“A champion named Goliath…came out from the Philistine camp. He was over nine feet tall. He had a bronze helmet on his head and wore a coat of scale armor of bronze weighing [125 pounds]; on his legs he wore bronze greaves, and a bronze javelin was slung on his back. His spear shaft was like a weaver’s rod and its iron point weighed [15 pounds].”
So… anybody interested in fighting this guy?
While most scientists and physicians don’t wear armor these days, they still can emanate an aura of invincibility and impenetrable knowledge.
“Unarmed” patients face little hope in changing the minds of these intellectual giants.
Or do they?
Patients are the most important stakeholders in the development of new therapies. Thus, one might conclude that patients must be involved in the process of developing these therapies. While there are some excellent examples of patient involvement improving both the execution and the focus of clinical trials, it is far from the norm. Why?
There seems to be consensus from all the stakeholders that patient involvement would enhance the development of new medicines and devices. There also seems to be consensus that this promise is not being realized. Why?
The purpose of this series is to identify and characterize some of the barriers to effective collaboration between patients and the innovative pharmaceutical industry.
Understanding the Problem
There are several factors that make it difficult to achieve the type of deep patient involvement in the drug development process and the shaping of the future of medicine. In this series we will explore five of these barriers and look for common elements that may provide a course of action.
- Patients Don’t Speak the Language of Drug Development
- Professional Experts Can Be Intimidating
- Patients and Researchers See the Journey Differently
- Many Stakeholders, Many Stakes
- Biopharma May Be Missing What is Most Important to Patients
This installment will focus on the challenges that arise because of the disparity between trained professional experts and patients who have found themselves trying to be heard.
Professional Experts Can Be Intimidating
During my 30 years working for a major pharmaceutical company, I was always amazed at the sheer intelligence and vast amount of knowledge my colleagues had amassed. Many of them have spent large portions of their lives in training to become recognized and credentialed experts in chemistry, biology, toxicology, statistics, medicine or one of countless other specialties required to fully understand medical potential of specific compounds or proteins.
With this expertise comes a well-earned pride and confidence that is palpable. In fact, many of my colleagues would attest to the fact that this pride and confidence is the primary objective of their training; the ability to stand up to intense scrutiny and skepticism without losing intellectual integrity. The successful “defense” of a doctoral thesis can be a traumatic, cathartic, and life-changing accomplishment. It is meant to be. And many researchers have multiple graduate-level degrees.
Their depth of knowledge is extensive, but relative few of these specialists develop an appreciation for the full breadth and complex interdependencies in the development of a medicine. This is why they must work together. When these talented and highly prepared individuals come together in teams, they are able to solve the extremely complex problems inherent to disease. They are a force to be reckoned with.
Patients, on the other hand, have been unwillingly thrust into the role of “expert.” Receiving the news of a diagnosis can be a traumatic, cathartic, and life-changing event. Nevertheless, their expertise is probably the most valuable because they alone know what it is like to experience the disease every day. Patients are able to identify what is most important to study and whether there has been a meaningful change in those variables. They also know what risks they would really be willing to take in order to find an effective treatment. They have a firsthand understanding of the burden of the disease and the burden of current therapies.
However, unless they happen to work in the field, they are unlikely to have had much formal training in science or even in engaging in the types of intellectual sparring that moves science forward. Once again, they can be at a serious disadvantage when trying to influence decisions. The cards are stacked against them.
Newly diagnosed patients start a long learning journey through which they cross a number of thresholds; diagnosis, current treatment options, community involvement, experimental treatment options, trial involvement and activism. The things they must learn grow in difficulty, complexity, and depth with each step. The farther they go, the harder it is to find, let alone understand, what they need to learn.
And, if they find sufficiently successful treatment at any point, they probably drop out of the journey. So, what does this mean? There are very few patients who have the will, perseverance, or even health to make this long journey successfully to the point where they are adequately prepared to make a real difference in the development of future medicines. It just takes too long!
Currently, learning about medicine development lags the motivation that patients have to take this journey. No one gets diagnosed and immediately asks, “Well what does it take to find, develop and gain regulatory approval for a new medicine?” That urge comes later. Thus, there is an opportunity to shorten the lag time between when a person is diagnosed, starts to learn and when he or she is fully prepared by developing effective educational tools and providing them sooner in the journey.
In addition, some patients are faced with a short runway. Their ailment may be progressive, debilitating or even terminal. Even if their disease is manageable, they cannot escape the inevitable march of time. At some point, these patients may not be physically, mentally, or emotionally able to continue the good fight. Their motivation declines.
Thus, we need to change the trajectory of their learning curve so that informed patients have more time to exert their influence once they are fully prepared. Again, we need to provide more effective educational tools that speed up the learning curve. One way to accomplish this is to make the learning journey exciting, enticing, and fulfilling. In this way, patients will go beyond their own circumstances and seek learning either for learning’s sake or for the sake of other patients like themselves.
In addition to the time it takes to be fully prepared, we cannot ignore that this is multifaceted preparation. Patients need knowledge, skills, and confidence to engage fully with this system. Thus, any educational tools or system need to provide this full-thickness education. There need to be elements of study, of practice, of experimentation, and of achievement built right into the program.
There is Hope
There is an example of this type of education already in existence in Europe. The European Union Patient Academy for Innovative Therapies (EUPATI) was formed in 2012 as a five-year directive of the European Innovative Medicines Initiative (IMI). EUPATI has been highly successful in bringing together experts from all corners to develop web-learning, resources, events, and tools for educating patients about the drug development process. However, this effort has been exclusively focused on the EU processes. While the scientific framework is roughly the same, the regulatory, legal, and ethical controls on drug development in the EU are much different than those in the US. Additionally, there are some cultural and political advantages to creating such a government-sponsored project in Europe.
The challenge remains to develop world-class educational content, tools, and systems to prepare patients in the US to fully exert their influence on shaping medicines of the future. Since there is no organization like EUPATI in the US, it has been suggested that drug developers partner with patient advocacy organizations (PAOs) and interest groups. While this is certainly a necessary step, it will only lead to further fragmentation and dilution of the education. There is also a high likelihood that effort and investments would be duplicated.
It has also been pointed out previously that there is no overarching governance structure for the world of patient education. As a result, there may emerge different product-based or disease-based narratives within the various education programs. As a patient seeking the definitive word on this topic, I would be troubled by all the different stories I could find.
A more efficient, effective, and truly patient-centered approach would be to develop a common set of educational tools that the PAOs could then distribute. This would ensure the design meets the needs of patients and would also cement the PAO as the “Go To” source of information.
That common set of tools, like EUPATI, could have versions or add-ons for each disease or set of related diseases. The cost of building a single source, or common set of core courses would be much lower and could be shared across the healthcare system, leaving only the cost of customization by disease to be borne by the PAO. This would put a reliable, relevant, and robust set of educational tools within reach of all PAOs, large and small. Further development, maintenance, and growth of these tools could be funded through PAO subscriptions based upon the size of a particular patient population or on a fee-for-usage basis.
The benefits to patients and to society are real and substantial if we can find a way to unlock their wisdom.
 1 Samuel 17:4-7 Holy Bible (NIV)
 Lowe MM, Blaser DA, Cone L, Arcona S, Ko J, Susane R, Wicks P. Increasing Patient Involvement in Drug Development. Value in Health 2016:19:869-878
 Lowe et al. 2016
 It’s time for a code of ethics in patient education. PharmExec.com May 2013