August 2nd, 2020
- Gene Therapy for Inherited Genetic Disorders Market size in terms of volume and value 2019-2025 - Market Research Correspondent - August 2nd, 2020
- Gene Therapy Market Professional Industry Overview 2020 Segmentation and Development Analysis BY 2027 - Science Examiner - August 2nd, 2020
- Greenwich swimmers take the plunge to fight cancer - Greenwich Time - August 2nd, 2020
- Cell and Gene Therapy Market Overview, Growth & Advancement to (2020-2025) - Owned - August 2nd, 2020
- Movers & Shakers, July 31 | BioSpace - BioSpace - August 2nd, 2020
- REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights - Herald-Mail Media - August 2nd, 2020
- Catharpin mom helps raise national awareness for her young sons disease - WDVM 25 - August 2nd, 2020
- Spinraza-Zolgensma Combination Well-tolerated in Children with SMA... - SMA News Today - August 2nd, 2020
- Vineti and EVERSANA form strategic alliance to advance patient-centered services and supply chain automation for regenerative medicine - PRNewswire - August 2nd, 2020
- Orchard Therapeutics to Webcast Conference Call of Second Quarter 2020 Financial Results - GlobeNewswire - August 2nd, 2020
- Expression Therapeutics Announces Bill Swaney as VP of Manufacturing and the Construction of Cell and Gene Therapy Manufacturing - Yahoo Finance - August 2nd, 2020
- REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights - PRNewswire - August 2nd, 2020
- Global Gene Therapy Market Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2025 - Research Newspaper - August 2nd, 2020
- Cell and Gene Therapy Tools, and Reagents: Global Markets - PRNewswire - August 1st, 2020
- Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD - PharmiWeb.com - July 31st, 2020