Tag: Rare Disease

Consulting

NORD Summit, a Rare Disease Regulatory Wonderland

Rare disease is one of our main areas of focus here at Premier, so you’ll often see my colleagues and me talking about rare, through our webinars and white papers, and basically, almost any phone conversation with us. Last week, I attended the NORD Summit in Arlington, VA, and as usual, I found myself inspired...

Tales from the Road – Orphan Drugs Summit in Amsterdam

Introducing Tales from the Road, a series of blog shorts, where our Premier People report in from their travels outside the office. Because sometimes, not everything that happens on the road stays on the road! Today’s inaugural post comes from Juliet Moritz, Executive Director, Strategic Development Department, Rare Diseases, reporting back on her trip to Amsterdam...

Patient and Stakeholder Engagement

Register Now: Patient Advocacy Groups and Their Role in Orphan Drug Development

There are more than 7,000 known rare diseases, and these conditions afflict an estimated 30 million Americans (almost one in 10). Worldwide, some 350 million are living with a rare disease. But for 95 percent of these conditions, there are no FDA-approved treatments — not one. Conventional wisdom holds that innovation follows need: that if...

Clinical Research: Phase 1 - Phase 4

Getting It Right from the Start: Applying QbD to Rare Disease Studies

Quality and effective risk management are vital to every clinical trial, and their importance is greater still when working in rare indications. Your patient population is small, vulnerable, and geographically dispersed, so there’s limited data and little opportunity to replicate clinical trial results. It’s as if the phrase “get it right the first time” had...

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 Survey of Rare Diseases, Orphan Drugs Highlights Regulatory Challenges

A new survey of clinical trial decision makers commissioned by Premier Research reports that nearly two-thirds (64%) of respondents said that among the most difficult factors in recruiting for a rare disease or orphan drug clinical trial was not only finding patients to join and remain in trials, but identifying and setting up investigative sites for studies.

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.