Patient and Stakeholder Engagement


Long-Term Follow-Up in Gene Therapy Trials: Ensuring Patient Engagement & Regulatory Compliance

Gene therapy products are typically designed to achieve therapeutic effect through long-acting or permanent changes in the human body. Due to this extended period of exposure, patients who receive gene therapies may be at increased risk of delayed adverse events. To understand and mitigate the risk of these delayed adverse events, participants in gene therapy...

Patient and Stakeholder Engagement

Premier Insight 273: Conclusive & Positive Results Delivered Ahead of Time – By Eight Months

Background Patients rushed to enroll almost as soon as word of the study got out. We were dealing with a significant unmet need: Most patients with Binge Eating Disorder (BED) seek help from psychiatrists, nutritionists, or obesity specialists. But there was no approved, effective pharmacologic treatment. And patients desperately wanted one. Objective With unanticipated interest...

Patient and Stakeholder Engagement

Meeting the Patients Where They Are: A Case Study in Treatment Landscape Mapping

Meeting the Patients Where They Are: A Case Study in Treatment Landscape Mapping and its Impact on Site Selection as a Result of Partnering with a Patient Advocacy Organization March 3, 11:35am CESTPatient Partnering in Clinical DevelopmentBerlin, Germany The role of Patient Advocacy Organizations (PAOs) has been evolving over the past few decades and the...

Clinical Research: Phase 1 - Phase 4

Applied Clinical Trials – Patient Engagement and Advocacy: Advancing the Cause of Clinical Drug Research

Duchenne muscular dystrophy (DMD) patients almost universally lose the ability to walk as the disease progresses. Patients generally understand that loss of ambulation is to be expected, and as was learned from a patient preference study, they tend to put greater priority on maintaining use of their hands. Yet for many years, DMD drug trials...

Patient and Stakeholder Engagement

Chance Connections Become New Relationships at Global Genes

At Premier Research, we attend a lot of events throughout the year – and through these events, we have the opportunity to meet individuals from all sides of the clinical development process. At the recent Global Genes RARE Patient Advocacy Summit, we met countless patients, advocates, and their families, including Kim Tuminello and her daughter...

Patient and Stakeholder Engagement

PM360 – Working with Advocates: Understanding Patient Concerns

Patient advocacy groups have exploded in number and scope in recent years and in many ways are reshaping the drug development landscape, from trial design to recruitment support to participation in the regulatory approval process. Thousands of organizations now advocate for millions of patients, and while many patients and families eagerly engage with these groups,...

Patient and Stakeholder Engagement

Outsourcing Pharma – Patient centricity efforts becoming more wide-ranging and sophisticated, say CROs

Patient centricity efforts becoming more wide-ranging and sophisticated, say CROs contribution by Juliet Moritz, Vice President, Patient and Stakeholder Engagement

Patient and Stakeholder Engagement

Premier Insight 270: A Rosacea Trial Failed Many Enrollment Hurdles.

Two large, parallel trials with 107 sites spread across the United States. Heavy competition for patients because of multiple competing treatments already on the market. And an advertising strategy that needed to attract more than 1,400 subjects fast enough to meet the sponsor’s aggressive schedule. Those were just some of the challenges we faced when...

Medical and Regulatory Affairs

The FDA’s New Guidance on Natural History Studies in Rare Diseases: What You Need to Know

In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development,[1] addresses one of the major challenges sponsors encounter when developing therapies for rare disease: the lack...

Patient and Stakeholder Engagement

Patient Engagement Expert to Address Orphan Drug Congress April 11

DURHAM, N.C., March 25, 2019 — Premier Research’s head of patient engagement will discuss a patient-centered look at drug development at the World Orphan Drug Congress (WODC) USA in Oxon Hill, Maryland. Juliet Moritz, Premier Research’s newly appointed vice president of patient and stakeholder engagement (PASE), will present From the End to the Beginning: A Patient-Centric...