Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Extended Role of Scientific Advice as a Consequence of the New Medicines Legislation in the EU including a Comparison to the Situation in the USA ***

Ellen Güttler (Abschlußjahr: 2006)

Language: English

The meaning of scientific advice has rapidly increased since this initiative exists. Upon the New Medicines Legislation (NML), the EMEA for instance extended its task list and, due to an opening up to the public and the transparency initiative, the scientific advice procedure now should also involve groups outside the regulatory environment, such as public health institutions or patient organisations.

Looking at the United States (US), the role of SA has been standardised and established in the legislative earlier than in the European Union (EU). Especially the Prescription Drug User Fee Act (PDUFA) enhances the meaning of SA during the process of preparation of a New Drug Application (NDA) and lays down the right of companies to obtain it.

The scope of this thesis is to demonstrate the role of SA for drug development and subsequent Marketing Authorisation procedures, especially in the EU and US markets: The impact of the new NML and the resulting changes in SA activities should be shown. The most important procedures and types of SA should be described. The main focus of description is laid on the EU, because the role of SA increased after the NML implementation. In the discussion a comparison to the situation in the US will be provided.

The EMEA has provided SA to sponsors since 1996. The introduction of the new EU medicines legislation provides a grater mandate to give SA particularly regarding the development of new therapies with direct and clearly distributed responsibilities and effective structures for the provision of SA by the SAWP and its experts network. The new framework for SA allows for earlier and greater involvement of internal and external assessors. Therefore it is made possible to streamline the procedure to allow finalisation within maximally 70 days or earlier. Moreover the involvement of the CHMP will be consolidated by formalising of a peer review to ensure consistency in the provision of SA. More interaction and communication is included with stakeholders, through the extended scope and increased use of follow- up procedures, publication of standard Q&A documents and further measures to develop and improve existing guidelines, especially for rapidly evolving topics. Moreover, new measures such as incentives to SMEs, additional specific expertise and new competence are put in place to follow the extended scope as defined by the new regulation. In the context of cooperation with other non EU authorities EMEA and FDA have agreed upon a programme for companies to obtain parallel or joint SA from the 2 agencies. The parallel SA procedure includes a mechanism for the EMEA, FDA and companies to exchange views on scientific issues during the development phase of new breakthrough drugs, with a positive adoption from the industry as well as from the agencies.

SA in the USA is linked to different sorts of milestone meetings with the FDA, which are displayed in sections 5.2 and 5.3. The PDUFA initiative provides a framework for specific SA procedures (as outlined in table 3) such as SPA, SA in the setting of biotechnology procedures including independent consultants and the frequent scientific interaction in the scope of CMA (pilot 2), which are restricted to PDUFA products. For both regions scientific advice is the earliest source for upcoming scientific issues to be clarified in guidance documents with the examples for GT products and biosimilars or follow-on proteins. The same examples also show on the other hand that a different value and political and historical issues lead to different legal classification of certain types of biotechnology drugs, resulting in different possibilities for these drugs to be subject of SA procedures. In the discussion a comparison is provided, listing the main differences for SA in both regions.

Pages: 54, incl. Annexes: pages : 06