Rare Disease

Clinical Research: Phase 1 - Phase 4

Strategies for Finding ‘Hidden’ Patient Populations

A little less than five percent of the world’s population are estimated to suffer from more than 7,000 different types of known rare diseases. There are even more medical conditions where those who have them are too embarrassed to talk about what’s wrong or simply don’t know exactly what ails them. Yet, these individuals are...

Patient and Stakeholder Engagement

PM360 – Patient Advocates Assume a Large Role as Orphan Drug Development Expands

“Rare disease” may be the biggest misnomer in the pharmaceutical industry. Around the world, 350 million people live with conditions classified as rare because each afflicts a relatively small segment of the population. In the U.S., 30 million—nearly one in 10 Americans—suffer from one of about 7,000 known rare diseases. As it applies to this...

Clinical Research: Phase 1 - Phase 4

Natural History Studies: A Growing Force in Treating Rare Diseases

Knowledge of a disease’s natural history is essential to building the scientific foundation for an effective clinical development program. That’s especially the case when developing drugs to treat rare diseases, which by their very nature are poorly understood. Natural history studies don’t just track the course of diseases over time, but identify demographic, genetic, environmental,...

Consulting

Premier Insight 249: A High Unmet Need that No One Wanted to Talk About

What do you do when patients are too embarrassed to talk about what’s wrong with them? It was a trial for fecal incontinence, and patients wanted help – they just didn’t want to admit it. We needed a new outreach and recruitment process, so we tried radio, outdoor, and subway. We rigorously tracked what worked...

Clinical Research: Phase 1 - Phase 4

Natural History vs. Registry Studies in Rare Disease

Patient registry and natural history studies are often thought of as interchangeable, but there are major differences in these approaches to clinical drug development. Understanding the role of each is important, as well as understanding the distinctions on how these study designs are used in pursuing treatments for rare diseases. Patient Registries A patient registry...

Clinical Research: Phase 1 - Phase 4

Natural History or Registry Study? Conducting Rare Disease Research

Sound drug development requires a comprehensive understanding of the disease being treated. To design reliable clinical studies and achieve meaningful outcome measures, researchers must apply known etiology and thorough knowledge of the disease’s progression. For rare diseases, this information is often minimal at best: patient numbers are small and historical data is spread across treating...

Consulting

The Rise of Patient Advocacy

The effects of rigorous patient advocacy are easily seen today throughout the pharmaceutical industry. Patients who suffer the debilitating effects of rare diseases are banding together to be heard in an attempt to have their conditions studied to further the development of treatments and cures. However, the influence of patient advocacy groups has not always...

Clinical Research: Phase 1 - Phase 4

The Biggest Hurdles in Rare Oncology Research (And How to Overcome Them)

Translational research has the potential to help all patients, but the principles behind bench-to-bedside research hold special promise for patients with rare diseases — many of which have no standard treatments. Here’s how changes to the way rare oncology studies are performed are getting treatments to the patients who need them at a faster rate. What Makes...

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 trial design use only a priori inputs to obtain final trial results. From first patient in to last patient out, trial execution proceeds without change, following a black box approach. While a standard for clinical research, this strategy doesn’t always suit rare oncology research, which must contend with very small patient populations that are often difficult to treat....