Development in harmony with official requirements

The pharmaceutical industry is the most regulated industry there is. Authorities in various countries oversee pharmaceutical companies in the development, testing, production and marketing/selling of their products. Before a pharmaceutical company can begin to market and sell its products, it must also obtain approval in the country in which the product is to be introduced.

As government authorities raise their requirements, pharmaceutical companies´ costs increase substantially, and the official requirements are often felt to be complicated and bureaucratic. While every research project is unique, government requirements are often general. In situations that are unclear, the simplest route is to fulfill all requirements that could conceivably be applicable. However, such a strategy increases bureaucracy. Active Biotech is a small company engaged in developing innovative drugs using limited resources in the shortest possible time, and is not in a position to choose such a strategy.

The fundamental principles of Regulatory & Quality Affairs

The main task of Regulatory & Quality Affairs is to ensure that all of Active Biotech´s activities comply with drug legislation while maintaining the capacity to effectively develop new drugs.

Government requirements and harmonisation

Legislation in the pharmaceutical area follows common principles, but varies among countries and regions. Initially, different sets of regulations were based on the same fundamental principles of quality, safety and effectiveness. In the 1960s and 1970s, however, national regulations were developed and divergent detailed requirements defined. As a result of the requirements on the pharmaceutical industry, many lengthy and expensive studies had to be duplicated to permit global marketing of new products. This was considered a problem, even by government authorities, the healthcare system and patients, who wanted speedy access to new, safe drugs.

The first step toward harmonisation was taken by the EEC in the 1980s. In 1990, the ICH was founded, a body that created a set of common rules for the collection and presentation of technical data. The work of the ICH is on-going, and has now reached the final stage of an agreement on the structure of a global registration application (CTD).

These harmonisation efforts are extremely helpful. However, much remains to be done before we may actually look ahead to a uniform global market.

Discovery phase

During the discovery phase, the drug authorities´ monitoring requirements are low. Most of Active Biotech´s research operations take place in laboratories and involve testing of new substances in test tubes. Animal trials only occur to a limited extent.

However, the company has high internal standards regarding its procedures and documentation, since they form the basis of patent applications. Active Biotech´s research and development files contain all research reports with references to the corresponding raw data.

Pre-clinical phase

Most pre-clinical-phase studies are carried out to investigate and document the safety of a substance. Drug authorities stipulate exactly what studies are required and how that are to be performed. Requirements include a quality assurance function that is independent of the units performing the study and ensures that all studies are carried out correctly and that reports correctly reflect the data generated.

The Golden Rule of every drug development program is to ensure that the individuals participating in the clinical studies are not exposed to improper risk and that their personal integrity is respected. This is achieved through pre-clinical safety tests that include toxicological and pharmacokinetic studies.

Clinical phase

Studies involving humans require prior permission from government authorities. Harmonisation is not as well advanced in this area as in the pre-clinical area. The EU recently announced a new directive regarding uniform rules. This particularly improves harmonisation with the US – although practical discrepancies will remain for a long time.

Clinical studies on humans are conducted in close cooperation with physicians and hospitals, and are carefully regulated. The quality assurance function examines and approves critical components of both the manufacture of the medicine and the implementation of the study.

If the initial studies yield favourable results, further studies are done involving increasingly large groups of patients. Each such progression requires a new application to the relevant authorities.

Registration phase

Once the clinical studies have been completed with positive results, all facts are compiled – that is, everything from the design and manufacturing of the new drug to safety tests on animals and investigations of effect and safety in clinical studies. This takes the form of an application for permission to market the new drug in a particular country. For the EU, it is possible to make a single application that applies to all member states.

Active Biotech´s registration strategy

Government requirements are designed to provide a basis for registration. Even if the develpopment chain is sequential, with clearly defined intermediate goals, the rules involved have not necessarily been devised to support the purpose of demonstrating proof of principle – that is, that the candidate drug has an effect on a particular biomarker.

Consequently, in cooperation with internal and external experts, and government authorities, Active Biotech designs project programs that make it possible to clearly test researchers´ hypotheses while focusing on patient safety. Accordingly, the company allows itself to postpone certain studies until the substance has demonstrated proof of principle. Active Biotech´s approach is, however, implemented in compliance with the applicable rules and guidelines, and meets registration requirements.

Projects well prepared

Active Biotech´s development strategy is based on independently driving the development of new products to the point of proof of principle. In each individual project, it focuses on relevant guidelines, taking the project´s current development phase into consideration. An overall development strategy that complies with applicable and anticipated government requirements is essential. This ensures that Active Biotech´s projects are well prepared for delivery to the next phase where they can be further developed in cooperation with partners.

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