Tag: orphan drug development

Consulting

5 Operational Considerations for Rare Disease Trials

From researchers to sponsors to patients to advocacy groups to clinicians, rare disease research is full of passionate, driven, and determined people. As the greatest asset to the field, all people involved must be utilized to their greatest potential for a study to be successful. That’s why significant attention must be paid to the operational challenges within...

Consulting

Optimizing Regulatory Considerations for Orphan-Drug Development on a Global Scale

Rare disease and orphan drug research has a long history of serious challenges, thanks to both intrinsic factors—fewer patients means less available data; less available data means a less complete picture—and market forces—it’s more difficult to find investors when a smaller pool of patients may mean a more uncertain potential return on investment. Luckily, recent changes...

The Outlook for Orphan Drug Research: A Live Webinar

DURHAM, N.C., DECEMBER 5, 2017 — The outlook for orphan drug research is more promising than ever, with regulators providing a progressive development platform and government reforms putting new emphasis on the need to treat rare diseases. Premier Research will examine the outlook for biotech and pharma companies at a live webinar on Tuesday, December...

Premier Research Names Kimberly Glen Executive Director for Rare Disease

DURHAM, N.C., JULY 24, 2017 — Premier Research has named Kimberly Glen an Executive Director for Rare Disease, Strategic Development Department. She brings to the company nearly three decades of experience in contract research, drug development, and pharmaceutical consulting. “Rare disease is an extremely demanding field of study, imposing unique challenges in patient recruitment, deriving...

Consulting

4 Historical Orphan Drug Development Barriers, Have We Broken Through?

Both understanding the role of patient advocacy groups in building better clinical trials and improving the research process mean first taking a look at the history of orphan drug development in the United States and Europe. An “orphan drug” is a drug that treats a condition that fewer than 200,000 people have. Because rare diseases...

Patient and Stakeholder Engagement

The Future of Patient Advocacy and Orphan Drug Development: Challenges and Opportunities

Despite advances in the orphan drug development process and the more prominent role that patient advocacy groups now play, researchers in this area still face a number of challenges. But, fortunately, increased collaboration between patient groups, industry leaders, and regulatory bodies can help overcome these hurdles. Barriers to orphan drug research include: Small population By definition,...

Patient and Stakeholder Engagement

Gaining Strength: Webinar to Explore Advocates’ Role in Drug Development

DURHAM, N.C., August 10, 2016 — Patient advocacy groups play an important and growing role in developing drugs to treat cancer and rare diseases, contributing to study design, recruiting patients, and even directly funding clinical trials. At a September webinar, Premier Research will team with rare disease advocacy organization Global Genes to explore this important...

Medical and Regulatory Affairs

What’s Ahead in Orphan Drugs? Attend Our June 1 Webinar

DURHAM, N.C., May 31, 2016 — Treatment of rare indications is turning a promising corner as regulators and lawmakers boost incentives and guidance in these areas of unmet medical need. Three experts from Premier Research will look at financial aid, expedited reviews, and other avenues available to orphan drug developers in a webinar on Wednesday, June...

Premier Research to Highlight Rare Disease Expertise at WODC 2016

DURHAM, N.C., April 13, 2016 — Premier Research will have a large presence at this year’s World Orphan Drug Congress, April 21-22 in Washington, D.C. The company is a platinum sponsor of the 2016 conference, and its top medical expert will moderate a panel discussion on drug development. At WODC, more than ever a top...