Why future medicines must be co-created with patients

Neil Bertelsen, member of Patient Focused Medicines Development (PFMD) and former chair of Health Technology International (HTAi),  explores how to improve the barriers and limitations in the drug development process? 

For too long, the established research community, in both academia and industry, have dictated the direction, shape and outcomes of medical research. Today, people living with conditions demand and expect a role in shaping the science that will deliver meaningful impacts to their future lives.

We know partnering with patients leads to better research and will ultimately produce a new generation of medicines geared towards their needs. Yet, there is still reluctance and perhaps even resistance in some quarters to collaborating with patients in health research. The most common fear is that it will slow down progress. 

Speed does not have to be sacrificed, and Covid-19 research is a prime example. In the rush to develop Covid vaccines and treatments, the UK’s National Institute for Health Research (NIHR) noticed a staggering fall in the number of research projects involving patients compared to previous averages. Yet, after offering a service to connect researchers to patients, the NIHR noted that patient and public involvement in research rose to 85%, even for urgent Covid-19 studies. So, it is possible to conduct time-critical research and involve patients and the public. 

In fact, patient involvement speeds up the drug development process. Work by the Economist Intelligence Unit showed that clinical studies designed with patients to be more patient-centric recruit participants almost twice as fast as others. 

It is unbelievable that patient involvement in research is still not routine today. One concern is that patients will not understand the research and will be unable to contribute. This is simply not true. Over the last decade the patient community has come together with academia and industry to develop a range of training courses to learn about research and drug development. Perhaps the most famous of these, EUPATI, provides global training to empower patients with the expertise required to understand and contribute to the research and development process.

Patients have unique knowledge and understanding that can really improve research. People living with a disease can perfectly explain how the condition affects their day-to-day life. They can compare those experiences with what is planned to be measured in a clinical study and be vocal if they feel the study will not capture the true impact of their disease. 

With these insights we will be able to make a better assessment of new medicines using criteria that patients have highlighted as important to their daily lives. Let’s take the example of a common measure used in clinical trials to assess how mobile someone is – a measure of how far a person can walk in six minutes. The Duchene muscular dystrophy community highlighted that this test excludes people already using wheelchairs, and it is the use of arms and hands that is most important to many people with the condition. Working alongside the research community, the Duchenne patient community co-developed a new measure for arm and hand function to be used in future clinical studies.   

Fortunately, there is a wealth of guidance, support, and networks available to help. Patient Focused Medicines Development (PFMD), an independent global coalition of health stakeholders, has spent the last five years building a library of tools, resources, and how-to guides to get the research community started.  The public-private consortium, PARADIGM, published a wide range of guides and practical tools, too. These are all freely available for the global community to make use of. 

Change is already happening. In this field, we learn by doing and being honest about what works well and what doesn’t. I am seeing a new openness, particularly from pharmaceutical companies, about sharing their approaches publicly. The Book of Good Practices contains examples such as Astra Zeneca’s and Novartis’ use of patient partners to impact their clinical study designs. Bristol Myers Squibb (BMS) has publicly announced a program called PEER to bring in patient insights into all stages of clinical research. 

But for those of us working in this field, we know there is so much more to do to make patient involvement in medicine development seamless and systematic. I facilitate a monthly working group as part of the Advocacy Exchange, a virtual collaboration space for the patient community. What is clear from this work is that while some expert patient advocates are now being routinely approached to collaborate on research, the vast majority of the patient community are still left out in the cold. We are missing a chance to encompass the full diversity of the patient community. It is a challenge we are ready to embrace, and this platform is one example of how we are starting to do so.  

So, I call on the research community to embrace these challenges with us. Let’s build the medicines of tomorrow together, learning from one another to advance the health of all.