Challenges Facing Rare Disease Clinical Trials

The USA Orphan Drug Act of 1983 created incentives for pharmaceutical companies to research and create new drug therapies for rare diseases. These incentives include tax breaks, exclusive marketing rights, unique research grants and fast-tracked FDA review processes. As a result, the clinical costs associated with the development of an orphan drug are less than those associated with non-orphan drugs1. Despite these research advantages, there are also unique challenges associated with running rare disease clinical trials:

Patient Recruitment. About 30% of Phase III clinical trials fail because they can’t meet enrollment targets2.This statistics is especially pronounced in rare disease clinical trials where the patient population is not only limited, but also scattered around the globe. Furthermore, about 50% of rare diseases affect pediatric patients, which leads to more ethical and legal considerations when recruiting and enrolling patient in clinical trials2 leading to slower recruitment timelines. Finally, given the small patient population size, it is more difficult to perform replication studies for rare diseases.

Clinical Trial Design. Because the study population is small and often heterogeneous in terms of age, sex and/or disease severity, common methods for clinical trial design fail to show statistical significance and are thus not suitable to use. As a result, orphan clinical trials are often nonrandomized, un-blind and do not use a placebo control. Instead, cross-over, N-of-1 and adaptive design studies are used for rare disease research3. Adaptive designs are most frequently used and consist of analyzing interim data of a study to decide how to modify or proceed with the clinical trial without compromising the integrity of the research4.

Site Suitability. As the rare disease patients are scattered around the globe, it is necessary to establish more research sites that are complementary to patient locations. With more research sites, there is a requirement to recruit and train more clinical personnel to correctly administer interventions and track patient progress. Similarly, with each additional site, the sponsor must make sure that the trial locations are equipped to handle the requirements of the investigational product. For example, if a drug requires cold-chain storage, the site must have appropriate refrigeration equipment to handle the strict temperature needs.

As you can tell, planning rare disease clinical trials can get complicated, so let us handle your clinical trial logistics needs and use your valuable time to focus on research design and recruitment. At Bay Area Research Logistics, we have experience in supporting clinical trials of all sizes in more than 30 countries around the world. Whether you are looking for phase I, II, III or IV logistics support, Bay Area Research Logistics will help you execute the planning, labelling, packaging, storage and distribution needs. Contact us today to learn more about our services and experience!

1 Jayasundara, K., Hollis, A., Krahn, M., Mamdani, M., Hoch, J. S., & Grootendorst, P. (2019). Estimating the clinical cost of drug development for orphan versus non-orphan drugs. Orphanet journal of rare diseases, 14(1), 12.
2 Khaleel, S. L. (n.d.). Rare Disease Patient Recruitment And Retention. Retrieved from
3 Kempf, L., Goldsmith, J. C., & Temple, R. (2018). Challenges of developing and conducting clinical trials in rare disorders. American Journal of Medical Genetics Part A, 176(4), 773-783.
4 Hilgers, R. D., K?nig, F., Molenberghs, G., & Senn, S. (2016). Design and analysis of clinical trials for small rare disease populations.?Journal of Rare Diseases Research & Treatment, 1(1), 53-60.

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