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The Role of Key Opinion Leaders in Rare Disease Clinical Trials

Written by Angela Vinken and Patti Arsenault

 

Key Opinion Leaders (KOLs) — i.e., trusted, well-respected experts — are crucial in clinical research, especially in rare diseases. They have the expertise, network, and experience to add great scientific value to clinical research and can influence and shape the trajectory of clinical research within their professional communities.

The challenge, however, is how to use KOLs wisely — they are often clinicians first and researchers second, and their time is in high demand. Here, we discuss how KOLs can shape clinical trials, best practices for working with KOLs, and key considerations and potential challenges.

 

How key opinion leaders can shape clinical research

Key opinion leaders are often the (lead) principal investigator on a trial or data monitoring committee, which lends credence to the trial and the sponsor. In rare disease communities, strong links exist between treating physicians, patients, and caregivers or advocates, and KOLs. Having the endorsement or involvement of a relevant KOL for rare disease clinical research can therefore have several important impacts:

  • KOLs can help with patient recruitment and site participation. KOLs reassure potential participants of the value of the research and the quality of the care during the trial, thus encouraging more patients to enroll in the trial.

  • KOLs can attract other clinicians to participate in the trial. KOLs are highly regarded in their fields and so can often attract other clinicians to participate in clinical trials, which can be crucial in today’s competitive landscape for both enrollment and site participation.

  • KOLs can improve trial design, analyses, and dissemination of results. KOLs use their experience to ensure the endpoints considered are meaningful to clinicians, patients, and regulators, and that effect sizes are clinically and medically relevant. With their wide networks, KOLs can help ensure results reach as wide an audience as possible. 

  • KOLs can potentially decrease clinical trial timelines. The combination of the above points (a KOL’s scientific input, standing in their community, and trust fellow clinicians and patients have in the KOLs opinion and input) can impact trial timelines, which in turn would get products faster to patients. This is particularly important in rare diseases, where the patient population is limited.

 

How to select the best KOL for your study

To maximize the potential impacts stated above, it’s important to ensure that your outreach is tailored to a KOL’s expertise, interests, and priorities, and to consider how these align with your research. A good value proposition will highlight how their specific knowledge and expertise can contribute meaningfully to the trial and, more important, how this could improve the clinical environment for the entire patient community.

To use their time most appropriately, it is crucial to confirm their exact role and involvement in your trial from the beginning: provide enough time to allow for their input on the trial design and protocol, and site and patient recruitment strategies. This early engagement will foster ownership and solidify their continued involvement.

 

Important considerations when working with KOLs

There are several important considerations a sponsor will need to make when working with KOLs to ensure the trial runs as smoothly as possible.

  • Avoid any conflicts of interest. A solid Conflict of Interest is required to ensure both their and your needs around integrity and trust are covered.

  • Their involvement should be focused. A KOL’s time is often in high demand, so flexibility will be required, and their involvement should be focused. Concise and frequent communication will align needs and regular realignment of their engagement is important. Consider using virtual meetings rather than face to face to minimize efforts.

  • Include diverse perspectives and avoid potential bias. As KOLs are often very close to their patients and their community, they may inadvertently introduce bias or unduly influence decisions by focusing on their own area of specialty. A combination of KOLs will allow for diversity (e.g., of different areas of expertise and focus) and inclusion to ensure representation from a variety of perspectives and patient populations. An open dialogue that encourages multiple viewpoints will be important; any decisions will need to be evidence-based and confirmed within the team — with proper discussions and decision rights taken into consideration. This will strengthen the research, and results will be more impactful.

  • Promote the KOL’s involvement in your trial. KOLs often want to be involved in related publications, speaking engagements at conferences, or leadership roles in trial-related committees, which increases their stance in their scientific community. Acknowledge and celebrate the contributions of KOLs to the trial’s success, especially since you will want to keep the KOLs on board for the next project!

  • Consider relevant regulatory requirements. Any involvement of KOLs in trial design, execution, analyses, and/or dissemination of results has to follow regulatory requirements, especially around remuneration and promotional activities. A clear contract will protect both the sponsor and the KOL. Patient welfare and ethical considerations should, of course, come first in any interaction with and involvement of KOLs.

 

Key takeaways

By adhering to these principles or best practices, pharmaceutical companies can build strong and mutually beneficial relationships with KOLs, fostering collaboration, innovation, and, ultimately, better healthcare outcomes for patients.

 

 

 

Angela Vinken_cropAbout Angela Vinken

Angela Vinken is Global Head, Portfolio Leadership, at Cytel. She has 25+ years of experience in the CRO industry, in varying leadership roles across Phase 2–4 clinical research, including leading teams in project and clinical management, resource management, and records management. Angela is a process-driven business leader enabling the achievement of full business potential through business development/operational leadership to accelerate business growth and product-line and client-base expansion.

 

 

Patti Arsenault_crop

 

About Patti Arsenault

Patti Arsenault is Vice President, Quality Assurance, at Cytel. With a background in programming and data management, she has more than 20 years of experience in understanding quality in clinical trials. Cytel’s global team of quality specialists help teams bring continuous improvement in all areas.

 

 

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