Probably one of the most significant developments in sterilization and aseptic practices in pharma is that of vaporized hydrogen peroxide (VHP) as a contact or “cold” sterilizing agent. The use of VHP in contained environments has allowed us to decontaminate working spaces, equipment, and materials and has led to the development of the reliable isolator.
If we take for granted that there will always be new and changing processes, systems, and digital platforms and the organizational complexity within Big Pharma will not abate, how do we ensure we optimize the entire clinical trial process and make the best use of our technology and process investments?
The EMA Q&As and their updates mark an important turning point in the pharmaceutical industry regarding cleaning validation and quality risk management. The new ASTM standards will provide guidance to the industry on the appropriate development of health based exposure limits, for the implementation of science- and risk-based approaches to cleaning validation, and on the use of statistical techniques for measuring the risk in cleaning and assessing the effectiveness of cleaning processes.
The true effect of an intervention is often not seen until real-world usage takes place, but with such a delay between R&D and healthcare delivery, how can the industry close the gap? And what is needed to deliver more effective interventions that patients really want?
The new trade regime unleashed by President Trump has not only heightened trade tensions, it has the potential to affect patient access to affordable medicines. His administration’s Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs highlighted foreign governments “freeriding” off of American investment in innovations.
Imagine your company just received FDA approval of a new pharmaceutical, the result of years of clinical research and difficult regulatory scrutiny. The product is being manufactured and is shipping to distributors and wholesalers. Providers and patient advocacy groups seem excited for the launch, and sales goals are considered aggressive. However, one key variable remains: coverage.
While the designation for what constitutes a combination product is clearly defined by the FDA, industry continues to push the boundaries for disease treatment with new and innovative breakthrough drug therapies. As regulatory professionals, the challenge we face is to align these new therapies with application-filing strategies including investigational new drug applications, new drug applications, biologics license applications, and medical devices.
Progress in development of gene and cell therapies around the world has potential to transform standards of care for a range of diseases and address significant areas of unmet need in healthcare over the coming years. In the U.S. alone, almost 20 gene and cell therapy products have been approved thus far,1 with many other development programs reaching later clinical stages. The technology platforms of many of these drugs also offer the potential for curative efficacy and expansion for use in multiple indications.
In recent years, a growing number of pharmaceutical companies have recognized the potential benefits of pre-launch access programs, either as components of later-stage clinical development or early access programs or to support importation of drugs into countries recognizing an FDA or European Medicines Agency (EMA) approval. These programs help patients access investigational therapies prior to regulatory approval or commercial launch and are generally reserved for therapies that have demonstrated an acceptable level of proof of concept and safety. In addition to expanding treatment options for patients, often in indications that have limited or no approved therapies available, pre-launch access programs are also widely seen to play a significant role in advancing many drug development programs.
Fall is in the air, and Halloween is just around the corner. I always associate Halloween with “The Wizard of Oz.” When I grew up, Halloween meant reruns of the movie and was a highly anticipated annual family viewing event. Dorothy, the Wicked Witch of the West, those creepy flying monkeys, and, of course, the Wizard. We all had our favorite characters, and my poor parents had to contend with my post-movie nightmares for days. But it’s the Cowardly Lion who’s been on my mind recently.