It is Time to Equip Patients to Shape Drug Development

Equip Patients to Shape Drug Development


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.[1] Why?

It is only over the past couple of years that the concept of patient involvement in drug development has been gaining momentum. It goes under names like “patient-centric”, “patient-centered” or “patient-led” research and seems to be important to all the various stakeholders.

There seems to be consensus 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. This is not meant to be a comprehensive list. Instead, it focuses on some of human issues that motivated patients may face.

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. Each of these factors could warrant a full discussion of their own. However, here we will explore five of these barriers and look for common elements that may provide a course of action.

1.    Patients Don’t Speak the Language of Drug Development

The language used to describe the development of new medicines is quite dense with medical terminology, regulatory requirements, and industry jargon. The words used can have multiple meanings and when the layperson, the lay-patient, is suddenly submerged in this new language, they can find it very difficult and confusing. It also can be quite scary. Patients who learn the lingo would be better equipped to understand, contribute to, and shape the conversations about emerging therapies.

2.    Professional Experts Can Be Intimidating

Many researchers have spent large portions of their lives in training to become recognized and credentialed experts in chemistry, biology, toxicology, statistics, medicine or one of the countless other specialties required to fully understand the medical potential of specific compounds or proteins. Patients, on the other hand, have been unwillingly thrust into the role of “expert.” 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 intellectual sparring that moves science forward. Once again, they can be at a serious disadvantage when trying to influence decisions.

3.    Patients and Researchers See the Journey Differently

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. 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.

Researchers grow accustomed to the ups and downs of R&D. They can always go back and pursue another target.

But patients are likely to face a roller coaster of hopefulness and despair like they have never experienced. When a program fails, the patients may not have time, strength, or energy to ramp up for the next “shot on goal.”

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 a line of research fails and there are such disparate underlying perspectives.

4.    Many Stakeholders, Many Stakes

One concern researchers mention is that patients find it hard to be objective. The truth is, no one at the table is truly objective.[2] If the goal is to have patients and researchers collaborating directly on drug development strategies and clinical trials, then it needs to be recognized that they have different stakeholders with different needs. All the various stakeholders have an important role to play in this complex ecosystem, but it takes time and effort to learn them well enough to influence. Both patients and researchers need to develop the skills to have honest, effective conversations around the sensitive topic of stakeholders so that the sensitivity itself does not become a barrier to progress. Fortunately, this is a skillset that can be learned and practiced.

5.    Biopharma May Be Missing What is Most Important to Patients

Operational efficiency is critically important to the biopharmaceutical companies from a strictly business standpoint. However, operational efficiency probably is not the primary concern for patients. Patients really want better treatments. The promise of patient-involvement that researchers will be able to design better drugs based on patients’ unique insights. Biopharma may be missing the big win because it is so much harder to manage and they have little experience letting patients drive the discussion. Maybe, it is not up to the companies, but rather patients to take the initiative. The key will be to equip patients so that they are able to step up to this challenge.

The general consensus is that to truly accomplish patient-involvement, -centricity, or -leadership it is important to get patients involved at the very beginning of a drug development program. It is believed that the sooner the patient is involved, the more they will be able to shape the research, and the better the outcomes will reflect patient needs.[3] But how?

There are emerging regulatory efforts to further this agenda and answer this question. The US FDA is working on developing guidance for Patient-Focused Drug Development and published an 8-part framework for this work in May 2017[4].

There is a huge opportunity for patients to get involved in the shaping of this guidance. All US Patient Advocate Groups should be building this work into their strategic plans for the next 5 years. Part of this strategy should be to provide drug development education to their constituents so that those patients are better prepared to effectively contribute.

There is a mature model of patient education in the EU. 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) to educate patients about the European drug development process. EUPATI has developed educational materials in 6 key areas, and serves patients in 12 EU countries in 7 different languages.[5] 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.  Patients in the US deserve a similar program.


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 to the point where they are adequately prepared to make a real difference in the development of future medicines. It just takes too long!

There are two strategies that can improve this situation:

1.     Refocus education efforts toward patients and their families

2.     Accelerate the learning journey so that “trained” patients can have a bigger impact

Upcoming installments of this series will flesh out each of these barriers and examine what it will take to use these strategies to overcome them.

Related Articles in the Series

Posted by Kevin Freiert


Posted in

Kevin Freiert

Kevin is the CEO 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] 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

[2] Lowe MM et al. 2016

[3] Lowe MM et al. 2016



Photo by Harry Sandhu on Unsplash