A version of this article was previously published in the Journal of mHealth.
These days, clinical trials are in the news pretty much daily. Whether successful or not, they can impact not only the perception of a pharmaceutical company but also their stock prices. While still the gold standard of research, clinical trials are often riddled with issues that limit their applicability to broader populations or delay market access.
In this article, we will explore a few of these issues, along with how getting early and timely feedback from all relevant stakeholders can help prevent them from occurring or minimize their impact.
The complexity of trials
Clinical trials are becoming more complex. For example, there are now 2.5 times as many clinical endpoints and procedures compared to 15 years ago, increasing the burden on patients and the trial sites. As a result, the learning curve for both clinical trial participants and site staff can be extraordinarily steep. If the clinical trial experience can be simplified and improved, studies will become faster, more efficient, and–ultimately–better for the patients.
The need for robust and flexible protocols
One of the greatest challenges facing clinical trial investigators and sponsors is developing a robust and flexible trial protocol. In fact, most experimental drugs do not fail because the drug is bad, they fail because of poor clinical trial design. This indicates a need for multi-stakeholder review of the protocol early in the clinical trial planning process, including statisticians, clinicians, regulators, and patients.
Diversity (or the lack thereof)
Clinical trials are known to have a major diversity problem. Although this issue has gained substantial attention in recent years, it remains largely unresolved. In the US, approximately 75% of clinical trial participants of approved drugs from 2020 were white, even though minority racial and ethnic groups make up around 40% of the population. Of course, ethnicity is not the only area where diversity is lacking. Factors such as age, sex, gender identity, socioeconomic status, disability, pregnancy and lactation status, and the presence of comorbidities should all be factored in to ensure a clinical trial population that mirrors the real-world situation.
Suboptimal patient recruitment, retention, and clinical trial experiences
Patient recruitment can be a major rate-limiting step, especially now that more focus is (rightfully) being placed on participant diversity. Once recruitment is complete, participant retention is another hurdle that can impact the trial’s timelines and outcomes. Reportedly, less than 5% of clinical trial participants are asked for their feedback on the trial and as many as 8 out of 10 do not feel empowered or like they are making a difference. Understanding the participant experience before, during, and after clinical trials is key and should be the first step to addressing the issues with recruitment and retention.
Understanding the nuances of the patient journey
Before moving a molecule to the next clinical trial phase, sponsors and investigators need to understand where the product will fit in the treatment algorithm and how it will impact the patient journey. This includes gathering information of the current standard(s) of care that physicians are prescribing and where they perceive the treatment gaps to lie. However, to get a holistic view, insights-gathering initiatives should not only include prescribers, but also the end users: the patients.
Overcoming the barriers
If you haven’t guessed it already from the title, when used wisely, virtual insight-gathering can help overcome many of the issues highlighted above.
Robust clinical trial protocols
To overcome the issue with poor study designs, robust clinical trial protocols can be co-created or optimized by seeking input from all relevant stakeholders early on in the process. Feedback from patients will ensure that the primary and secondary endpoints are relevant to them and that potential barriers to participation are minimized. Physicians and other prescribers should be consulted to make sure there is buy-in. Further, their input is crucial for understanding whether the control arm in the trial reflects the standard of care in their clinical practice, the protocol is comprehensive, the “right” people are enrolled, and the inclusion and exclusion criteria are reflective of the real-world patient population that would be candidates for the drug.
Regulators and payers should also be consulted before the Phase 3 clinical trial protocol is finalized; getting their feedback is key to gaining clarity on what data and endpoints to focus on in order to maximize the chances of successfully getting the drug to market. Finally, other key stakeholders such as clinical pharmacologists should be consulted to help determine the right dose and administration schedule, and independent statisticians should be asked for their input on the data collection and analysis.
Efficient clinical trial recruitment
When it comes to overcoming the issues plaguing clinical trial recruitment and retention, virtual insight-gathering can help understand how and where patients learn about clinical trials and what factors will impact their decision to enroll. Patient advocates can be engaged to co-design outreach strategies and recruitment campaigns. Virtual insight-gathering can also be conducted during or after a trial to gain a deeper understanding of the patient experience with the trial. In turn, collecting this feedback and acting upon it can improve patient empowerment and satisfaction with the trial itself, as well as retention in future trials.
Of course, “virtual insight-gathering” can look many different ways. Typically, it involves a mix of real-time (i.e., web meetings) and asynchronous (over-time, anytime) touchpoints. The latter is useful for engaging international and diverse groups of stakeholders for whom finding a time to meet in real-time can be challenging or there may be language barriers preventing everyone from contributing fully. Activities can be planned at regular intervals or be ongoing in the form of online community boards and portals. Hosting parallel or multidisciplinary virtual touchpoints ensures that all relevant stakeholders can share their unique points of view. This is also much easier asynchronously from a scheduling perspective. Further, some junior clinicians and patients may not feel comfortable with providing verbal feedback in front of investigators, sponsors, and their peers; the asynchronous format provides a layer of anonymity and psychological safety, encouraging honest feedback.
Clinical trials are notoriously complex and expensive, not to mention riddled with numerous issues. While it’s an uphill battle to solve these issues, virtual insight-gathering, especially when started early and when including all relevant stakeholders, can help mitigate many of them. Considering the enormous time- and monetary cost of a failed trial, the upfront cost of seeking insights from clinical experts, patients, and payers is a drop in the ocean and an investment well spent.