The research-based pharmaceutical industry is looking at the draft of pharmaceutical legislation presented by the European Commission in April this year with some concerns. In their view, the package would accelerate Europe’s decline in R&D investments, reducing the availability of innovative therapies for patients in the region. In an interview with the Healthcare Daily, which took place on the sidelines of the international conference „Value and Role of Incentives in Life Sciences,“ Nathalie Moll, Director General of the European Federation of Pharmaceutical Companies and Associations (EFPIA), shared her thoughts.
In a recent interview for the Healthcare Daily, the director of the AIFP, David Kolář, described the proposed pharmaceutical package as the way of a stickrather than a carrot. How do you see it?
I always go back to what this legislation was created to do in the 1960s. Its aim was to ensure the safety of all medicines in Europe. When it was revised in the early 2000s, elements of incentives were added to encourage research in areas where there was less. Now attempts to address access is being added, even though access is a national competence, making its inclusion in European legislation problematic and likely to fail.
As for what David Kolář said, the idea that enforcing legislation would stimulate innovation to fix patient access is very optimistic – it is not the right tool. Access is a complicated, multifaceted, and different reality in each Member State, and it is too simplistic to try to legislate for it at the European level by adapting instruments that were designed to promote research.
How likely is it that there would be a mass exodus of manufacturers of innovative medicines if the proposal were adopted in its current form?
I don’t think this is a mass exodus, but a gradual decline that this new legislation is likely to accelerate. It is a trend that has been ongoing for the last 20 years. Twenty years ago, the US spent two billion more on research and development than Europe, and today it is 25 billion – Europe’s share of clinical trials has also fallen.
Advanced therapies in Europe are a third of those in China in terms of clinical trials. And over the last 20 years, we have lost 1/4 of global investment in research and development.
According to the recently published Dolon report, we would lose another third of our research base to the Commission’s proposals, which would mean a loss of EUR 2 billion in R&D investment in Europe every year. So, it will not be an exodus, but a clear acceleration of existing trends.
The European Commission has repeatedly called for more EU medicine self-sufficiency. To what extent do you think it is actually able to deliver this, for example with regard to its environmental objectives?
If we want to be self-sufficient and have medicines, we need innovation. Resilience starts with research.
We need to find solutions that do not hamper our ability to research and develop new treatments and vaccines here in Europe. That is the concern of the innovative industry. We are fortunate because around 64% of active substances come from Europe, including Switzerland and the UK, so we are less dependent on the rest of the world.
If you look at the environmental aspects within the EU’s Green Deal policy, there are some concerns, such as the ban on PFAS chemicals. This would really impact medicine production in the EU, causing it to stop within a few years which in turn would create a huge dependency on the rest of the world, going against the EU policy of Open Strategic Autonomy.
We need to have the right pragmatic policies, including the European list of critical medicines, to reduce dependence on countries outside the EU.
First amendments to the pharmaceutical package have already appeared in the European Parliament, for example, to remove the conditioned duration of regulatory data protection. How confident are you that the final proposal will meet your expectations?
It is more about meeting the objectives of the EU Pharmaceutical Strategy – generating faster, more equitable access to medicines across Europe and supporting the industry in Europe to be a world leader in medical innovation. That is the opportunity in front of us. Members of the European Parliament are already reflecting on the Commission’s proposals trying to assess if they move us towards or away from those two goals we all share. Our hope is that the amendments can help return Europe to the forefront of medical research. We need everyone involved in getting medicines to patients around the table to find some evidenced based solutions to the access problems now, not in five years via legislation.
Your organization has commissioned an analysis of the impact of the draft pharmaceutical legislation, and its results are said to be significantly different from those commissioned by the European Commission. What are the main differences between these analyses, and how do you explain them?
The European Commission has not conducted a competitiveness check. It has assessed the impact, how much it would cost the innovative industry, and how much the generic industry and the Member States would gain. It is a very superficial assessment and does not address the impact on competitiveness, the ecosystem where innovation is created, small and medium-sized enterprises, access, and the environmental proposals. It is dangerously simplistic and because of that it is misleading policy making.
What are the main conclusions from your analysis?
According to our analysis, investment in R&D, which we have already lost by 25% in the last 20 years, would fall by another third. About 32% of global investment now comes to Europe, and by 2040, we would be at 21%. We would also lose 2 billion in research and development investment a year, which is a huge amount – and almost all Member States would be impacted.
Around a third of new medicines rely on regulatory data protection – an important part of last stage intellectual property. Often these are the most innovative products. The research from Dolon shows that around one in five of projects to research these medicines (22%) would no longer be economically viable in Europe.
And what do you expect to be the impact on medical treatments?
We could see the research and development of around 50 out of 225 expected new treatments foregone over the next 15 years – medicines which may not be researched elsewhere. This equates to around 50 of the 225 treatments expected over the next 15 years.
Also, 9 out of 10 projects in small and medium-sized enterprises that depend on regulatory data protection would be at risk, and according to these projections, we would lose 45 treatments for rare diseases that may not be developed elsewhere.
It is really worrying. And even more troubling is that there has been no formal review of institutional competitiveness. We can offer our numbers, but ideally, institutions should conduct their own studies before making such important decisions that will affect Europe for the next 20 years.
Extremely expensive innovative drugs are appearing on the market. For example, in the Czech Republic a drug costing 100 million crowns recently caused a stir. In your opinion, should the pharmaceutical industry be more transparent about the pricing of such expensive drugs if it wants the public system to pay for them?
The reality is that over the last 20 years spending on medicines by health systems has remained at around 15% of healthcare costs. The data tells us that new medicines have not been the principal driver, but are actually part of the solution to help health system cope with an ageing population and increased levels of chronic disease and increasing demand on health services.
We need to consider the case you mentioned in the broader context of how many patients it benefits and what health technology assessments have been conducted for a particular product. In Europe, we have a system in place where health technology assessments ensure that cost and clinical effectiveness criteria align. Innovative medicines often also contribute to cost reduction. For example, when you use an innovative medicine, you may not need to undergo a transplant or surgery. It’s not so much about the price of the medicine but rather its value to the healthcare system.
So what do innovative manufacturers propose?
Aware of the existing inequalities to access to innovative medicines across Europe, EFPIA members have taken concrete actions and made proposals to reduce unavailability and access delays. These include a commitment to file for pricing and reimbursement in all 27 EU countries within 2 years of marketing authorization and measuring that commitment through a portal as well as proposing a conceptual framework for Equity-Based Tiered Pricing (EBTP), to ensure that countries that can afford less pay less, and anchored in a principle of solidarity between countries. Also, novel payment and pricing models, when used appropriately and tailored to the situation, can accelerate patient access, allowing payers to manage clinical uncertainty, budget impact and sustainability of the healthcare system, whilst providing sufficient incentives for innovation.
Filip Krumphanzl
