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The Trials of Rare Disease Clinical Research

Thoroughly explaining a clinical trial can alleviate a patient and increase the number of participants in a trial, especially for a rare disease. Image courtesy of Fishers Hypnosis.3

While we previously discussed how rare disease research has dramatically increased in both academic and industrial research, clinical trials of potential rare disease drugs are difficult to conduct. Typical common disease clinical trials can select from a large pool of candidates. Rare disease clinical research has multiple disadvantages including having a smaller sample size, less understanding of the actual disease, and a scarcity in clinical researchers in the rare disease fields. Despite these challenges, there are still opportunities for researchers to delve into the world of rare disease research.

One of the major challenges of rare disease clinical trials is the shortened life span of many rare disease patients. There are many ethical concerns in obtaining placebo-controlled results in these trials, since parents may also be reluctant to enroll their children in trials where they potentially may not receive treatment. However, clinical research CROs have understood that working closely with patient advocacy groups will increase the number of recruits in their clinical trials, especially when these advocacy groups are able to thoroughly explain the trials to both patients and their families. Increasing the number of patients in trials will allow for better data, and ultimately more effective results in the search for rare diseases cures.

Scientist has always advocated rare disease research, and has raised awareness for rare diseases through last year’s Rare Disease Challenge. An important aspect of orphan drug development is the need for these potential drugs to successfully undergo clinical trials. In fact, Scientist has many clinical trial CRO vendors. One in particular is Agility Clinical, whose mission is to help pre-clinical and clinical companies develop drugs and devices, particularly in the rare disease and pediatric disease fields. This and other clinical trial CROs understand the challenges that face rare disease research and because they are specialized towards clinical research with highly trained scientists, they are able to guide biotech companies or pharmaceutical companies towards a more efficient trial design. This is just another example of how CRO services can increase the efficacy of the drug discovery pipeline at the clinical trial stage, especially in the rare disease field!

  1. Augustine E, Adams H, Mink J. Clinical Trials in Rare Disease: Challenges and Opportunities. J. Child Neurol. Sept 2013 vol. 28 no.9: 1142-1150.