Tag: Rare Disease

Real-World Science and Late Phase

The Expanding Role of RWE in Rare Studies

Real-world evidence is increasingly important in drug development as the medical and pharma communities recognize the data’s potential for predicting the benefits of treatment interventions. Accelerating this trend is the Food and Drug Administration’s growing understanding of how RWE has helped researchers better understand and use COVID-19 treatments. While placebo-controlled trials continue to have a...

Clinical Research: Phase 1 - Phase 4

In Silico Modeling Unveils a New Era in Rare Disease Drug Development

Progress in advanced computational methods using modeling and simulation has been enabled by advancements in computing technologies and the rise of artificial intelligence and machine learning. This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of...

Rare Disease Research Embraces the Unknown. Let’s Take It On. Together.

Premier Research is built on a foundation of rare disease research. With decades of relevant experience in developing novel therapies, devices, and diagnostics, we make rare a common focus. Our aim is to provide Sponsors of novel rare disease therapies with proactive solutions to ensure trials stay on course and fast-track clinical outcomes to deliver...

Separating the Good from the Great: A Checklist for Evaluating a CRO for Rare Disease Studies

Small patient populations. Limited data on disease. Patient enrollment and retention challenges. Narrow site pools. These are some of the most common hurdles sponsors face in rare disease drug development. The right CRO partner can help turn these challenges into solutions. When launching your next RFP, keep this checklist on hand to ensure you’re covering...

Clinical Research: Phase 1 - Phase 4

To Placebo or Not to Placebo? The Great Debate in Rare Disease Trials

Small patient populations. Geographic dispersion. Competing studies. Sound familiar? If you’re a sponsor of a rare disease study, you know all too well the challenges that come with recruiting patients. These challenges lead to immense pressure to design and implement studies that limit the burden of participation, while keeping patients engaged and generating high-quality data...

Clinical Research: Phase 1 - Phase 4

Realizing the Full Value of Patient Centricity in Rare Disease: 5 Ways to Maximize Your Engagement Efforts

As industry interest in rare diseases increases, so does competition within an already challenging clinical research environment where patient pools are inherently limited and often geographically dispersed. To differentiate their trials and inspire interest among patients and other key stakeholders, sponsors need to be proactive in demonstrating their commitment to addressing unmet therapeutic needs.    Beyond...

Premier Research and InSilicoTrials Partner to Leverage In Silico Modeling and Simulation and Optimize Regulatory Pathways for Rare Disease Therapies

MORRISVILLE, N.C., MILAN, ITALY, TRIESTE, ITALY — Premier Research, whose mission is to help the most innovative biotech and device companies take their best ideas from concept to commercialization, and InSilicoTrials, a leader in the use of artificial intelligence (AI) and computational modeling and simulation (CM&S) to accelerate development of new therapies and medical devices, have...

Clinical Research: Phase 1 - Phase 4

At the Intersection of Rare Disease and Precision Medicine: A Road to Growth

Individually, rare diseases by definition have a low prevalence, but collectively, the societal burden and impact of these uncommon conditions is significant. The majority of rare diseases are genetic in origin, and advances in genomic sequencing tools and technologies have driven an increase in the identification of rare disorders. Currently, one out of every 10...

Clinical Research: Phase 1 - Phase 4

Rare Disease Patients as Partners: Value Beyond Enrollment

In 2020, the European Joint Programme for Rare Diseases developed a guide on patient partnerships in rare disease research projects. This guide still rings true today, and we summarize in this blog the key points for sponsors of rare disease studies to consider when engaging patients and their representatives in study planning. If you’re developing...

Clinical Research: Phase 1 - Phase 4

Charting the Path: How Patient and Caregiver Journeys Guide Rare Disease Study Design

It comes as no surprise that putting patients first is imperative for achieving success in rare disease development programs. However, to what extent can sponsors integrate patient input into their study design while still securing the high-quality data they need?  Although a bit of a balancing act, it is possible to design a study that...