Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Scientific Advice - Value Adding Strategies for Regulatory Agencies and Pharmaceutical Companies ***

Dr. Bianca Krafft (Abschlußjahr: 2007)

Language: English

During the lifecycle of a medicinal product Scientific Advice (SA) is a vital strategic regulatory tool. The competitive global environment and high development costs justify the demand for an efficient global drug development programme appropriately proving safety and efficacy thus providing access to all major markets. Essentially, SA meetings maximise the chance of securing subsequent approval and market access but also maximise the quality of the approval and Time to Market (TtM). On the other hand such meetings can minimise the costs of development as they help to avoid conducting clinical studies which are not necessarily needed and/or flawed (or incomplete) studies which need to be repeated.

In defining a global regulatory strategy for a project, SA at the appropriate time is a key element in order to establish and validate the project-specific strategy. To create such global strategy, different aspects need to be taken into consideration. Hard facts or constrains like legal requirements, regulatory guidelines and SA already received, build the framework for the global regulatory strategy. However, in the context of SA not only the hard, regulatory and legal facts come into play but also soft skills like communication, negotiation, strategic thinking, planning ahead and assuming what regulators think and want. The Regulatory Affairs Manager in charge of the SA is the key contact person who mediates and translates between the company and the regulatory authority. The preparation and conduct of the SA procedure requires thorough planning and coordination by the responsible Regulatory Affairs Manager and the product development team. Particular emphasis needs to be given to timing, request, Briefing Book, rehearsal, debriefing, minutes, interpretation and implementation of results but also possible follow-up advice. The SA procedure generally is a way of close interaction between the sponsor/pharmaceutical company and the respective national Competent Authority (CA) or responsible Ministry of Health (MoH). Not only during the initial development phase, but also after licensure when line extensions (i.e. new indications, new dosage forms) or expansion of the existing license (i.e. second or third wave in MRPs) are planned, the opportunity to meet with the responsible CAs via SA in advance can be vital to ensure positive outcome of the later MAA/NDA/BLA and early market entry as planned.

SA is particularly key to clinical development; as it enables the generation of a prediscussed dataset and improves the chance of meeting regulatory needs to support Marketing Authorisation Application (MAA) / New Drug Application (NDA) / Biologics License Application (BLA) approval later. From experience there is clearly a positive correlation between prior SA and eventual later MAA/NDA/BLA success. Generally, companies should always establish and maintain credibility. Much of the help authorities offer to a sponsor, like SA meetings, often relies only on the good will and courtesy of the authority. The granting of SA meetings by countries where no national, legal obligation to offer SA exists is one example.

In a SA meeting the sponsor/company should demonstrate profound knowledge of the drug under development and of the development process. Companies should interact and respond to authorities with honesty in a timely fashion and as complete as possible. One of the most frequent reasons for seeking SA is "programme approval", to get an answer to the question "Is what we are planning to do OK?".

During the SA meetings questions concerning the details of the entire process can be adressed. Among others, these can concern:

  • future pharmaceutical development
  • later licensure
  • regulatory procedures / submission strategy
  • clinical protocol design
  • statistical analysis
  • clinical endpoints
  • manufacturing
  • labelling


To visualise and summarise the various different impacts on a SA meeting and its success, Figure 1 below has been created.
This thesis does not focus on the citation and discussion of regulatory guidelines and legal requirements regarding SA, but has an emphasis on demonstrating how industry uses strategic SA based on "hard facts" in combination with data and "soft facts" to create a win-win situation.

Pages: 48 Annexes, Pages: 7

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