Rare Disease

PharmaLive – Incentives, Opportunities Abound as Orphan Drug Demand Grows

If ever a drug development effort called out for priority review and approval, this was it. A U.S. pharmaceutical company was developing a treatment for hypophosphatasia, a progressive, ultra-rare metabolic disease for which only supportive therapy was available. Asfotase alfa, an innovative enzyme replacement drug, was the first real hope for those diagnosed with HPP,...

Clinical Research: Phase 1 - Phase 4

Rare Oncology Research in the 21st Century

Speakers: Sachin KulkarniExecutive Director, Strategic Development, Oncology & General MedicinePremier Research Juliet M. MoritzExecutive Director, Strategic Development, Rare DiseasesPremier Research Study of rare cancers tends to look more like rare disease research than standard oncology research. This webinar will highlight some of the challenges in designing and conducting trials for rare cancers and explore positions...

Patient and Stakeholder Engagement

When Rare Diseases Take Research Far Afield: Dec. 15 Webinar

PHILADELPHIA, December 15, 2015 — 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 examine the challenges of conducting trials in such unconventional locales at a webinar on Tuesday, Dec. 15. Opening sites in countries where trials are...

Clinical Research: Phase 1 - Phase 4

Rare Disease Trials: The Beginning, the Middle and the End

Speaker: Angi Robinson, Executive Director, Pediatrics and Rare Diseases, Premier Research This session includes a regulatory overview of rare diseases and orphan drug status as well as practical considerations for varying stages of the development process. Viewers will learn more about conducting successful Natural History studies and the presenters will review how to avoid common...

Medical and Regulatory Affairs

Regulation of Orphan Drugs: May 19 Webinar Offers Important Insights

Join Nach Dave and Carol Huntington of Premier Research for Orphan Drug & Rare Disease Development: Understanding the European and US Regulatory Landscape. The hour-long event, beginning at 11am EDT, 4pm BST, and 5pm CEST, is aimed at researchers and pharma companies tackling some of the 7,000 rare diseases identified by the National Institutes of Health.

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.

Premier Research Experts to Share Rare Disease Insights at World Orphan Drug Congress

Premier Research will present a speaker and host two roundtable sessions at the World Orphan Drug Congress, April 22-24 in Washington, DC. The Philadelphia-based contract research organization is a platinum sponsor of the fifth annual event, a top meeting place for biopharma, biotechs, government, patient groups, and others.

Achieving Uncommon Success in Rare and Ultra-Rare Disease Studies Webinar

Premier Research and Alexion Pharmaceuticals will co-present a webinar on achieving uncommon success in rare and ultra-rare disease studies on Wednesday, March 11, at 11am EDT (NA) / 3pm GMT (UK) / 4pm CET (EU-Central).

Premier Research Employees to Support Rare Disease Awareness

7,000 rare diseases, many untreatable. 50% of patients are children. 35% of deaths in the first year of life are due to rare diseases. To everyone at Premier Research, that’s a call to action. So Premier Research employees worldwide will join together on Friday, February 27, to raise awareness of Rare Disease Day 2015.

Premier Research Augments Staff in Fast-Growing Pediatrics and Rare Disease Clinical Trials Division

PHILADELPHIA, March 18, 2013 — Premier Research today announced the addition of four talented clinicians to key leadership roles in its fast-growing pediatric and rare disease clinical trial research disciplines. The Philadelphia-based global contract research organization (CRO) named Dr. Susan P. Tansey, MBCHB, MRCP, CCST, medical director, pediatrics. Her medical specialty areas include vaccines, cardiovascular, and oncology, although her initial training was in neonatology and pediatric medicine. She has considerable experience in pediatric clinical research and trial design.