Tag: Rare Disease

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...

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

Tales from the Road – World Orphan Drug Congress

Last week, I attended World Orphan Drug Congress in Belgium. It was my first WODC, and it was a productive three days in Brussels – even if it was held at the airport hotel, and we never left the building! The congress agenda covered a wide range of topics in the rare and orphan disease...

Consulting

NORD Summit, a Rare Disease Regulatory Wonderland

Rare disease is one of our main areas of focus here at Premier, so you’ll often see my colleagues and me talking about rare, through our webinars and white papers, and basically, almost any phone conversation with us. Last week, I attended the NORD Summit in Arlington, VA, and as usual, I found myself inspired...

Tales from the Road – Orphan Drugs Summit in Amsterdam

Introducing Tales from the Road, a series of blog shorts, where our Premier People report in from their travels outside the office. Because sometimes, not everything that happens on the road stays on the road! Today’s inaugural post comes from Juliet Moritz, Executive Director, Strategic Development Department, Rare Diseases, reporting back on her trip to Amsterdam...

Patient and Stakeholder Engagement

Register Now: Patient Advocacy Groups and Their Role in Orphan Drug Development

There are more than 7,000 known rare diseases, and these conditions afflict an estimated 30 million Americans (almost one in 10). Worldwide, some 350 million are living with a rare disease. But for 95 percent of these conditions, there are no FDA-approved treatments — not one. Conventional wisdom holds that innovation follows need: that if...

Clinical Research: Phase 1 - Phase 4

Getting It Right from the Start: Applying QbD to Rare Disease Studies

Quality and effective risk management are vital to every clinical trial, and their importance is greater still when working in rare indications. Your patient population is small, vulnerable, and geographically dispersed, so there’s limited data and little opportunity to replicate clinical trial results. It’s as if the phrase “get it right the first time” had...