Rare Disease

Patient and Stakeholder Engagement

The Future of Patient Advocacy and Orphan Drug Development: Challenges and Opportunities

Despite advances in the orphan drug development process and the more prominent role that patient advocacy groups now play, researchers in this area still face a number of challenges. But, fortunately, increased collaboration between patient groups, industry leaders, and regulatory bodies can help overcome these hurdles. Barriers to orphan drug research include: Small population By definition,...

Clinical Research: Phase 1 - Phase 4

From Laboratory to Bedside: Expediting Development of Novel Rare Cancer Treatments

Study of rare cancers poses special challenges for drug developers, who often must draw on their experience in both oncology and rare disease. Current strategies and processes for general oncology drug development don’t always apply to rare oncology, a field that today accounts for up to 20 percent of new cancer diagnoses. To optimize drug...

Clinical Research: Phase 1 - Phase 4

4 Phases of Biomarker Method Validation in Rare Disease Research

Rare diseases present some unique obstacles for researchers that significantly contribute to the high cost of drug development. Fortunately, innovations in biomarker use have the potential to significantly cut back on these costs while improving data quality. However, before biomarkers can be used in this research, their associated collection, measurement, and evaluation methods must be validated. The key phases of biomarker...

Consulting

From Lab to Bedside: February 28 Webinar Examines Treatment of Rare Cancers

DURHAM, N.C., FEBRUARY 22, 2017 — Premier Research will share new insights on rare oncology drug development at a webinar on Tuesday, February 28. From Laboratory to Bedside: Expediting Development of Novel Rare Cancer Treatments (11 a.m. EST / 4 p.m. GMT / 5 p.m. CET) will examine how new processes and regulatory pathways are...

Consulting

Rare Disease Day 2017: With Research, Possibilities Are Limitless

One of the most rewarding efforts I’ve seen in my years working with Premier Research is watching our Rare Disease Day activities unfold. Every year we ask our employees to support Rare Disease Day in some way, so we can all remember how crucial our role is in clinical development, especially in this area of...

Clinical Research: Phase 1 - Phase 4

The Science and Art of Conducting Clinical Trial Feasibility in Rare Disease and Pediatric Studies

Rare disease and pediatric studies present significant challenges, including country selection, site selection, and patient recruitment. A thoughtfully-designed, well-executed feasibility study can help sponsors obtain a realistic assessment of the best path forward for developing and conducting successful global clinical trials. Performing a thorough assessment of clinical trial feasibility is an important early step in...

Quality

Applying Quality by Design to the Rare Disease Population

When applied to clinical development, Quality by Design is an approach that focuses effort on prospectively identifying and managing risk to improve clinical trial quality and outcomes. The application of Quality by Design principles is particularly important in rare diseases, where the limited, geographically-dispersed, and often vulnerable patient pool leaves little margin for error. By...

Clinical Research: Phase 1 - Phase 4

Clinical Trials in Rare Diseases: Every Patient Counts

In many rare and ultra-rare disease studies, sponsors do not have the luxury of choosing the countries or sites where the study will be conducted. Instead, they must use the sites where those patients are already being treated, regardless of geography. Additional protocol criteria – such as treatment-naïve patients or general prevalence of a disease...

Clinical Research: Phase 1 - Phase 4

Proven Strategies for Rare Disease and Orphan Drug Development in the U.S.

Orphan drugs are medicinal products intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. Orphan drugs are a growing market, due in large part to tax incentives, longer periods of market exclusivity, and shorter, smaller clinical trials, as well as the premium pricing associated with many orphan...

Clinical Research: Phase 1 - Phase 4

The Science of Hope

Despite the inherently small market for orphan drugs, orphan drug development has recently undergone significant growth, with global sales of over $100 billion in 2015. In 2015, more than 20 new drugs were approved by the U.S. Food and Drug Administration (FDA) and 18 new drugs were approved by the European Medicines Agency (EMA) for...