To ensure consistency in the quality, safety, and efficacy of medicinal products derived from complex biological materials, emphasis must be placed on their standardized production and quality control testing. The establishment of general requirements applicable across a diverse range of product classes governing starting materials, manufacturing, and regulatory oversight is an essential aspect of this process.
Since the middle of the 20th century, medical science has found ways to prepare therapeutic products derived from human blood and plasma for the treatment of many life threatening diseases, as well as for complex surgical procedures.
Processing blood into various types of medicines or products is a highly specialized process because blood products and biological technologies are inherently variable due to the nature of the source materials as well as the methods to test them. Only blood products of demonstrated quality, safety and efficacy should be used, which is the overall goal of the National Regulatory Authorities (NRA) worldwide.
Cell and Tissues
Well-characterized cells and tissues are employed not only as substrates to produce other biological products such as vaccines, but increasingly as therapeutic products themselves. As growth substrates, the uniformity of these materials and their freedom from adventitious agents is essential. As therapeutic products, especially using recent advances in genetic engineering, they can provide much more targeted and compatible interventions than the chemical and mechanical techniques available until now.
Cytokines are proteins which mediate and regulate immunity, inflammation, and haematopoiesis. One important group of cytokines regulates immune cell proliferation and differentiation, important therapeutic targets for a variety of proliferative diseases, and another group, that includes the interferons, mediates the cellular responses to infections. The large number of cytokines, their short half life in plasma, and the redundancy of their effects make this complex family of therapeutic products one of the most challenging and promising in the field of medicine.
National Regulatory Agencies (NRA) are responsible for evaluation, control and approval of certain in vitro diagnostic devices (IVD). WHO provides international biological reference standards for applications in the standardization of materials and approaches used in medical diagnostics such as diagnosing disease, monitoring therapy, blood safety, and public health applications (e.g. monitoring immune status, screening for disease or susceptibility) or otherwise characterizing biological material from individuals. The current focus of WHO's work in this area is for IVDs that are either used in blood screening, clinical diagnosis or plasma pool testing prior to entering the blood fractionation process.
Biological products once prepared in minute amounts from crude biological materials can increasingly be produced on well characterized yeast, bacterial, or mammalian cell lines transfected with the recombinant DNA of the target protein. DNA and RNA itself are also being developed as therapeutic agents, such as in the fields of gene therapy and nucleic acid vaccines. This area of work concentrates on defining the quality and safety aspects of this emerging but very promising new area in the field of biologics.
Preventive vaccines remain one of the most cost-effective and efficient means of combating diseases, and in certain cases such as smallpox and polio, vaccination can result in the complete eradication of a major human health threat. Transmissible diseases continue to claim millions of lives yearly, and the development of new vaccines for old and emerging pathogens, new antigen combinations, and new methods to improve their quality, potency, safety, and efficacy remains one of the major challenges and promises in the field of public health.
WHO Implementation Workshop: Characterization of cell banks for the production of biologicals, 27-29 May 2013 and WHO Informal Consultation on regulatory risk assessment in the case of adventitious agent finding in a marketed vaccine, 30-31 May 2013, Beijing, Republic of China
WHO Informal Consultation on the Scientific and Regulatory Considerations on the Stability Evaluation of Vaccines under a Controlled Temperature Chain, Langen, Germany, 4-6 June 2013
Expert Committee on Biological Standardization, Geneva, Switzerland, 21-25 October 2013
Quality, Safety and Standards (QSS)
Immunization, Vaccines and Biologicals (IVB)
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