Why Is The Reality Of Patient Engagement Not Living Up To Its Theory?
By Pauline Frank, Patient Co-Next
Unless you have been living under a rock in the biopharma industry for the past few years, you likely have heard about the benefits patient engagement can have on drug development. Even a quick search on ChatGPT for the most common statements about its importance in drug development and clinical trials gives solid reasons how patient engagement “enhances drug development,” “improves clinical trial design,” and “boosts innovation.”
However, despite these sound arguments, there is still hesitation from those actually conducting clinical trials. The theory of patient engagement does not necessarily translate into successful implementation. Why?
Below is a summary of eight key insights that I have gained from speaking and listening to many people across the industry who run clinical trials (noting that there may be exceptions, but these common themes come up consistently). Why is it important to hear their perspectives? When any change is intended to be implemented in healthcare, understanding the motivations, concerns, and experiences of those doing the actual activity is important to get to the root of the reasons for the barriers to change.
1. Many Speak Of Patient Engagement, Few Fully Implement It
First, let’s consider who the patient engagement enthusiasts are in the industry —excluding patient advocates and organizations in the not-for-profit sector, as we try to understand the challenges for implementation specifically among trial sponsors. Even in companies with seeming support from management, patient engagement stands on the shoulders of a handful of people who are personally very passionate about it. They typically sit within a function charged with patient engagement responsibilities. But outside of this function, other supporters are often scattered throughout the organization. There are too few supporters working in clinical trials to make a lasting difference. Then once these supporters leave, the momentum is lost.
Key takeaway: Clinical teams must also champion patient engagement.
2. Thinking It’s Only Possible In Big Pharma
The support and belief in patient engagement may not be deeply embedded within organizations, even those that cite themselves as “patient-centric.” Especially within large organizations, there are multiple layers between management and associates, who have many competing priorities. When patient engagement is not fully integrated into their normal activities, and instead treated like something special or additional, company reorganizations and downsizings may reverse any progress made. Thereafter the effort to implement patient engagement starts again.
If smaller companies look at the bigger companies and think, “They’ve got it all worked out, we don’t have a chance!”, rest assured that they haven’t. It is a continual learning process, and what is presented externally is “the best of, right now” and not necessarily implemented company-wide.
Key takeaway: Patient engagement must be part of a drug development company’s DNA to be sustainable, no matter its size.
3. A Lack Of Shared Learnings
Everyone is in a learning phase, and it will take time to build confidence, expertise, and the (shareable) case studies. The most reliable and well-documented examples are published in peer-reviewed journals, mostly by healthcare organizations and academia. Although there is some sharing from biopharma, these are surprisingly few and often lack adequate details of real challenges and results, including neutral or negative outcomes. This is likely due to concerns about confidential information (related to the target product profile or compound positioning) and/or sensitive topics (for example, the dynamics between the sponsor and the patient advocates). It is possible that with alignment between all stakeholders — clinical trial teams, patient advocates, company legal and compliance, etc. — more detailed and helpful case studies can be created.
Key takeaway: Share all kinds of learnings, not just what looks good.
4. The Value Of Patient Engagement Remains Unclear
Arguably the biggest issue raised by trial sponsors who are reluctant to fully implement patient engagement, is that the value or return on investment (ROI) of patient engagement in clinical trials is not well-defined. Although reports indicated that patient engagement in clinical trials can save x amount of dollars and y amount of time for sponsors, it does not seem tangible enough. In every clinical trial, there are many factors and confounders, and measuring the direct impact of patient engagement alone is nearly impossible.
Historically, sponsors call in patient advocacy groups when the house is already on fire and the trial recruitment needs to be rescued. When this happens, demonstrating the value of patient engagement at this late stage becomes even more challenging, and the expectations are unfair to patient groups. Even though it is common knowledge that most trials fail to recruit patients on time, leading to increased costs and delayed or canceled studies, the willingness to embrace patient engagement as an integral strategy early in the trial design remains low.
Key takeaway: Value cannot be demonstrated when it is unclear to stakeholders what to measure. Don’t just tick the “patients were engaged“ box. Be clear on what successful engagement means for the study and what impactful actions came out of it.
