Can Patient Centricity Help Drive Innovation?

A patient-driven R&D model is able to deliver the same results much faster, and much cheaper.

Let’s face it, clinical research has not exactly been patient-centered in the past: clinical trials and the selection of end-points have always been designed for the benefit of scientists, not patients. Take multiple sclerosis: the scientist measures disease progression by looking at microscopic lesions and how they grow over time. That doesn’t mean anything to patients; for them, what matters is mobility and how it becomes impaired as the disease progresses.

This disconnect directly affects enrolment in clinical trials. Improving patient enrolment is a big issue, because it’s the number one source of delays and the primary reason some clinical trials take forever. One third of clinical trials sites enroll zero or one patient and the infrastructure that you’ve had to maintain for nothing is really huge. Unfortunately, this issue is made even worse when patients see what is offered to them in those trials, it really doesn’t make them feel like participating.

So, if instead of tracking microscopic lesions, we could track mobility or other things that are important to patients, I think their willingness to enroll would be much greater.


Of course, patient-centricity is not only about trial design and selection of endpoints; take a look at patient consent. How many times do people go to medical institutions where they are asked to sign forms that run to 15 pages and have been Xeroxed a million times? They’re barely readable. You’ve got to sign those forms right there in those few seconds; clearly you don’t have time to read them and you’ve got to take everything at face value. This is really an awful way to do it. Why can’t it be done online, like Research Kit is starting to offer?

Data collection

Then there’s data collection. Patients volunteer their time and sometimes put themselves at risk; they certainly subject themselves to pain and side-effects; but what do they get in exchange? Nothing. They don’t have rights to the data that is collected; they don’t have rights to the tissues that are taken through biopsy, for instance. So, it is not a very attractive proposition.

If clinical research is to be made more attractive, it is going to have to become much more patient-centered. It really is surprising that researchers have not understood that. There is a sense among the patient community of being fed-up with being used as guinea-pigs. Essentially it’s a one-way street so why should they contribute anything in return?

However, new bio-sensing technologies is an area that is showing a lot of promise by enabling us to collect a lot more clinical data of a much-higher quality – this is potentially transformative.  But clinical research needs to go beyond the collection of clinical data where it has lived in the past to collect every bit of data that can possibly be collected: genomic, phenotypic, behavioral, environmental, microbiome – all those data sets play a role in disease etiology and today we have the technology to do that, effortlessly and without cost.

It’s only after we have that data that we can start trying to understand the knowledge that is embedded in that data, and we’ll have the chance to make significantly faster progress in our understanding of a disease. These are not just great ideas for the future; you’ve got people in hospitals, in academic medical centers, actually busy making this happen already, so this is going to be upon on us this decade.

Lack of interest hampers innovation

We should also note that there are plenty of diseases that the industry has no interest in because they are too small or because they are ill defined scientifically. There are all kinds of medical reasons why the industry won’t consider many diseases. Yet these diseases touch a lot of people: the rare disease space in the US affects 30 million people plus their families.

Until very recently, there was no hope for most of those patients: they had the disease and they had to put up with it or treat it with some drug that was approved for something else that might or might not do some good.

Patient power

What has changed in the past 20 years – and FasterCures has been a very strong driving force – is that we have seen patients starting to take things into their own hands. They will do whatever it takes to find a cure even though they may not have any background in drug R&D or biology; they may not even have any money. However, when patients are determined, they get results; it is difficult to stop them.

Patients are very good at raising money. Rare diseases are very emotive and, with the Internet, the patient community can assemble: a community of 5,000 patients around the world can connect, work together and act together. Once they are connecting, they can raise significant funding.

But they can do much more than that; they can use the money they have raised to create the tools that you need to undertake drug R&D. For instance, patients can do genotyping and put that in a database; they can do tissue banking; they can fund research in universities in order to create a mouse model of a disease. Then, once they’ve done that, they can put all the data in free, open access – typically, scientists will flock to that, because having high-quality, curated data is valuable. In a typical research project it takes 80% of the time and money to get to that point. If someone else is offering to do that for free, it is a very powerful magnet. With those tools the scientists can move things forward a lot faster.

An example of this is multiple myeloma, where research was started by a woman almost 20 years ago; she came down with the disease and was told she had no more than three years to live and probably would never see her daughter graduate from high school. But she was determined that would not happen and she raised money and launched research, and she went into remission eventually. As of last year when I checked it, she had raised $225 million and had funded all sorts of research that produced six drugs that were eventually approved and a lot more that are currently in the pipeline. 

