Best Practices for Patient Engagement in Drug Development: A Brief Background on Patient Engagement

Clinical Researcher—December 2018 (Volume 32, Issue 10)


Debra Michaels


The expectation of patients to be engaged in healthcare decision making and medical product development has been on a steady increase since the early 1980s, when AIDS activists demanded the dedication of adequate resources to research treatments for a disease that was then a death sentence. The expansion of that expectation to the population at large has been stimulated by the availability of widespread and multidirectional communication, and by scientific advances making it possible to better understand the mechanisms of disease and the basis for treatment and cure.

Empowered patients increasingly see themselves as equal stakeholders in a collaborative health system with medicines developers, regulatory authorities, healthcare providers, and payers—and rightfully so, for their insights are the best way for us to understand their experiences, their needs, and what constitutes the value of a medical therapy.

In Search of Clarity

Medical product researchers understand the importance of involving patients in the product development process, but progress toward optimal engagement has been slow. Even with good intentions, we’re often unsure about how to engage patients and how to be “patient-centric.”  Nobody today has the full answer, but the good news is that best practices are evolving from the experiences of early adopters, and we can learn from them.

A note about terminology: the terms “patient engagement,” “patient involvement,” and “patient-centric” or “patient centricity” have been used in these opening paragraphs. In this article, “patient centricity” is used as an umbrella term meaning all patient-centered efforts and activities of an organization to engage patients.

Under this umbrella falls “patient engagement,” meaning direct and constructive interaction with patients in the full medicines life cycle to enable practices that are based on patient perspectives and that result in outcomes that meet patient needs as well as the developer’s needs. “Patient advocacy” also falls under this umbrella, and refers to the actions of specific healthcare professionals, known as patient advocates, to advocate for and support the needs of patients, caregivers, and families.

Other terms you’ll see in the literature and social media include “patient-centered,” “patient-focused,” and “patient-dedicated,” and new ones are being coined all the time. An insightful industry colleague brought a lengthy terminology discussion to a close by proclaiming, “Let’s stop worrying so much about what we call it—the important thing is to just do it.”

How Do We Start to Engage Patients?

Patient involvement in medical product research and development (R&D) can occur at many places throughout the life cycle of the product, as shown in Figure 1 from the EUPATI Guidance for Patient Involvement in Industry-Led Medicines R&D. The ideal would be to engage patients for their input in all of these areas, but to date, that remains an aspiration for most (if not all).

Figure 1

Each organization must start with what is consistent with its own goals and objectives. Often, one functional area within a company decides on its own to seek patient involvement, based on its specific objectives, and this is fine. To paraphrase my industry colleague, the important thing for getting started is to just do it; however, taking the time to follow a few best practices at this stage can lay the foundation for later benefits. For example:

  • Bring your team together and discuss where your function fits within the R&D continuum. What outcomes are you charged with? How could patient insights help to improve those outcomes? What would be your team’s “win” for successful engagement with the patients you serve?
  • Who are the patients you serve, and what does their patient experience look like? What do you know about their patient community? Which patient organizations represent them? What would the “win” be for this community to engage with you? Some companies conduct comprehensive “patient landscapes” to answer these questions and better understand those with whom they should engage.
  • Are there start-up or ongoing patient engagement activities elsewhere within your company? What are these, and could collaboration or information sharing be beneficial?

With this basic information, you can approach patient engagement more strategically. Brainstorm with your team to identify a simple starting initiative that will meet patient objectives as well as yours. Win/win efforts are more likely to be successful.

However, sometimes it’s not apparent what a meaningful engagement might be. It’s always appropriate and helpful to begin building relationships with patient groups representing the patient communities of interest (see the section on best practices for more discussion on this point).

Which Patients or Patient Groups Should We Work With?

Meaningful patient engagement starts from the premise that the patient is an equal stakeholder and brings a specific expertise to the medical product development process. “Patient” is a general term, and it’s important to engage patient representation with the skills and expertise required by the objectives of the project.

A patient may be an individual patient, caregiver, patient group or representative, or a patient advocate. These different types of “patients” have different types of knowledge and abilities to represent the broader patient community that you need to engage. Patient groups vary widely as well—in their purposes, functions, services provided, organizational maturity, staffing, and other characteristics.

Product sponsors often find they must work with more than one type of patient to meet the needs of their research, and more than one group within each type to ensure broad representation of the patient community for whom the therapy is intended. A comprehensive list of questions and considerations for assessing patient group fit with your engagement effort can be found in the “DIA Considerations Guide to Implementing Patient-Centric Initiatives in Healthcare Product Development.”

What Are Some Best Practices for Engaging with Patient Groups?

One practice in particular sets the foundation for successful engagement with the patient group(s) you believe can be valuable partners, and that is to establish an ongoing relationship as early in the product development process as possible. If you have responsibilities for recruiting patients to trials and want to engage patient groups to provide outreach advice or assistance, you should be relying on relationships that were formed before the protocol was written and even before the trial was designed.

Experienced patient groups share that they have been asked at the last moment to help rescue under-enrolled trials, and may not be successful in helping if the protocol design is too burdensome for patients or the product does not meet patient needs. Had they been involved at the time key decisions were being made, they may have been able to guide the sponsor to a better product or protocol design.

Engaging with patient groups in long-term relationships with the deeper purpose of open communication and sharing of experience from both perspectives can have intangible, but significant, benefits. According to the Clinical Trials Transformation Initiative (CTTI), the most valuable insights will come from ongoing, bi-directional communication within this type of trusted relationship.

Partnerships for initiatives with patient groups should be approached similarly to any other stakeholder collaboration, beginning with clear definitions for the following:

  • Mutually beneficial goals and objectives
  • Expectations of each party in terms of roles and responsibilities
  • Information/data that will be shared
  • Specifics about resources that will be provided

This is also the time for both parties to define how they will measure the success of the project, including achievement of objectives, impact of the effort, and quality of the engagement.

As with unique and expert input provided by other stakeholders (e.g., key opinion leaders), some initiatives amount to the provision of services by the patient group; for example, participation on patient advisory boards, provision of information on diseases or treatments, or informing product development processes. Patient representatives providing services have the right to expect appropriate compensation for time and expenses, and there is ongoing discussion among stakeholders about how to determine the fair market value of such compensation.

Further, ethical principles, including maintaining independence of the collaborating parties, must be strictly observed (a good discussion of this can be found in EFPIA’s (European Federation of Pharmaceutical and Industry Associations) “Ethical Rules for Collaboration between Patient Groups, (etc.) and the Pharmaceutical Industry.”

It Takes a Village

Best practices for communication during and after the project may seem like common sense, but multi-directional communication is sometimes overlooked or only assumed to be taking place. Clear communication between the collaborating partners should be frequent and ongoing throughout the project for addressing progress, points of difficulty, or changes of direction.

At the conclusion of the project, outcomes should be shared, as should appreciation for the commitment and efforts of the partnering group in the collaboration. Share the project outcomes within your company as well—successes encourage further adoption of patient engagement, and lessons learned help you and others to improve the next engagement project.

Which brings us to one last best practice in engaging with patients: Don’t expect perfect outcomes, and don’t be discouraged by mistakes. The most important outcome of patient engagement is establishing lasting relationships with the patient communities you serve.

Every sponsor-patient relationship is unique, but sincerity, openness, and mutual respect between partners is generally rewarded, regardless of specific project outcomes. Further, the art and science of patient engagement are in the early stages of development, and though most of us are still learning, we’re at a tipping point. Patient engagement will advance as a practice if we all keep working at it.

Debra Michaels ( is Associate Director of Scientific Programs for DIA.