Regulatory requirements for the approval of medicines
Definition
In both the pharmaceutical and the biotech industry, the approval of a new medicine is subject to strict regulatory requirements (regulatory affairs). These are supervised by the authorities and reviewed at regular intervals. Especially in view of the frequent corporate collaborations in research and development (R&D), compliance with the specific requirements presents companies with a major challenge. Looking at the entire life cycle of a medicine, these issues become particularly apparent in the interplay between quality assurance, manufacturing and approval.1
Review of the documents
The main focus of the approval procedure is the review of the documents submitted by pharmaceutical companies, which are intended to demonstrate quality, efficacy and safety. Smooth document management is therefore essential for companies and their quality assurance. In addition to the required documents, the medicines authority also monitors the various phases of the clinical trials. These must comply with the prescribed testing guidelines for medicinal products, the recommended international guidelines and the rules of Good Practice (GxP), and should reflect the current state of scientific knowledge. Such verification is usually carried out through on-site inspections ordered by the authorities.2
Pharmaceutical quality
For approval, the medicinal product must be of an appropriate quality in accordance with recognised pharmaceutical rules. These regulatory requirements are laid down, among other places, in pharmacopoeia monographs. In addition to the composition of the medicinal product, the documents to be submitted also include detailed documentation of the entire manufacturing process, the control of starting materials, the packaging materials used, the intermediate and finished products, and the stability studies carried out. Furthermore, the manufacturer must obtain a manufacturing authorisation before a medicine is approved. The medicines must be manufactured in accordance with the rules of Good Manufacturing Practice (GMP). On-site inspections ordered by the authorities are carried out for verification purposes. If necessary, samples of the medicinal product may also be taken. These are tested for their pharmaceutical quality in the laboratories of the medicines authority.3
Pharmaceutical efficacy
To obtain approval for a medicine, adequate efficacy in the intended indication is also required. This refers to the probability that therapeutic or preventive results can be achieved with the medicinal product. The medicines undergo various studies, which must comply with the rules of Good Clinical Practice (GCP).4
Safety of medicinal products
The safety or potential harmfulness of a new medicine is also assessed before approval. The safety of a medicinal product must be demonstrated in clinical and non-clinical studies. Non-clinical testing includes a detailed determination of toxicity, which is carried out in suitable in vitro and animal studies. In clinical studies, all side effects and serious adverse outcomes are carefully documented. The formal framework for conducting such clinical and non-clinical studies, and for the corresponding documentation, is set by the rules of Good Laboratory Practice (GLP)5