As researchers seek to harness the human immune system to fight cancer, they’re looking at several emerging opportunities to expand use of biomarkers. Among them: Human leukocyte antigen typing. Microbiome analysis for determining risk of inflammatory complications with immune therapeutics. Tumor mutation burden, measured via whole genome sequencing, whole exome sequencing, or comprehensive gene panel...
Vaccines were once thought to have great potential for combating some types of cancer, but reality has failed to match those expectations. To date, vaccines have failed to play a major role in the pursuit of immune response for oncology patients. There have been two notable successes — sipuleucel-T (marketed as Provenge) is approved to...
DURHAM, N.C., MAY 23, 2017 — Interleukin-2, interferon, and other immune-modulating agents have long been used to treat some solid malignancies, but their efficacy is generally limited to immunogenic cancers such as melanoma and kidney cancer. Until now. Today, multiple immuno-oncology pathways are under development, and we’ll explore this promising trend in a webinar on...
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...
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,...
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...
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...
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...
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...
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,...