Patient and Stakeholder Engagement

Patient and Stakeholder Engagement

Seasonality: A factor for successful patient recruitment

Patient enrollment Managing clinical trials requires extensive planning of patient enrollment. Clinical trials may be more cost-effective with better forecasts of recruitment. A variety of circumstances influence the enrollment of subjects. Several factors to consider, including the rarity of the condition being examined, the capacity of the trial sites to recruit participants, and the degree...

Clinical Research: Phase 1 - Phase 4

Patient Enrollment and Retention: Overcoming Challenges in Observational Research

The challenge of enrollment for observational research Patient enrollment and retention for investigational trials are notoriously difficult. How is enrollment affected when there is no direct benefit to the patient: no breakthrough study drug with the potential to cure, no added caregiver time, no meaningful compensation for participating? Observational studies, by design, demand a continuation...

Clinical Research: Phase 1 - Phase 4

Addressing the Top 7 Challenges in Decentralized Dermatology Trials

Each year, nearly 85 million Americans see a physician for at least one skin disease, with $75 billion in direct costs to the U.S. healthcare system.[i] While the FDA has approved more than 110 dermatology drugs, these treatments address only about 30 indications, leaving the vast majority of known skin conditions unaddressed. In recent years,...

Clinical Research: Phase 1 - Phase 4

7 Tips for Designing and Operationalizing Rare Disease Natural History Studies

Natural history studies play a crucial role in rare disease drug development, providing insights that advance discovery and shape clinical trial design. The benefits of these studies also extend to understanding patient journeys, clarifying the standard of care, identifying disease-specific centers of excellence, and surfacing opportunities to improve patient care. In this blog post, we...

Clinical Research: Phase 1 - Phase 4

How to Use Patient Registries and Natural History Studies in Rare Disease Development

Successful drug development requires a deep understanding of the disease of interest—its etiology, epidemiology, presentation, manifestations, and progression. In rare diseases, however, much of this information may be unknown. Patient populations are small and historical data are collected inconsistently and dispersed across treating physicians practicing in diverse geographies. Patient registries and natural history studies are...

Clinical Research: Phase 1 - Phase 4

5 Tips for Operationalizing Gene Therapy Studies

Gene therapy clinical trials are logistically complex studies that require highly qualified sites and engaged patients for success. The ability to select the right sites and patients can be the difference between product failure and market approval. In this final part of a three-part blog series on gene therapy development, we provide tips on operationalizing...

Clinical Research: Phase 1 - Phase 4

Designing Robust Gene Therapy Studies

To develop a successful gene therapy, sponsors must conduct research studies that balance the need for safety oversight and robust clinical evidence with the challenges of finding the right patients, pairing them with qualified sites, providing informed consent, and confirming they are capable of — and committed to — fulfilling all protocol requirements.    In...

Clinical Research: Phase 1 - Phase 4

Pursuing the Promise of Gene Therapy: Pathways to Successful Clinical Trials

The promise of gene therapy is to provide transformative treatments that meaningfully improve quality of life for patients, many of whom are currently living with debilitating diseases. To bring these treatments to market, sponsors are tasked with conducting clinical trials that generate robust evidence with appropriate safety oversight. In addition, gene therapy trials are faced...

Clinical Research: Phase 1 - Phase 4

Maximizing Oncology Study Outcomes: Overcoming Enrollment Challenges in a Phase 2 Prostate Cancer Clinical Trial

Prostate cancer (PC) is one of the most commonly diagnosed cancers overall and the most frequently diagnosed cancer in men. The 2018 global estimate of new PC cases exceeded 1.2 million, and more than 350,000 men died from the disease worldwide.1 Over the last decade the emergence of new treatments, combined with better use of...

Clinical Research: Phase 1 - Phase 4

Premier Insight 281: Overcoming Enrollment Challenges in a Phase 2 Prostate Cancer Clinical Trial

Background Prostate cancer (PC) is among the most commonly diagnosed cancers overall, and the most frequently diagnosed cancer in men. The 2018 global estimate of new PC cases exceeded 1.2 million with more than 350,000 deaths worldwide.  Over the last decade, new treatments combined with better use of existing therapies in early-stage disease have transformed...