Feature articles during July focused on global regulatory strategy for cell and gene therapy, with articles on US and EU regulations and guidances and the development and manufacture of the therapies. Also included were articles on recasting the corrective and preventive action (CAPA) process as a continuous improvement process, a military-civilian perspective on real-world evidence (RWE) to support regulatory decision making, and regulatory reporting in multinational trials during COVID-19.Advanced therapy medicinal products (ATMPs), including cell therapies, gene therapies, and tissue-engineered products, are highly complex treatments that differ from traditional medicines, both in how they are made and administered and in the benefits they may provide. Regulations for these products were established relatively recently and are still evolving in many jurisdictions globally. The novelty of these products, the inherent complexities of cell and gene therapy products, and the lack of experience with such products pose many challenges for developers.In part one of this two-part series, this months expert authors address these challenges and offer hands-on, practical advice and guidance on regulation and production of ATMPs. If there is one clear, take-home message to developers, it is that early and frequent collaboration with regulatory agencies, during both the approval and development phases, is paramount. It saves time and money, and it reduces the risk of a negative impact on the trajectory of a clinical trial. Part 2 of the series will cover upstream manufacturing and process controls for biologics, the ATMP regulatory landscape in China, EU GMP requirements for autologous cell therapies and parenteral biologics, and regulatory challenges and opportunities in the US.Regulations and guidancesThe regenerative medicine advanced therapy (RMAT) field has the potential to provide profound benefits to patients with serious diseases and disorders, and the RMAT designation is helping drive these transformative technologies to market. In Update on RMAT designations, William K. Sietsema and Janet Lynch Lambert discuss the scope and purpose of the special designation for RMATs created by the passage of the 21st Century Cures Act and provide a tally of products that have received the special designation to date. However, while the promise of regenerative medicines to cure disease is propelling the field at a rapid pace, developing these therapies requires a rigorous, carefully planned approach to ensure a seamless progression to regulatory approval and commercial success.Siegfried Schmitt expands on that point about carefully navigating the complex and nuanced regulatory environment in two articles on US and EU regulations for RMATs and ATMPs, respectively. In US regulations for regenerative medicine advanced therapies, he provides a user-friendly, quick-access list of RMAT-related regulations and guidances. The article includes a useful introduction to the application process and descriptions of the features and criteria for various expedited program options, including breakthrough therapy, fast track, advanced approval, and priority review.In Regulation of advanced therapy medicinal products in the EU, Schmitt explains some of the terminology relating to ATMPs before documenting the key EU regulations and guidances for each therapy type. He concludes with discussions on marketing authorization, accelerated regulatory pathways, and market access. Again, he urges companies and developers to engage with the regulatory agencies early and often throughout the approval process and to seek external regulatory support, especially if the developer has limited in-house regulatory resources.Development pathways and manufacturingTwo articles shift the focus from the regulatory landscape to development and manufacturing pathways. The drug manufacturing facility environment presents one of the major sources of potential contaminants in the final biologic drug product, so it is critical to design facilities with cleanroom environmental controls and monitoring that adhere to the highest standards of current good manufacturing practice (cGMP) quality guidelines, write Mo Heidaran and colleagues. In Designing a biologics manufacturing facility: Early planning for success, the authors lay out the planning steps for compliance with cGMP to readiness for chemistry, manufacturing, and controls (CMC). The authors warn that the pressure to reduce time to market put considerable stress on all aspects of commercial operations and commercial-scale manufacturing development, so yet again, early tactical and strategic planning essential.In Advanced therapies: Trip hazards along the development pathway, Kirsten Messmer and Richard Dennett focus on the challenges and complexities of ferrying advanced therapies along the developmental pathway, which they call the trip. They examine the importance of some of the fundamental building blocks for the development program and highlight some commonly encountered challenges, or trip hazards, for cell and gene therapies. The suggest developers establish sound technical and regulatory strategies to better anticipate and avoid the trip hazards, which could prove costly, both in time and money, and have a negative impact the overall clinical study program.CAPA, RWE, and COVID-19Todays CAPA process has become highly focused on compliance, which has manufacturers struggling to determine which issues require a structured CAPA process and which can be resolved in alternative ways, writes Kathryn Merrill in Recasting CAPA