Rare Disease

Consulting

5 Endpoints for Rare Oncology Trials

In general, greater certainty about the clinical benefit of a drug correlates with an increased time to achieve needed results — the best evidence can take years of careful follow-up. But an unnecessarily long time to market isn’t good for sponsors and patients alike. This is especially true for patients battling rare cancers: With many of these conditions lacking standard treatments,...

Consulting

Adaptive Design Strategies in Rare Oncology

Traditional trials apply a frequentist strategy, which rely on the accuracy of pre-defined design assumptions (or inputs) to construct an effective design that yields robust final trial results. From first-patient-in to last- patient-out, trial execution proceeds without change, following a black box approach. While this strategy is common in clinical research, it is not well suited to...

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

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

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...

Patient and Stakeholder Engagement

Rare Cancer Research from the Patient’s POV

The purpose of clinical research is ultimately to help patients. That may sound obvious, but all too often we forget the human side when it comes to clinical trials. For patients with rare cancers, taking their perspectives into consideration is invaluable to quality research. But what specific unmet needs do rare oncology patients have? What...

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...