Rare Disease

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Recruiting from small populations, deriving valid conclusions from limited data, and helping customers achieve orphan drug status are challenges we know very well.

Rely on our mastery of rare disease

Our rare disease work covers all aspects of clinical trials and drug development. We’ve conducted 115 studies in 50-plus indications in the past five years.

What We Do Best

We have the hands-on experience and the seasoned pros to anticipate and overcome rare disease obstacles.

Challenge No. 1: Ensure feasibility and scientific rigor of trial design

The solution is more a matter of broad experience than of learning a set of rules.



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Challenge No. 2: Identify, recruit, and retain patients

It’s always important, but when there may be only 50 or 100 qualified patients in the whole world, the word “challenge” takes on a whole new meaning.

Challenge No. 3: Select effective sites — and properly train and motivate investigators

One-on-one relationships really matter.

Challenge No. 4: Achieve orphan drug status

Orphan drug designation is not automatic — it’s earned. We can help you over the regulatory hurdles.

Challenge No. 5: Stay on track, even in unfamiliar territory

It’s tricky, and often there are no precedents or well-established guidelines to show the way.

Rare disease resources

Watch our webinars, read informative white papers and case histories, and check out a fact-filled infographic.



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