Assembled Chaos

Working together to accelerate innovation in the life sciences

An area where industry and regulators are ahead of academic medical researchers.

An area where industry & regulators are ahead of academic medical researchers

A quick review of pharmaceutical company websites reveals a lot of messaging around patient engagement:

“Patient driven”

“Patient lives matter”

“There is only one way to succeed - together."

Is this just the savvy use of patient engagement buzzwords, or an indication of something more substantial.

The corporate vice-president of Parexel, one of the major contract research organisations, Alberto Grignolo recently said:

“Companies appear to now have greater sensitivity to the ability of patients to contribute valuable knowledge to the development of new medicines, as opposed to patients being given lip-service and token patronising credit.”

This is perhaps nowhere else more evident than in some of the Innovative Medicines Initiative (IMI) projects.

The IMI is a European Union funded public private partnership. Since its inception there has been an emphasis on patient engagement.

In the Unbiased Biomarkers in the Prediction of REspiratory Disease outcomes (U-BIOPRED) project we engaged patients deeply including them as participants in the work package conference calls and consortium meetings.

We have taken that further in the European Translational Research Information and Knowledge management management Services (eTRIKS) project engaging patients and other stakeholders on societal level issues such as the re-use of medical research data.

The commitment of the IMI to patient engagement is perhaps most evident in the project European Patients Academy (EUPATI) that is focused on "education and training to increase the capacity and capability of patients to understand and contribute to medicines research and development”

The Faster Cures consortium recently held a ‘Expanding the science of patient input: pain points and potential’ workshop. In the workshop 2 concrete examples of how patient engagement is achieved in the pharmaceutical industry. What was perhaps more telling was that it was reported in the workshop that academics have been slow to engage patients. For example NIH funding programmes focused upon patient outcomes are under-subscribed.

Perhaps what is driving this attention is that both the European Medicines Agency and the Federal Drug Administration have substantially increased the number patients on their regulatory panels. In fact the EMA increased the number of patients on their panels from 76 in 2007 to 633 in 2014.

A bold viewpoint on patient engagement

Leonard Kish made a bold statement about patient engagement he called it:

"...the blockbuster drug of the century.”

When I first read this I wanted to rise out of my chair and march through the streets heralding the fact that the concept of patient engagement was starting to get the recognition it should. However, it is not so simple.

The manner in which patient engagement is typically implemented complicates the matter.

Many researchers have a bias against meaningful engagement of patients

Often when the decision is made to engage patients and someone asks how it is going to be done there is a long 'uhhh….’.  Then as ideas are discussed you get these types of reactions:

"We can't use that language it's too complicated for patients."

“Answering a survey is about all we can expect from patients. They won’t understand the more scientific aspects of the research."

“Oh, I got it. We will have patients read the informed consent form and tell us what they think. Then we can say we engaged patients in our research."

These types of reactions are more typical for academic medical researchers. I can say that because I was an academic medical researcher. It was an eye opening conversation that I had 17 years ago to alter my own bias against the meaningful inclusion of patients in research. I will describe that conversation a bit later in this post.

For now it is important to realize the bias against meaningful engagement of patients in research is the result of a fallacy.

The fallacy is that patients are simple minded. Of course if patients are simple minded it would not make sense to engage them in a meaningful way in research. But that is not the way it is.

At some point almost everyone becomes a patient.

Clinicians, scientists, engineers, philosophers, politicians or physicists all become patients. It is, however, not just these highly educated professionals who are motivated to understand science.

A conversation that ended my bias

In 1999 at the University of Vermont I helped form a Community Medical School. Professors from the medical school gave lectures on research to the public.

A major concern we had was that the content would be too technical. We were wrong.

One of the lectures was given by a very dynamic professor who marched up and down the stairs of the auditorium to demonstrate how the molecules that make up your muscle, actin and myosin, worked. Clearly he was an engaging and entertaining presenter. However, I was concerned.

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He used some very technical terms and tried to explain complex concepts. He was prsenting material that actual medical students struggle with.

After the presentation I went immediately to a couple of our stalwart attendees. They were a retired couple that routinely cancelled their bridge night to attend.

"What did you think?"

"It was very good."

"Did you understand all of it."

"No, but that is okay. We like the fact that he did not dumb it down. We enjoy the challenge of trying to understand the difficult concepts."

That was a watershed moment for me. It was the moment that I realized that it is a fallacy to think patients are simple minded. My own personal bias against involving patients in research ended.

Most people can engage at some level with even the most complex scientific topics. This is especially true when your own health and wellbeing are at stake. This is key for making major medical research breakthroughs for one simple reason.

Patients are experts too.

Everyone's an expert in something.  Many other disciplines are converging with the life sciences particularly with the emergence of ‘mHealth’. We are now able to capture medically relevant data 24/7. It is now feasible to study personal aspects of human life. There is not only just personalised medicine. Medical research is also personalised.

So pretty much any type of expertise is relevant.

An untapped resource for medical research

The expertise of patients is an untapped resource in medical research. If we are going to make use of this resource the whole research community including academic medical researchers should endeavour to engage patients in a meaningful way.

Real dialogue.

The energizing fact is that once a real dialogue takes place patients and researchers are usually enthusiastic about the value of the interaction. Both sides gain substantial knowledge and understanding from such interactions.

Surveys or one way feedback sessions cannot create that type of enthusiasm.

Meaningful patient engagement in research is not exactly itself a blockbuster drug.

It is a paradigm shift in how research should be done - a paradigm shift that is necessary for next generation of breakthroughs in medical research.

Like any paradigm shift there is more needed than just buzzwords and slogans. There needs to be a real commitment to invest effort in having a real two way dialogue between patients and researchers. Moreover academia needs to catch up with industry, non profit organisations, and regulatory agencies.

The convergence of the creative energy of all stakeholders can be a powerful force for making real change.

Concrete examples where meaningful patient engagement made a difference will help drive the paradigm shift forward. Do you know of any examples? Describe them in comments below.