With their own unique challenges, studies within rare indications require a creative and proactive clinical trial team that has experience in the rare disease domain.
In this article, we discuss a handful of tactics that sponsors may deploy to improve their chances of a successful study.
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When setting up a rare disease clinical trial, sponsors must ensure the study design is both scientifically rigorous and practically feasible given the challenges of rare disease research.
Two key elements to consider during this planning phase include research and collaboration with Patient Advocacy Groups (PAGs) and working closely with experienced biostatisticians to explore innovative statistical designs.
PAGs often play a pivotal role in rare disease trials. These groups are often the backbone of the rare disease community and can provide invaluable insights into the disease’s natural history, patient experiences, and treatment needs. Early engagement with PAGs can enhance your trial’s design and execution, ensuring it is patient-centric and more likely to succeed.
Begin by researching and profiling PAGs in your study space, as different groups may have various levels of resources, goals, and involvement in research or clinical trials.
Given the complexities of rare disease trials, traditional statistical methods may not always be appropriate. Early collaboration with a consulting biostatistician who has ample experience within the rare disease space can help your team explore innovative statistical design that may significantly enhance a study’s feasibility and scientific rigor.
Early, collaborative engagement and strong relationships with Key Opinion Leaders (KOLs) can give study teams a wealth of insight.
Consider conducting interviews with KOLs on a project manager to KOL or sponsor to KOL level. These interviews may result in recommendations such as other PIs to involve or relevant advocacy or support groups to be aware of.
During the study enrollment period, remain involved with site staff – including working with staff to identify and attend potential events where the rare disease community may be coming together.
Additionally, you may consider maintaining a media presence. While your approach should depend on study specific details, your media presence may include campaigns such as Google Ads, social media marketing, or content marketing via featured blog posts.
PharPoint is a US-based CRO that has supported a large number of rare disease trials. To learn more about our capabilities and how we can help you confidently conduct an effective rare disease trial, reach out to our team.
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