ACT: What are some key considerations sponsors should be mindful of when aligning their decision-making process with the timeline of a trial?
Lwoff: Ultimately, understanding the need to have some flexibility with timelines. It’s a good idea to include built in buffers for unexpected delays or changes that happen often, such as delays in the supply chain additional time needed for study, and document preparation. They must understand that the timelines for regulatory submissions, review, and approval to measure the interactions with regulatory agencies accordingly and prevent potential delays in obtaining the necessary approvals.
Budgeting and financial planning should align with the study timeline to ensure the financial resources are available when needed throughout the study and the time required for data collection, monitoring and analysis should be considered and should be realistic. Sponsors must ensure that data management processes are efficient and that there are provisions for monitoring and addressing any data issues promptly.
When talking about timeline, it’s also important to consider that recruitment rate in a rare disease study will decrease throughout the course of the study, because, again, the population is limited in a rare disease clinical trial. As recruitment progresses, the number of available participants is reduced.
ACT: What do you think are currently the greatest obstacles for patient enrollment in the rare disease space?
Lwoff: There are a small number of individuals diagnosed with rare diseases, so it can be challenging to recruit enough eligible participants to a robust clinical trial. As noted before, the limited participant pool can extend the timeline for reaching enrollment targets. The criteria for enrolling patients in rare disease can be very specific, which can further limit the pool of eligible participants a rare disease trial, and very often involves complex procedures, frequent visits or other long-term commitments that patients and caregivers may be hesitant to enroll due to the potential impact that these may have in their quality of life. We also need to consider that currently, there may be no available treatments for most rare diseases. Depending on the severity of the condition, patients may be open to participate in clinical trials due to the clinical benefit that they may obtain in their condition.