Rare Disease

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...

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...

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...

Clinical Research: Phase 1 - Phase 4

Choices, Choices: Which Type of Natural History Study Meets Your Needs?

When you’re looking into conducting a natural history study, you’ve got two things to consider up front: the premise that underlies all of these studies, and which of several types suits your purpose. We tackled these and other topics in our last rare disease webinar. Natural History vs. Registry Studies in Rare Disease considers a...

Premier Research Sponsors ‘Disorder,’ a Rare Disease Film Festival

DURHAM, N.C., AUGUST 14, 2017 — Rare diseases afflict tens of millions of people, and for professionals seeking to treat these conditions, it can be tempting to regard patients more as diagnoses and outcomes than as folks seeking to lead otherwise normal lives. That’s why Premier Research has signed on as a presenting sponsor of...

Clinical Research: Phase 1 - Phase 4

4 Potential Advantages of Adaptive Design

Adaptive design is a type of clinical trial methodology that incorporates prospectively planned opportunities for modification of one or more aspects of a study’s design or its hypotheses based on interim analysis of study data. Explicitly planning these pre-specified changes helps to maintain scientific integrity while also introducing greater flexibility in the clinical research environment. The major...