Our educational components provide a glimpse into our culture, medical perspectives, future vision and direction. How we influence our audience reflects the impact on our sponsors and their patient, not only at the present, but also in the future.
There may be as many as 7,000 rare diseases. The European Union defines a disease as rare when it affects less than one in 2,000 people. According to the Orphan Drug Act, a rare disease affects less than 200,000 people in the United States. Support from FDA, EMA and other regulatory agencies, combined with technological and scientific advances, have made it more feasible for pharmaceutical companies to give dedicated attention to orphan drug development. In doing so, they're developing medications for rare diseases that often have no cure and no available treatment. To ensure novel therapies advance from the lab to regulatory approval, a blockbuster-drug approach won't work. Managing rare disease studies to completion requires innovative thinking in nearly every phase of program development. Those willing to become trailblazers have the highest odds of success.
To execute precision medicine clinical trials, sponsors have adopted a range of new approaches, especially in early-stage studies. Many of these models allow sponsors to recruit more patients and bring drugs to market faster— goals drug developers in all therapeutic areas work toward.
As clinical trials become more complex and costly, the need for skilled project managers to oversee them is more important than ever. Project management methodologies drive success for enterprise organizations in all sectors. These same principles can help pharmaceutical and biotech companies run more efficient, successful clinical trials.
Gaps in dermatological research present potentially lucrative market opportunities for pharmaceutical companies. Patients that suffer from skin conditions and diseases also benefit. Effective treatments relieve irritating, chronic, and often painful symptoms, which improves their quality of life and lifts the psychological and social burden of a visible dermatological issue.
The CDISC set of standards revolutionized data management in clinical trials by introducing a standardized model for data interoperability. The standard has been shown to decrease study data resource requirements by 60% overall, and 70-90% in start-up stages when implemented early. This results in dramatic decreases in cost, but also increases data accuracy and time to lock, to say nothing of the tremendous benefits having a standard dataset available in the course of the study. Download our whitepaper and find out how to make early CDISC work for your trial.
A team from Biorasi joined more than 900 leaders from the FDA, NIH and the pharma and bio-pharma industries for the National Organization for Rare Disorders (NORD) & Orphan Products Breakthrough Summit.
Throughout two packed days of keynotes, breakout sessions and panel discussions, we gained valuable insight into the most urgent rare disease topics. Read about the a few takeaways from the event in this article.
As digital therapeutics are integrated into every aspect of the healthcare landscape, patients, payers, and providers are seeing an increasingly complex set of software-driven therapy options reaching the market. This rapidly expanding field has enormous potential but faces challenges at every stage, from design to patient perception. Focusing on the clinical trial stage, how does the trial of a digital therapeutic compare to that of a pharmaceutical? What are the specific challenges of running a digital therapeutics clinical trial, and how can sponsors adapt their approach for the highest chance of success?
In my previous article, I explained the types, causes and symptoms of Acute Kidney Injury (AKI). Here, I’ll share how physicians treat and manage AKI, as well as what to expect.
CRRT or intermittent hemodialysis? To treat AKI, nephrologists primarily use renal replacement therapy (RRT), which includes intermittent hemodialysis, peritoneal hemodialysis, various forms of CRRT, and “hybrid” therapies such as prolonged intermittent renal replacement therapy (PIRRT) or sustained low-efficiency hemodialysis (SLED). A few clinical trials are taking a look at CRRT and intermittent hemodialysis under different conditions.
What is AKI? AKI is a sudden, reversible decline in the kidney’s glomerular filtration rate (GFR); in other words, its ability to filter metabolic waste. This kidney damage causes an elevation of serum urea and creatinine, which affects the body's ability to function properly. Most AKI cases are reversible. However, it's important to treat AKI as early as possible to avoid its progression to chronic kidney disease or kidney failure.
Researchers are studying a few new and existing drugs to treat FSGS. FSGS needs a novel medication that regulates both high body fats and inflammasome inhibition via upstream innate immune system to block intracellular initiation of inflammatory cascade and extra cellular inflammation. Over the past five years, nearly 30% of Biorasi-initiated or ongoing studies involved chronic kidney disease. Our experience allows us to develop successful nephrology clinical trials.
To unlock more targeted, effective treatments, genetics researchers are exploring mutations responsible for some of the most common motor neuron diseases. These discoveries may lead to gene therapy treatment routes that do a better job of slowing disease progression than current medications. Much like gene therapy in oncology has brought improvement in cancer regression in recent years, we believe gene therapy—particularly therapy that targets and modulates RNA—is the future of neurology.
Biorasi’s Oncology Center of Excellence remains at the forefront of this exciting field. Our team of clinicians, program managers and other experts stay up to date on the latest developments, from Tumor Infiltrating Lymphocyte (TIL) therapy to gene therapy advances. This experience, combined with Biorasi’s worldwide site network and data-driven approach, allows us to guide oncology programs to success—on time and on budget.
A clinical trial's operational efficiency depends on a solid oversight and response mechanism. Without vigilant monitoring, patient safety, data integrity, and the overall health of your trial come into jeopardy.