5. Current Trial Planning Processes And KPIs Do Not Encourage Patient Engagement
Linked to the question of value, is the question of cost. When sponsors plan trial budgets, they plan them by deliverables. If the value or ROI of patient engagement is not well defined, this method makes it challenging for trial teams to justify the investment.
As for KPIs, traditional measures such as time to first patient recruited mean there is less willingness to take any steps that may cause delay.
What could this look like? Not engaging with patients at all in the study design/protocol or applying the “any patient will do” approach.
What are the consequences? Not allowing or optimizing the right patient input can be short-sighted. Opportunities to address the trial burden and identify solutions to optimize engagement with the target patient population then get lost. This, in turn, leads to challenges in full patient cohort recruitment, overall delays, and poor data quality.
Key takeaway: Adapt processes and test new KPIs that can better reflect the value of patient engagement.
6. Regulations, Rules, & Roles Are Unclear
Engaging with HCPs is within the comfort zone for clinical trial teams. Engaging with patients is not. People have even expressed fear about this. Is it fear of the unknown? Fear of delays or budget blowouts? Fear of not being “allowed” to interact with patients?
The truth is a mix of all the above, and the only way to know is to ask clinical trial teams, who will likely only share if they feel trust. There is also uncertainty about regulations or bureaucracy in different countries (e.g., U.S. versus EU) on engaging with patients, with misperceptions that it is difficult to do in Europe.
There is also the question of who should be involved. Clinical trial teams need to be actively part of the engagement end-to-end, and not expect the patient engagement professional supporting them (whether internal or external) to manage it alone, as they wouldn’t have enough knowledge about each trial.
Key takeaway: Identify barriers/motivators among trial teams, then co-design processes (including legal and compliance) to operationalize the engagement as part of the clinical trial process.
7. Patient Feedback — What To Do With It?
There are many cases of great efforts to engage with patients for a clinical trial… and then the team does not know what to do with the feedback or is unwilling or unable to implement the feedback. Some points for reflection should be: Did we select the right patient advocates? Were our questions clear enough? Did we provide enough information and do so in an appropriate manner? Have we made enough effort to assess why a recommendation cannot be changed? Can we explain the rationale to the patient advocates?
8. Expectations Are Not Managed
When the expectations of clinical teams and patient advocates are not well managed, there may be disappointment and/or frustration, which then leads to reluctance for future engagements and even mistrust. An internal or external patient engagement professional has a key role in supporting this. The trial team should not expect that every engagement will provide clinically profound insights from patients. On the other hand, they should not dismiss recommendations that seem trivial because, for patients, such recommendations may be profound. They should also pay attention to recommendations that seem difficult or impossible. Even if they cannot be addressed in this trial, they could be in future trials.
For patient advocates, avoid over-selling the trial to them to avoid potential damage to the relationship if their expectations were not met (for example, the trial results were not positive or all their feedback was not implemented). Ensure that there is clear communication at the end of the engagement to explain what was implemented versus what was not, and why not.
“If You Want To Go Fast, Go Alone. If You Want To Go Far, Go Together.”
When I reflect on these challenges, I think of this old proverb. There are solutions to these challenges. As with any change in ways of working, it will take concerted and purposeful steps to properly implement patient engagement in clinical trials and research. Just telling people what ChatGPT already knows is not enough, and that trial teams cannot just “do patient engagement” effectively without help in overcoming these barriers.
About The Author:
Pauline Frank has worked for 17 years in the pharmaceutical industry and has led global initiatives in medical information and communications, technology platforms and digital solutions, and healthcare professional engagement across multiple organizational change initiatives. In the past six years, she has led patient engagement and insights for oncology and hematology disease areas, where she worked closely with clinical trial teams to instill patient centricity as a key driver of clinical trials that are truly patient-focused. Her mission is to make patient engagement in clinical research a gold standard. Her passion venture — Patient Co-Next — provides biopharma companies and clinical trial service providers with strategic guidance on pragmatic approaches to implement patient engagement sustainably. She believes in walking the talk!