Patient-centered innovation: Can Europe lead the way?

Since a young age growing up in Italy, the workings of the human body have fascinated me — a curiosity that brought me to medical research. I’ve always wanted to help people. And through discovering new medicines, we can potentially help millions of patients.

What drives me — and so many other scientists in Europe and around the globe — is a desire to find new therapies that will improve people’s lives, especially in areas where there is currently an unmet need.

As a personal example, my passion for psychiatric diseases stems from witnessing the profound suffering they cause not just to the patient, but also within families. The global rise of mental health illnesses deeply concerns me and demands urgent attention.

Focusing on what matters to patients

In current European policy discussions, the term ‘unmet medical need’ is often defined by a simple question: Will the treatment keep the person alive for longer? Survival is obviously an important marker, but it is not the only one. The burden of the disease and the patient’s quality of life must be considered too.

For example, a skin disease might not be life-threatening or affect a person’s ability to function autonomously, but it can have an enormous impact on their mental state and productivity.

There are many layers to the burden of a disease on a personal and emotional level.

At the company I work for, Boehringer Ingelheim, we look at an unmet need in its totality to help us make decisions about where to focus our research and development (R&D).

We work with patients at every possible step of the R&D process, helping us to incorporate their feedback, and to focus on what the actual unmet patient need is.

We can’t make true progress on unmet needs unless we understand from the patients themselves what they need or what they think is missing from their treatment options.

This is why I am concerned about the European Commission’s proposals in the EU pharma package regarding unmet medical need. As currently written, the definition is too narrow and fails to reflect the diverse challenges faced by patients. This limited scope will not only leave many health conditions unaddressed, it also risks stifling innovation by discouraging R&D efforts aimed at tackling the broader, more complex needs of patients.

For example, an injection that can be done at home rather than in a hospital or a pill replacing an injection could make a world of difference for patients. For people with scleroderma, who often suffer from swelling and limited mobility in their fingers, a tool that assists in removing a pill from its packaging could significantly enhance their independence and ease of medication management, improving both comfort and quality of life.

By narrowing the concept of unmet medical need to strict metrics, we risk drifting away from real patient need.

Advances in science will guide our decisions on where to invest in R&D

At Boehringer Ingelheim, we look for areas of unmet patient need, how they have changed and progressed and where we might enter or leave an area of research, either because the science has advanced, or because there are already treatments in the pipeline.

We use the World Health Organization’s International Statistical Classification of Diseases and Related Health Problems — which categorizes thousands of disease areas, from cancer to infectious diseases. We look at where the breakthrough science matches our capabilities and expertise and what patients need the most.

For Boehringer Ingelheim, this includes, for example, oncology, inflammatory diseases and serious mental health illnesses, as well as research into the broader cardio-renal metabolic diseases.

The challenges of research into unmet patient need — and why we keep going

My hope as a research scientist is that we can solve some of the areas of unmet patient need we are working on today and progress toward a future where science empowers us to treat even more diseases.

Scientific research is never linear, and looking for a first-in-class treatment can take us into uncharted territory. Our work relies on hypotheses based on the scientific knowledge we have at the time. We’re constantly learning from failure, and we apply this knowledge to future research.

In clinical research, serendipity often plays a part. We follow the science, which sometimes leads us to finding treatments for diseases that were not originally targeted.  We must learn from the science and readjust accordingly to bring new treatment options forward. 

The more we progress, the more we also want Europe to remain in the global innovation race and at the forefront of pharmaceutical R&D.

Once the global powerhouse of pharmaceutical innovation, Europe has sadly seen a significant decline in its global share of R&D investments over the last two decades, with troubling consequences: 25 years ago, one in every two new treatments originated from Europe. Today, it is fewer than one in five. In clinical trials, Europe’s share of clinical trials has dramatically reduced from 22% in 2013, to 18% in 2018 and to 12% in 2023. These trends underscore the urgent need for action and a more supportive environment, as recently stipulated by the EU’s political leadership, who now acknowledge the pharmaceutical industry as a strategic sector for the future of Europe.

It will be vital that Europe takes the right political decisions to ensure that tomorrow’s technologies and treatments are also researched and developed in Europe, and swiftly reach patients wherever they live.  

As an optimist by nature, a passionate scientist and committed European, I am confident that nurturing scientific progress and a patient-first mindset in Europe can pave the way for more transformative therapies and help millions of people lead healthier and happier lives.