It is Time to Equip Patients Part 4

Fellow Travelers on a Long, Cold Road

Fellow Travelers on a Long Cold Road

The journey to a new medicine is a challenging, uphill, and winding road. Whenever a patient gets involved in medicines development, they are likely to face a roller coaster of hopefulness and despair like they have never experienced. Since only about 5% of the compounds and proteins put into advanced testing a make it through to market approval, chances are that they will see one or more potential new treatments fall short. If they are fortunate, a safe and effective treatment will be found in time for them.


There seems to be a consensus from all the stakeholders that increased patient involvement would enhance the development of new medicines and devices. There also seems to be a 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.

This installment will contrast how patients and researchers see the progress of medicines development.

Patients and Researchers See the Journey Differently

Patient Journey

Patients have the greatest stake in the success of our healthcare system and in the development of new and better treatments. They want to feel better and they want to feel better now. Because their lives are being directly affected, patients are impatient…and rightly so.

Depending on their ailment, there may or may not be an effective treatment available. A newly diagnosed patient has the reasonable expectation that there is something their physician can prescribe to alleviate their symptoms, fight the infection, or reverse the course of the disease. And in all likelihood, there is something. However, the available treatment may not be as effective as it needs to be or it may come with trade-offs like side effects, changes to diet, or many other disruptions of the patient’s life.  When faced with this reality, some patients reach a point when they get interested in exploring the available experimental treatments.[1]

To some of these patients, the breakthrough they need may seem like it is right around the corner. They may read about a new experimental treatment or hear the hype on the news or from the sponsor company and conclude that a treatment is coming soon. This will surely lift their spirits and give them hope. If there is a clinical trial recruiting subjects, they may even talk to their physician about participating.

And then there are patients with disorders that do not have any emerging treatments on the horizon. Either the science has not led to any promising approaches or, worse, no one has invested in looking for a treatment in this area. These patients find themselves entering the drug development process very early on and thus face very different challenges. The biggest hurdle for them is to gain the attention of researchers and those who fund them.

Researcher Journey

Contrast this with the researcher or physician who has spent many years searching for a new treatment. They likely have been at this point before and experienced the disappointment of the failure of a promising new treatment to meet expectations. Depending on their role in the 12-15 year-long process, researchers may have seen hundreds of failures and never worked on a “successful” medicine. And some of those failures occur right at the very end of the process when the goal line is in sight. This repeated failure tends to breed a certain callousness. It also breeds patience, determination, and hope. You see, these scientists, like Thomas Edison, know that with every failure we are one idea closer to the solution.[2]

Once the basic biology of a disease is understood, even partially, researchers from biotech and pharmaceutical companies begin to look for ways to intervene biochemically.  These experiments start in the lab, progress to animal or tissue models, and eventually are performed in human beings in clinical studies. Eventually, some promising results are seen and science advances. At any point in this incremental process, results can demonstrate that the research is on the wrong path. This may not mean going back to the drawing board, but it does mean taking a step or two back to try something else. This is a painfully slow journey.

The Shared Journey

Both of these groups share the same goal – to find a better treatment for the disease in question and improve the lives of patients. Both of these groups will get renewed excitement when that next new idea begins to show promise.  But when a program fails, the patients may not have time, strength, or energy to ramp up for the next “shot on goal.”  Likewise, both private and government organizations may choose to step away from programs that do not show enough progress. Unfortunately, the patients’ disease will continue to progress.

This reality can lead to conflict and misunderstandings. Patients may see the researchers as cold, uncaring, and calculating. Researchers may see the patients as not objective, unrealistic, and short-sighted. These characterizations can make it difficult for either group to have productive conversations. Trust, sometimes built up over years, can evaporate in minutes because of an impulsive comment during times of stress.  It takes education and skill to manage the fragility of a team’s cohesiveness when there are such disparate underlying perspectives.

One way to facilitate this collaboration and keep it healthy is to educate patients about the drug development process and the way progress is made incrementally. Informed patient advocates will understand the enormous odds of any given experimental treatment to succeed. This will help them deal with the swings in the research and not lose all hope when there are setbacks. They will also be able to better educate those they represent about the differences between the knowledge-seeking mission of clinical research and the patient-centered mission of medicine.[3]

Additionally, education must help patient advocates learn the skills needed to build and maintain trust, understand motivations, and to surface concerns. Researchers will need these skills. A shared set of techniques could help both groups approach stressful times when hard decisions more constructively. This type of education has to be experiential. It has to not only communicate the reality of the process, but it also has to evoke the emotions and reactions that setbacks do. The hope would be that these experiences would help teams of patients and researchers practice maintaining momentum in spite of disappointment.

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Kevin Freiert

Kevin is the Principal of Salem Oaks Enterprises, LLC. Committed to working with patients, their advocates, biopharmaceutical companies, regulators and other stakeholders to develop educational tools that help patients bridge the gap between themselves and Biopharma R&D.

Works Cited

[1] DasMahapatra et al. BMC Health Services Research (2017) 17:166 DOI 10.1186/s12913-017-2090-x

[2] In response to a question about his missteps, Edison once said, “I have not failed 10,000 times—I’ve successfully found 10,000 ways that will not work.”

[3] Dresser, Rebecca. When Science Offers Salvation: Patient Advocacy and Research Ethics. Oxford University Press, New York, 2001 p.9

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