Study Design

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...

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...

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...

IVDR Survival Guide Part 2: Preparing for the NB Bottleneck

The In Vitro Diagnostic Regulation (IVDR) has fundamentally changed how in vitro diagnostics (IVD) are regulated in the EU. As a result, scrutiny for review and documentation has increased substantially, including a dramatic increase in required reviews by notified bodies (NB). For IVD companies, the challenge of adapting to a significant set of regulatory updates...

IVDR Survival Guide Part 1: A Deep Dive into Classification, Clinical Evidence, and Performance Requirements

The EU has been working to strengthen its regulations for better patient protection and more effective implementation of the rules for in vitro diagnostic medical devices. As a result, in May 2017, a five-year in vitro diagnostic regulation (IVDR) transition plan went into effect. While some of the dates have been extended1 to allow for...

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...

PM360 | New Developments in Gynecological Cancer Research

Substantial unmet needs exist in the diagnosis and treatment of gynecologic cancers. Often, these tumors are detected in later stages when treatment options are limited, and prognosis is poor. However, the gynecologic cancer space is on the cusp of change. The recent introduction of novel medicines, namely targeted therapies, has improved patient care and outcome....

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...

Resource Document: 8 New Elements of the IVDR

If you develop or manufacture in-vitro diagnostic products, you know the May 2022 roll-out date for the In Vitro Diagnostic Medical Devices Regulation (EU) 2017/746 (IVDR) is just around the corner. But what, exactly, does that mean? In this resource document, we highlight eight new elements of the IVDR.

PharmaLive | Advancing Gene Therapy Development: Key Regulatory and Clinical Trial Considerations

Insights into the human genome have created myriad therapeutic opportunities for previously untreatable diseases. Gene therapy offers promise for addressing unmet medical needs across therapeutic areas and even the potential for curing certain genetically based conditions. As research tools and technology become more sophisticated, gene therapy development is accelerating at an unprecedented pace. According to...