Let’s Talk About Patient Engagement

Why does everyone talk about “Patient Engagement” in clinical trials?

As trials are becoming longer and more expensive and many of them fail to meet their recruitment goal, patient engagement plays a key role in the success of a clinical trial. But what is exactly patient engagement and why is it so important?

Getting to know the person behind the “subject” title

Times have changed, and nowadays, more than ever before, patients are more involved in their own clinical care. Equipped with knowledge gained from a variety of available resources, they bring their own views coupled with high expectations regarding their treatment options.

There is an increasingly growing understanding that it’s important to pay much more attention to our main stakeholder – the patient. Patients who are experiencing the illness first-hand, are an invaluable resource, capable of clarifying their priorities and true needs. These insights help to better shape the trial’s design and protocol long before you embark on the long path that is R&D. They can provide a unique point of view that will help optimizing the development process, identify knowledge gaps and better shape the protocol and the trial’s endpoints.

Patients can provide information about the things that actually matter to them – the daily burden of the disease, their unmet needs, therapeutic burdens, risk-benefit ratio and more, directing the researcher to ask the correct questions. The way there is clear – by creating a true and meaningful partnership between the patient and the researcher. This partnership can transform the clinical development process from one that is directed by sponsors and investigators to a process driven by the needs of patients and their caregivers.

Current state of affairs

Recent efforts focus on formalizing the partnership process by creating designated models. These are three suggested models:

  • Patients as advisory board members – it consists of a group of patients with similar characteristics to the expected trial population who meet regularly with the clinical staff to help improve performance. They provide their experience and advise on aspects such as eligibility criteria and the follow-up phone call schedule to help researchers design a trial that would be both useful for patients and easy for them to participate in.
  • Patients as steering committee members -The selected individual would give input on protocol design, anticipated burden for study participants, recruitment and retention tactics, and other key areas. The organization who is responsible for the study or the sponsor would also be responsible for the engagement and education of this patient representative, whose views would carry as much weight as those of other steering committee members.
  • Patients as co-investigators – patients and caregivers are included as co-principal investigators (PI). As a co-PI, they oversee patient engagement efforts and work to address potential barriers to patient involvement by openly discussing concerns about data security and anonymity. They are also in charge of creating a patient- and family-friendly glossary of acronyms commonly used within the medical community.

These efforts are very important as although there are increased levels of patient interest in treatment options, appreciation of clinical trials as a viable care alternative is still limited. The partnership models can offer a patient centric framework and help raise awareness and willingness to participate in clinical trials.

A great example for the significance of patients in the trials design is the CONNECT-HF trial funded by Novartis. It’s a special kind of study, not testing a new medication but trying to understand what kind of care and resources people with HF use after leaving the hospital. The researchers’ goal is collecting information from patients to better understand what kind of support and care is the most helpful during the time they leave the hospital and recover at home. This is a unique study with the potential to open a window for HF patients’ true needs and pains. Those insights can help researchers better define HF trials outcomes and patient centric design.

In this study, the team worked closely with a patient advisory group. The selected group had similar characteristics to the trial group (age, sex, race/ethnicity, and geographical location) but they never participated in a clinical trial. This advisory group offered their experiences and advice on aspects such as eligibility criteria and the follow-up phone call schedule to help researchers design a trial that would be both useful for patients and easy for them to participate in.

Looking ahead

True patient engagement and patient centricity models will need to reflect the factors that drive patients’ decisions to enroll and remain in clinical trials. Protocol design, site selection, site staff training, employed technologies, provided information will all need to be examined through the eyes of the patients and their needs.  The bottom line is that patients find

in-person interactions during the trials as the most effective way to contribute to their engagement. That is why including them in the process is so essential.

The present conception shows that patient engagement activities will increase over the next few years. Considering that patient centricity and patient engagement weren’t given much attention until five or six years ago, we should expect to wait a little while longer to witness a true change. New effective models of engagement are still necessary.

Remembering why we are doing what we are doing

In the end, the purpose of drug development is to help people in need and improve their lives. If we want to perfect our ability in doing so, we need to learn to better tailor our trials to the true needs of the people they are designed for.

If we want to elevate enrollment rates and improve retention, we need to change our perception and start thinking of patients as our most valuable asset. We must emphasize the personal relationship between the patient and the clinical staff alongside the development of mobile and digital technology.


  1. “Models of Engagement: Patients as Partners in Clinical Research” from applied clinical trial. http://www.appliedclinicaltrialsonline.com/models-engagement-patients-partners-clinical-research
  2. http://connectheartfailure.org/about-connect-hf
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