Scientific journals are crucial for reporting new research and assessing scientific breakthroughs. But imagine for a moment what would happen if a journal, like Nature, published national editions reporting different findings and conclusions for the same research. How would that be received?
The result would be an outcry in the academic community over scientific evidence effectively adapted to national preferences. Yet this absurd scenario is common practice when it comes to assessing the benefits of innovative medicines. More pertinently, it is happening with the so-called Health Technology Assessments (HTA) in EU Member States.
HTAs are supposed to systematically evaluate the relative effectiveness, cost-effectiveness and likely budgetary impact of health technologies. They cover medicines, medical devices, diagnostic tools and surgical procedures, as well as measures for disease prevention, diagnosis and treatment. But the way HTAs are conducted in EU Member States is sometimes questionable, in particular when it comes to innovative medicines.
Let’s use another analogy, this time to explain the role and importance of HTAs. Say you want to buy a new car. We can take as given that the cars on the market have all gone through rigorous checks to ensure they are safe to drive. But as a buyer, you will want additional information about things like fuel consumption, speed and how it compares to other cars in a variety of areas. Ideally, you wouldn’t have to do the work yourself, but rely on a neutral information provider (Step 1). However, taking a final informed purchase decision represents a different step. It requires a subjective evaluation and conclusion about the ability of the car to satisfy the buyer’s specific needs and preferences e.g. how it can fit and transport the family, and whether it is within the person’s budget in light of other priorities (Step 2).
For innovative medicines, HTA is focused on summarising information about the clinical benefits in a systematic, transparent, unbiased and robust manner. This scientific-clinical assessment can be likened to Step 1 in the car-purchasing process above. Based on that, healthcare systems and payers then take a value decision that resembles Step 2 of buying a car: is it worth paying for? In other words, should patients have access to the new medicine? This second part — assessing value and taking the decision— depends heavily on the specific context of a healthcare system. As these can vary widely from one country to the next, there are good reasons to leave final purchasing decisions to the individual Member States.
But the fact that scientific clinical benefit assessments of the same medicine conducted in parallel and based on the same evidence come to different conclusions across the EU-27 jeopardises the functioning of the Single Market. There should be a consensus on what constitutes good science in comparative clinical assessments of a health technology. Ideally, resources would be pooled across EU Member States so that medicines can be jointly assessed for their relative effectiveness on the basis of clinical evidence.
But this is not the case today. National HTA bodies conduct assessments of the same clinical evidence for a medicine in parallel to each other but reach different conclusions. For example, for oncology medicines the situation has been extensively researched and reported. The extent to which clinical evidence from oncology trials is considered robust or acceptable varies greatly between HTA bodies of different Member States. To illustrate, every national agency looks at the use of surrogate endpoints – a clinical trial endpoint used as a substitute for a direct measure of how a patient feels, functions, or survives – in a different way. These are accepted in Poland and often accepted in Sweden; not accepted in the Netherlands and often not accepted in Portugal. England and Italy determine acceptance on a case-by-case basis.
This is not just about diverse valuation criteria: some HTA bodies do not even apply the same basic assessment principles consistently to different medicines. Furthermore, many countries, notably some of the smaller EU Member States, lack the capacities and capabilities – staff, expertise and resources – to conduct high-quality HTAs. It is therefore unsurprising that these parallel assessments differ in their conclusions on the clinical benefit of new medicines. For patients, doctors and healthcare authorities, this bouquet of contradictory conclusions about the clinical outcomes of the very same medicine is confusing.
The costs of duplication
For taxpayers, the duplication of national processes is a waste of scarce resources. It can also contribute to substantial delays in pricing and reimbursement negotiations in Member States, which in turn delays accessibility of treatments that might bring highly relevant benefits to patients. Manufacturers seeking to introduce the same medicine in various EU countries lose time and money trying to satisfy an array of divergent and inconsistent requests for additional evidence (usually at a time when new clinical evidence can no longer be reaped from trials that ran their course). The result is, of course, higher costs. In the long run, this will hurt the EU’s competitiveness and dent its attractiveness for pharmaceutical innovation.
The European Commission has recognised this problem. In 2018, it proposed a EU HTA Regulation that would scrap parallel assessments by means of an obligation for Member States to use jointly established EU HTA reports. However, after more than three years of negotiations, the legislative compromise that has been established between the EU institutions substantially dilutes this obligation for Member States and comes with a range of unanswered questions. The coming years will show whether Member States are willing to let science lead the way.
As the health sector evolves and expands in the years to come, the EU will need to step up its collaborative efforts and remove market access barriers to innovative medicines, technologies and processes. European patients are put at risk when their access to medicines for unmet medical needs is delayed by unnecessary duplication of administrative procedures. Good science should know no borders.
National HTA bodies conduct assessments of the same clinical evidence for a medicine in parallel to each other but reach different conclusions.
For taxpayers, the duplication of national processes is a waste of scarce resources. Manufacturers seeking to introduce the same medicine in various EU countries lose time and money trying to satisfy an array of divergent and inconsistent requests for additional evidence.
