Expert Insights

Expert Insights

Planning a Rare Cancer Study

Planning a Rare Cancer Study
19 April 2021

Of all cancer types, 22% are considered rare, according to Rare Cancers Europe (RCE). Those rare cancers correspond to more than 200 different tumor types, a higher diversity than the most frequent breast, colon, prostate, and lung cancers put together.

Typically, the differing histologies of various cancers have informed therapy development. With some of the more recent advances in molecular biology, clinical trial design involves different medical and protocol considerations. And as cell and gene therapy progresses, researchers also need to consider genetic variants when targeting a rare cancer subgroup.

Today, sponsors and biotech CROs can get specific with their clinical trials. Rare malignancies now represent about a quarter of all cancers. Pediatric studies are increasing. And researchers have identified rare groups such as triple-negative breast cancer.

Designing Your Rare Cancer Protocol

Because of the small populations available, data access, organization, and management and statistical design are key in rare cancer studies. Other protocol considerations include the following:

  • Shorter-term endpoints, such as three- or six-month progression-free survival, to increase detection of benefit
  • Retrospective database review for insight into disease patterns and management
  • Designs like Bayesian, adaptive, and continuous reassessment methods for more reliable data and faster trial completion in small populations
  • Next-generation sequencing to detect mutations that help guide new treatments

Early Feasibility

Assess feasibility during the earliest stages of study planning and throughout development. Identify where patients reside, how to access those patients within the context of the trial, and what they need to participate. A global feasibility assessment is paramount for rare cancer studies.

Establish Partnerships

Given the operational complexities and limited patient populations for rare cancer studies, a strong network is key to success. Establish relationships with partners that can provide access to data and to potential participants. Tap social media networks, patient advocacy groups, and similar communities to communicate the value of participating in clinical trials — including yours.

Assessing feasibility, designing protocols, and later, gaining access to real-world data, all require a different approach when developing rare cancer treatments. For the full story on how to refine this approach, download our Guide to Rare Cancers: What to Consider for Complex Clinical Studies.

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