The question of how a novel therapy can be optimally positioned within a specific national market is addressed differently by different national healthcare systems. This is the only means by which both the manufacturer and all stakeholders involved can ensure that they strike a fair balance with regard to their expectations. This process not only includes having a precise knowledge of national regulations but also demands optimal planning of the scientific programmes used to deliver the results required for different phases of a novel drug’s life cycle. Dialogue with payers and other stakeholders as well as knowledge of national evaluation systems and markets are essential.
The health systems of the individual EU countries face the challenge of providing patients with access to new treatment methods within their limited budgets. The evaluation of the added value of an innovation is becoming more and more important and is carried out from the perspective of the various political and social structures of a country. Methods of Health Technology Assessment (HTA) are used. The results determine the reimbursement of new products and thus their financial impact on a healthcare system. Notwithstanding Europe-wide efforts to harmonize HTA methods, knowledge of country-specific pricing and reimbursement regulations will continue to be important.
Economic as well as clinical aspects must always be taken into account when making decisions about market access and negotiating pricing of therapies. The main challenge is to extrapolate data from small study populations with a short study duration to cover the entire population over a much longer period of time. In this context the use of health economic models is crucial for obtaining satisfactory results, but only possible in the hands of experts.
Real World Evidence & CRO Services
Real world Evidence (RWE) results from the evaluation of data obtained in real healthcare situations. In addition to clinical studies, it is becoming increasingly important to answer scientific questions regarding new therapies. RWE has therefore become an essential tool to demonstrate the value of a new drug in the context of approval and benefit assessment. Once a new treatment has arrived in everyday care, RWE helps to show the perspective of patients and the impact on professional care, which is relevant for insurance funds. RWE‘s generation requires a precise overview of which stakeholder needs which information at which point in time. Planning and efficient implementation of such studies is essential and is supported by close cooperation with a CRO.