Regulatory Context

Biological medicinal products contain an active substance that results from a biological based production process or derived from a biological source, rather than being chemically synthesised. A combination of physicochemical and biological testing procedures are required for characterisation and the determination of quality of the substance. There are many types of biological medicines which range in complexity, diversity and innovation. Examples of biological medicines include:

  • Immunological medicinal products or vaccine
  • Medicinal products extracted from living systems, for example, derived from human blood and plasma, stem cell therapy or antibodies
  • Medicinal products developed by recombinant DNA technology, mimicking endogenous proteins, controlled expression of genes in prokaryotes and eukaryotes or hybrid cell lines and monoclonal antibody methods
  • Advanced therapy medicinal products such as gene therapy medicinal products

Regulatory Roadmap

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Key issues in this Sector

Immunogenicity and preclinical testing
There has been a focus on this issue since the TeGenero TGN1412 incident in 2006. Immunogenicity of the product in humans is a key issue with the impact being either neutralising through induction of antibodies after repeated administration or stimulating where the product is immunostimulating. Because the biological product may have limited activity in animals it can be difficult to produce a good candidate for preclinical testing with biotech products, especially if the product is human specific. Standard toxicology programmes are generally inappropriate and a prediction of immunogenicity is necessary.

A biosimilar is a biological medicinal product that is highly similar to another biological medicine which already has a marketing authorisation and has been appoved for use in patients (reference medicinal products). Biosimilars contain a version of the active substance of an already approved medicine. Biologicals are molecularly much more complex than small chemical molecules and this complexity creates a challenge for manufacturers looking to match a substance without access to the original developmental data. Additionally, as the complex proteins that comprise biologics are derived from genetically modified living organisms, biosimilars are more expensive than chemical drug ingredients and represent a bigger investment for companies. As patent expire on biological medicines increased numbers of biosimilars will likely come available in the coming years and thus there is an opportunity for companies to invest in this area. Real Regulatory are here to help guide and support companies who are looking to invest in this area.

Issues to be aware of

Raw materials during manufacture of animal or human origin
Assuring the viral safety of biotechnological medicinal products is a complex process and any materials of human or animal origin used as ingredients, excipients or during manufacture need to be assessed. Since the use of animal-derived materials is unavoidable for the production of some medicinal products and complete elimination of risk at source is rarely possible, the measures taken to manage the risk of transmitting animal Transmissible Spongiform Encephalopathies (TSEs) via medicinal products represent risk minimisation rather than risk elimination. Risk can be minimised by considering the source of the animals, the nature of the animal material, cross-contamination and production processes.

Risk management and pharmacovigilance
There is a greater need for customised risk management plans to play a vital role in maintaining safety standards for all biotech medicines. Key components of these risks include long stage follow up, assessment of immunogenicity and batch identification for adverse events among others.International Conference on Harmonisation (ICH) guidelines are designed for conventional products so risk management plans for biotech products must be designed on a case by case basis. Efficient planning for risk management and pharmacovigilance during preclinical stages should ensure that all issues can be anticipated in advance.

The most efficient route for your regulatory approval

Real Regulatory has up-to-date knowledge in key therapeutic areas throughout the product lifecycle and can assist you in dealing with current issues in drug development and in anticipating costly delays your application might encounter. Our highly experienced team of consultants can ensure you follow the most efficient route to regulatory approval for your biotech product.

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