Before starting Phase 1 trials, an Investigational New Drug (IND) application must be approved by the FDA. This critical early step in clinical trial development grants an exemption to laws prohibiting the transportation of drugs across state lines prior to market approval. The three major required areas of information in an IND include: Animal pharmacology...
There are many pitfalls that come with preparing an Investigational New Drug (IND) application for FDA submission, but with the right plan – and the right partner – there are also plenty of ways to avoid them. The most important strategy? It all comes down to one thing: Always remember to keep your audience –...
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 discuss their unique development challenges and determine where regulatory flexibility can be justified. While the...
Use of long-acting injectable antipsychotics is often reserved for patients with chronic or treatment-refractory schizophrenia or repeated non-compliance issues. However, recent studies have consistently found a role for these treatments both soon after diagnosis and in the treatment of chronic disease. A severe and often debilitating neurodevelopmental disorder, schizophrenia affects a person’s ability to think...
Data monitoring committees and clinical endpoint committees can provide independent, expert review of study data and opinions to standardize outcomes and optimize data quality to help sponsors meet complex trial monitoring requirements. As global, multi-center medical device studies become increasingly common, trial monitoring is growing more complex. Regulators require that sponsors perform site monitoring to...
Advances in medical technology have opened the door for dermatology mobile health (mHealth) programs, and these same technologies have the potential to radically change dermatology trial designs. Here are four ways digital disruption in the dermatology field is benefiting both patients and providers: 1. Reduced wait times: Digital health assessments make time spent scheduling, traveling,...
Small- to mid-sized biotech and pharma companies face many challenges when planning and executing an early-phase oncology trial, from performing a regulatory gap analysis and developing a target product profile to identifying the right patients and selecting efficacy endpoints. Sponsors of first-in-human trials of novel oncology compounds face significant challenges. Beyond the typical circumstances of limited budget,...
The marketplace for orphan drugs is growing, and changes in the regulatory landscape are providing favorable conditions for collaboration in the area of drug development in rare diseases. Understanding the regulatory and operational nuances of orphan drug development can help sponsors position their promising compounds for clinical and commercial success. Today more than ever, researchers...
To achieve operational excellence in gene therapy trials, sponsors must understand – and overcome – obstacles ranging from start-up regulations and site selection to patient recruitment and retention. Gene therapy holds promise for treating a wide range of diseases, from cancer and diabetes to rare genetic disorders. It has also sparked great interest because it...
Understanding the obstacles inherent in Alzheimer’s clinical trials, from high screen failure rates to lengthy trial durations that are demanding for both patients and caregivers, can help sponsors plan for – and overcome – these challenges. Despite intensive research, nearly 15 years have passed since the lastnew Alzheimer’s disease medication was approved. For those living...