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International Clinical Trials

Needle in a Haystack

If there is one hot zone in drug development, it is the area of rare diseases. Formerly the province of small biotechnology companies and pharmaceutical startups, this area is now attracting the attention of increasingly bigger players, as most recently exemplified by Shire’s establishment of a separate rare disease innovation hub in Cambridge, Massachusetts, US. As we have moved beyond the blockbuster era, and with the declining productivity of Big Pharma pipelines, many larger firms are looking to rare diseases as a potential alternate revenue stream – hence the increased pace of partnering and acquisition activity, as well as efforts to expand ‘organically’ into the field.

Despite the rush of entrants into the category, conducting clinical trials in this area remains a complex proposition. Much of the difficulty is derived from the fact that many rare diseases are not as well understood as more common ailments like diabetes and hypertension. The relatively small patient populations often do not easily lend themselves to defining efficacy endpoints, inclusion and exclusion criteria or other study design elements. Such a lack of clarity makes it especially difficult to devise a protocol that will meet the FDA’s scrutiny, and could even discourage people from participating.

Incentives and Challenges

Nevertheless, pharmaceutical developers have numerous motivations for conducting these studies, including Prescription Drug User Fee Act waivers, tax credits and extended exclusivity for orphan treatments; these can give companies extra time to recoup trial-associated costs. That said, many start small – initially focusing on indications with small patient populations, which may be a springboard to expanded indications or, if they are lucky, off-label usage.

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Scott Treiber, PhD, MBA, is Executive Vice President of Biopharmaceutical Development at Chiltern and oversees all aspects of biopharmaceutical development including Phase 1-4 trials across a wide range of therapeutic areas. Before joining Chiltern, he held Vice President positions at Theorem, inVentiv Health, Hospira and PharmaNet. Scott is on the board of the Center for Healthcare Innovation.
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