market authorisation . 5 , 8 , 9 EMA conditional market authorisation may be granted to medicines where the benefit of providing immediate access to the drug outweighs the risk of less comprehensive data . 8 The manufacturer is required to complete specific obligations such as ongoing or new studies to provide further data relevant to the product . 8 The EMA Adaptive Pathway builds on the conditional market authorisation approach and is specifically applied to therapeutic areas where there is a high unmet need . 9 In the United States , the FDA published the ‘ FDA ’ s Real-World Evidence Program ’ in late 2018 . This document outlines the methods and considerations to be used by the FDA to assess RWE for regulatory decisions . 6 This includes indication extension / modification , changing route of administration or dose and safety information . The guidance also included direction for industry on the use of electronic source data in clinical investigations , with the FDA acknowledging the huge potential to gather source data from technologies such as computers , wearables , mobile devices and biosensors . 9 The publication of the FDA ’ s RWE framework is expected to accelerate the use of RWE for approval and coverage decisions in the US .
HTAs in Europe France In France , RWE is gaining momentum as the Haute Autorité de Santé ( HAS – High Health Authority ) calls for more dynamic HTA ratings for high cost drugs . 10 In January 2020 , an action plan for the assessment of innovative medicines was announced ( although this is not yet a compulsory regulation ). Currently the plan applies to oncology as well as gene and cell therapy products but is likely to involve all innovative therapies once officially implemented . The rationale for such plan comes from the fact that innovative therapies are often given early access in France despite the large uncertainties and risks at the time of market authorisation . HAS noted that there is a shift in supplying evidence toward post-market authorisation reassessments . This highlights the growing need for the assessment of real-life outcomes ( effectiveness , true respondent
populations , toxicity , safety , impact on health care organisation and appropriate use ) where RWE will play a key role . HAS have stressed that the high prices of innovative drugs are a challenge and thus the use of RWE in post-market authorisation setting will help identify those truly innovative drugs .
Germany In January 2020 , IQWIG produced a report providing guidance on the types of RWE it will accept for value assessment and how to generate scientifically acceptable RWE . 11 The report provides detailed guidance on research methods and highlights that RWE should be produced in connection with the existing regulations ( GSAV law – law for more safety in the supply of medicines ). IQWIG also demonstrates a strong preference for data sourced from patient registries stating that data coming from electronic patient records and claims data from health insurance funds is not considered ‘ realistic ’. Overall , while RWE is receiving increasing support in Germany , IQWIG takes a conservative and restrictive position , staying closer to traditional study designs but still opening the door to RWE . This is demonstrated by IQWIG only considering RWE in comparison with the appropriate comparator therapy specified by the G-BA for benefit assessment . Thus , single arm studies will not be considered . This is in part due to the strict respect for the German legal market access framework and the legal framework under which IQWIG operates .
Italy Italy is a pioneer in the use of RWE in managedentry agreements , but scientific societies push for a more formal inclusion of RWE across all technologies . 12 This is highlighted in a position paper published by leading Italian scientific societies on the importance of RWE and urging the need to implement appropriate regulations for observational studies . 12 The aim of the paper was to help ensure the scientific and ethical quality of observational research through the review of the current authorisation pathway . The paper makes specific reference to the assessments introduced by AIFA for observational drug studies but broadens its
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