Is it possible to get more efficiency in your conventional and specialty tests while maintaining ongoing quality? ISO 15189:2012 accreditation answers this question by delivering a comprehensive approach to quality management in medical, central and referral laboratories. Not only can these standards ensure quality, but they can reduce your risk of costly delays and ultimately save money in your trials.
“It’s no surprise that sponsors are concerned about the risk of regulatory findings and increasingly requesting ISO-accredited labs,” said Paul Kirchgraber, Vice President and Global General Manager at Covance Central Laboratory Services. “They need a demonstration of increasing quality—and savings—across their outsource vendors. Adopting ISO 15189:2012 is a powerful way to highlight our broad quality standards that meet or exceed their expectations.”
Like my colleagues at Covance, my work ultimately contributes to improving healthcare and patients’ lives. Our efforts to accelerate fresh approaches towards effective treatments became deeply personal in 2007 when my oldest sister, Vicki, was diagnosed with advanced Triple Negative Breast Cancer (TNBC).
From her initial diagnosis, my sister was full of vitality and enjoyed a high quality of life due in part to several of the novel treatments that Covance had helped develop. She even participated in a long-term clinical trial involving an angiogenesis-inhibitor, in addition to traditional chemotherapy, during her early treatment.
Vicki became a student of her diagnosis, reading countless journal articles on the rapid advances in scientific understanding. When her health took a sharp decline, she asked for my support in helping her gain access to a new trial focused on the immune system. She wanted to do whatever she could to help others find a better treatment, or even a cure, for TNBC. Ultimately, she was too weak to make the journey to participate in a cutting-edge clinical trial. Vicki died on October 1st, 2013.
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
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.
Iteration is the key for earlier and better decisions
The great inventor Thomas Edison once said, “I have not failed. I’ve just found 10,000 ways that won’t work.” While he was not talking about compounds, he could have been. The likelihood of success for any given compound is less than one percent. Aggravating the situation are several other factors, including constant pressure on decreasing pricing, quickly diminishing point of return at the 20-year mark, and along the way, inherent risk and large investments.
Still, we persevere. But to be successful in today’s environment requires a different, non-linear approach. The status quo will not work. We recommend taking a holistic view of the drug development process and an integrated approach. Continue reading
A five-point checklist for early, positive studies
Now more than ever, there is increasing pressure to bring new drugs to market more efficiently. With so much at stake when introducing a drug into the human body for the first time, you need to consider safety and efficacy and an ability to make a decision whether and how to proceed at every turn. As a result, you want to improve the earlier stages of drug development to enhance the quality of compounds that progress to Phase III. The end goal? To increase the success rate of molecules in Phase III. Continue reading
Regulatory strategy hinges on having the end in mind from the outset
“Begin with the end in mind” has become a popular catchphrase, but only because it proves itself to be true time and time again. You can more easily comply with regulatory requirements by expending increased effort into your target product profile. This is the foundation of your regulatory strategy and sets the stage for future development considerations. It is the second of ten elements of regulatory strategy:
- Summary of guidelines/precedents from multiple agencies
- Target product profile
- Agency interaction plan and timeline
- Identification of regulatory data requirements
- Assessment of registration routes and mechanisms globally
- Consideration of country registration timelines to meet business objectives
- Early market access and reimbursement plan
- Consideration of external influencing
- Lifecycle support and supplemental application plan
- Risk management and post-marketing plans
As device technology significantly improves and becomes more reliable and user friendly, the range and quality of measurable modalities—from actigraphy (movement or sleep patterns) to respiration—continues to expand. Wearable medical devices are being depended on for gathering and transmitting objective, experiential data in real time. These devices could enable researchers to go from looking at a very small number of data points to analyzing hundreds of readings per second from thousands of patients. Continue reading
The integrity of any Phase I clinical program relies heavily on the production, supply and analysis of quality dose formulations. Historically, drug doses have been formulated, manufactured and analyzed at the contract manufacturer, which typically adds significant time and expense given the small volume and the regulatory expectations at Phase I. One solution that is becoming widely practiced is combining extemporaneous preparations at the clinical research unit with independent dose analysis. It’s a one-two punch that not only saves time and money, it provides an extra assurance of quality.
Pharmacies are nimble
Contract manufacturers are built for the scale and regulatory requirements of a large Phase III clinical trial. Volume is their game. In Phase I, volume is overkill and leads to additional time and cost. Continue reading
Failures of Phase III programs after successful Phase II programs is probably the worst outcome of a clinical development program, as it failed in the most costly way. Nevertheless, these failures occur not infrequently. In psychiatry, highly publicized Phase II success stories ended in discontinuations of development programs, such as the NK1-antagonist program in depression several years ago. More recently, other examples have emerged. Some skip the Phase II process altogether with designs, which are supposed to provide “pivotal” data for regulatory purposes in large Phase III-like studies, which are just labeled as Phase II. These failures do not come out of the blue. Sometimes it is important to go back to basics and consider the purpose of Phase II trials.
What is the purpose of a Phase II trial?
The purpose of Phase II trials, besides gaining insights into the safety of a compound, is broadly exploratory, i.e. to generate data, which help with the design of the pivotal Phase III program. In a therapeutic area, a reasonably performed Phase II study can provide insights into clinical and biological patient characteristics, which match the properties of the drug under study. With an increased interest in personalized medicine, these boundaries between patient populations have to be understood in order to be successful. This approach is in direct contradiction to the urge to generate a “pivotal” Phase II outcome. Continue reading