as a continuous improvement process. Merrill summarizes a white paper developed by the Medical Device Innovation Consortium, in which the CAPA process is recast as a continuous improvement process for driving higher product quality and improved patient safety. It is intended to enable organizations make a greater number of improvements more quickly, and over time, which will have a favorable impact on product quality in the field.During the Afghanistan and Iraq wars, the US Military Health System used an approach known as focused empiricism to develop new approaches for casualty care. In doing so, it implemented real-world data (RWD) and RWE into a system of performance improvement and product development to achieve historic rates of survival, write Todd E. Rasmussen, a colonel in the US Air force, and Brian J. Young. In A military-civilian perspective on real-world evidence to support regulatory decision making, the authors summarize the framework promoting the collection and analysis of RWD in the healthcare system and describe a new era of collaboration between the US Department of Defense and the FDA, within the context of a new Public Law 115-92, to coordinate on the delivery of military-relevant medical products. The article reviews the FDA evidentiary standards for medical product approval and gives examples of how RWE can help meet those standards.COVID-19 continues to disrupt and redefine the regulatory process and activity. In Managing uncertainty: Regulatory reporting in multinational trials during COVID-19, Ioana Ionita discusses regulatory reporting challenges for multinational clinical trials during the pandemic, as well as the challenge of assessing what is reportable and how to submit COVID-19 risk mitigation measures. She offers real-world experience on how she and her colleagues stopped and restarted recruitment in ongoing multinational clinical trials, and how those actions were reported globally. Ionita concludes that close collaboration between sponsors, CROs, local affiliates, investigational sites, and health authorities is important in choosing strategies under challenging circumstances and when no precedent applies.Whats coming in August?Articles during August will focus on global clinical trials and clinical trial applications. Despite ICH efforts to produce guidelines for the development of drugs and biologics and to standardize the format of marketing applications, there remain considerable differences among countries in the format of clinical trial applications and health authority review processes. This collection of articles will address these divergent formats and processes and provide options for navigating the regulatory aspects of clinical trials. Look for these topics and more throughout August at http://www.raps.org.October call for articlesFor October, Regulatory Focus will look at the regulatory toolboxthe tools regulatory professionals need and where to find them, with an emphasis on websites, guidances, meeting minutes and FDA correspondence, including warning letters, enforcement actions, 483s, and notices. Articles will discuss how to interpret the meaning behind regulatory agency actions and available options for documentation. The submission deadline for articles is 1 September 2020. To contribute to the October issue or suggest a topic, contact Rene Matthews at rmatthews@raps.org.Citation Matthews R. Regulatory Focus, July issue: Cell and gene therapy. Regulatory Focus. July 2020. Regulatory Affairs Professionals Society.
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Regulatory Focus, July issue: Cell and gene therapy - Regulatory Focus
- After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it - Endpoints News - December 17th, 2020
- Gene Therapy in One Eye Improves Vision in Both Eyes - The Scientist - December 17th, 2020
- After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News - December 17th, 2020
- Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,... - December 17th, 2020
- Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire - December 17th, 2020
- Passage Bio Invests In Gene Therapy Manufacturing R&D Site - Contract Pharma - December 17th, 2020
- Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -... - December 17th, 2020
- Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds - Forbes - December 17th, 2020
- What is gene therapy? - The Star Online - December 17th, 2020
- They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News - December 17th, 2020
- Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire - December 17th, 2020
- Gene Therapy Injection in One Eye Improves Vision in Both - Technology Networks - December 17th, 2020
- Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace - December 17th, 2020
- Worldwide Gene Therapy Industry to 2025 - Cancer is Expected to Hold Significant Market Share in the Indication Segment - ResearchAndMarkets.com -... - December 17th, 2020
- Taysha Gene Therapies Set to Join Russell 2000 Index on December 21, 2020 - Business Wire - December 17th, 2020
- Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - BioSpace - December 17th, 2020
- Single gene therapy injection surprisingly boosts vision in both eyes - New Atlas - December 17th, 2020
- Freeline takes the haemophilia B gene therapy fight to Uniqure - Vantage - December 17th, 2020
- Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial - PRNewswire - December 14th, 2020
- ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies - FierceBiotech - December 14th, 2020
