- degree of EMA use of post-marketing authorization (post-launch) efficacy studies and adaptive licensing;
- degree of pan-European health technology assessment (HTA) body coordination in reviewing pre-launch evidence and demanding post-launch studies (for example, through the EUnetHTA initiative designed to reduce duplication);
- nature of regulator – health technology assessment body interaction.
The most likely scenario combining different pathways for these factors entails a dominant role for the regulatory body (EMA) as it becomes more involved in commissioning studies post-launch. This would stimulate further dialogue but not necessarily agreement between companies, HTA bodies and the EMA around post-launch relative effectiveness evidence. In relation to pre-launch requirements, there would be a degree of convergence in requirements as between HTA bodies and a greater dialogue between the EMA and HTA bodies as to what was expected at-launch. Together with some progress in the availability of electronic health records and of pan-European disease registries, there could be a very different expectation of and ability to supply relative effectiveness evidence.
In the second paper, focussing on the USA, the key factors influencing the future environment for generating and using comparative effectiveness evidence are the degree to which:
- integration of healthcare delivery occurs;
- electronic health records are made useful for research purposes;
- large databases and mixed data sources become tractable for CER and generating new insights;
- patient involvement in research increases.
Increased integration of the healthcare system, specifically the degree to which healthcare integration shifts risk acceptance for patient care from payers to provider organisations, is the key driver of the USA scenarios. The USA FDA was not seen as a key driver for change in the USA, unlike the EMA in the EU. No formal FDA policy changes are expected to impact expectations for comparative effectiveness during this period and the FDA was assumed not to coordinate scientific advice for drug developers with payers. In our scenarios, the USA makes greater progress than the EU in creating a data-rich environment and exploring the potential of ‘big data’.
In the third paper, comparing EU and the USA, the authors conclude that drug companies will conduct studies for relative/comparative effectiveness in both the USA and the EU. Companies will want to meet regulator and payer concerns through pre-launch studies where possible. However, an increasingly data-rich environment in the USA could promote use of post-launch observational studies aimed at payers, although the latter retain a strong preference for randomized controlled study evidence to support key elements of value. In Europe, likewise, while there is development in non-experimental methods, multi-country registries, and increased acceptance of observational data by payers and regulators, both prefer randomized controlled studies.
The papers were the results of a research collaboration between OHE and the Center for Medical Technology Policy (CMTP). The papers about EU and USA presenting plausible scenarios of future demand for and ability to produce relative effectiveness and comparative effectiveness evidence used semi-structured key informants interviews and three rounds of modified Delphi. The research was funded by a consortium of five pharmaceutical companies: Amgen, GSK, Lilly, Novartis, and Sanofi.
Posted in Drug Development/R&D, Health Technology Assessment, Pricing and Reimbursement | Tagged External publications