Transfusion medicine is the branch of medicine that encompasses all aspects of the transfusion of blood and blood components including aspects related to hemovigilance. It includes issues of blood donation, immunohematology and other laboratory testing for transfusion-transmitted diseases, management and monitoring of clinical transfusion practices, patient blood management, therapeutic apheresis, stem cell collections, cellular therapy, and coagulation. Laboratory management and understanding of state and federal regulations related to blood products are also a large part of the field.
Current good manufacturing practices (cGMP) are those conforming to the guidelines recommended by relevant agencies. Those agencies control the authorization and licensing of the manufacture and sale of food and beverages, cosmetics, pharmaceutical products, dietary supplements, and medical devices. These guidelines provide minimum requirements that a manufacturer must meet to assure that their products are consistently high in quality, from batch to batch, for their intended use. The rules that govern each industry may differ significantly; however, the main purpose of GMP is always to prevent harm from occurring to the end user. Additional tenets include ensuring the end product is free from contamination, that it is consistent in its manufacture, that its manufacture has been well documented, that personnel are well trained, and that the product has been checked for quality more than just at the end phase. GMP is typically ensured through the effective use of a quality management system (QMS).
The regulation of therapeutic goods, defined as drugs and therapeutic devices, varies by jurisdiction. In some countries, such as the United States, they are regulated at the national level by a single agency. In other jurisdictions they are regulated at the state level, or at both state and national levels by various bodies, as in Australia.
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or European Medicines Evaluation Agency (EMEA).
Natalizumab, sold under the brand name Tysabri among others, is a medication used to treat multiple sclerosis and Crohn's disease. It is a humanized monoclonal antibody against the cell adhesion molecule α4-integrin. It is given by intravenous infusion. The drug is believed to work by reducing the ability of inflammatory immune cells to attach to and pass through the cell layers lining the intestines and blood–brain barrier.
Cell therapy is a therapy in which viable cells are injected, grafted or implanted into a patient in order to effectuate a medicinal effect, for example, by transplanting T-cells capable of fighting cancer cells via cell-mediated immunity in the course of immunotherapy, or grafting stem cells to regenerate diseased tissues.
A bioprocess is a specific process that uses complete living cells or their components to obtain desired products.
A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, they include vaccines, whole blood, blood components, allergenics, somatic cells, gene therapies, tissues, recombinant therapeutic protein, and living medicines used in cell therapy. Biologics can be composed of sugars, proteins, nucleic acids, or complex combinations of these substances, or may be living cells or tissues. They are isolated from living sources—human, animal, plant, fungal, or microbial. They can be used in both human and animal medicine.
The process of establishing documentary evidence demonstrating that a procedure, process, or activity carried out in testing and then production maintains the desired level of compliance at all stages. In the pharmaceutical industry, it is very important that in addition to final testing and compliance of products, it is also assured that the process will consistently produce the expected results. The desired results are established in terms of specifications for outcome of the process. Qualification of systems and equipment is therefore a part of the process of validation. Validation is a requirement of food, drug and pharmaceutical regulating agencies such as the US FDA and their good manufacturing practices guidelines. Since a wide variety of procedures, processes, and activities need to be validated, the field of validation is divided into a number of subsections including the following:
The European Directorate for the Quality of Medicines & HealthCare (EDQM) is a Directorate of the Council of Europe that traces its origins and statutes to the Convention on the Elaboration of a European Pharmacopoeia.
A biosimilar is a biologic medical product that is almost an identical copy of an original product that is manufactured by a different company. Biosimilars are officially approved versions of original "innovator" products and can be manufactured when the original product's patent expires. Reference to the innovator product is an integral component of the approval.
