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• What is 21 CFR part 11?
21 CFR Part 11 is a section in the Code of Federal Regulations (CFR) that sets forth the United States Food and Drug Administration’s (FDA) guidelines on using electronic records and electronic signatures. Each title of the CFR addresses a different regulated area, 21 CFR relates to Pharmaceuticals and Medical Devices and Part 11 being applicable to electronic records and electronic signatures.
Read more about 21 CFR part 11:
An introduction to 21 CFR Part 11
• What is CMC?
A chemistry, manufacturing and controls (CMC) strategy is an approach to providing the FDA with scientific data characterizing the therapeutic molecule, its stability and formulation, the manufacturing process, and how the manufacturer is planning to ensure consistency and control of the product throughout the product life cycle. Each IND and subsequent BLA will include a CMC section to ensure drug products are consistently effective, safe and high quality for consumers when they are manufactured.
Read more about CMC:
Leave no CMC stone unturned
• What is an electronic record?
As defined in 21 CFR Part 11, an electronic record is any combination of text, graphics, data, audio, or pictorial information represented in digital form that is created, modified, maintained, archived, retrieved or distributed by a computer.
• What are ICH guidelines?
The International Conference on Harmonization represents the countries interested in unified standards. ICH developed more than 60 guidelines to eliminate duplication in the development and registration process, so that a single set of studies can be generated to demonstrate the quality, safety and efficacy of a new product. The ICH facilitates international electronic communication through the provision of Electronic Standards for the Transfer of Regulatory Information (ESTRI), giving the Electronic Common Technical Document (eCTD), allowing for the electronic submission of the Common Technical Document from applicant to regulator.
• What is PDUFA VI?
On Aug. 18, 2017, the President signed into law the FDA Reauthorization Act (FDARA). This new law includes the reauthorization of the Prescription Drug User Fee Act (PDUFA), intended to provide the FDA with the necessary resources to maintain a predictable and efficient review process for human drug and biologic products. Aside from the more visible changes to the fee structure and fees, PDUFA VI also aims to do more to integrate patient perspectives into the development and regulatory review of new medicines.
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• What is the 21st Century Cures Act?
The 21st Century Cures Act, signed into law in December 2016, is intended to provide the FDA with tools aimed at modernizing regulatory programs. In July 2017, the FDA announced a detailed work plan for the steps the agency is taking to implement different aspects of Cures, which included elements that further the goals of the personalized medicines initiative.
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• What is PIC/S?
The Pharmaceutical Inspection Co-operation Scheme (PIC/S) aims to harmonize inspection procedures worldwide by developing common GMP standards and providing training opportunities to inspectors. In 2017, the organization published a revised GMP guide, as well as unveiled a new strategic plan with a strong emphasis on training and better communication with heads of regulatory agencies.
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• What is the DSCSA?
Signed into law in 2013, the Drug Supply Chain Security Act (DSCSA) combats the spread of counterfeit prescription drugs. As part of the legislation, the FDA outlined a long list of traceability and security requirements that pharma stakeholders must abide by. The first phase of DSCSA required all products to be traceable at the lot level. Phase two requires pharma manufacturers, distributors, wholesalers and dispensers are required to incorporate interoperable exchange, verification and tracing.
• What is an IND application?
Before drug sponsors can dive into First in Human (FIH) studies, they first have to demonstrate the safety and efficacy of the proposed drug in their IND application. The application should give a full synopsis of the drug, including all salient data collected from the development and testing phases, along with manufacturing information.
Read more about how to understand and prepare an IND application:
Keys to submitting an successful IND application to the FDA
• What are ATMPs?
Advanced Therapy Medicinal Product (ATMP) is the umbrella term for three drug product classes: Somatic cell therapies, gene therapeutics and tissue engineered products as well as a combination of these technologies with a medicinal product. All ATMP classes contain either living cells or viral vectors and are therefore characterized by a high degree of complexity.
Read more about the vexing issues behind production of gene therapies:
Gene therapies: On the rise
• What is an ADC?
An antibody-drug conjugate (ADC) consists of a cytotoxic agent — or ‘payload’ — linked to a monoclonal antibody and directed toward a specific cell surface target overexpressed by tumor cells. The modality is designed to kill cancer cells while leaving healthy tissue unharmed. The drugs offer the best of both worlds in cancer care, overcoming the limitations of many current therapies by combining the toxic properties of chemotherapy with the accurate targeting of antibody-based treatments.
Read more about ADCs:
The great reappearing act
• What is an Incretin mimetic?
Incretin mimetics are a fast-growing class of drugs originally developed to treat type 2 diabetes, currently being used for weight loss in obesity care. As their name implies, the drugs work to mimic the glucose-lowering actions of incretins —hormones released by the small intestines — by using long-acting stable receptor agonists of the GLP-1 hormone to stimulate insulin secretion.
Read more about incretins and GLP-1 inhibitors:
Pharma's weight loss challenge
• What is product drift?
Even minor changes in any component of manufacturing or quality control could lead to changes in the biologic product, which may ultimately impact the quality, safety, efficacy, or interchangeability of biologics. This change in the product and its characteristics that can occur over time as a result of manufacturing changes is referred to as “product drift.”
Read more about product drift:
Will product drift cause a rift?
• What is an unproven cellular therapy?
