Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

The Impact of the EMA-EUnetHTA Collaboration on Drug Development ***

Dr. Georg Tschank (Abschlußjahr: 2014)

Summary
Language: English
Current drug development occurs in the field of tension between the requirements of regulatory authorities in the context of licensing and health technology assessment (HTA) bodies in the context of reimbursement and pricing. Whereas the regulatory field is highly harmonized in Europe, HTA assessment is still done at the national level and Europe-wide efforts for harmonization in the HTA-field are just emerging. A major step towards this goal was the foundation of EUnetHTA in 2006, a voluntary network of national HTA bodies with the aim to develop methodologies and tools for standardized evaluation and reporting that allow the transnational sharing of HTA information. An important aspect of HTA is relative effectiveness assessment (REA) which seeks to determine the value of a medicinal product in relation to an established standard of care under real world conditions. In the field of REA there is big overlap between the activities of HTA bodies and Regulatory Authorities, especially in the context of so-called rapid REA which relates to HTA at the time of marketing authorisation. Here both Regulatory Authorities and HTA bodies use the same set of clinical data for their analysis. Accordingly, the collaboration between the EMA and EUnetHTA started out in 2010 as a project for the adaptation of the contents of the European Public Assessment Report (EPAR) to the needs of HTA bodies for REA. In the meanwhile the scope of the collaboration has been extended to all aspects of drug development including the evolving area of post authorisation evidence generation e.g. in the context of post-authorisation efficacy studies. A particularly important aspect of the collaboration is the procedure of Parallel Scientific Advice by the EMA and HTA-bodies. It serves as a hub for information sharing between the EMA, EUnetHTA and industry and thereby helps uncover areas for potential alignment and areas of conflict of interest. The three year joint work program between the EMA and EUnetHTA will end in 2015, as will EUnetHTA´s own work program “Joint Action 2”. In 2013 a European HTA Network has been established by the European Commission. This network has the task to elaborate a strategy for collaboration of HTA-bodies and EUnetHTA currently has the role of a scientific advisory platform. The future of EUnetHTA will depend on the availability of funding, possibly under the umbrella of HORIZON 2020.The EMA-EUnetHTA collaboration is embedded in a network of interactions between regulatory authorities and HTA bodies at all levels, national, European and international. This makes it sometimes difficult to sort out the specific effects of the EMA-EUnetHTA collaboration on drug development. Some of the joint projects of EMA and EUnetHTA are in the planning phase, others are pilots or have just moved into field testing. Nevertheless the EMA-EUnetHTA collaboration has already fulfilled an important part of its purpose: It has helped to detail the shortcomings of the current drug development model concerning a close collaboration of industry, regulatory authorities and HTA-bodies/payers and in addition it is proving that alternatives are possible. The EMA-EUnetHTA collaboration occurs at a time where the old drug development paradigm is fading because it is no longer sustainable for the following seasons: Industry is faced with a significant number of dropouts in late stage development and commercial failures causing a constant rise in development costs. Medical practice is adopting the concept of stratified/personalised medicine which leads to clinical development strategies dealing with smaller patient populations and narrower indications which can be commercially very risky. Payers and patients are gaining more and more influence on everyday health care practice.
At the same time the new pharmacovigilance legislation with the possibility to mandate post authorisation efficacy studies and regulatory strategies like adaptive licensing as well as pricing and reimbursement procedures according to the concept of coverage with evidence generation promote the concept of drug development as a process of continuous evidence generation. There is general agreement among all stakeholders that these challenges can only be mastered by close interaction of Regulatory Authorities, HTA-bodies and industry.
In this context the EMA-EUnetHTA collaboration is welcomed by industry as an import step towards the alignment of regulatory and HTA needs and expectations are high that it will become one of the driving forces for the creation of more sustainable ways of drug development and health care provision.
As many of these collaborative projects have been launched only recently their impact on drug development is difficult to assess, but industry is ready to adopt the idea of drug development as a process that covers the whole life cycle of a medicinal product where continuous generation of evidence is becoming a prerequisite to support a positive benefit/risk ratio and to justify reimbursement and pricing in an ever changing health care environment.
Pages:59