Patient and Stakeholder Engagement

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...

Clinical Research: Phase 1 - Phase 4

Pharmaceutical Commerce: Empowering Patients in Oncology Trials

Patient engagement is critical to the success of oncology clinical trials, many of which have endpoints that require study participants to be followed for many years to evaluate side effects and long-term survival data. And yet, sponsors often postpone careful consideration of strategies for patient engagement and retention until after the treatment phase of their...

Clinical Research: Phase 1 - Phase 4

Pharmaceutical Outsourcing: Decentralized Clinical Trials

Patient centricity—also known as patient-centered care—has been a growing force across healthcare. Like the consumer-driven change in other industries, patient centricity upends established models. Whereas traditionally, healthcare professionals dictate care decisions with little consideration for the patient’s desires, patient-centric care puts the patient first. Its tenets include:  • Empowering patients through technology • Reducing inconvenience...

Clinical Research: Phase 1 - Phase 4

Improving Birth Control for All: Opportunities and Obstacles in Male Contraception

Introduction There are currently more than a dozen categories of female birth control with proven efficacy on the market. For men, the only options available today are condoms and vasectomy. Despite decades of research and discussion about male contraception, no new products have entered the market since the condom was created in the 1800s. As...

Clinical Research: Phase 1 - Phase 4

Clinical Researcher: Preparing for the Future

In July 2021, the Industry Standard Research (ISR) Report on Hybrid/Virtual/Decentralized Clinical Trials Market Outlook surveyed 109 industry leaders worldwide who had been involved in DCTs over the past year.{1} Respondents anticipated a 12% increase in hybrid trials over the next two years—and predicted that DCTs would outstrip traditional trial models within three years. They...