31.2 million people across Europe suffer from depression. In some countries, close to 1 in 8 people are afflicted, a number which probably has been exacerbated by COVID-19. 20% of those affected  do not respond to current treatments, but potential new treatments under development reported in EFPIA’s 2022 Pipeline Review offer hope to those with hard to treat depressive disorders. Today there are over 280 clinical trials for so called psychoplastogens, which unlike traditional anti-depressants can have sustained positive effects following a single administration. Overall, they promise a paradigm shift in treatment of mental health disorders.

This is one striking example contained in the 2022 pipeline review of how research and development efforts under way in pharmaceutical companies are seeking to respond to unmet medical needs in patients in Europe and all over the world.

Some are for conditions that touch many of us, such as Alzheimer’s or cancer. Others are for conditions that touch fewer of us, such as ALS, but are equally  devastating. In some cases, the therapies in development are for diseases where there are currently no existing effective treatments, such as Alzheimer’s, and in other cases (such as for many cancers), new therapies can add new targeted tools and options that can be used by physicians when treating their patients, improving the outlook for many.

The Pipeline Review paints a picture of an exciting innovation landscape. The report’s description of the potential impact of these innovative treatments on healthcare systems and society is striking. But results of medical innovation are not just about the numbers, they are about the personal impact on the lives of people.

Take CAR-T therapies, for example. They are a form of advanced cell therapy  for blood cancers, that teaches a person's own immune system to fight diseases such as non-Hodgkin lymphoma, leukemia and multiple myeloma. The Pipeline Review reveals an impressive 621 clinical trials for CAR-T treatments - an increase of 185% since 2020! The results from different trials are remarkable. One trial show that patients are 2.5 times more likely to be alive at two years without cancer progression or need for additional cancer treatment. But the search for new and better treatments against cancer is about much more than CAR-Ts -  a new class of cancer immunotherapy called BiTEs could allow people to live longer, healthier, more productive lives, with significant downstream impacts on healthcare systems and society.

Another example is innovation in gene therapy. Gene therapy treats or prevents disease by correcting the underlying genetic problems that causes the disease instead of treating the symptoms through drugs or surgery. There are already seven gene therapy treatments available in the EU and with more than 500 clinical trials ongoing  worldwide, they could revolutionize treatment prospects for thousands of people living with rare diseases. For patients with Hemophilia A, a disease caused by a faulty gene, gene therapy can mean a radical life change. Instead of the unpleasant, weekly intravenous treatments that 75% of Hemophilia A patients need, gene therapy would be a one-time treatment that would substantially reduce spontaneous bleedings. In addition to the improved lives of patients, this would also reduce the pressure on  healthcare systems, and society would benefit as patients and their families would miss fewer days from work.

However, amidst the excitement there is cause for concern. A closer look at  exactly where pharmaceutical innovation is happening reveals that Europe is loosing ground. 25 years ago, new treatments from Europe far exceeded those developed in  the US. Today, in a complete reversal of the situation,  only 22% of new treatments originate from Europe, compared with 47% from the US. While we can and should celebrate  the 185% increase in clinical trials across the world for CAR-T therapies, only 2 out of 10 happen in Europe.

The worrying trend where more and more investments in pharmaceutical R&D are happening outside of Europe is bad for European patients. By participating in a clinical trial, patients take a more active role in their own health care, and they also gain early access to new treatments not yet available as standard of care. The investments in clincial development also nurture a broader public-private innovation eco-system of academic institutions, research hospitals and SME’s, and when this is weakened there is risk of creating a downward spiral, making Europe even less attractive for future medical research and innovation. The EU’s pharmaceutical strategy provides an opportunity for the political leaders of Europe to reverse this trend. First, Europe needs to maintain a robust framework for  Intellectual Property protection, which is the incentive sine qua non for private investments in lengthy and risky medical R&D. Reducing existing protection periods or making them conditional on issues that are far removed from the time and place where the original investment decision is made would further erode Europe’s innovation eco-system and send the wrong signals to the world about Europe’s future ambitions as a region where medical breakthroughs happen (and are not merely imported).  Secondly, the process for approving new medicines must sped up. At an average of 426 days to approve a new medicine, we’re trailing far behind the US (244), Canada (306) Japan (313) and Australia (315). The rapid and effective approval of COVID-19 vaccines showed us that a faster and more agile system is possible without compromising on safety and quality, and we should make sure to learn from this when updating, resourcing and future-proofing the EU regulatory system. Thirdly, we need new models and a better collaboration between industry, health systems and patients to improve access to medicines throughout Europe. More on that in a coming blog.

Our industry spends around €42 billion in research and development in Europe every year, and is the industry that spends most of its revenue on R&D overall, but in a world with increased global competition and rapid scientific developments we cannot rest on our laurels. We hope that policymakers and all stakeholders share our vision that Europe’s share of global R&D investments should increase, not decrease, and that this would be positive for patients, health systems and the broader society and economy. The EU pharma strategy and the upcoming revision of EU’s pharmaceutical legislation provides a once in a generation opportunity to start realising this vision. Let’s take it.