Another example is the Chordoma Foundation, started by a student at Duke University who came down with the disease. That patient determined that he was not going to allow the disease to rob him of his life. He essentially decided to create a one-man pharmaceutical company and was successful in getting research going, raising money and getting the tools needed for scientists to make progress; he made them available with open, free access, and now the research programs are working on clinical drug candidates.

New perspective on the R&D model

These are examples of patients taking things into their own hands, accomplishing on a shoestring and to a much shorter timeline than industry. It puts a new perspective on the whole drug R&D model, because we keep hearing that it takes billions of dollars and 10-15 years to come up with a new drug. A patient-driven R&D model is able to deliver the same results much faster, and much cheaper. Who’s right? Who’s wrong? Those are questions that I think are worth addressing.

The other factor in this equation is passion; in drug R&D, passion changes the odds and the results. It lowers the costs, shortens the timeline and increases the probability of success. The corporate drug R&D model can learn from the patient-driven R&D model; pharma needs to do this because I can see that the patient-driven research model could eventually supplant the current corporate R&D model.

Citizen science

Can citizen science play a part? It’s certainly a possible evolution and frankly, I think there is a very high probability that it will be tried. Whether or not it succeeds is another matter. 

I’m in touch with some companies that are developing new technologies around rapid assessment of toxicology. It doesn’t really cost that much; a person could afford it. So you’re in the lab and you come up with a molecule – or repurpose one – and use this rapid toxicology assessment; you basically get a read-out of the risk attached to self-administering that drug. I can imagine, in a number of cases, that risk might be quite tolerable and worth taking, for patients who don’t have another option. You will be able to use an automatic synthesis lab basically to make those compounds or there will be a market for making them and you could administer the drug to yourself; nobody can stop you.

So you could see a patient community that has no therapy and no hope, engaging in that sort of thing and doing their own research. Because you’re running the trial on single individuals, it raises some statistical issues but it also has some benefits over and above the cost.

How will the regulatory authorities react? It’s difficult to say but I think there’s no question that we’re headed in that direction, especially as drugs produced by the industry tend to be priced systematically at $100,000 or above. I think patients are so fed up with this that they have an incentive to take things into their own hands, and discover their own drugs.

So today we’re seeing the R&D model being transformed in multiple ways at the same time: transformed by the involvement of patients; and transformed by the emergence of new technology such as ResearckKit, such as the $1k genome, and the fast and economical toxicology assessment.

Patient lifestyles

Finally, looking at R&D through a patient’s eyes also enables us to take account of factors like lifestyle. For instance, when a patient only has to take a drug once a day instead of twice a day, or once a week instead of every day, that can be a breakthrough innovation as far as they’re concerned. And the industry needs to look at it this way, instead of looking at innovation simply as a different chemical structure; the industry is beginning to recognize this but it’s going to take a radical change of culture. Part of that culture is that, oftentimes, the industry would prefer to pursue a complicated, expensive solution to a problem rather than a change in formulation, which tends to be a lot easier – because for them complexity often equates with innovation.

Problems with patient connectivity

There are, of course, challenges around the concept of patient-centricity in the context of innovation. Imagine one patient on a treatment arm and the other on a placebo arm, and the treatment arm has red capsules and the placebo arm has blue capsules. In the past, the patient would have been on their own but, today, patients in a trial can connect and share their experiences.

So all the red capsules can post about what they’re going through and the people with the blue capsule can do the same thing. Pretty soon it becomes obvious what color the placebo is and what color the treated arm is. Further, if patients don’t want to rely on the data aggregated from the Internet, they can just break open a capsule, go to a chemist and have the content analyzed, and they know very quickly whether they’re getting sugar or the real drug. So, under these conditions it becomes difficult to do clinical research, but that’s the reality and we’re going to have to deal with that.

Bernard Munos studies pharmaceutical innovation, what causes it and how to get more of it. Several of his papers, published in Nature & Science have helped stimulate a rethinking of the industry business model. In 2012, FiercePharma named him one of the 25 most influential people in biopharma. In 2013, he became a Senior Fellow at FasterCures (a center of the Milken Institute). Besides doing research, he spent a lot of time helping organizations – big, small, public, private, US and non-US – become better innovators.

He learned his trade in the 30 years he spent in the industry at Eli Lilly. Before that, he trained as an animal scientist in France and earned additional degrees in business and economics from Stanford and UC-Davis. Bernard serves on various boards and advisory bodies, including at NCATS, the Institute of Medicine, Glenmark Pharmaceuticals, think tanks and foundations.

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Patient-Centered Clinical Trials USA 2015

Oct 19, 2015 - Oct 20, 2015, Philadelphia

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