•Unclear scientific rationale to suggest potential efficacy
• Lack of understanding of the mechanism of action and/or the biological function to support clinical use
• Insufficient data from in vitro assays, animal models and clinical studies
• Lack of a standardized approach to confirm product quality and ensure consistency in cell manufacturing
• Inadequate information disclosed to patients to enable proper informed consent
• Uncontrolled experimental procedures in humans
Read more about unproven stem cell therapies:
Bio's bad apples
• What is aseptic processing in pharmaceutical manufacturing?
According to the FDA’s 2004 “Guidance for Industry Sterile Drug Products Produced by Aseptic Processing — Current Good Manufacturing Practice,” in aseptic process: “the drug product, container and closure are first subjected to sterilization methods separately, as appropriate, and then brought together.” It is critical that containers be filled and sealed in an extremely “high-quality” environment.
Read more about aseptic processing:
No Man’s Land
• What are isolators in pharma manufacturing?
Isolators are an arrangement of physical barriers that provide an enclosed working space that is detached from the surrounding environment. This enables manipulation to be undertaken within the space from the outside the enclosure without compromising integrity.
Read more about isolators and sterile processing:
Sizing Up the Benefits of Sterile Drug Manufacturing Techniques
• What is a cleanroom in pharma manufacturing?
In simple terms, a cleanroom is a controlled area within a wider facility that maintains a specific level of air particles and other contaminants.
Read more about cleanroom essentials:
12 essential steps for setting up a pharma cleanroom
• What are biological safety cabinets (BSC)?
Biological safety cabinets are enclosed, ventilated laboratory workspaces designed to protect the operator and the environment.
Read more about BSCs
Integrating BSCs into cGMP environments
• What are Rapid Transfer Systems (RTS)?
Rapid Transfer Systems have become a popular method of transferring items in an era where containment systems are needed more than ever. These systems make it possible to aseptically transfer items like tools, waste, supplies, and byproducts or yields before, after or mid-campaign.
Read more about RTS
Maintaining sterility in aseptic processing
• What are the most common tablet production problems?
Although there are quite a few issues that may arise during tablet manufacture, by far the most common tablet production problem is sticking. Sticking is when material adheres to the surface of the punch tip face.
Read more about tablet production:
Q&A: Tablet production
• What is CAPA?
Corrective action, preventive action consists of improvements to an organization's processes in order to eliminate causes of non-conformities or other undesirable situations.
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• What are CGMPs?
Current good manufacturing practice (CGMP) requirements, which define the manufacturing standards to legally market drug products in the U.S., are the bedrock of pharmaceutical quality regulation. Congress first mandated the application of quality assurance and control principles to drug manufacturing in 1968. The discussion of CGMPs initially centered on satisfying compliance standards for material systems, equipment and facilities, production, laboratory, packaging and labeling — standards that set only a threshold for in-house quality systems.
Read more about CGMPs:
Beyond 'good' practices
Key considerations for CGMP raw materials compliance
• What is QbD?
Quality by Design (QbD) brings a systematic approach to drug development that aims to ensure quality by applying analytical and risk-management methodologies to the design, development and manufacturing of new medications
Read more about QbD:
The advantages of a ‘Quality by Design’ approach in pharma drug development
• What is data integrity?
A critical aspect of data integrity is documenting who did what, why and when. Safeguarding data integrity (DI) — making sure data is complete, consistent, and accurate throughout its life cycle — is vital to data reliability and quality. The FDA and other regulatory agencies have highlighted that complete, consistent, and accurate data need to be ‘ALCOA’ — attributable, legible, contemporaneous, original and accurate.
• What is lean?
Lean is a business improvement approach that focuses on process improvement in new product development, manufacturing and distribution in order to cut lead times, improve quality and customer responsiveness, resulting in enhanced revenues, reduced investment and costs. Pioneered by Toyota in the 1950s and widely adopted across industries, lean’s objectives include using less human effort, less inventory, less space and less time to produce high-quality products as efficiently and economically as possible.
• What is continuous manufacturing?
Unlike batch manufacturing, where APIs or finished medicines are made in intermittent steps that often take place in different locations, continuous processing allows each element of manufacturing to take place in an uninterrupted process in a single facility. Continuous manufacturing provides several significant advantages over conventional batch manufacturing including increasing output and efficiency, lowering costs and accelerating scale-up.
Read more about continuous manufacturing:
The promise of PCM: Getting to maturity
Continuous bioprocessing technology: Adoption and future trends
• What is glass delamination?
Delamination is a response primarily observed in converted tubing glass vials in which thin flakes release from the interior surface of the vial into the liquid formulation. The propensity for delamination is dependent on multiple factors.
Read more about glass packaging:
Stabilizing the drug supply
• What is procurement?
Procurement relates to the process under which goods and services are acquired and consumed by a business — both upstream and downstream. On the other hand, sourcing relates to the process of supplier selection and management. Historically, both sourcing and procurement processes have been incredibly laborious with teams spending hours or often days sifting through the internet searching for new suppliers to engage with.
Read more about procurement:
AI beyond drug development
• What is natural language processing?
NLP is an area of artificial intelligence and computational linguistics, and essentially a way in which a computer can extract meaning from written or spoken language. It includes information retrieval and extraction, lexical and semantic analysis, pattern recognition, tagging and annotation, and data mining techniques.
Read more about natural language processing:
Improving the risk-reward calculus for clinical trials
• What is next generation sequencing?
Next generation sequencing (NGS) is a powerful technology that has completely transformed biology and has equipped researchers with more knowledge about how cells work — and what happens when they don’t.
Read more about NGS:
Paving the way for NGS in biomarker testing