Companion diagnostics are evolving quickly as novel technologies emerge and new drug targets appear. Dr. Mark Roberts, Director of Diagnostics Development, recently shared his thoughts on the industry trends with the summit leaders at the World CDx Boston 2015.
How much progress has been made in the area of companion diagnostics? Continue reading
To drive change, medicine requires hard data to supply evidence of clinical benefit. However, the studies we rely on to make decisions about a drug’s efficacy are often statistically underpowered – that is, therapeutic trials may fail to show the benefit of agents or devices when a benefit does, in fact, exist. This is due to limited data from smallpatient populations or too much variability in the data.
We performed analyses of studies of anticoagulation in electrical cardioversion to examine this problem more clearly. We also show how proactive data pooling could help to mitigate limitations in statistical power. Continue reading
Change is in the Air
There’s reason for new hope in the ongoing battle against cancer. From standing-room-only presentations of provocative data at cancer conferences, to landmark publications and new drugs approvals, the signs are multiple and clear. Harnessing the immune system as an anti-cancer therapy–a strategy that has yet to fully deliver on its promise– is now the most exciting area of oncology drug development.
Immune Surveillance: an Invisible Malignancy Sentinel
The first chapter in the story of cancer immunotherapy is a tale that provides perspective on how evolving scientific insight serves as a backdrop to the interplay between human hopes and the sometimes capricious nature of medical advances. But that story is well beyond the scope of the next 800 or so words. However, we can take advantage of hindsight to consider some of the key lessons learned. Continue reading
Companion Diagnostics: The New Engine
Companion diagnostics’ impact on pharmaceutical development is like dropping a new engine into a classic car. Faster speed. Better performance. More efficiency. Companion diagnostics is changing the way we develop, test and market new therapies—with full-throttle power.
Today, we often have the ability to test a patient to see what drugs will work—or not work—and watch for mutations and triggers down the road. But back up a bit: we can also design drug trials to include subjects with the correct biomarkers for the treatment. And back up a bit more: we can develop drugs and biomarker tests together for the most effective combinations of disease targets, drugs and patients. Continue reading
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