Rare Disease

Consulting

Ashanthi DeSilva’s Story: A Look Back at the First Gene Therapy Trial – TEST

In 1976, pop culture icon John Travolta starred in the made-for-television movie The Boy in the Plastic Bubble about a young man with a compromised immune system. The premise of the film is that Travolta’s character has to decide between living in isolation behind a protective bubble or dying engaged with the physical world. Hollywood...

Clinical Research: Phase 1 - Phase 4

LSX 2021: The Path From Preclinical to and Through Clinical

Presented by: Peter Larson, Vice President, Medical Affairs for Hematology-Oncology Angi Robinson, Vice President, Specialty Areas April Marquick, Cell and Gene Therapy Expert Originally presented at the 2021 LSX World Congress virtual conference, this webinar series by Premier experts in oncology, rare disease, and cell and gene therapies focuses on development considerations for advanced therapies,...

Medical and Regulatory Affairs

Clinical Data Trends: Transforming Data Access/Analysis

The clinical trial industry has been steadily undergoing a data evolution. Data from remote capture devices, such as wearables and other novel sources (e.g., increasingly large health data repositories and electronic healthcare data), have gained importance. Proper management and utilization of such data allows sponsors to untether from a set roster of trial sites, focus...

Clinical Research: Phase 1 - Phase 4

Rethinking Data Quality Best Practices in the Era of Decentralized Clinical Trials

Pandemic-related disruptions have accelerated much-needed change in clinical operations, but this change has been accompanied by questions about data collection and data quality. In a recent survey commissioned by Oracle Health Sciences, more than 75 percent of industry respondents indicated that limitations in patients’ ability to attend on-site visits sped up their adoption of decentralized...

Medical and Regulatory Affairs

Long-Term Gene Therapy Follow-Up: Patient Considerations

When developing a long-term follow-up strategy for gene therapy trials, safety is the main concern, closely followed by the patient perspective. Digital technologies and monitoring have proven essential to this, easing the burden on the patient, and providing accurate, time-saving methods of data management. Momentum is increasing in the field of gene therapy, with over...

Patient and Stakeholder Engagement

“It Motivates Me Every Day”: Our Premier Research Team on Why Rare Disease Research Matters

There are as many as 7,000 distinct types of rare and genetic diseases, and an estimated 400 million people suffer from a rare disease globally. In addition, three of 10 children with a rare disease won’t live to see their fifth birthday. Rare Disease Day, celebrated around the world on the last day of February,...

Study Design

Establishing Competitive Advantage in Dermatology Trials: Critical Study Design and Operational Considerations for a New Age

Skin diseases are the fourth leading cause of health afflictions, affecting nearly 900 million people worldwide.1,2 At the same time, there has been a remarkable increase in dermatology drug development activity in recent years. For conditions such as atopic dermatitis, psoriasis, and melanoma, there are hundreds of ongoing or planned clinical trials.3 In this new...

Clinical Research: Phase 1 - Phase 4

Premier Research to Present on Development Considerations for Advanced Therapies at LSX World Congress

DURHAM, N.C., January 25, 2021 — Premier Research, the clinical research company that delivers outcome-focused insights which support sponsors in developing life-changing therapies, will join the healthcare and life science industries’ most forward-thinking executives at the LSX World Congress virtual conference from February 1 to 5, 2021. The conference will include a presentation by Premier...

Medical and Regulatory Affairs

How COVID-19 Is Making Operationalizing Gene Therapy Trials Even Tougher – And How We Handled It

Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the science of precision medicine. In our previous blog post, we discussed site selection, manufacturing, long-term...

Medical and Regulatory Affairs

Site Selection, Manufacturing & Long-Term Follow-Up: Looming Challenges for a Rescue Phase 1/2 Gene Therapy Trial

Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. In this blog post, we introduce a case study as a framework for exploring critical study design considerations of...