Tag: Orphan Drug

Clinical Research: Phase 1 - Phase 4

Proven Strategies for Rare Disease and Orphan Drug Development in the U.S.

Orphan drugs are medicinal products intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. Orphan drugs are a growing market, due in large part to tax incentives, longer periods of market exclusivity, and shorter, smaller clinical trials, as well as the premium pricing associated with many orphan...

Patient and Stakeholder Engagement

When Rare Diseases Take Research Far Afield

Researching rare and ultra-rare diseases sometimes means taking the study to the patients, setting up sites in countries unaccustomed to hosting clinical research. Premier Research will examines the  challenges of conducting trials in such unconventional locales in this webinar. Opening sites in countries where trials are uncommon and not thoroughly regulated requires that sponsors really...

Patient and Stakeholder Engagement

Premier Research’s New Rare Disease and Orphan Drug Survey Accentuates Difficulty of Recruiting Patients and Motivating Patients to Take Part in Study

A new survey of clinical trial decision makers commissioned by Premier Research and involving 50 biotech and pharmaceutical firms in North America and Europe reveals that more than two-thirds (69%) of respondents said that among the most difficult factors in recruiting patients into a rare disease clinical trial was not only finding and motivating patients to join and remain in trials, but identifying and setting up investigative sites for studies.