Achieving a sponsor’s goals, though of utmost importance, should only be one area of emphasis during a clinical trial. One must also consider the broader team of stakeholders—the patients, their caregivers and their physicians. This is never more true than in the study of rare diseases.

By their very nature, rare con­ditions are not always well under­stood, and the endpoints for study success are not always well estab­lished. This, often combined with small, scattered patient populations and a high proportion of younger subjects, calls for well-thought-out protocols and teams that under­stand how to operationalize these challenging studies.

Patient recruitment for rare dis­ease trials can be difficult, which makes the retention of both the pa­tient and their care team critical. In­vestigators should work in tandem with patients, their caregivers, phy­sicians and patient advocacy groups to consider each patient’s individual experience. Rare diseases patients and their caregivers are typically well-informed about their condi­tions, meaning their insights should be considered in the study design; this helps foster a feeling of owner­ship in the outcome of the study on behalf of the subjects. Finally, clear communication of the study plan is important to both recruit and retain patients.

Understanding the patient ex­perience during the design phase can ensure the intent of the trial is met. For example, in a study in­volving young Duchenne muscu­lar dystrophy patients, one of the standard assessments was an early morning walk test. Through en­gagement with an advocacy group, it was found that children with this condition may suffer from stiffness in the morning and preform this assessment at a disadvantage, lead­ing to less accurately representative results. A simple adjustment to the assessment schedule resulted in a better outcome.

Gathering input from as many stakeholders as possible during the clinical study design phase and em­bracing a larger and more inclusive team can be integral to success. By working together, these teams will be able to deliver outcomes for future treatments that are fully in­formed by those most eager to re­ceive the benefit

Written by Guest Writer Dr. David Frakes, M.D. Frakes is the senior vice president of Project Management for Neuroscience at Worldwide Clinical Trials. Dr. Frakes leads the global Neuroscience project management unit at Worldwide, with full accountability for development and execution of a wide range of Neuroscience studies, including those in Psychiatry, Neurology, Alzheimer’s, Pain and Orphan Diseases. A Board Certified Psychiatrist, Dr. Frakes has more than 26 years of industry experience, with extensive expertise in central nervous system clinical drug development. Please visit www.worldwide.com to learn more.

This article was reprinted from Volume 23, Issue 12, of The CenterWatch Monthly, an industry leading publication providing hard-hitting, authoritative business and financial coverage of the clinical research space. The Action Items section features short columns  focusing on actionable or how-to advice from clinical trial professionals. To submit an Action Item, please contact editorial@centerwatch.com. Subscribe >>