How aligned are the perspectives of EU regulators and HTA bodies? A comparative analysis of regulatory‐HTA parallel scientific advice

Abstract

BackgroundIn 2010, the European Medicines Agency (EMA) initiated a pilot project on parallel scientific advice with Health Technology Assessment bodies (HTABs) that allows manufacturers to receive simultaneous feedback from both the European Union (EU) regulators and HTABs on their development plans for medicines.AimsThe present retrospective qualitative analysis aimed to explore how the parallel scientific advice system is working and levels of commonality between the EU regulators and HTABs, and among HTABs, when applicants obtain parallel scientific advice from both a regulatory and an HTA perspective.MethodsWe analysed the minutes of discussion meetings held at the EMA between 2010, when parallel advice was launched, and 1 May 2015, when the cutoff date for data extraction was set. The analysis was based on predefined criteria and conducted at two different levels of comparison: the answers of the HTABs vs. those of the regulators, and between the answers of the participating HTA agencies.ResultsThe analysis was based on 31 procedures of parallel scientific advice. The level of full agreements was highest for questions on patient population (77%), while disagreements reached a peak for questions on the study comparator (30%). With regard to comparisons among HTABs, there was a high level of agreement for all domains.ConclusionsThere is evident commonality, in terms of evidence requirements between the EU regulators and participating HTABs, as well as among HTABs, on most aspects of clinical development. Indeed, regardless of the question content, the analysis showed that a high level of overall agreement was reached through the process of parallel scientific advice.

Inconsistent approaches of the G-BA regarding acceptance of primary study endpoints as being relevant to patients - an analysis of three disease areas: oncological, metabolic, and infectious diseases

“…Considering that the majority of these PEPs were accepted by regulatory authorities for marketing authorisation, the classification of such a large proportion of PEPs as not relevant to patients by the G-BA highlights marked differences in data interpretation by regulatory and health technology assessment (HTA) bodies. A similar finding was also demonstrated in a comparative analysis of parallel scientific advice of different European HTA bodies and the EMA [10]. …”

“…A recent publication has identified considerable heterogeneity in regulatory and HTA approaches, even among different European HTA bodies [10]. Regulatory authorities have adapted their approval pathways for innovative and promising new medicines to facilitate early patient access to new treatments, for example via conditional approval or adaptive pathways [10].…”

“…A recent publication has identified considerable heterogeneity in regulatory and HTA approaches, even among different European HTA bodies [10]. Regulatory authorities have adapted their approval pathways for innovative and promising new medicines to facilitate early patient access to new treatments, for example via conditional approval or adaptive pathways [10]. In order to effectively establish early access to medicines, HTA procedures need to follow the EMA’s footsteps and provide harmonised, transparent, flexible, conditional, and adaptive methods that adopt the level of evidence accepted by medicines agencies [10].…”

Between January 2009 and April 2016, 134 novel anticancer therapies were approved: what is the level of knowledge concerning the clinical benefit at the time of approval?

“…Further, the exchange of information, methodology and the joint development of tools on an European level between regulators (EMA), HTA networks (eg, European network of Health Technology Assessment, EUnetHTA) represented by scientific institutes (eg, Institute for Quality and Efficiency in Health Care, IQWiG; National Institute for Health and Care Excellence, NICE) can support the alignment of perspectives and sustainable evidence-based decisions for healthcare systems. 32 Our findings in combination with recent studies emphasise the need for a systematic tool to evaluate the benefit of novel drugs in a standardised and transparent way, as well as the importance of the systematic assessments of follow-up trials 3-6 years after approval of all anticancer drugs on a national level.…”

Challenges for Relative Effectiveness Assessment and Early Access of Cancer Immunotherapies in Europe

“…In general, the choice of adequate endpoints and comparators to support both (accelerated) assessment and (early) access to market is crucial in any drug development. The recent analyses of parallel EMA – HTA scientific advice with regard to requests for the trial population, study design, endpoints, and comparators, show an agreement, important for trial population and study design and rather weak for the choice of endpoints and comparator(s) (10) 6 ; on the contrary, the agreement among HTA bodies was rather high on most items (10). …”