Featured Trial Design Editorial

  1. “Acceptable Risk” Reduces Clinical Development Time

    We learn about risk from a very young age. If you take a good risk and it pays off, you are rewarded. Bad risks can often result in punishment. Can the use of “acceptable risk” also allow us to cut the duration of clinical trial timelines?

  2. A Practical Framework For Assessing Risk In Clinical Trials

    The purpose of a comprehensive assessment system is to support structured, systematic, objective, and rational decision making. This holds true even when assessing something as difficult to pin down as risk. 

  3. Ensure A Smooth Start-Up In Gene Therapy Trials

    Preparations for a large Phase 3 clinical trial will always present challenges. In the area of gene therapies, those challenges can be magnified. Gene therapy trials can add more time and cost to a trial, and will also require specific expertise not needed in small molecule and biologics trials.

  4. Are You Properly Engaging Your Clinical Pharmacology Team?

    Robert Homolka, Director of Clinical Pharmacology Operations at Shire, feels the support the Clinical Pharmacology group provides to the clinical development programs is one of the more important aspects of his job. Studying and ensuring the safety of a drug is of primary importance to the sponsor, and clinical pharmacology is front-and-center in leading that effort.

  5. BMS Invests Record $3.6 Billion In Immuno-Oncology Pathway

    Bristol-Myers Squibb (BMS) and Nektar Therapeutics have announced a partnership deal worth up to $3.6 billion that will lay out a new path for immuno-oncology (I/O) combination therapies.

  6. Cell Therapy Trial Creates Skin Cells For Rare Disease Patients

    Abeona Therapeutics recently conducted a Phase 1/2 clinical trial for individuals with Recessive Dystrophic Epidermolysis Bullosa (RDEB). RDEB is an often fatal connective tissue disorder marked by skin blistering and caused by a lack of a collagen protein responsible for anchoring the dermal and epidermal layers together.

  7. The Father And CEO Who Climbed A Mountain To Raise Awareness

    How far would most CEOs go to raise awareness of the diseases they are attempting to treat? Tim Miller, CEO of Abeona Therapeutics, climbed more than 14,000 feet to the top of Mt. Rainier to raise awareness for Sanfilippo Syndrome, a rare and deadly genetic disease that impacts children.

  8. Beat AML Utilizes Innovative Master Trial Configuration

    Acute myeloid leukemia (AML) is a cancer that develops in bone marrow where new blood cells are created. It quickly moves into the blood, where it can be fatal in a matter of months. A new trial using a Master Trial configuration will provide hope for patients.

  9. The Role Of Modeling & Simulation In Clinical Trials

    Modeling and simulation (M&S) in the drug development process is an industry-proven scientific approach used to inform crucial drug development decisions such as dosing, drug-drug interaction (DDI), and other critical safety and efficacy questions. The FDA has shown a strong commitment to utilizing M&S in the drug development process.

  10. Cell Therapy Trials Present Unique Challenges For Pharma

    Maria Fardis, CEO of Iovance Biotherapeutics, has spent 18 years working in the pharma industry. Although most of her experience has been in small molecules and proteins, she now works in the cell therapy space and notes these treatments are a new and exciting space with meaningful differences in how clinical trials are conducted.