Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Choice of study location for a First-in-Human study with a biological product - a critical decision for a biotech company

Marcus Baur (Abschlußjahr: 2012)

Language: English

Over the past few years, a lot of mergers and acquisitions have taken place in the pharmaceutical industry. As a result, there are more and more multinational companies established (not uncommonly “Global Players”) which have employees in various parts of the world. Thus, the different conditions and requirements for conducting Clinical Trials within these companies are well known at least in Europe and the US.

Nevertheless, if comparing the figures of First in Human (FiH) trials either on official websites (e.g. clinicaltrials.gov and EudraCT homepage) or by contacting pharmaceutical companies, two outcomes are very obvious:
  1. The majority of trials are conducted in the US, and
  2. Companies prefer to conduct the trials in the region of their origin.

First-in-Human (FiH) trial means that the new drug is tested in human beings for the first time and is the step out of preclinical testing to clinical development. For the research-based pharmaceutical company, this is a very important milestone as the outcome of this trial can be crucial to the further development of the product - or at the worst can signify the end. Therefore it is absolutely essential to conduct the trial according to the required standards and specifications and thus create reliable data.
In addition to the creation of quality, the researching companies have had to struggle against the steep increase of development costs within the last couple of years and this trend is set to continue. A third important point to consider in this special field of clinical development is the development time. Due to very aggressive generic companies in the market today, the researching companies have to compensate their development costs and generate income within the duration of the patent protection. The faster the development process, the longer they can profit from patent protection.

Since the TeGenero accident in 2006, there have been a lot of discussions about the conduct of FiH trials and how to avoid such disasters in the future. As a first step, the European Medicines Agency has created a new Guideline “to identify and mitigate risks” which can be seen as a very good basis for planning and conducting FiH trials.
Both regions, Europe and the US, are the most important regions for the pharmaceutical industry, both for volume of sales and research and development. With the high standards on both sides and ICH membership, there should only be if at all- very minor differences in requirements for conducting FiH trials. However, the United States seem to be the preferred location for pharmaceutical companies to conduct their FiH trials.

In order to investigate the companies choice of study location, this master thesis weighs up the most relevant criteria for decision making in both regions.
Europe as a region has a common economic area as well as a centralised approval body, the European Medicines Agency (EMA), which has authority to issue a Marketing Authorization for a new drug. Nevertheless, clinical trial authorizations are under the responsibility of the single member states and Europe as a whole cannot be compared with the US. Representative for Europe, a number of important FiH trial countries as well as the EMA and its Guidelines have been used for comparison.

A large part of this comparison rests on regulatory aspects like approval process and timelines on the part of Competent Authorities, liaising with authorities, regulatory guidelines and dossier requirements.

On the other hand there are important criteria from a clinical perspective like the selection of study site and investigator or just organizational criteria like drug supply which also play a significant role. In the end, a criteria that becomes more and more important are the costs for conducting trials.

The conclusions that could be drawn from this thesis were that neither of the two regions is superior to the other one and there are no regulatory reasons to preclude the conduct of a FiH trial study in one of the regions. The decision where to go for the respective trial is always a case-by-case decision that needs to be made within the project team.

Nevertheless, each sponsor should take the effort to perform a trial specific assessment of the existing requirements as the decision can be very trial- or company dependent.


Pages: 49

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