There is always an excited buzz around one or two hot topics at statistics conferences, and PSI 2023 was no exception. Previously, it’s been estimands, or real world evidence. This year we were all talking about HTA. But what is it? And why the buzz now?
According to the European Commission (EC), a health technology assessment (HTA) is a “procedure for assessing the added value, effectiveness, costs and broader impact of health care interventions including medicines, medical devices, surgical procedures”.1
The EC describe two HTA domains – clinical (relative clinical effectiveness, relative safety, description of health technology under assessment) and non-clinical (economic evaluation, ethical aspects, organizational aspects, social aspects, legal aspects).1 Methods and assessment criteria can vary across countries to suit the clinical and legal requirements leading to a large number of HTAs for a single medicine. There are important differences between regulatory assessment and HTA but perhaps a suitable analogy is that imagine you have created a new variety of strawberry (it’s Wimbledon season after all!). You develop it and the conclusion from your market research is that people like it! So you decide to try to sell it to a large supermarket. Now it needs to be better than your competitors, much better. Better on taste, better on price, as well as ensuring you’ve positioned your product in the right aisle. It’s not easy! The difference is in the product, strawberries compared to interventions, however both have the goal of ensuring patients get access to the best available option.
HTAs have been around for a long time so why the increased levels of interest now? In the EU, the European Medicines Agency (EMA) provides clinical assessment and regulatory approval for new medicines. All member states, or countries, have their own HTA process but soon the clinical aspect of HTAs will be harmonized and conducted by a Joint Scientific Consultation (JSC), followed by a Joint Clinical Assessment (JCA) coordinated by a consortium: EUnetHTA.2 The first JCA was published on 9th June 2023 and new oncology medicines and advanced therapy medicinal products will be assessed using the process from 12th January 2025 onwards.3 There will be many benefits and efficiencies in conducting a single assessment that encompasses the requirements of all the member states, as well as some challenges for drug companies, for example the dossier preparation will need to start earlier and the submission may require different resourcing strategies.
At the most recent PSI conference, various aspects of HTA were discussed in a TownHall meeting, invited talks, contributed sessions, an interactive session joint with the HTA European Special Interest Group and Launch and Lifecycle Special Interest Group, posters and with fellow conference attendees at coffee breaks. Discussions ranged from methodologies needed for JCAs, particularly around the specification of relevant PICOs (population, intervention, comparison, outcome), to statistical questions of multiplicity, treatment switching, indirect treatment comparisons and extrapolating survival curves to estimate long term treatment effects. While much of the discussion was on the EUnetHTA 21 guidelines, we heard about other HTA initiatives such as collaborations between NICE, Australia and Canada.
At Phastar we are committed to collaborating with our clients on their dossier preparations, maintaining our focus on quality.
References
- https://health.ec.europa.eu/system/files/2023-06/hta_regulation-implementation_factsheet_en.pdf
- https://www.ema.europa.eu/en/documents/presentation/presentation-european-network-health-technology-assessment-eunethta-health-technology-assessment_en.pdf
- https://www.eunethta.eu/publication-of-first-joint-clinical-assessment/
