Reading two thoughtful articles recently prompted me to muse on the ever-increasing overlap between regulation and health technology assessment (HTA).
The first
article explores the merits and potential pitfalls of hard-wiring regulatory and HTA processes in medicines as well as the geographical variation of adoption across the EU and speculates as to whether the regulatory changes will make the EU a more (or less) attractive place to introduce new oncology drugs. The second
article, in our own
Regulatory Rapporteur (
March 2025 issue) surveyed the practical impact of the recent changes in EU medicines regulation which introduced parallel and linked processes under the EU HTA regulation (HTAR).
There has long been a debate that formally connecting HTA to regulatory approval will accelerate market access for new medicines, whereas others believe that this link would create too large a hurdle and delay market introduction. For medical devices, the argument for separation is much stronger. In my view, the balance between pre- and post-market information needs is different for medical devices due to the iterative process of development and pricing being substantially more dynamic, so fixing HTA calculations is much more difficult. In both domains, local contexts need to be considered as the cost drivers can be substantially different from one country to another. Equally, health priorities and affordability play a role which means that centrally produced data may not automatically translate to simple decisions at a local level.
Regardless of which view you side with, regulatory professionals will increasingly need to understand and engage with HTA processes. Data relating to safety, performance, efficacy and cost-effectiveness is, and always has been, a continuum which generates evidence both for safety and cost-effectiveness from pre-market to real-world post-market. Efficient and effective evidence building requires these overlaps to be recognised and its utility maximised. This is no easy task, especially for large and complex organisations where specific activities along the product lifecycle are often quite siloed, while regulatory affairs and HTA might sit in completely different parts of the organisational structure. All of this may demand new approaches to structure and cross-cutting collaborations that may challenge the skills and knowledge of all concerned.
For medicines, new collaborative processes need to be developed to meet demanding timelines both in HTAR and the revised pharmaceuticals legislation. Meanwhile with medical devices, there is huge change continuing as medical device regulations and in vitro diagnostic regulations move into the latter phases of implementation, but surely questions will soon be asked about the relationship between devices and HTA. A ‘canary in the mine’ for future challenges lies in the handling of combination products where the various regimes come together in often uncomfortable ways. The discomfort may increase as digital technologies become more prevalent in combination with drugs, conventional devices and diagnostics.
So, what are the implications for TOPRA? Should we look to provide a broader offering for members in the domain of HTA? Or should we look to collaborate with organisations that can complement our in-house offerings?
To help us understand how we can support our members and the profession with these evolving issues, please share your views with us by emailing
publications@topra.org.