Tag: Rare Disease

Patient and Stakeholder Engagement

Premier Voices #8: Engaging Patients in Clinical Trials w/ Alan Thomas

Clinical research certainly has evolved over the past three decades. There are more trials crossing a broader spectrum of indications, and advances in study design and execution have dramatically changed the way drugs and medical devices are evaluated and sold. But perhaps no change has been more significant than the growth in engagement on the...

Patient and Stakeholder Engagement

Premier Voices #7: Rare Disease Advocacy & International Ataxia Day w/ Alan Thomas (Ataxia & Me)

This episode, we’re proud to share the first part of our conversation with Alan Thomas, patient advocate and founder of Ataxia & Me, ahead of International Ataxia Awareness Day on September 25. Alan talks about his personal journey with ataxia and how he first got started with patient awareness. Follow Alan and Ataxia & Me...

Clinical Research: Phase 1 - Phase 4

Premier Voices #2: Registry Studies & Natural Histories in Rare Disease Trials w/ Angi Robinson & Juliet Moritz

 Patient registries and natural history studies are vital tools for orphan drug researchers, who often struggle with limited understanding of diseases and sparse data due to small patient numbers. In the second installment of the Premier Voices podcast with Angi Robinson, Vice President, Clinical Development Services, Rare Disease & Pediatrics, and Juliet Moritz, we...

Study Design

Premier Voices #1: Rare Oncology w/ Colin Hayward & Peter Larson

The inaugural edition of Premier Voices focuses on rare oncology drug development and takes a looks at: Using adaptive design to limit patient exposure to ineffective treatments and increase the overall likelihood of success Making the most of small and geographically dispersed patient populations Dealing with limitations such as a lack of defined biomarkers and baseline...

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...