Rare Disease

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

Biomarker Method Validation in Rare Disease Drug Development

While drug discovery has seen major advances over the last few years — especially in the domain of rare disease research — the number of new drug approvals has not kept pace with increasing development costs. However, the innovative use of biomarkers has the potential to change that trend. Biomarkers in Rare Disease Research Biomarkers are objectively measurable characteristics...

Medical and Regulatory Affairs

8 Programs That Get Rare Cancer Treatments to Patients Faster

A variety of factors can make it difficult to conduct traditional full-scale clinical trials for new treatments of rare cancers. Consequently, because so little information is available, treatments for rare oncology patients are inadequate or nonexistent. Luckily for both patients and researchers, the FDA and the European Medicines Agency (EMA) have introduced regulations that expedite review and approval of certain investigative drugs. Expedited FDA...

Clinical Research: Phase 1 - Phase 4

Want to Study Rare Disease? Get Ready for These Roadblocks

A rare disease, also referred to as an orphan disease, by definition affects a small percentage of the population. Most are genetic and appear early in life, with one in three children with a rare disease dying before their fifth birthday. While the definition of “rare” varies from country to country, the US defines a rare disease as...

Clinical Research: Phase 1 - Phase 4

Innovations in Rare Oncology Trial Design

A rare cancer is often defined as one with an incidence of less than 15 per 100,000. The patient populations of these conditions are so small that traditional clinical trial design strategies can be unfeasible. But, with such a low incidence, just why is rare oncology research so important? Why Rare Oncology Needs Innovation Keep in mind...

Clinical Research: Phase 1 - Phase 4

Rare Disease Research: 3 Biggest Hang-Ups to Patient Recruitment

When developing treatments for a relatively common illness or disease, patient recruitment is relatively straightforward.  Screening and data collection can generally be done in a centralized location in a highly controlled environment. Now, imagine the concentration of people with the disease is not high enough in your local area, or even your country for that...

Medical and Regulatory Affairs

Life Science Leader – Orphan Drug Incentives & Innovations On The Rise

The year 2015 was a productive one for introduction of drugs that target rare diseases. U.S. regulators approved 21 new orphan drugs, a 40 percent increase from the previous year. European regulators approved a record 18 orphan compounds, a small increase over 2014. Any progress is a good thing, but these advances pale when weighed...

Patient and Stakeholder Engagement

Tales from the Road – World Congress Patient Advocacy Summit

This week’s Tales from the Road is from Angi Robison, Executive Director, Strategic Development Department, Rare Disease & Pediatrics, who gives us a bit about what she took away from the World Congress Patient Advocacy Summit back in October. Greetings from the road! I just had an excellent two days at the World Congress Patient Advocacy...

Clinical Research: Phase 1 - Phase 4

Rare Disease Studies: How to Run Successful Programs

Speakers: Patricia Nowowieski, Head of Global Clinical Operations for Alexion Pharmaceuticals. Patricia’s work in global clinical trials involves specialized populations and complex logistical and regulatory challenges. Angi Robinson, Executive Director and Scientific Account Leader for Premier Research. Angi’s extensive experience includes oversight of global studies in pediatrics and rare diseases. Mallory Bissett, Associate Director of...