Developing, executing, and overseeing clinical trials is a complex process. Gaining reliable evidence from clinical trials is essential for appropriate decision-making activities regarding trial participants’ safety and the reliability of trial results. As clinical trials have become more complex, the...
Expertise: Rare Disease 79 results
Risk-Based Monitoring (RBM) makes an impact. The US Food and Drug Administration (FDA) requires that clinical trial sponsors “provide oversight to ensure adequate protection of the rights, welfare, and safety of human subjects and the quality of the data submitted...
Patient advocacy groups have exploded in number and scope in recent years and in many ways are reshaping the drug development landscape, from trial design to recruitment support to participation in the regulatory approval process. Thousands of organizations now advocate...
In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development,[1] addresses...
Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. The approvals of voretigene neparvovec-rzyl (Luxturna) for inherited vision loss, tisagenlecleucel (Kymriah) for lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta) for...
Careful planning is important for all early drug development programs, but it is particularly critical in rare diseases where study populations are limited and precedents for drug development are lacking. Pre-IND meetings with the FDA give sponsors the opportunity to...
In 1976, pop culture icon John Travolta starred in the made-for-television movie The Boy in the Plastic Bubble about a young man with a compromised immune system. The premise of the film is that Travolta’s character has to decide between...
Quality and effective risk management are vital to every clinical trial, and their importance is greater still when working in rare indications. Your patient population is small, vulnerable, and geographically dispersed, so there’s limited data and little opportunity to replicate...
What do you do when patients are too embarrassed to talk about what’s wrong with them? It was a trial for fecal incontinence, and patients wanted help – they just didn’t want to admit it. We needed a new outreach...