Europe has already missed the boat on the last technology revolution, especially in pharma and ICT
Between 2014 and 2019, large European companies underperformed compared to companies from other regions in terms of for example [1]Â :Â
This gap is mainly caused by low performance in the tech-creating industries: pharmaceuticals and ICT. These two sectors have a strong interface, especially when it comes to biotech in general and ATMPs in particular. Europe may be strong in terms of biotech science, talent, and innovation, but is trailing in production and adoption. Â
Hence, it does not seem surprising that European patients are about to miss the boat on ATMPs
Let’s look at some numbers:Â
- Profitability –Â Europe is characterised by a high rate of ATMP marketing authorisations, but 1 out of 3 approved ATMPs were subsequently withdrawn from the market, all for commercial reasons; something which is not seen in the US for example.[2] [3] [4] [5] [6] [7] [8]Â Â
- R&D spending –
Europe underperforms compared to other regions in terms of the total number of ongoing industry-led clinical trials in 2022, as shown in Figure 1.[9]Â Â
Between 2014 and 2022, Europe’s share of newly initiated industry trials decreased from 21% to 13%, as shown in Figure 2.[9] [10] Â
Figure 1 Total number of ongoing ATMP-trials in Europe, Asia-Pacific and North America, at the end of June 2022 (Source: Alliance for Regenerative Medicine, 2022).
Figure 1 Total number of ongoing ATMP-trials in Europe, Asia-Pacific and North America, at the end of June 2022 (Source: Alliance for Regenerative Medicine, 2022).
The EU Joint Clinical Assessment is unlikely to stem the tide
Theoretically, the EU Joint Clinical Assessment (JCA) could help stem the tide, by aligning clinical evidence requirements and thereby accelerating reimbursement timelines and expanding reimbursement across (smaller) European countries.[11]
But … The challenge is that the JCA starts with those therapies for which clinical assessments tend to be inconclusive: ATMPs and oncology therapies.
Where traditional clinical assessments are meant to seek validity and certainty before reimbursement, ATMPs and oncology therapies are precisely the types of medicine that inherently come with thin data packages and clinical uncertainty stemming from methodological challenges, related to:
- Ethical problems related to setting up a control group, due to the life-threatening or highly debilitating nature of the disease (hampering comparison with a control group, or leading to cross-over from the control group to a follow-up treatment, hampering the demonstration of the significant difference between the groups)
- Very small trial populations due to the rarity of the disease (hampering a well-powered study for measuring a significant difference between the intervention group and the control group)
- Slow progression of the disease due to therapy efficacy and/or the nature of the disease (hampering data collection on final outcomes within the timespan of the trial)
In other words, when the JCA is based on the traditional framework of providing certainty on effectiveness and price before reimbursement, it will further delay reimbursement timelines in Europe.
The risk of moving towards the lowest common denominator
As part of harmonizing clinical evidence requirements in Europe, Member States are likely to be tempted to move towards the lowest common denominator, i.e., the most traditional assessment framework. This would annul some of the best practices developed so far, such as the automatic added benefit for orphan drugs in Germany (which acknowledges the methodological challenges related to small trial populations), or context-specific evidentiary requirements related to e.g. single-armed trials, indirect treatment comparisons, cross-over in trials, absence of statistical significance, or alternative endpoints.
A need for innovative pricing and reimbursement pathways
The JCA would not provide a solution to the most critical patient access barrier to ATMPs: innovative pricing and reimbursement pathways to ensure early patient access (adoption) while gathering real-world evidence and managing risks (ICT). This need is growing with the growing number of ATMPs being developed to treat chronic diseases. Â
But unfortunately, it’s exactly in the two performance areas of adopting biotech innovations and ICT that Europe is trailing…Â
What do you think? Will Europe miss the boat on ATMPs?
We’re happy to discuss in more detail how best to navigate this landscape, using:Â
- Early and cross-functional value & access strategies to address key barriers timelyÂ
- Our i-VBP model to price rightÂ
- Excellent value communication to start discussing anticipated barriers with stakeholders
- Feasible and impactful proposals for co-creating innovative access solutions
ABRREVIATIONS
ATMP – Advanced Therapeutic Medicinal Products
EU – European Union
ICT – Information and Communication Technology
JCA – Joint Clinical Assessment
P&R – Pricing & Reimbursement
REFERENCES
[1] McKinsey, 2022. Securing Europe’s competitiveness: Addressing its technology gap.
[2] EMA, 2013. Maci. Overview. Closure of EU manufacturing site
[5] EMA, 2017. Public Statement. Glybera. Expiry of the marketing authorisation in the European Union
[9] Alliance for Regenerative Medicine, 2022. Regenerative Medicine: The Pipeline Momentum Builds
[11] Inbeeo, 2022. How should pharmaceutical companies prepare for the upcoming joint EU HTA?
