Good news for the Duchenne Muscular Dystrophy (DMD) community. On June 8th, BioMarin announced the filing of a Marketing Authorization Application to the European Medicines Agency for Drisapersen, an antisense-mediated exon 51-skipping compound able to target the most prevalent genetic mutations responsible for the lack of production of functional dystrophin. The European filing follows the submission of a New Drug Application to the US FDA for Drisapersen back in April 2015.
Normally, dystrophin bridges cytoskeletal proteins to extracellular matrix and stabilises muscle fibres during contraction. The lack of its production in DMD leads to muscle damage, progressive muscle wasting, severe disability and premature death between the second and third decades due to cardiac or respiratory failure.
Who do you trust with your brand? If you are not properly testing your raw materials, your brand’s reputation may be at risk.
Infant formula is one of the most highly regulated products in the world—and with good reason. As often the sole source of nutrition for infants, your end products must be unquestionably safe and consistent.
Often, manufacturers test only their end products for adherence to quality and safety standards. This approach can be inefficient and does not offer solutions to recurring problems. By assuring proper testing of your raw materials, you gain confidence in your suppliers’ Certificates of Analysis, minimize risk and increase efficiency of production.
When manufacturing infant formula, you have more than food safety to think about. Managing regional differences in your global supply chain, maintaining consistency through seasonal variability and refining your process for efficient production are only a few of your challenges. The following five steps will help you strengthen management of your supply chain.
Despite the growing use of flow cytometry, there are currently no official regulatory guidance documents governing its validation. Having recognized the gap, stakeholders from the pharmaceutical industry and clinical testing laboratories have proactively published recommendations.
Scientists helping scientists with guidelines
In 2005, biomarker research was gaining momentum but the lack of clear validation guidelines made biomarker data difficult to interpret, hampering its usage. Existing validation paradigms applied only to PK data. Scientists from the American Association of Pharmaceutical Scientists (AAPS) realized that one set of rules could not fit all and that new standards were needed. They issued Fit-for-Purpose papers, addressing the need for accuracy, compliance and fitness for intended use and introducing the concept of iterative method validation to track biomarker development phases.
Makers of dietary supplements are keeping a close eye on New York after several major retailers were recently accused of selling mislabeled store-branded herbal supplements.
The allegations came after testing initiated by the state’s Attorney General’s office found that supplements, such as ginseng and echinacea, apparently did not contain the labeled ingredients. Although dietary supplements do not have to be approved by the U.S. Food and Drug Administration (FDA) before being sold to consumers, they must be labeled correctly and safe for consumption. They also must, according to current good manufacturing practice, be tested using scientifically valid methods–in other words, “accurate, precise, and specific for its intended purpose.”1
If you walk into a McDonald’s restaurant anywhere in the world, one thing is certain: your hamburger will taste the same. Ensuring that level of consistency in tens of thousands of restaurants is no small feat—and is a testament to the stringent quality and safety practices followed by their suppliers.
Today increasing numbers of food manufacturers seek to achieve that same consistency worldwide. Achieving consistency takes regular, global product testing. However, finding a partner that can providethis confidence, anywhere in the world, requires some investigation of its own.
So what do you look for in a lab? And how do you compare one to another? After more than 20 years of managing safety and quality programs, these are my top four suggestions:
In the effort to reduce attrition rates and improve approval rates of new molecular entities by regulatory agencies, there’s no doubt that biomarkers can make a big impact. But it’s not as simple as tacking on additional studies. Biomarker development requires an insightful strategy and consideration of specific opportunities and needs throughout the drug development pipeline.
A quality biomarker starts at the source—the sample itself. Sample collection and handling protocols must be standardized to specify the minimum volume requirement in the proper container along with the most optimal temperature during transportation and storage. These requirements should be backed and driven by validated processes. To further ensure biomarker stability, it’s equally critical to include the maximum allowed time in transportation. Continue reading
There’s no denying that studies are only as strong as their resulting data—and nonclinical studies produce a lot of data. A two-week study can easily generate more than 2 million results, while a two-year study can contain upward of 500 million data points. In an effort to process and easily interpret these massive data files, the FDA has developed SEND (Standard for Exchange of Nonclinical Data).
While it’s easy to think of SEND as another obstacle, it’s actually an opportunity to reveal new insights and gain efficiencies in data management. Exchanging information in a standard format can ease knowledge transfer between internal and external databases, as well as provide a common framework to support a more robust submission process. Coupled with the emergence of new tools for visualization and statistical analyses, SEND has the power to revolutionize the way data drive drug development decision making. Continue reading
When a patient reads the label on their medicine bottle, he or she naturally relies on the medicine to contain the correct drug, be safe, work as intended and list the correct dosage. The pharmaceutical companies that produce these medicines similarly must rely on their internal manufacturing processes and quality control testing to generate the medicine responsible for this patient trust.
For the development of biologic medicines, the process of generating a quality product is less straightforward than that of a small-molecule medicine, like pain relievers such as aspirin. Selecting the right partner, such as Covance and its ‘Central GMP Testing Laboratory’ model, can smooth the path to validation and consistent manufacturing quality for your biologic. Continue reading
“The whole is greater than the sum of its parts” is often quoted to inspire teamwork and synergy but it can also apply to drug development. Studies that assess endpoints in isolation have value and can achieve the desired outcome. Yet, many times a more complicated picture emerges and assessing multiple endpoints in a combined study reveals a more holistic view.
Inspired by the 3Rs—reduction, refinement and replacement of animals used in safety testing—the possibility of integrating multiple endpoints into one study is shaping new best practices in early drug development. Integrated solutions can maximize the value of each study to provide a better understanding, reveal earlier decision points and produce greater confidence in clinical outcomes.
While the concept seems straightforward, it’s not only a combination of otherwise standalone studies. Integrated solutions require a unique blend of fit-for-purpose experimental strategies tailored to each unique drug development program and the relevant endpoints. Continue reading
It seems every few months there’s another announcement about a new rapid method to test for pathogens. These developments have food manufacturers talking, but why the growing need for speed when it comes to food pathogen testing? And how do you know which method is right for you?
Faster answers, minimizing impact
The benefits of rapid testing are most obvious when you consider the investigation of a food-borne illness outbreak caused by a pathogen such as Listeria monocytogenes, E.coli O157:H7 or Salmonella. These methods allow investigators to quickly link case strains and screen more samples. They also can accelerate root cause investigations so manufacturers can get to a resolution more quickly. Continue reading