Rare Disease

Clinical Research: Phase 1 - Phase 4

Tackling Common Issues in Gene Therapy Trial Operationalization

Between navigating relatively uncharted territory and the practical limitations of most gene therapy delivery systems, ensuring successful clinical trials of these products is a serious challenge for even the most experienced researchers. Compounding this is the long-term nature of most gene therapy trials — it’s not unusual to have follow-up periods of a decade or more....

Medical and Regulatory Affairs

The Guardrails of GMOs: Regulatory Considerations for Gene Therapy Trials

Like any new medical product, gene therapies must undergo rigorous approval processes. However, there are a number of additional regulations for gene therapies that don’t apply to conventional pharmaceuticals. This is especially true within the European Union. (It’s important to note that while most gene therapy studies are based in the U.S. and Europe — which...

Clinical Research: Phase 1 - Phase 4

Gene Therapy 101: From the 1960s to Today

Gene therapy is a hot topic in clinical research today — and for good reason! These technologies have the potential to treat — and in some cases even cure — a wide range of conditions, including rare genetic disorders that previously had no effective therapies. What Is Gene Therapy? Gene therapies are a diverse group of...

Planning Rare Disease Studies: Our Top Expert to Address WODC

DURHAM, N.C., APRIL 23, 2018 — Premier Research’s top rare disease and pediatrics expert will discuss clinical trial planning at the World Orphan Drug Congress in Oxon Hill, Maryland. Angi Robinson, Executive Director, Strategic Development, Rare Disease & Pediatrics, will present Early Development Planning for Orphan Drugs: Tasks, Timelines, and Takeaways on Thursday, April 26,...

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...

Clinical Research: Phase 1 - Phase 4

Improving Regulatory and Operational Performance in Orphan Drug Development

Today more than ever, regulatory bodies are providing a progressive platform for rare disease drug development. Extensions to existing regulations and incentives and attempts to reduce orphan drug designation applications provide a collaborative environment for companies developing orphan drugs. Understanding the regulatory landscape in orphan drug development — specifically the various pathways, incentives, and engagement...

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...

Consulting

PharmaVoice – Trending 2018: Rare Diseases

PharmaVoice – Trending 2018: Rare Diseasescontribution by Angi Robinson, Executive Director, Strategic Development Department, Rare Disease & Pediatrics

Consulting

Expert Co-Authors Article on Wolf-Hirschhorn Syndrome

DURHAM, N.C., OCTOBER 17, 2017 — Patients with Wolf-Hirschhorn syndrome, a congenital malformation disorder caused by variably sized chromosomal deletions, may have fewer motor-skill and communicative impairments than commonly believed. That’s the conclusion of an article in the European Journal of Medical Genetics co-authored by an expert at Premier Research. Dr. Susanne Schmidt, Premier Research...