Tag: Rare Disease

Consulting

Planning a Gene Therapy Trial? Ask an Expert — and Pay Attention

It’s been 46 years since researchers Theodore Friedmann and Richard Roblin published a landmark paper in the journal Science that posed a provocative question: “Gene therapy for human genetic disease?” And while clinical trials for gene therapy drugs have become much more common in the past 20 years — more than 2,400 had been conducted...

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...

Patient and Stakeholder Engagement

Stories of Courage and Hope: Key Takeaways from Disorder: The Rare Disease Film Festival

Every so often, you stumble onto a marketing partnership that just seems to fit. When we were approached earlier this year about a rare disease festival happening in October, we knew immediately that we had to be a part of it. Who could pass up the opportunity to be part of a first-of-its-kind event that...

Patient and Stakeholder Engagement

Why the time’s right for a Rare Disease Film Festival

Premier Research is proud to be a presenting sponsor of “Disorder: A Rare Disease Film Festival,” being held in Boston in October. Festival co-founder Daniel DeFabio shares with us why having such an event is important for raising awareness, sparking conversations and hopefully, maybe even a cure or two. A couple years ago, I made...

Clinical Research: Phase 1 - Phase 4

Fragile X: The Quest to Treat a Complex, Little-Understood Condition

We worked on a Phase II study of a drug to treat Fragile X syndrome that, like most rare work we do, was a challenge from the start. Fragile X is a rare and not fully understood genetic disorder, typically resulting from an expansion of the CGG triplet repeat within the Fragile X mental retardation 1 (FMR1) gene...