Oncology & Hematology

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 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...

Medical and Regulatory Affairs

Rapid Change, Real Promise: The Future of Rare Oncology Research

As our understanding of the genetic and molecular basis of cancer advances, rare oncology research is accelerating at an unprecedented pace. In fact, in 2014, more than 40 percent of U.S. Food and Drug Administration’s orphan drug designations were for rare cancers. Moreover, the trend toward increasing international cooperation among big pharma, biotech, and academia...

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

Maximizing Success in Early Stage Oncology Trials

The success rate of investigational compounds eventually approved for clinical use in cancer remains the lowest among all diseases. Of the more than 750 drugs currently under development for the treatment of cancer, it is predicted based on past performance that only a few will ultimately demonstrate sufficient efficacy and safety for regulatory approval and...

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

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...

Premier Research Bolsters Rare Disease and Oncology Drug Development Expertise with New Hires

DURHAM, N.C., June 13, 2016 — Premier Research is expanding its international organization of experts in rare disease amid continued growth in the field — especially in rare oncology, which has surpassed one-third of the company’s rare disease work. “Development of novel compounds for treatment of unmet medical needs historically has been overlooked by big pharma,...

Clinical Research: Phase 1 - Phase 4

Premier Insight 265: Exceeding Expectations: Durable Remission in Diffuse Large B-Cell Lymphoma

When a developer of a CD19-directed cytolytic antibody for B-cell Lymphoma engaged Premier Research to assist with screening and enrollment in a global Phase 2 study, the Premier project team devised an operational strategy with a goal to exceed expectations. Despite several challenges, the enrollment was completed three months ahead of schedule thanks to consistent...