The new EFPIA Pipeline Review reveals that infectious, inflammatory, cancer, Alzheimer’s and rare disesases areas are among the top priorities of researchers running clinical trials today to investigate new medicines and vaccines. The comprehensive report found that in 2020 alone, around 5,000 clinical trials were launched – despite the disruption caused by the COVID-19 pandemic. In fact, the volume of trials has increased over the past five years.  

The Review paints a picture of a healthy innovative pipeline, focused on major unmet needs: 40% of trials are on substances targeting rare diseases, while ground-breaking cell and gene therapies continue to grow in importance.  

The example of gene therapies is particularly inspiring. By replacing defective or missing genes, scientists aim to cure life-long and highly debilitating diseases such as haemophilia. CAR-T treatments, a type of cell therapy which are already transforming the lives of people with blood cancers, are now being studied also against solid tumours. This could be a future game-changer for many forms of cancer.  

The report also points to progress on Alzheimer’s treatments which researchers hope will delay the onset and progression of dementia; remyelinating therapies with the potential to restore mobility and vision in people with multiple sclerosis; and curative therapies for HIV and hepatitis B. And let’s not forget breakthrough mRNA technologies which have given us the first COVID-19 vaccines and could one day be used to treat aggressive brain cancer.  

Preparing for the future 

The Pipeline Review surely inspires optimism about the overall trajectory of medical innovation and comes with a caveat. Our capacity to continue to develop and deploy the results of these research efforts requires fresh thinking about Europe’s research ecosystem and healthcare infrastructure. Many times, however, clinical trials which are both vital for the research eco-system as well as the first chance for many patients to get access to ground-breaking therapies, do not take place in Europe, but in the U.S., China and other regions. While it is clear that past innovations have improved patient outcomes, and the coming wave of advances can go much further, now is the moment for preparation as well as celebration.  

The report identifies areas where industry can work with other stakeholders to pave the way for the new therapies and vaccines that the pipeline can deliver. It is a recipe for making Europe a global leader in medical innovation while ensuring that our patients have access to interventions that could improve – or save – their lives.  

For a start, we need to adapt our regulatory processes to better suit the kinds of therapies coming down the track. The clinical development and production of the new advanced therapies on the horizon require new approval pathways.  

Equally, today’s approaches to Health Technology Assessment (HTA) and pricing and payment may be ill-suited to tomorrow’s therapies. New approaches to funding truly innovative treatments will be essential to their use, as well as robust systems for data capture to monitor the effectiveness of new treatments in real life.  

Together, we also need to think about healthcare infrastructure and diagnostic capacity to prepare the ground for therapies that change the current treatment paradigm.  The first generation of CAR-Ts, for example, involve harvesting cells from a patient, transporting them to a lab for processing, and returning them to the patient. To do this in a timely manner, we need to integrate care and manufacturing facilities. New Alzheimer’s therapies would require capacity and procedures including neuroimaging to correctly diagnose patients at an early enough stage.  

Collaboration is key 

Reading the report, I was also struck by how many of these improvements will require partnership. It is a recurring theme in every chapter – from regulatory reform and new financing schemes to horizon scanning and priority setting. Complex challenges require collaboration between a constellation of healthcare actors.