- Gene Therapy Market Analysis by Vector Type, Application, Region - Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -... - December 14th, 2020
- Retinal Gene Therapy Market to Witness Sales Slump in Near Term Due to COVID-19; Long-term Outlook R - PharmiWeb.com - December 14th, 2020
- Gene Therapy, Absolutely and For Real | In the Pipeline - Science Magazine - December 14th, 2020
- Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe... - December 14th, 2020
- Rocket Pharmaceuticals in orbit after gene therapy read-out - - pharmaphorum - December 14th, 2020
- Bluebird trumpets long-term data from beta-thalassaemia gene therapy - - pharmaphorum - December 14th, 2020
- ASH 2020: Novel Gene Therapy Found to be Safe and Effective in Treatment of Hemophilia B - OncoZine - December 14th, 2020
- Gene Therapy Unexpectedly Improves Vision In Both Eyes Of Patients Suffering A Form Of Blindness - IFLScience - December 14th, 2020
- Innovative payment models to support cell and gene therapies on the rise - MedCity News - December 14th, 2020
- Better education needed to give patients improved understanding of gene therapies, new review highlights - University of Birmingham - December 14th, 2020
- Global Gene Therapy Market Report 2020: Market is Expected to Recover and Reach $6.84 Billion in 2023 - Forecast to 2030 - GlobeNewswire - December 14th, 2020
- Navigating the Complexities of AAV Scale-Up and Manufacturing - Genetic Engineering & Biotechnology News - December 14th, 2020
- EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan - Business Wire - December 14th, 2020
- New CSL gene therapy could trigger a stock rerating - The Australian Financial Review - October 19th, 2020
- Cancer Gene Therapy Market Size & Share Insights on Growing Applications by 2026 - re:Jerusalem - October 19th, 2020
- Gene Therapy: Healing remedy or harmful hoax? The Knight News - The Knight News - October 19th, 2020
- Polyplus-transfection presents latest solution portfolio for gene therapy market to Alliance for Regenerative Medicine's Meeting on the Mesa -... - October 19th, 2020
- Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome - BioSpace - October 19th, 2020
- Merck's New VirusExpress Platform Speeds Development of Cell and Gene Therapies - PharmiWeb.com - October 19th, 2020
- Axovant Gene Therapies Receives Rare Pediatric Disease Designation for AXO-AAV-GM2 for Tay-Sachs and Sandhoff Disease - GlobeNewswire - October 19th, 2020
- Ori Biotech's new cell and gene therapy platform raises 23m - BusinessCloud - October 19th, 2020
- Foundation to Fight H-ABC, University of Massachusetts Medical School and Yale University Initiate Gene Therapy Study Targeting Cure for Rare Disease... - October 19th, 2020
- 5-Year-Old Girl, Being Treated In French Gene Therapy Trial, Dies In US - NDTV - October 19th, 2020
- Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire - October 19th, 2020
- Castle Creek Biosciences Announces First Patient Dosed in Phase 1/2 Clinical Trial of FCX-013 Gene Therapy for Treatment of Moderate to Severe... - October 19th, 2020
- Longview family rallies around 3-year-old with rare, fatal genetic condition - Longview News-Journal - October 19th, 2020
- Child dies in US during gene therapy trial - The News International - October 19th, 2020
- Tag: Gene Therapy - The Think Curiouser - October 19th, 2020
- Gene Therapy for Inherited Genetic Disorders Market Robust pace of Industry during 2018-2028 - The Think Curiouser - October 19th, 2020
- Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation - BioSpace - October 19th, 2020
- Ori looks to advance closed cell therapy tech - BioProcess Insider - October 19th, 2020
- Histogen Appoints Moya Daniels as Executive Vice President and Head of Regulatory, Quality and Clinical Operations - BioSpace - October 19th, 2020
- EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum - October 10th, 2020
- American Gene Technologies to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Unchained Labs launches the ultimate gene therapy tool; tackle AAV titer, empty/full ratio and more with Stunner! - PRNewswire - October 10th, 2020
- Smartphone-connected pacemaker devices, experimental gene therapy among top 10 innovations at annual event - BioWorld Online - October 10th, 2020
- CellGenix Completes Facility Expansion to Stay Ahead of the Increasing Demand for GMP Raw Materials - b3c newswire - October 10th, 2020
- Cleveland Clinic Names Top 10 Medical Innovations; Sickle Cell Therapy Tops the List - HealthLeaders Media - October 10th, 2020
- Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference - October 10th, 2020
- Flexion Therapeutics to Present at the 2020 Cell & Gene Virtual Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Taysha Gene Therapies Partners with Invitae to Enable Rapid Access to Genetic Testing and Earlier Diagnosis of Patients with CNS Disease for Rare and... - October 10th, 2020
- Cancer Gene Therapy Market 2020 by industry trends, statistics, key companies growth and regional forecast - News by Decresearch - October 10th, 2020
- Bone Therapeutics, Link Health and Pregene to develop and commercialize the ALLOB allogeneic bone cell therapy platform in China and Southeast Asia -... - October 10th, 2020
- Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - GlobeNewswire - October 10th, 2020
- Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference - October 10th, 2020
- Genprex to Present at the Alliance for Regenerative Medicine's Virtual Cell and Gene Meeting on the Mesa - Business Wire - October 10th, 2020
- Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- 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