Featured Trial Design Editorial

  1. Can ICER Help Design Clinical Trials But Stay Independent?

    According to a report, the Institute for Clinical and Economic Review (ICER) is considering offering a service, for a fee, to pharmaceutical manufacturers whereby ICER would provide guidance on clinical trial design.

  2. Virtual Registries: How To Bring Patient-Centricity Into Clinical Research

    Our rapidly increasing genetic-level disease knowledge and exponential technology advances are changing the way we manage health. But we may never fully seize these opportunities if we limit our clinical research approach to past models. Changing societal attitudes around sharing personal data and new consumer technology make innovative study designs and operations possible. This includes new possibilities in designing and operationalizing patient registries by moving to “virtual” setups.

  3. Overcome The Challenges Of An Ultra-Rare Disease Trial

    ArQule is a personalized medicine company working in the oncology space to develop treatments that target specific mutations. While attempting to overcome a challenge with its product, ArQule was approached by a group at the NIH which wanted to partner to help patients with an ultra-rare disease known as Proteus syndrome.

  4. A Patient’s Journey To Join A CAR-T Cell Therapy Trial

    Nicole Gularte is currently battling cancer for the 8th time. The last time she had to do so, it seemed a CAR-T cell therapy trial might be the solution. This article is about her long battle with cancer, the struggle to get into a CAR-T cell trial, and the ups and downs she has faced along the way. Her story is an inspiration to others.

  5. Real World Evidence And The Collision Between Pharma R&D And Healthcare

    The true effect of an intervention is often not seen until real-world usage takes place, but with such a delay between R&D and healthcare delivery, how can the industry close the gap? And what is needed to deliver more effective interventions that patients really want?

  6. Designing Clinical Trials With The Payer In Mind

    Imagine your company just received FDA approval of a new pharmaceutical, the result of years of clinical research and difficult regulatory scrutiny. The product is being manufactured and is shipping to distributors and wholesalers. Providers and patient advocacy groups seem excited for the launch, and sales goals are considered aggressive. However, one key variable remains: coverage.

  7. Incorporating Patient-Centric Outcome Metrics In Rare Disease Trials

    Commercial viability of pharmaceuticals depends on getting medicines to patients, which requires developers to evaluate investigative therapies using outcomes that are quantifiable, easy to interpret, and clinically relevant. However, clinical relevance is often a matter of stakeholder perspective: what developers view as meaningful may not appear as such to patients and caregivers.

  8. A New Immunotherapy Paradigm: It’s Not Just For Cancer Anymore

    When Bruce Gillis, CEO of EpicGenetics, decided to look into fibromyalgia, he drew blood from 17 patients and analyzed 35 different immune system factors. When compared to a control group, he was astounded by the results. There was a huge disparity between the immune system markers for the 17 patients and those of the control group. That launched his quest for a cure.

  9. An Introduction To Financial Benchmarking In Biopharma Clinical Development

    The high cost and failure rate of new drug candidates going through clinical trials are well documented and a recurring subject of research both by industry and academia.1 Further, the cost of drug development is a debated topic, and there is no consensus on what the “true cost” is because of the different methods used for these calculations.2 The lack of adequate comparable cost data and value measures also makes it difficult for pharmaceutical sponsors and trial sites to implement financial benchmarking for planning, costing, and budget management.

  10. Why All The Hype Around Real-World Evidence? Here’s What You Need To Know

    This is part 2 of a two-part roundtable Q&A on real-world evidence (RWE) in the 21st century. In this installment, our experts discuss how RWE is utilized, why real-world data (RWD) analyses are getting more attention, and what the recent attention means for the future of medical practice and drug development. RWD analyses are utilized to generate insights about a medicine’s effectiveness, safety, and associated costs.