PRO59 ANALYSIS OF THE G-BA BENEFIT ASSESSMENTS OF ORPHAN DRUGS AND GERMAN PAYER OPINIONS ON THE NEW "LAW FOR THE IMPROVEMENT OF SAFETY IN DRUG SUPPLY"

Abstract

Since 2011, manufacturers of orphan drugs with sales exceeding €50m have been required to undergo assessment by the Joint Federal Committee(G-BA). Currently, an “additional benefit” is assumed for orphan drugs by the G-BA; however, the “Law for improvement of safety in drug supply”, expected to be implemented in Jul-2019, is likely to impact the assessment. According to the Mar-2019 draft, the G-BA would be able to commit manufacturers to carry out data collection, if not enough evidence is available to assess the benefit at the time of the dossier submission. If no added benefit is demonstrated or no additional data is collected, then this can impact the to be agreed reimbursement price1. The research aimed to evaluate the rationale behind the benefit assessment ratings of orphan drugs and to explore payer’s opinions on the impact of this new law. All completed orphan drugs G-BA assessments between Jan-2011 to May-2019 were reviewed. Drugs receiving a non-quantifiable additional benefit were further studied to identify reasons behind their rating, using the G-BA justification of the resolution documentation. Furthermore, via the IPSOS Dimensions software, 38 payers in Germany were asked about their opinion on the new law, regarding orphan drugs. 115 orphan drug patient-populations were evaluated by the G-BA. 65 drugs were granted a non-quantifiable added benefit. The primary reasons observed were related to the study design, mainly the lack of a direct comparator(n=51) but also the short length of trials, small target population and lack of blinding. Moreover, the new law is expected to worsen the conditions for new orphan drug evaluations. The lack of a direct comparator was the main reason for unquantifiable additional benefit score in orphan indicated. The new law, which addresses the benefit assessment for orphan drugs, will likely add hurdles to manufacture’s developing orphan drugs, according to payers.