close

What is innovation in medicines?

It’s the breakthroughs that make the headlines but from a patient perspective, the impact of incremental innovation can be monumental too. Advances in care are often labelled as one or the other whereas in reality they are often linked, part of the same continuum which can revolutionise outcomes for patients over time. 

When we hear the word ‘innovation’, it’s radical technology or tools that transformed how we live our lives that spring to mind. There are numerous classic examples such as the wheel, the printing press, the Internet and the iPhone.  

In medicine, the headlines have been dominated by innovations that have saved millions of lives or dramatically improved people’s quality of life. Think of antiretrovirals for HIV or the vaccine that eradicated smallpox.  

Now and in the future, we recognise the personal and societal costs of diseases like dementia or multiple sclerosis and understand the desperate need for new diagnostics and treatments. Indeed, these are the areas that feature strongly in the EFPIA Pipeline Review which provides an overview of treatments currently in development.  

Small steps and giant leaps 

However, most innovations – in medicines or in other research-intensive fields – are not hailed as paradigm shifts that entirely solve major problems. Most of the innovation is incremental innovation. That is, advances that move an area forward, step by step.  

Breakthrough innovation and incremental innovation are closely linked. In fact, they are interdependent. The iPhone was not invented in a vacuum; it was the result of multiple smaller, meaningful steps forward in several areas of technology over time. And, of course, a breakthrough does not mark the end of product development: incremental innovation continues to refine and improve disruptive technologies. The smartphone you have today is superior to the one you carried a decade ago.  

Incremental innovation in medicines 

The story of any breakthrough is the sum of multiple step-by-step innovations that laid the foundation for one or more major advances: the quiet revolutions in basic science and in medical therapies that underpin progress.  

Let’s return to HIV – a disease which, in the 1980s, was widely viewed as a death sentence. Today, thanks to several waves of medical progress, people with HIV can expect to live approximately as long as the general population. Antiretroviral therapies have extended and improved people’s lives and stripped the disease of its terror.  

However, it is easy to forget that while the picture today is radically different to that of four decades ago, HIV treatment was not an overnight success. The first antiretroviral was approved in 1986; several more followed in the early 1990s, as well as new class of HIV drugs which acted much faster than its predecessors. Through incremental innovation, scientists developed new approaches to treating the patient, until there were half a dozen classes of HIV drugs. From there, clinicians had multiple options from which to build and test combination treatment regimens. And research in this field continues. 

The ongoing push for better MS therapies  

The history of multiple sclerosis (MS) treatment is a little different, but it follows the same theme. MS, a chronic autoimmune disease that affects the central nervous system, was initially treated with strong immunosuppressants when patients’ symptoms worsened. This approach evolved and has eventually led to a variety of disease-modifying therapies to reduce symptoms and delay disease progression. This is a very personal example to me as I watched one of my best childhood friends go through the incremental innovation in the treatment type and administration over the past nearly 30 years since discovering she suffered from the disease. A trained architect, she was unable to practice what she loved to do in the first years of her disease because of how debilitating the daily injections of her treatment were and instead worked part-time in a call centre and had to rest for the second part of each day. She started working as an architect part time when her treatment evolved to a once a month, week long drip-treatment and today, she is a full-time architect who can take an oral medication and do what she really loves: create. 

From the approval of interferon beta in the early 1990s to the first monoclonal antibody in 2004 and several new treatments in the 2010s, medical innovation has delivered new medicines and new modes of delivery. There has also been a strong focus on improving the safety profile of MS treatments, to find ways to stop – rather than delay – disease progression, and to address some of the organ damage caused by the disease.  

This story is far from over and, as the EFPIA Pipeline Review shows, we will not rest until new therapies meet the needs of the MS patient community.  

Improving melanoma survival rates, one step at a time 

The goal of medical research is to meet the needs of patients. Ideally, this would happen with a single breakthrough treatment. But it rarely works like that. Survival rates for advanced melanoma have seen several stepwise increases in recent years – and there is still room for improvement.  

Ten years ago, only 10-15% of patients were still alive five years after diagnosis, despite receiving chemotherapy and other interventions. Immunotherapy delivered some progress, but the first waves of treatments were expensive and had strong side effects. Then came new kinds of immunotherapy: first-generation checkpoint inhibitors and highly targeted kinase inhibitors. Five-year survival rates shot up to 30-40%, with both classes of drugs hailed as breakthroughs.  

The advent of second-generation checkpoint inhibitors – a clear example of incremental innovation – improved five-year survival rates to 50%. Now, combination therapies have pushed survival rates up to 60%.  

This, of course, is not enough. Patients, families, doctors, researchers – all of us – dream and work for 100%. The lesson from recent history is that success will come in a series of steps rather than in one giant leap.  

The shoulders of giants 

How we define and incentivise medical innovation will shape future waves of medical progress. That is why the active conversation about updating the EU’s pharmaceutical legislation is fundamental. We can’t design a system entirely focused on waiting for that eureka moment, the near miraculous breakthrough for a disease where there are no treatment options. We need a system where the early-stage research and incremental innovation that really fuels progress for patients over time are valued and rewarded.

You can access the EXON report here

Patients living with a host of conditions have seen improvements in their quality of life, how their treatments fit in to daily life through monumental, incremental innovation. Together with the dramatic breakthroughs we hope to see, let’s make sure that Europe and Europeans can benefit from both.  

To find out more about why we need a broad, patient-centric definition of unmet medical needs, click here 

Nathalie Moll

Nathalie Moll joined the European Federation of Pharmaceutical Industries and Associations (EFPIA) as Director...
Read Morechevron_right