Clinical quality management systems (CQMS) are systems used in the life sciences sector designed to manage quality management best practices throughout clinical research and clinical study management. A CQMS system is designed to manage all of the documents, activities, tasks, processes, quality events, relationships, audits and training that must be administered and controlled throughout the life of a clinical trial. The premise of a CQMS is to bring together the activities led by two sectors of clinical research, Clinical Quality and Clinical Operations, to facilitate cross-functional activities to improve efficiencies and transparency and to encourage the use of risk mitigation and risk management practices at the clinical study level.
Moxetumomab pasudotox, sold under the brand name Lumoxiti, is an anti-CD22 immunotoxin medication for the treatment of adults with relapsed or refractory hairy cell leukemia (HCL) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. Moxetumomab pasudotox is a CD22-directed cytotoxin and is the first of this type of treatment for adults with HCL. The drug consists of the binding fragment (Fv) of an anti-CD22 antibody fused to a toxin called PE38. This toxin is a 38 kDa fragment of Pseudomonas exotoxin A.
Limbal stem cells, also known as corneal epithelial stem cells, are unipotent stem cells located in the basal epithelial layer of the corneal limbus. They form the border between the cornea and the sclera. Characteristics of limbal stem cells include a slow turnover rate, high proliferative potential, clonogenicity, expression of stem cell markers, as well as the ability to regenerate the entire corneal epithelium. Limbal stem cell proliferation has the role of maintaining the cornea; for example, by replacing cells that are lost via tears. Additionally, these cells also prevent the conjunctival epithelial cells from migrating onto the surface of the cornea.
Non-biological Complex Drugs (NBCDs) are medical compounds that cannot be defined as small molecular, fully identifiable drugs with active pharmaceutical ingredients. They are highly complex and cannot be defined as biologicals as they are not derived from living materials. NBCDs are synthetic complex compounds and they contain non-homomolecular, closely related molecular structures with often nanoparticular properties. This is, for instance, the case with the iron sucrose and its similars. But also with other drug products, e.g. polypeptides (glatiramoids), swelling polymers, liposomes as the NBCD class is growing. Hence and due to their complexity and specific composition mix, such colloidal iron carbohydrate drugs cannot be fully identified, characterized, quantitated and/or described by physiochemical means to define their pharmaceutical properties. Therefore, contradictory to the generic paradigm pathway, relying on a full pharmaceutical identity and sameness in vitro evaluation exercise, they need additional evaluation with a reference product to assess comparability e.g. in tissue targeting in the body. This requires an appropriate, yet to be defined and be harmonized regulatory approach for these new class of medicinal products. The profile and the performance of NBCDs is defined by the multi-step manufacturing process, which is laborious, difficult to control and not disclosed by intellectual property. Minimal changes in for instance the starting materials or the process conditions might result in significant clinical differences affecting therapeutic effects or safety.
Pemigatinib, sold under the brand name Pemazyre, is an anti-cancer medication used for the treatment of bile duct cancer (cholangiocarcinoma). Pemigatinib works by blocking FGFR2 in tumor cells to prevent them from growing and spreading.
Advanced Therapy Medicinal Products, or ATMPs, are advanced therapeutic drugs that are based on cell therapy or gene therapy. The criteria to which a drug must conform to be classified as an ATMP, are defined in Article 17 of Regulation (EC) No 1394/2007 by the European Commission.
SCTbio is global contract development and manufacturing organization (CDMO) providing cGMP services of Advanced Therapy Medicinal Products (ATMPs). It operates in Europe and North America. The company has strong expertise in the development of autologous cell-based products, cell banking and all needle-to-needle GMP operations, including a validated apheresis collection sites network, product manufacturing, QC, GMP storage, QA/QP release, and worldwide drug products supply for clinical and commercial scale.
Tabelecleucel, sold under the brand name Ebvallo, is a medication used for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease. Tabelecleucel is an allogeneic, EBV-specific T-cell immunotherapy which targets and eliminates EBV-infected cells in a human leukocyte antigen (HLA)-restricted manner. It is made of cells of the immune system called T-cells that have been taken from the recipient (allogeneic) and are then mixed with EBV-infected B-cells from the same donor.
