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EUCRAF POST--‐GRADUATE MASTER “REGULATORY AFFAIRS FOR BIOPHARMACEUTICALS”
Candidate name MichaelaNahler
Examinername Gab...
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the EUCRAF seminars and has put them into perspective of the HTA requirements. Very helpful are
the tabular listings of ...
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of 3

Nahler_MSc_Thesis_GDallmann_140507[1]-1

Published on: Mar 3, 2016
Source: www.slideshare.net


Transcripts - Nahler_MSc_Thesis_GDallmann_140507[1]-1

  • 1. 2 EUCRAF POST--‐GRADUATE MASTER “REGULATORY AFFAIRS FOR BIOPHARMACEUTICALS” Candidate name MichaelaNahler Examinername Gabriele Dallmann Examinercontact email details Gabriele.dallmann@eucraf.eu Title of the MSc thesis Health Technology Assessment requirements and the implications on Global Development of Biopharmaceuticals Assessment of the Master Thesis of Michaela Nahler Michaela Nahler has selected the topic of her Master Thesis related to a topic that is of high actual interest due to the importance the Health Technology Assessment (HTA) has for the definition of the price assigned to a new medicinal product. In contrast to the regulatory approval of novel medicines, which is performed on a commonly accepted legal and procedural basis, HTA is performed nationally. Mrs. Nahler has collected and reviewed the formal requirements for approval and HTA assessment, the parameters and the methods used in both areas. Approval identifies the benefit--‐risk ratio whereas HTA identifies the additional effectiveness, which is then used as the basis for the price negotiations. Approval is done centrally whereas the HTA bodies act nationally due to the autarkic function of the health systems of the EU member states. It is obvious that this separation of the two areas and mainly the national heterogeneity in HTA assessments create an additional hurdle to pharmaceutical companies. Furthermore, thinking globally, both, regulatory approval and HTA assessment is even more heterogeneous when companies want their products bring on the markets in other regions of the world. Another serious concern is that the collection of relevant information on the legal basis, methods, procedures, parameters etc. is extremely difficult as there is no common source of information available to which pharmaceutical companies can be referred to. In addition, due to the lack of harmonization or agreement on criteria, study design, parameters and comparators used to investigate clinical efficacy and effectiveness, a common scientific advice would be desirable which, however, is also not yet in place very effectively. Taking this situation, the Master thesis of Mrs. Nahler is thus an important work as it provides an excellent overview on the main details relevant for both areas, the regulatory approval and the HTA assessment. She has reviewed literature databanks and websites for the relevant information and has prepared a questionnaire to gather information on HTA in US, Canada, Brazil and Australia. While listing the main regulatory guidelines she has recollected the knowledge she has developed in
  • 2. 3 the EUCRAF seminars and has put them into perspective of the HTA requirements. Very helpful are the tabular listings of the benefit/risk vs relative effectiveness criteria. The structure is suitable and provides unique information. Mrs. Nahler has taken benefit from the opportunity to interview managers of the Merck Serono organisation in different countries of the world and the fact that Mercko Serono is actively involved in the alignment efforts at the industry side. The results of her review and the questionnaire are presented very clearly. Due to the high grade of uncertainty is this work a very valuable source of information to the reader and to the team at Merck Serono on: • Complexity of HTA across Europe and harmonisation efforts • Benefit--‐risk and relative effectiveness as key areas of interactions between regulatory authorities and HTA bodies • Regulatory requirements • HTA requirements • Benefit--‐risk vs. relative effectiveness assessment: agreements, deviations • Alignment efforts between regulators and HTA bodies (governmental harmonisation) • Alignment efforts from industry incl. an example from Global Headquarter of Merck Serono The topic of the Master Thesis is thus of high relevance and the Thesis could be used in many respect as an excellent reference to where we stand at the moment and what will be the most urgent activities to align the regulatory and HTA systems. The Master Thesis is very clearly structured and has been well edited. All references are adequately recorded and a comprehensive list of abbreviations provides the reader with all necessary explanations. Tables are well referred to in the text. Overall, the Master Thesis is a very good work as it is discussing a highly interesting topic, presents the scope and content in a very clear way and draws valid conclusions. Dr. Gabriele Dallmann Biopharmaceutical Expert EUCRAF Study Director
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