Feasibility in Uncommon Populations

Clinical trial feasibility determinations in rare disease

Michael Murphy, MD, PhD

How do feasibility determinations for interventional studies in pharmaceutical research differ when addressing rare diseases? The process has changed radically over the last few decades as incorporating the perspectives of many different stakeholders in trial design and operations has become more important. Protocol development addressing rare disorders with significantly unmet clinical needs is no longer the isolated remit of those sponsoring the trial. Design now begins with the patient and extends to incorporate the input of the patient’s extended support group as well as contributions from multiple healthcare providers. Design also accommodates ongoing changes in healthcare delivery as well as the evolving regulatory sentiments that enable these trials.

Today, feasibility starts during protocol design: innovative study designs and end points, novel treatment options, and expanded regulatory options are all explored at a very early stage. The timing and the propriety of expanded access programs for patients are considered as part of a process that attempts to maximize access to the active treatment and minimize exposure to the control. The potential for accelerated approval pathways, fast track designations, and breakthrough therapy designations when data are appropriately supportive add additional dimensions for review when considering strategic program options.

This paper will examine ways in which the approach to feasibility in rare, geographically dispersed populations has evolved, with particular attention to the perspectives of those stakeholders responsible for facilitating trial design and operations. How the research and development process approached that goal previously, modified the process recently, and continues to examine new directions for innovative solutions in the future is a focus of review.

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Michael Murphy, MD, PhD
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Michael Murphy, MD, PhD

Chief Medical and Scientific Officer
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