Treatments that are safe and effective for adults may be ineffective or even dangerous for children. But infants and children are often prescribed medications with “off-label” use, where the treatment’s safety, dosage and efficacy are based solely on adult studies. To address this issue, both drug developers and regulators are working to boost clinical trials in children and include this underserved market in their studies.
Challenges with pediatric trials
A number of factors work against studying pediatric populations. As a highly fragmented and dynamic population, children and infants undergo rapid developmental changes over time, complicating study design and interpretation.
In addition, small sample sizes and potentially low incidence rates can make it difficult to find a treatment group—as well as a suitable control group with an approved active control. Finally, ethical considerations, such as informed consent can be more complex in pediatric trials. Continue reading
The biologics sector continues to offer lucrative opportunities for future growth, but with relatively few contributions for the treatment of CNS indications. High-growth CNS market segments generally share one feature: biologics play a pivotal role in the treatment paradigm, or soon will. Neuromuscular-blocking biologics recently secured regulatory approval for chronic migraine. Alzheimer’s disease is a high unmet need indication currently addressed only by symptomatic treatments, with potential for disease modification biologic therapies to play an important future role.
The example of biologics’ success in the CNS market is highlighted largely by the success of therapies that target the multiple sclerosis (MS) disease process. This success has spurred some drug developers to increase their assets in this space and seek future opportunities for biologics in other CNS diseases. However, one important new reality that is slated to reshape the future landscape of this market is the introduction of biosimilars. Continue reading
Signed into law in 2011, the Food Safety of Modernization Act (FSMA) is the most sweeping change to the way food safety is regulated in the U.S. since the adoption of the Food Drug and Cosmetic act itself in 1938. Due to the sheer size and magnitude of this legislation, the FDA is taking time to get the rules correct. Continue reading
In 2013, oncology represented the largest segment of the clinical trial market. In 2014, it is estimated to grow by 4.9%,1 reaching nearly $100 billion. The number of oncology clinical trials stands well above those in other therapeutic areas and most major biopharmaceutical companies are involved in oncology to some degree.
Yet, with such compelling numbers, why do only 6.7% of these trials lead to FDA approval from Phase 1?2 Possibly because of difficulties recruiting patients for oncology trials, but more likely due to the complexity of cancer as a disease. Continue reading
US health care is exponentially more complicated today than just 5 years ago. Even the most competent, experienced marketing teams may not anticipate or clearly understand the complex forces that are changing how we select, dispense, and reimburse for drugs and devices.
Changes in Traditional Customers
Pharmacy Benefit Managers (PBM) are your primary contracting conduit into commercial and Medicare Part D plans to assure formulary market access. The FTC approved the $29.1 billion ESI-Medco merger, further consolidating the PBM group. If your new product is not granted preferential access on a national PBM, you may be losing access to 70 million patients. Continue reading
Running along the side of your thigh, the vastus lateralis muscle works with three other muscles within your “quad,” permitting actions crucial for forward movement. In the laboratory, clinicians are also relying on muscles like the vastus lateralis to move their research forward in the study of neuromuscular agents.
Muscle biopsies hold the key to understanding many aspects about a potential treatment or therapeutic agent. From pharmacokinetics to stem cell therapy, there are several intriguing areas of interest in the field. Continue reading
An estimated 1 million infants in the U.S. are fed formula from birth, and by the time they are three months old, about 2.7 million rely on formula for at least part of their nutrition.
On June 9, 2014 the U.S. Food and Drug Administration released a final rule regarding the manufacturing standards of infant formula. The final rule—which amends the FDA’s quality control procedures, notification, and record and reporting requirements for manufactures of infant formulas—is meant to ensure that formulas for infants without unusual medical or dietary problems are safe and support healthy growth. The rule also establishes current good manufacturing practices (CGMPs), and sets a date of September 8, 2014 for manufacturer compliance. Continue reading
A six-step systematic, proactive approach to your study can help transform risks into returns
Recently, the major regulatory agencies—FDA and EMA—have highlighted their concerns that current monitoring approaches are not only inefficient, but they also fail to support the overall integrity of the trial itself. In the light of recent regulatory guidances for risk-based monitoring (RBM), the industry is re-thinking the way clinical trials are conducted.
However, even before your study commences, the opportunity for RBM is present. By viewing your study as a holistic process—and by leveraging prior experiences and insights—you can more effectively manage risk while simultaneously optimizing quality. Continue reading
Drug discovery and development is inherently risky. Recent figures indicate that less than 11% of new pharmaceutical agents entering clinical development reach the marketplace across all therapeutic areas. For those working in CNS drug development, we know well that the prospect of seeing our compounds passing through all phases of drug development and successfully reaching the market is even more challenging.
This low success rate in CNS drug development has been attributed to a number of reasons, among them the complexity of the brain physiology, the limited characterization of the pathological mechanisms leading to neuronal dysfunction or the suboptimal knowledge about the mechanism of action of drug candidates by the time the compound enters clinical development. Continue reading
Since the 1960s, much medical research has been conducted on placebo, an inert agent that does not contain any active therapeutic substances. It looks like a real medicine, but it is not; and it is used to diminish the suffering of the patient through expectation rather than through the exertion of a specific medical effect. Given the importance of this false drug in the treatment of psychiatric and neurological diseases, these investigations have become one of the most interesting and critical aspects of neuroscience. However, rather than placebo itself, it is precisely the placebo response, defined as the patient’s condition or symptoms resulting from placebo (natural history minus placebo condition), that has attracted attention. A problem exists for clinical trials in major depression, anxiety, neuropathic pain, or Parkinson’s disease arises when this placebo response becomes high enough that it’s impossible to differentiate between the true effect or clinical response of the investigational drug and the placebo effect itself.
The placebo response constitutes a paradox. While in clinical development, placebo responders are excluded or treated with special attention to minimize and keep under control their response to placebo. Continue reading