PHP134 Biosimilar Pricing: Past, Present and Future

Abstract

tions on epidemiological data; 12 specified that the uncertainty surrounding epidemiological data should be addressed, especially in terms of the transferability of international data. The party responsible for the conduct of the analysis (therefore responsible for providing the epidemiological data) was named by 20 guidelines, of which 14 explicitly referred to the marketing authorization holder. Furthermore, an acceptable level of evidence was mentioned only by 4 guidelines (Australia, Austria, Poland, and Scotland) and included surveys, registers, databases and experts' opinions. The relevance of epidemiological data for final reimbursement decisions was explicitly mentioned by 5 guidelines (Australia, Israel, Russia, Scotland, and South Africa). However, consequences for unacceptable epidemiological evidence (i.e., cases not following recommendations or requirements) were only indicated by Russia and Scotland in the form of refraining from giving positive reimbursement advice. CONCLUSIONS: Population-level epidemiological data is mentioned in 77% of the guidelines, but those focus mostly on issues of data transferability. Only few countries (19%) address the role of population-level epidemiological data for reimbursement decisions. To reduce decision uncertainty, approaches to address the often occurring paucity of epidemiological evidence should preferably be part of all pharmacoeconomic guidelines. OBJECTIVES:We investigated commonalities and significant differences in HTA evidence requirements in the EU5 (Germany, France, UK, Italy and France) and the implications on phase III programmes, focusing on three key, rapidly evolving elements of HTA: choice of the comparator, Health-Related Quality of Life (HRQL) and effectiveness. METHODS: In-depth, semi-structured 60-min telephone interviews were undertaken with 34 HTA experts across the EU5 (17 from HTA bodies and Academia, 17 from pharma). RESULTS: An active comparator in phase III is mandated or clearly preferred in all EU5 countries for HTA/payer purposes. It tends to be the therapy most commonly used in clinical practice, although best practice or cheapest therapy may be selected. Countries vary in the degree of flexibility in the approach to comparative evidence. UK appears to be the most flexible in the choice of comparator and acceptance of indirect evidence. Germany is the most demanding (e.g. head-to-head data vs. multiple, specific comparators for multiple sub-populations). HRQL is increasingly important in HTA, yet all respondents agreed that, in practice, HRQL is still only supportive to 'hard' end-points such as morbidity and mortality. Challenges in capturing HRQL as a measure of clinical benefit and/or a way to derive utilities, were discussed. Demonstrating effectiveness is becoming increasingly relevant to HTA, although efficacy data from phase III trials still have the major role -the greatest challenge is the generalisation of results to the wider, real-world patient population. Countries differ in their flexibility to addressing the demons...