We Know Rare Disease

Explore our resources drawing on more than 190 orphan drug clinical trials.

Position compounds for clinical and commercial success

Despite aggressive orphan drug development over the past 15 years, the pharma industry — dissuaded by small patient populations, high costs, and limited commercial potential — has barely put a dent in the 7,000+ known rare diseases.

In this white paper, we look at opportunities in the field of rare disease treatments and outline strategies to maximize the odds of success.

Click here to access the white paper.


You may be interested in these additional resources:

Podcast: Engaging Patients in Clinical Trials w/ Alan Thomas & Juliet Moritz

Alan Thomas & Juliet MoritzAs Premier Research’s Vice President, Patient and Stakeholder Engagement (PASE), Juliet Moritz has viewed this evolution up close — and so has Alan Thomas, founder of an organization that advocates on behalf of people afflicted with ataxia, a rare neurological disorder. Listen to the podcast now.


Blog Post: 5 Operational Considerations For Rare Disease Trials

For studies in support of orphan drug designations, operational concerns can be broken down into five major issues: patient identification, patient engagement, patient retention, investigative site selection, and site team support. Read more here.


Case Study: Using Every Tool To Fight A Rare Disease

Close-up of two people grayscaleOur recruiting target was 18 patients, but even that was a challenge for a condition that afflicts just one in 31,000 people. It required making full use of what few resources were available, including key opinion leaders (KOLs) at the two trial sites and a national patient advocacy group. Read the case study.

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